Kerry Dooley Young

U.S. regulators canceled a July 31, 2019, advisory committee about lumateperone, an experimental schizophrenia drug that has had some mixed results in testing.

On July 23, the Food and Drug Administration announced the cancellation of the Psychopharmacologic Drugs Advisory Committee meeting it had previously called for to review the new drug application for lumateperone. The agency said the meeting was canceled because of “new information regarding the application.” The FDA said it was continuing to evaluate the application and would, as needed, announce a future meeting on it.

The developer of lumateperone, Intra-Cellular Therapies, issued its own statement on July 23, noting that a meeting had been scheduled with the FDA “shortly” and that an update would be provided after the meeting. The New York–based firm also said it recently had provided additional information to the FDA to meet agency requests. This information was related to “nonclinical studies.”

“The FDA canceled the advisory committee meeting to allow sufficient time to review this new and any forthcoming information as they continue” to review the new drug application for lumateperone, Intra-Cellular said in the July 23 statement. The company also said there may be an extension of the FDA’s Sept. 27, 2019, target action date on the lumateperone application.

Investors viewed this as bad news. Shares of Intra-Cellular on July 23 dropped from an opening price of $11.90 to a closing one of $8.19. On July 29, they closed at $8.12.

Still, it is unclear how the FDA will decide on the lumateperone application and whether the agency will call another advisory committee meeting on it.

Last year, the FDA accepted the application for lumateperone, a once-daily treatment, Intra-Cellular said. The agency had in 2017 given a fast-track designation to lumateperone for the treatment of schizophrenia.


Lumateperone is the lead product for the company. Intra-Cellular describes lumateperone as having a novel mechanism of action, and is said to act on the serotonergic, dopaminergic, and glutamatergic systems.

On the company’s website, Intra-Cellular says three large randomized, double-blind, placebo-controlled trials have been done for lumateperone as a schizophrenia drug. In two of these studies, results for lumateperone at a 60-mg dose showed a “statistically significant separation from placebo on the primary endpoint, the Positive and Negative Syndrome Scale or PANSS total score.”

In a recent routine filing with the Securities and Exchange Commission, Intra-Cellular said it was having an “ongoing dialogue” with the FDA about lumateperone. The company in 2016 had announced that, in a phase 3 study known as ITI-007-302, lumateperone had not separated from placebo on the primary endpoint, change from baseline on the PANSS total score, in the predefined patient population. The active control for ITI-007-302, risperidone, did separate from placebo.

In the recent SEC filing, Intra-Cellular said the FDA already has confirmed that the results of ITI-007-302 did not preclude the submission of a new drug application.

Intra-Cellular also said “lumateperone was statistically significantly better than risperidone on key safety and tolerability parameters, and exhibited a safety profile similar to placebo” in the 302 study. Lumateperone’s failure to best placebo in the 302 test was “in part due to an unusually high placebo response at certain sites.”

U.S. regulators canceled a July 31, 2019, advisory committee about lumateperone, an experimental schizophrenia drug that has had some mixed results in testing.

On July 23, the Food and Drug Administration announced the cancellation of the Psychopharmacologic Drugs Advisory Committee meeting it had previously called for to review the new drug application for lumateperone. The agency said the meeting was canceled because of “new information regarding the application.” The FDA said it was continuing to evaluate the application and would, as needed, announce a future meeting on it.

The developer of lumateperone, Intra-Cellular Therapies, issued its own statement on July 23, noting that a meeting had been scheduled with the FDA “shortly” and that an update would be provided after the meeting. The New York–based firm also said it recently had provided additional information to the FDA to meet agency requests. This information was related to “nonclinical studies.”

“The FDA canceled the advisory committee meeting to allow sufficient time to review this new and any forthcoming information as they continue” to review the new drug application for lumateperone, Intra-Cellular said in the July 23 statement. The company also said there may be an extension of the FDA’s Sept. 27, 2019, target action date on the lumateperone application.

Investors viewed this as bad news. Shares of Intra-Cellular on July 23 dropped from an opening price of $11.90 to a closing one of $8.19. On July 29, they closed at $8.12.

Still, it is unclear how the FDA will decide on the lumateperone application and whether the agency will call another advisory committee meeting on it.

Last year, the FDA accepted the application for lumateperone, a once-daily treatment, Intra-Cellular said. The agency had in 2017 given a fast-track designation to lumateperone for the treatment of schizophrenia.


Lumateperone is the lead product for the company. Intra-Cellular describes lumateperone as having a novel mechanism of action, and is said to act on the serotonergic, dopaminergic, and glutamatergic systems.

On the company’s website, Intra-Cellular says three large randomized, double-blind, placebo-controlled trials have been done for lumateperone as a schizophrenia drug. In two of these studies, results for lumateperone at a 60-mg dose showed a “statistically significant separation from placebo on the primary endpoint, the Positive and Negative Syndrome Scale or PANSS total score.”

In a recent routine filing with the Securities and Exchange Commission, Intra-Cellular said it was having an “ongoing dialogue” with the FDA about lumateperone. The company in 2016 had announced that, in a phase 3 study known as ITI-007-302, lumateperone had not separated from placebo on the primary endpoint, change from baseline on the PANSS total score, in the predefined patient population. The active control for ITI-007-302, risperidone, did separate from placebo.

In the recent SEC filing, Intra-Cellular said the FDA already has confirmed that the results of ITI-007-302 did not preclude the submission of a new drug application.

Intra-Cellular also said “lumateperone was statistically significantly better than risperidone on key safety and tolerability parameters, and exhibited a safety profile similar to placebo” in the 302 study. Lumateperone’s failure to best placebo in the 302 test was “in part due to an unusually high placebo response at certain sites.”

U.S. regulators canceled a July 31, 2019, advisory committee about lumateperone, an experimental schizophrenia drug that has had some mixed results in testing.

On July 23, the Food and Drug Administration announced the cancellation of the Psychopharmacologic Drugs Advisory Committee meeting it had previously called for to review the new drug application for lumateperone. The agency said the meeting was canceled because of “new information regarding the application.” The FDA said it was continuing to evaluate the application and would, as needed, announce a future meeting on it.

The developer of lumateperone, Intra-Cellular Therapies, issued its own statement on July 23, noting that a meeting had been scheduled with the FDA “shortly” and that an update would be provided after the meeting. The New York–based firm also said it recently had provided additional information to the FDA to meet agency requests. This information was related to “nonclinical studies.”

“The FDA canceled the advisory committee meeting to allow sufficient time to review this new and any forthcoming information as they continue” to review the new drug application for lumateperone, Intra-Cellular said in the July 23 statement. The company also said there may be an extension of the FDA’s Sept. 27, 2019, target action date on the lumateperone application.

Investors viewed this as bad news. Shares of Intra-Cellular on July 23 dropped from an opening price of $11.90 to a closing one of $8.19. On July 29, they closed at $8.12.

Still, it is unclear how the FDA will decide on the lumateperone application and whether the agency will call another advisory committee meeting on it.

Last year, the FDA accepted the application for lumateperone, a once-daily treatment, Intra-Cellular said. The agency had in 2017 given a fast-track designation to lumateperone for the treatment of schizophrenia.


Lumateperone is the lead product for the company. Intra-Cellular describes lumateperone as having a novel mechanism of action, and is said to act on the serotonergic, dopaminergic, and glutamatergic systems.

On the company’s website, Intra-Cellular says three large randomized, double-blind, placebo-controlled trials have been done for lumateperone as a schizophrenia drug. In two of these studies, results for lumateperone at a 60-mg dose showed a “statistically significant separation from placebo on the primary endpoint, the Positive and Negative Syndrome Scale or PANSS total score.”

In a recent routine filing with the Securities and Exchange Commission, Intra-Cellular said it was having an “ongoing dialogue” with the FDA about lumateperone. The company in 2016 had announced that, in a phase 3 study known as ITI-007-302, lumateperone had not separated from placebo on the primary endpoint, change from baseline on the PANSS total score, in the predefined patient population. The active control for ITI-007-302, risperidone, did separate from placebo.

In the recent SEC filing, Intra-Cellular said the FDA already has confirmed that the results of ITI-007-302 did not preclude the submission of a new drug application.

Intra-Cellular also said “lumateperone was statistically significantly better than risperidone on key safety and tolerability parameters, and exhibited a safety profile similar to placebo” in the 302 study. Lumateperone’s failure to best placebo in the 302 test was “in part due to an unusually high placebo response at certain sites.”

WASHINGTON – Kim Schrier, MD, the first pediatrician elected to the U.S. House of Representatives, describes her decision to seek a seat in Congress as a natural extension of her work caring for children.

Nick Piegari/MDedge News

Following the 2016 election, she said she became concerned about what she saw as rising threats to the health of families. The Republican congressional majority made several attempts to undo the Affordable Care Act. In an interview with Pediatric News, Dr. Schrier, a Democrat who represents Washington’s 8th district, said she worried about a rollback of the insurance protections the 2010 law created for people with preexisting conditions. She understood the need for these guarantees of access to care both as a physician and as a patient – Dr. Schrier was diagnosed with type 1 diabetes as a teenager.

Her concerns extended beyond the health care and insurance. Efforts to weaken environmental protections could create new risks for children’s health, she said. Dr. Schrier also worried about attempts to erode Roe v. Wade and to reduce funding for programs such as SNAP supplemental food assistance.

“I felt like every one of those elements was under assault and so who better to speak to that than the pediatrician with her own preexisting condition?” she said.

Dr. Schrier currently is the only female physician serving in Congress. “It’s exciting to be here and it’s exciting to bring that lens that no one else really has,” she said.

Still, the decision to run was not easy.

“I’ve left a practice that I love. I had no intention to run for office ever in my life.”

Dr. Schrier earned an undergraduate degree in astrophysics at the University of California, Berkeley. She worked for about a year at the Environmental Protection Agency before earning her medical degree at the University of California, Davis, and completing her residency at Stanford (Calif.) University. She was working as a pediatrician in the Seattle-based Virginia Mason Health System when she decided to run for Congress.

Her husband, David, and son, Sam, are staying on in the family’s home of Sammamish as she settles into her new work as a legislator. She said she returns home often.

“I go back and forth every week. I just didn’t see it working all that well with having them here,” she said. “I work from 8:00 or 9:00 in the morning until 9:00 at night and would never see them anyway.”

Dr. Schrier has long used an insulin pump and glucose monitors to keep her diabetes in check.

“It is a bit more difficult because my days are less predictable” when working in the Capitol, she said. “So I carry granola bars around in my purse.”
 

Bipartisan bill

Dr. Schrier said she is taking her time in deciding on the first health care legislation that she will introduce in the House. She’s considering measures that would focus on children’s health, possibly in connection with nutrition or with protection of those in immigrant communities.

“I’m looking at something that is uniquely me and know that a lot of my colleagues are going to be working on things like affordability of medications and I will partner with them,” she said.

Dr. Schrier said that she supports creating a way for people to buy into Medicare coverage, paying for this government insurance on a sliding scale. The American public is getting more comfortable with the idea of expanding access to Medicare, which “is incredibly popular but now exclusively available for seniors and other selected groups,” she said.

In her view, an expanded Medicare would stand among private insurance options, allowing for a gradual change.

“It’s a way that we could start tomorrow to let people buy in, as opposed to having a 5- or 10-year rollout that something as big and daunting as a Medicare-for-all plan would require,” she said.

But Dr. Schrier said she won’t start her legislative career with such a sweeping goal as a Medicare buy-in bill.

“I think it would be jumping the gun to come in right away with my own health plan because I really want to collaborate” with fellow lawmakers, she said.

Winning Republican support for her future legislation also is important to Dr. Schrier. “I want to make sure that I have the support of a lot of my colleagues,” she said. “I would like to see a successful first bill.

“The advantage of coming at this as a pediatrician and taking on a first bill or bills that relate to children is that both sides of the aisle can get behind what is good for families and children,” Dr. Schrier said. “I think that’s hard to really argue with.”

Dr. Schrier cosponsored her first piece of legislation in February, reaching across the aisle to work with Rep. Dan Newhouse (R-Wash.) to introduce H.R. 1048, a bill to authorize the next phase of a water resource management plan for Washington’s Yakima River basin.

Beyond that effort, Dr. Schrier said she’s seeking areas of common ground with Republican members of her state delegation. She had dinner in January with two Republican members of the Washington delegation – Rep. Cathy McMorris Rodgers and Rep. Jaime Herrera Beutler. Rep. McMorris Rodgers’ website describes her as a leader in the disabilities community and the only member of Congress to give birth three times while in office, while Rep. Herrera Beutler has spoken openly about her daughter’s battle with Potter’s syndrome, in which the kidneys fail to develop.

Within her party, Dr. Schrier is allied with the moderate New Democrat Coalition. Also in the coalition are two fellow physicians, Ami Bera, MD, and Raul Ruiz, MD, both from California. The New Democrat Coalition also has several lawmakers who flipped House seats from Republican control to Democratic in the 2018 election, a group that includes Dr. Schrier and Reps. Jennifer Wexton (D-VA) and Mikie Sherrill (D-NJ). It was the victories of these moderates that gave the Democrats control of the House. Yet, they get far less media attention than do House Democratic freshmen who are politically further to the left. These members, including Rep. Alexandria Ocasio-Cortez (D-N.Y.), tend to hail from districts where Republicans stand little chance of winning.

Dr. Schrier said the Democratic party, and Congress as a whole, should focus on issues such as affordable health care, on which the country can unite.

“If you’re looking for collaboration, that just doesn’t make the headlines and yet it is the absolute best thing for the country,” she said, adding that most Americans have more centrist views. “So we should really be governing to the middle and that’s how we can move our country forward.”

WASHINGTON – Kim Schrier, MD, the first pediatrician elected to the U.S. House of Representatives, describes her decision to seek a seat in Congress as a natural extension of her work caring for children.

Nick Piegari/MDedge News

Following the 2016 election, she said she became concerned about what she saw as rising threats to the health of families. The Republican congressional majority made several attempts to undo the Affordable Care Act. In an interview with Pediatric News, Dr. Schrier, a Democrat who represents Washington’s 8th district, said she worried about a rollback of the insurance protections the 2010 law created for people with preexisting conditions. She understood the need for these guarantees of access to care both as a physician and as a patient – Dr. Schrier was diagnosed with type 1 diabetes as a teenager.

Her concerns extended beyond the health care and insurance. Efforts to weaken environmental protections could create new risks for children’s health, she said. Dr. Schrier also worried about attempts to erode Roe v. Wade and to reduce funding for programs such as SNAP supplemental food assistance.

“I felt like every one of those elements was under assault and so who better to speak to that than the pediatrician with her own preexisting condition?” she said.

Dr. Schrier currently is the only female physician serving in Congress. “It’s exciting to be here and it’s exciting to bring that lens that no one else really has,” she said.

Still, the decision to run was not easy.

“I’ve left a practice that I love. I had no intention to run for office ever in my life.”

Dr. Schrier earned an undergraduate degree in astrophysics at the University of California, Berkeley. She worked for about a year at the Environmental Protection Agency before earning her medical degree at the University of California, Davis, and completing her residency at Stanford (Calif.) University. She was working as a pediatrician in the Seattle-based Virginia Mason Health System when she decided to run for Congress.

Her husband, David, and son, Sam, are staying on in the family’s home of Sammamish as she settles into her new work as a legislator. She said she returns home often.

“I go back and forth every week. I just didn’t see it working all that well with having them here,” she said. “I work from 8:00 or 9:00 in the morning until 9:00 at night and would never see them anyway.”

Dr. Schrier has long used an insulin pump and glucose monitors to keep her diabetes in check.

“It is a bit more difficult because my days are less predictable” when working in the Capitol, she said. “So I carry granola bars around in my purse.”
 

Bipartisan bill

Dr. Schrier said she is taking her time in deciding on the first health care legislation that she will introduce in the House. She’s considering measures that would focus on children’s health, possibly in connection with nutrition or with protection of those in immigrant communities.

“I’m looking at something that is uniquely me and know that a lot of my colleagues are going to be working on things like affordability of medications and I will partner with them,” she said.

Dr. Schrier said that she supports creating a way for people to buy into Medicare coverage, paying for this government insurance on a sliding scale. The American public is getting more comfortable with the idea of expanding access to Medicare, which “is incredibly popular but now exclusively available for seniors and other selected groups,” she said.

In her view, an expanded Medicare would stand among private insurance options, allowing for a gradual change.

“It’s a way that we could start tomorrow to let people buy in, as opposed to having a 5- or 10-year rollout that something as big and daunting as a Medicare-for-all plan would require,” she said.

But Dr. Schrier said she won’t start her legislative career with such a sweeping goal as a Medicare buy-in bill.

“I think it would be jumping the gun to come in right away with my own health plan because I really want to collaborate” with fellow lawmakers, she said.

Winning Republican support for her future legislation also is important to Dr. Schrier. “I want to make sure that I have the support of a lot of my colleagues,” she said. “I would like to see a successful first bill.

“The advantage of coming at this as a pediatrician and taking on a first bill or bills that relate to children is that both sides of the aisle can get behind what is good for families and children,” Dr. Schrier said. “I think that’s hard to really argue with.”

Dr. Schrier cosponsored her first piece of legislation in February, reaching across the aisle to work with Rep. Dan Newhouse (R-Wash.) to introduce H.R. 1048, a bill to authorize the next phase of a water resource management plan for Washington’s Yakima River basin.

Beyond that effort, Dr. Schrier said she’s seeking areas of common ground with Republican members of her state delegation. She had dinner in January with two Republican members of the Washington delegation – Rep. Cathy McMorris Rodgers and Rep. Jaime Herrera Beutler. Rep. McMorris Rodgers’ website describes her as a leader in the disabilities community and the only member of Congress to give birth three times while in office, while Rep. Herrera Beutler has spoken openly about her daughter’s battle with Potter’s syndrome, in which the kidneys fail to develop.

Within her party, Dr. Schrier is allied with the moderate New Democrat Coalition. Also in the coalition are two fellow physicians, Ami Bera, MD, and Raul Ruiz, MD, both from California. The New Democrat Coalition also has several lawmakers who flipped House seats from Republican control to Democratic in the 2018 election, a group that includes Dr. Schrier and Reps. Jennifer Wexton (D-VA) and Mikie Sherrill (D-NJ). It was the victories of these moderates that gave the Democrats control of the House. Yet, they get far less media attention than do House Democratic freshmen who are politically further to the left. These members, including Rep. Alexandria Ocasio-Cortez (D-N.Y.), tend to hail from districts where Republicans stand little chance of winning.

Dr. Schrier said the Democratic party, and Congress as a whole, should focus on issues such as affordable health care, on which the country can unite.

“If you’re looking for collaboration, that just doesn’t make the headlines and yet it is the absolute best thing for the country,” she said, adding that most Americans have more centrist views. “So we should really be governing to the middle and that’s how we can move our country forward.”

WASHINGTON – Kim Schrier, MD, the first pediatrician elected to the U.S. House of Representatives, describes her decision to seek a seat in Congress as a natural extension of her work caring for children.

Nick Piegari/MDedge News

Following the 2016 election, she said she became concerned about what she saw as rising threats to the health of families. The Republican congressional majority made several attempts to undo the Affordable Care Act. In an interview with Pediatric News, Dr. Schrier, a Democrat who represents Washington’s 8th district, said she worried about a rollback of the insurance protections the 2010 law created for people with preexisting conditions. She understood the need for these guarantees of access to care both as a physician and as a patient – Dr. Schrier was diagnosed with type 1 diabetes as a teenager.

Her concerns extended beyond the health care and insurance. Efforts to weaken environmental protections could create new risks for children’s health, she said. Dr. Schrier also worried about attempts to erode Roe v. Wade and to reduce funding for programs such as SNAP supplemental food assistance.

“I felt like every one of those elements was under assault and so who better to speak to that than the pediatrician with her own preexisting condition?” she said.

Dr. Schrier currently is the only female physician serving in Congress. “It’s exciting to be here and it’s exciting to bring that lens that no one else really has,” she said.

Still, the decision to run was not easy.

“I’ve left a practice that I love. I had no intention to run for office ever in my life.”

Dr. Schrier earned an undergraduate degree in astrophysics at the University of California, Berkeley. She worked for about a year at the Environmental Protection Agency before earning her medical degree at the University of California, Davis, and completing her residency at Stanford (Calif.) University. She was working as a pediatrician in the Seattle-based Virginia Mason Health System when she decided to run for Congress.

Her husband, David, and son, Sam, are staying on in the family’s home of Sammamish as she settles into her new work as a legislator. She said she returns home often.

“I go back and forth every week. I just didn’t see it working all that well with having them here,” she said. “I work from 8:00 or 9:00 in the morning until 9:00 at night and would never see them anyway.”

Dr. Schrier has long used an insulin pump and glucose monitors to keep her diabetes in check.

“It is a bit more difficult because my days are less predictable” when working in the Capitol, she said. “So I carry granola bars around in my purse.”
 

Bipartisan bill

Dr. Schrier said she is taking her time in deciding on the first health care legislation that she will introduce in the House. She’s considering measures that would focus on children’s health, possibly in connection with nutrition or with protection of those in immigrant communities.

“I’m looking at something that is uniquely me and know that a lot of my colleagues are going to be working on things like affordability of medications and I will partner with them,” she said.

Dr. Schrier said that she supports creating a way for people to buy into Medicare coverage, paying for this government insurance on a sliding scale. The American public is getting more comfortable with the idea of expanding access to Medicare, which “is incredibly popular but now exclusively available for seniors and other selected groups,” she said.

In her view, an expanded Medicare would stand among private insurance options, allowing for a gradual change.

“It’s a way that we could start tomorrow to let people buy in, as opposed to having a 5- or 10-year rollout that something as big and daunting as a Medicare-for-all plan would require,” she said.

But Dr. Schrier said she won’t start her legislative career with such a sweeping goal as a Medicare buy-in bill.

“I think it would be jumping the gun to come in right away with my own health plan because I really want to collaborate” with fellow lawmakers, she said.

Winning Republican support for her future legislation also is important to Dr. Schrier. “I want to make sure that I have the support of a lot of my colleagues,” she said. “I would like to see a successful first bill.

“The advantage of coming at this as a pediatrician and taking on a first bill or bills that relate to children is that both sides of the aisle can get behind what is good for families and children,” Dr. Schrier said. “I think that’s hard to really argue with.”

Dr. Schrier cosponsored her first piece of legislation in February, reaching across the aisle to work with Rep. Dan Newhouse (R-Wash.) to introduce H.R. 1048, a bill to authorize the next phase of a water resource management plan for Washington’s Yakima River basin.

Beyond that effort, Dr. Schrier said she’s seeking areas of common ground with Republican members of her state delegation. She had dinner in January with two Republican members of the Washington delegation – Rep. Cathy McMorris Rodgers and Rep. Jaime Herrera Beutler. Rep. McMorris Rodgers’ website describes her as a leader in the disabilities community and the only member of Congress to give birth three times while in office, while Rep. Herrera Beutler has spoken openly about her daughter’s battle with Potter’s syndrome, in which the kidneys fail to develop.

Within her party, Dr. Schrier is allied with the moderate New Democrat Coalition. Also in the coalition are two fellow physicians, Ami Bera, MD, and Raul Ruiz, MD, both from California. The New Democrat Coalition also has several lawmakers who flipped House seats from Republican control to Democratic in the 2018 election, a group that includes Dr. Schrier and Reps. Jennifer Wexton (D-VA) and Mikie Sherrill (D-NJ). It was the victories of these moderates that gave the Democrats control of the House. Yet, they get far less media attention than do House Democratic freshmen who are politically further to the left. These members, including Rep. Alexandria Ocasio-Cortez (D-N.Y.), tend to hail from districts where Republicans stand little chance of winning.

Dr. Schrier said the Democratic party, and Congress as a whole, should focus on issues such as affordable health care, on which the country can unite.

“If you’re looking for collaboration, that just doesn’t make the headlines and yet it is the absolute best thing for the country,” she said, adding that most Americans have more centrist views. “So we should really be governing to the middle and that’s how we can move our country forward.”

BETHESDA, MD. – The risk of brain damage from sickle cell disease (SCD) merits more attention, even with progress made in recent decades to prevent strokes, according to Lori Jordan, MD, PhD, of Vanderbilt University, Nashville, Tenn.

“Whether we can see it or not, the same injury we’ve been talking about in the kidney and the liver and other places is occurring in the brain” with sickle cell disease, Dr. Jordan said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

The concern about long-term brain injury reflects major shifts in SCD treatment. Improved medical care has transformed SCD from a disease that often resulted in an early death to more of a chronic condition, Dr. Jordan said, citing research that shows a survival rate of roughly 99% to age 18 years (Br J Haematol. 2016 Jun;173[6]:927-37).

One of the major success stories in SCD treatment also has been using primary prevention steps to cut the risk of overt stroke at least 10-fold, Dr. Jordan said. Primary prevention includes annual scans with transcranial Doppler ultrasound to identify children with SCD at high risk of stroke.

“What’s not changing is that there is silent injury that accumulates” and can cause lifelong harm, she said. “We want to protect our patients long term so that they can have a successful adult life, not just a successful childhood.”

Research done by one of Dr. Jordan’s colleagues at Vanderbilt, Michael R. DeBaun, MD, showed that regular blood-transfusion therapy significantly reduced the incidence of the recurrence of brain infarct in children with sickle cell anemia. (N Engl J Med. 2014 Aug 21;371[8]:699-710).


But the lessons from the work of Dr. DeBaun and his colleagues with their Silent Cerebral Infarct Multi-Center Clinical (SIT) Trial have not yet been fully adopted, Dr. Jordan said. That’s partly due to the inconvenience and cost of routinely administered blood transfusions to prevent silent cerebral infarcts, which, when used long term, cause side effects, she said.

Dr. Jordan said there’s growing interest in identifying patients at high risk for stroke and moving them toward stem cell transplant, though studies are ongoing. She urged greater attention to the high lifetime costs of strokes and other cerebrovascular complications, particularly in children and young adults.

While some of the brain infarcts are small and don’t result in focal weakness of the body, these “silent infarcts” do produce cognitive effects that reduce function, school performance, employment, and quality of life, she said.

“The injury to the brain is present, whether we can see it or not,” Dr. Jordan said. “In these precious patients, slow cognitive decline isn’t acceptable, frankly.”

Dr. Jordan reported having received funding from the American Heart Association and the National Institutes of Health for stroke prevention studies in SCD.

BETHESDA, MD. – The risk of brain damage from sickle cell disease (SCD) merits more attention, even with progress made in recent decades to prevent strokes, according to Lori Jordan, MD, PhD, of Vanderbilt University, Nashville, Tenn.

“Whether we can see it or not, the same injury we’ve been talking about in the kidney and the liver and other places is occurring in the brain” with sickle cell disease, Dr. Jordan said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

The concern about long-term brain injury reflects major shifts in SCD treatment. Improved medical care has transformed SCD from a disease that often resulted in an early death to more of a chronic condition, Dr. Jordan said, citing research that shows a survival rate of roughly 99% to age 18 years (Br J Haematol. 2016 Jun;173[6]:927-37).

One of the major success stories in SCD treatment also has been using primary prevention steps to cut the risk of overt stroke at least 10-fold, Dr. Jordan said. Primary prevention includes annual scans with transcranial Doppler ultrasound to identify children with SCD at high risk of stroke.

“What’s not changing is that there is silent injury that accumulates” and can cause lifelong harm, she said. “We want to protect our patients long term so that they can have a successful adult life, not just a successful childhood.”

Research done by one of Dr. Jordan’s colleagues at Vanderbilt, Michael R. DeBaun, MD, showed that regular blood-transfusion therapy significantly reduced the incidence of the recurrence of brain infarct in children with sickle cell anemia. (N Engl J Med. 2014 Aug 21;371[8]:699-710).


But the lessons from the work of Dr. DeBaun and his colleagues with their Silent Cerebral Infarct Multi-Center Clinical (SIT) Trial have not yet been fully adopted, Dr. Jordan said. That’s partly due to the inconvenience and cost of routinely administered blood transfusions to prevent silent cerebral infarcts, which, when used long term, cause side effects, she said.

Dr. Jordan said there’s growing interest in identifying patients at high risk for stroke and moving them toward stem cell transplant, though studies are ongoing. She urged greater attention to the high lifetime costs of strokes and other cerebrovascular complications, particularly in children and young adults.

While some of the brain infarcts are small and don’t result in focal weakness of the body, these “silent infarcts” do produce cognitive effects that reduce function, school performance, employment, and quality of life, she said.

“The injury to the brain is present, whether we can see it or not,” Dr. Jordan said. “In these precious patients, slow cognitive decline isn’t acceptable, frankly.”

Dr. Jordan reported having received funding from the American Heart Association and the National Institutes of Health for stroke prevention studies in SCD.

BETHESDA, MD. – The risk of brain damage from sickle cell disease (SCD) merits more attention, even with progress made in recent decades to prevent strokes, according to Lori Jordan, MD, PhD, of Vanderbilt University, Nashville, Tenn.

“Whether we can see it or not, the same injury we’ve been talking about in the kidney and the liver and other places is occurring in the brain” with sickle cell disease, Dr. Jordan said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

The concern about long-term brain injury reflects major shifts in SCD treatment. Improved medical care has transformed SCD from a disease that often resulted in an early death to more of a chronic condition, Dr. Jordan said, citing research that shows a survival rate of roughly 99% to age 18 years (Br J Haematol. 2016 Jun;173[6]:927-37).

One of the major success stories in SCD treatment also has been using primary prevention steps to cut the risk of overt stroke at least 10-fold, Dr. Jordan said. Primary prevention includes annual scans with transcranial Doppler ultrasound to identify children with SCD at high risk of stroke.

“What’s not changing is that there is silent injury that accumulates” and can cause lifelong harm, she said. “We want to protect our patients long term so that they can have a successful adult life, not just a successful childhood.”

Research done by one of Dr. Jordan’s colleagues at Vanderbilt, Michael R. DeBaun, MD, showed that regular blood-transfusion therapy significantly reduced the incidence of the recurrence of brain infarct in children with sickle cell anemia. (N Engl J Med. 2014 Aug 21;371[8]:699-710).


But the lessons from the work of Dr. DeBaun and his colleagues with their Silent Cerebral Infarct Multi-Center Clinical (SIT) Trial have not yet been fully adopted, Dr. Jordan said. That’s partly due to the inconvenience and cost of routinely administered blood transfusions to prevent silent cerebral infarcts, which, when used long term, cause side effects, she said.

Dr. Jordan said there’s growing interest in identifying patients at high risk for stroke and moving them toward stem cell transplant, though studies are ongoing. She urged greater attention to the high lifetime costs of strokes and other cerebrovascular complications, particularly in children and young adults.

While some of the brain infarcts are small and don’t result in focal weakness of the body, these “silent infarcts” do produce cognitive effects that reduce function, school performance, employment, and quality of life, she said.

“The injury to the brain is present, whether we can see it or not,” Dr. Jordan said. “In these precious patients, slow cognitive decline isn’t acceptable, frankly.”

Dr. Jordan reported having received funding from the American Heart Association and the National Institutes of Health for stroke prevention studies in SCD.

BETHESDA, MD – While there are experimental treatments such as sevuparin and gene therapy in testing for sickle cell disease (SCD), researchers said this field is lagging because of a lack of funding.

Dr_Microbe/Thinkstock

Yogen Saunthararajah, MD, of the Cleveland Clinic, described what he called a “paltry” landscape of new drugs for SCD at Sickle Cell in Focus, a conference held by the National Institutes of Health.

There are only four main approaches taken by drugs now in clinical testing for addressing the root causes of SCD, despite decades’ worth of research of the genetic and mechanistic underpinnings of this disease, he said.

“It’s pretty sad,” Dr. Saunthararajah said, referring to the quantity of efforts, not their quality.

Within days of his presentation at the conference, one of the drugs he highlighted had officially fallen out of contention. An Oct. 26 post on NIH’s Clinicaltrials.gov site said Incyte had terminated its phase 1 study of INCB059872 in SCD “due to a business decision” not to pursue this indication. Incyte confirmed that it dropped development of INCB059872 for SCD but will continue testing it for other indications, including acute myeloid leukemia (AML).

Dr. Saunthararajah said that work on another approach, using decitabine (Dacogen), for which he has done a phase 1 study, is “struggling,” because of the search for funding.

Two other approaches that Dr. Saunthararajah cited in his presentation appear to remain on track. These are gene therapy and the once-daily voxelotor treatment from Global Blood Therapeutics.

In the field of gene therapy, Sangamo Therapeutics and Sanofi’s Bioverativ in May said the Food and Drug Administration had cleared the way for them to start a phase 1/2 clinical trial for the BIVV003 product that they are developing together. This uses zinc finger nuclease (ZFN) gene-editing technology to modifying a short sequence of the BCL11A gene, with the aim of reactivating fetal hemoglobin.

Bluebird Bio’s LentiGlobin gene therapy has advanced as far as phase 3 for transfusion-dependent beta-thalassemia and phase 1/2 for SCD. In October, the European Medicines Agency accepted the company’s application for approval of LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta⁰/beta⁰ genotype. The company has not said when it expects to file with the FDA for approval of this treatment.

In the field of oral therapies, Global Blood Therapeutics has said it’s in discussions with the FDA about a potential accelerated approval of voxelotor. The tablet is meant to inhibit the underlying mechanism that causes sickling of red blood cells. In June, the company completed a planned review of early data from its phase 3 trial, known as the HOPE study.

“On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1,500-mg and 900-mg doses after 12 weeks of treatment versus placebo,” Global Blood Therapeutics said in an August regulatory filing with the Securities and Exchange Commission.

The company has said that voxelotor may meet the FDA’s standard for accelerated approval under Subpart H program.

In his presentation at the NIH conference, Tom Williams, MD, PhD, of KEMRI/Wellcome Trust Research Programme highlighted a recent review of experimental SCD treatments by Marilyn Jo Telen, MD, of Duke University, Durham, N.C. (Blood. 2016;127[7]:810-9).

Also among the drugs being tested for SCD is Modus Therapeutics’ sevuparin, which Dr. Williams described as being “a heparin-like molecule without the anticoagulant complications.”

The compound originated as a “passion project” of Mats Wahlgren, MD, PhD, of the Karolinska Institute, Stockholm, who developed it for the treatment of malaria, Dr. Williams said. The company that’s developing sevuparin, Modus Therapeutics, is moving it forward first as a treatment for SCD for commercial reasons, Dr. Williams said.

“They think it’s a better first target,” he said.

An ongoing study of sevuparin for painful crisis is expected to be completed in December, with data then expected to be released in the middle of 2019, Ellen K. Donnelly, PhD, chief executive officer of Modus Therapeutics, said in an interview. She cited a mix of scientific, medical and commercial reasons for her company’s decision to advance sevuparin in SCD.

“First, and most importantly, there is proof of clinical benefit of a similar molecule (the low-molecular-weight heparin called tinzaparin) in patients with sickle cell disease,” Dr. Donnelly said. “Unfortunately, there is a bleeding risk with tinzaparin that limits use of the agent for the treatment of sickle cell disease. Sevuparin does not have the bleeding risk and thus is a strong candidate for SCD.”

Dr. Donnelly also noted the emphasis that the FDA’s Division of Hematology Products has put on development of therapeutics for SCD as a reason for proceeding first with this indication. The agency and the American Society of Hematology in early October held a workshop of experts, physicians, patients, and industry collaborators focused on identifying new endpoints for clinical studies.

Still, she noted that commercial reasons did factor into this decision.

“[I]t is possible for a small company like Modus to take an asset all the way to market when developing a therapeutic for a rare disease given the need for fewer patients and smaller trials,” Dr. Donnelly wrote. “As you can see from www.clinicaltrials.gov, we were able to run our phase 2 study with a small number of sites. In addition, in SCD it is possible to get approval with only one or two confirmatory studies.”
 

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

BETHESDA, MD – While there are experimental treatments such as sevuparin and gene therapy in testing for sickle cell disease (SCD), researchers said this field is lagging because of a lack of funding.

Dr_Microbe/Thinkstock

Yogen Saunthararajah, MD, of the Cleveland Clinic, described what he called a “paltry” landscape of new drugs for SCD at Sickle Cell in Focus, a conference held by the National Institutes of Health.

There are only four main approaches taken by drugs now in clinical testing for addressing the root causes of SCD, despite decades’ worth of research of the genetic and mechanistic underpinnings of this disease, he said.

“It’s pretty sad,” Dr. Saunthararajah said, referring to the quantity of efforts, not their quality.

Within days of his presentation at the conference, one of the drugs he highlighted had officially fallen out of contention. An Oct. 26 post on NIH’s Clinicaltrials.gov site said Incyte had terminated its phase 1 study of INCB059872 in SCD “due to a business decision” not to pursue this indication. Incyte confirmed that it dropped development of INCB059872 for SCD but will continue testing it for other indications, including acute myeloid leukemia (AML).

Dr. Saunthararajah said that work on another approach, using decitabine (Dacogen), for which he has done a phase 1 study, is “struggling,” because of the search for funding.

Two other approaches that Dr. Saunthararajah cited in his presentation appear to remain on track. These are gene therapy and the once-daily voxelotor treatment from Global Blood Therapeutics.

In the field of gene therapy, Sangamo Therapeutics and Sanofi’s Bioverativ in May said the Food and Drug Administration had cleared the way for them to start a phase 1/2 clinical trial for the BIVV003 product that they are developing together. This uses zinc finger nuclease (ZFN) gene-editing technology to modifying a short sequence of the BCL11A gene, with the aim of reactivating fetal hemoglobin.

Bluebird Bio’s LentiGlobin gene therapy has advanced as far as phase 3 for transfusion-dependent beta-thalassemia and phase 1/2 for SCD. In October, the European Medicines Agency accepted the company’s application for approval of LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta⁰/beta⁰ genotype. The company has not said when it expects to file with the FDA for approval of this treatment.

In the field of oral therapies, Global Blood Therapeutics has said it’s in discussions with the FDA about a potential accelerated approval of voxelotor. The tablet is meant to inhibit the underlying mechanism that causes sickling of red blood cells. In June, the company completed a planned review of early data from its phase 3 trial, known as the HOPE study.

“On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1,500-mg and 900-mg doses after 12 weeks of treatment versus placebo,” Global Blood Therapeutics said in an August regulatory filing with the Securities and Exchange Commission.

The company has said that voxelotor may meet the FDA’s standard for accelerated approval under Subpart H program.

In his presentation at the NIH conference, Tom Williams, MD, PhD, of KEMRI/Wellcome Trust Research Programme highlighted a recent review of experimental SCD treatments by Marilyn Jo Telen, MD, of Duke University, Durham, N.C. (Blood. 2016;127[7]:810-9).

Also among the drugs being tested for SCD is Modus Therapeutics’ sevuparin, which Dr. Williams described as being “a heparin-like molecule without the anticoagulant complications.”

The compound originated as a “passion project” of Mats Wahlgren, MD, PhD, of the Karolinska Institute, Stockholm, who developed it for the treatment of malaria, Dr. Williams said. The company that’s developing sevuparin, Modus Therapeutics, is moving it forward first as a treatment for SCD for commercial reasons, Dr. Williams said.

“They think it’s a better first target,” he said.

An ongoing study of sevuparin for painful crisis is expected to be completed in December, with data then expected to be released in the middle of 2019, Ellen K. Donnelly, PhD, chief executive officer of Modus Therapeutics, said in an interview. She cited a mix of scientific, medical and commercial reasons for her company’s decision to advance sevuparin in SCD.

“First, and most importantly, there is proof of clinical benefit of a similar molecule (the low-molecular-weight heparin called tinzaparin) in patients with sickle cell disease,” Dr. Donnelly said. “Unfortunately, there is a bleeding risk with tinzaparin that limits use of the agent for the treatment of sickle cell disease. Sevuparin does not have the bleeding risk and thus is a strong candidate for SCD.”

Dr. Donnelly also noted the emphasis that the FDA’s Division of Hematology Products has put on development of therapeutics for SCD as a reason for proceeding first with this indication. The agency and the American Society of Hematology in early October held a workshop of experts, physicians, patients, and industry collaborators focused on identifying new endpoints for clinical studies.

Still, she noted that commercial reasons did factor into this decision.

“[I]t is possible for a small company like Modus to take an asset all the way to market when developing a therapeutic for a rare disease given the need for fewer patients and smaller trials,” Dr. Donnelly wrote. “As you can see from www.clinicaltrials.gov, we were able to run our phase 2 study with a small number of sites. In addition, in SCD it is possible to get approval with only one or two confirmatory studies.”
 

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

BETHESDA, MD – While there are experimental treatments such as sevuparin and gene therapy in testing for sickle cell disease (SCD), researchers said this field is lagging because of a lack of funding.

Dr_Microbe/Thinkstock

Yogen Saunthararajah, MD, of the Cleveland Clinic, described what he called a “paltry” landscape of new drugs for SCD at Sickle Cell in Focus, a conference held by the National Institutes of Health.

There are only four main approaches taken by drugs now in clinical testing for addressing the root causes of SCD, despite decades’ worth of research of the genetic and mechanistic underpinnings of this disease, he said.

“It’s pretty sad,” Dr. Saunthararajah said, referring to the quantity of efforts, not their quality.

Within days of his presentation at the conference, one of the drugs he highlighted had officially fallen out of contention. An Oct. 26 post on NIH’s Clinicaltrials.gov site said Incyte had terminated its phase 1 study of INCB059872 in SCD “due to a business decision” not to pursue this indication. Incyte confirmed that it dropped development of INCB059872 for SCD but will continue testing it for other indications, including acute myeloid leukemia (AML).

Dr. Saunthararajah said that work on another approach, using decitabine (Dacogen), for which he has done a phase 1 study, is “struggling,” because of the search for funding.

Two other approaches that Dr. Saunthararajah cited in his presentation appear to remain on track. These are gene therapy and the once-daily voxelotor treatment from Global Blood Therapeutics.

In the field of gene therapy, Sangamo Therapeutics and Sanofi’s Bioverativ in May said the Food and Drug Administration had cleared the way for them to start a phase 1/2 clinical trial for the BIVV003 product that they are developing together. This uses zinc finger nuclease (ZFN) gene-editing technology to modifying a short sequence of the BCL11A gene, with the aim of reactivating fetal hemoglobin.

Bluebird Bio’s LentiGlobin gene therapy has advanced as far as phase 3 for transfusion-dependent beta-thalassemia and phase 1/2 for SCD. In October, the European Medicines Agency accepted the company’s application for approval of LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta⁰/beta⁰ genotype. The company has not said when it expects to file with the FDA for approval of this treatment.

In the field of oral therapies, Global Blood Therapeutics has said it’s in discussions with the FDA about a potential accelerated approval of voxelotor. The tablet is meant to inhibit the underlying mechanism that causes sickling of red blood cells. In June, the company completed a planned review of early data from its phase 3 trial, known as the HOPE study.

“On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1,500-mg and 900-mg doses after 12 weeks of treatment versus placebo,” Global Blood Therapeutics said in an August regulatory filing with the Securities and Exchange Commission.

The company has said that voxelotor may meet the FDA’s standard for accelerated approval under Subpart H program.

In his presentation at the NIH conference, Tom Williams, MD, PhD, of KEMRI/Wellcome Trust Research Programme highlighted a recent review of experimental SCD treatments by Marilyn Jo Telen, MD, of Duke University, Durham, N.C. (Blood. 2016;127[7]:810-9).

Also among the drugs being tested for SCD is Modus Therapeutics’ sevuparin, which Dr. Williams described as being “a heparin-like molecule without the anticoagulant complications.”

The compound originated as a “passion project” of Mats Wahlgren, MD, PhD, of the Karolinska Institute, Stockholm, who developed it for the treatment of malaria, Dr. Williams said. The company that’s developing sevuparin, Modus Therapeutics, is moving it forward first as a treatment for SCD for commercial reasons, Dr. Williams said.

“They think it’s a better first target,” he said.

An ongoing study of sevuparin for painful crisis is expected to be completed in December, with data then expected to be released in the middle of 2019, Ellen K. Donnelly, PhD, chief executive officer of Modus Therapeutics, said in an interview. She cited a mix of scientific, medical and commercial reasons for her company’s decision to advance sevuparin in SCD.

“First, and most importantly, there is proof of clinical benefit of a similar molecule (the low-molecular-weight heparin called tinzaparin) in patients with sickle cell disease,” Dr. Donnelly said. “Unfortunately, there is a bleeding risk with tinzaparin that limits use of the agent for the treatment of sickle cell disease. Sevuparin does not have the bleeding risk and thus is a strong candidate for SCD.”

Dr. Donnelly also noted the emphasis that the FDA’s Division of Hematology Products has put on development of therapeutics for SCD as a reason for proceeding first with this indication. The agency and the American Society of Hematology in early October held a workshop of experts, physicians, patients, and industry collaborators focused on identifying new endpoints for clinical studies.

Still, she noted that commercial reasons did factor into this decision.

“[I]t is possible for a small company like Modus to take an asset all the way to market when developing a therapeutic for a rare disease given the need for fewer patients and smaller trials,” Dr. Donnelly wrote. “As you can see from www.clinicaltrials.gov, we were able to run our phase 2 study with a small number of sites. In addition, in SCD it is possible to get approval with only one or two confirmatory studies.”
 

Financial interests

Other drugs in testing for SCD include rivipansel from GlycoMimetics, for which Pfizer is leading development. The sponsors expect to complete the so-called RESET trial by 2019, according to the clinicaltrials.gov. In this study, which is intended to enroll 350 participants, patients who have vaso-occlusive crises are randomly selected for treatment with either rivipansel or placebo.

Speaking during a question-and-answer session at the NIH conference, Robert Swift, PhD, said there’s a need for inexpensive oral drugs to treat SCD. Many other options will remain beyond the finances of people living in poor countries, he said.

“We need to focus not only on the root cause, but on something that is oral and inexpensive to solve the greater sickle cell problem,” Dr. Swift said.

Large drugmakers already have hospital-based sales forces, making SCD drugs administered in this setting attractive to them, he said.

“This is partly about where money is. The drug companies are going where the money is. It’s not oral drugs to treat everybody, it’s something else,” Dr. Swift said. “So someone else is going to have to fund the basic research” into treatments that could be more broadly used.

Dr. Swift said in an interview that he has received NIH funding for developing SCD-101, an oral drug, for which a placebo-controlled crossover study is underway.

Presenters at the NIH conference, including Dr. Saunthararajah, expressed frustration about what they see as relatively little work being done on SCD despite decades of knowledge about the root causes. Like Dr. Swift, he criticized the approach taken in selecting which treatments advance in this field.

“It’s not being driven by what is the most cost effective, what the patients need the most,” Dr. Saunthararajah said. “It’s driven by what will make the most money, not just for [the] drug company, but also for the hospital and also for the physicians.”

Dr. Saunthararajah reported having patents and patent applications around decitabine/tetrahydrouridine, 5-azacytidine/tetrahydrouridine, and differentiation therapy for oncology. He has also been a consultant for EpiDestiny, Novo Nordisk, and Takeda Oncology. Dr. Williams reported having no relevant financial disclosures. Dr. Swift is a managing member of Invenux and reported equity in Mast Therapeutics and SCD Development.

This article was updated on 11/9/2018.

BETHESDA, MD. – Obtaining an extended red cell antigen profile before a patient’s first transfusion can help improve outcomes and avoid complications in highly transfused patients, according to one researcher.

“We strongly feel that you should get an extended red cell type on the first encounter” for patients facing long-term transfusion support, Connie Westhoff, PhD, of the New York Blood Center, said at Sickle Cell in Focus, a conference held by the National Institutes of Health. “This can be a one-time test. It doesn’t have to be repeated.”

She also stressed the importance of ensuring that this information travels with patients, who may be seen at various hospitals. “One of the challenges here is making this part of the patient’s electronic medical record.”

Alloimmunization has been a major concern for chronically transfused patients, so there’s a real advantage to knowing a patient’s extended red cell antigen profile before the patient develops alloantibodies following transfusion, Dr. Westhoff said. Hemolytic transfusion reactions can sometimes destroy patients’ own RBCs in addition to the transfused RBCs. Having the patient’s profile to identify the potential cause of the incompatibility and guide transfusion support in an emergency can be lifesaving.

The implementation of genotyping by DNA-based methods has brought down the cost of this screening, making it no longer a barrier in care.


Dr. Westhoff cited a study she and her colleagues published on a study typing patients who have sickle cell disease (Transfusion. 2015 Jun;55[6 Pt 2]:1388-93). In that paper, they found that DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens, compared with hemagglutination methods. This led to its implementation as the primary method for extended RBC typing for patients with sickle cell disease at the Children’s Hospital of Philadelphia.

About 65%-70% of antibodies drop to levels that are not detectable by routine assays, further demonstrating the need for DNA-based methods. The drugs used to dampen the immune response in many of these patients may also hinder or impact detection of antibodies that remain at levels that continue to cause in vivo hemolysis, Dr. Westhoff said in an interview.

For patients with sickle cell disease in many Western countries, antigen matching for CEK, at a minimum, is routine. The United States is now moving in this direction.

“Modern transfusion practice is moving to knowing what antigens the patient is at risk to become immunized against. ... What antigens does the patient lack and what antibodies could the patient make,” Dr. Westhoff said in an interview. “It’s a major advantage in your transfusion service to expedite work-ups and patient care by having that extended antigen profile.”

Dr. Westhoff reported no relevant financial disclosures.

BETHESDA, MD. – Obtaining an extended red cell antigen profile before a patient’s first transfusion can help improve outcomes and avoid complications in highly transfused patients, according to one researcher.

“We strongly feel that you should get an extended red cell type on the first encounter” for patients facing long-term transfusion support, Connie Westhoff, PhD, of the New York Blood Center, said at Sickle Cell in Focus, a conference held by the National Institutes of Health. “This can be a one-time test. It doesn’t have to be repeated.”

She also stressed the importance of ensuring that this information travels with patients, who may be seen at various hospitals. “One of the challenges here is making this part of the patient’s electronic medical record.”

Alloimmunization has been a major concern for chronically transfused patients, so there’s a real advantage to knowing a patient’s extended red cell antigen profile before the patient develops alloantibodies following transfusion, Dr. Westhoff said. Hemolytic transfusion reactions can sometimes destroy patients’ own RBCs in addition to the transfused RBCs. Having the patient’s profile to identify the potential cause of the incompatibility and guide transfusion support in an emergency can be lifesaving.

The implementation of genotyping by DNA-based methods has brought down the cost of this screening, making it no longer a barrier in care.


Dr. Westhoff cited a study she and her colleagues published on a study typing patients who have sickle cell disease (Transfusion. 2015 Jun;55[6 Pt 2]:1388-93). In that paper, they found that DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens, compared with hemagglutination methods. This led to its implementation as the primary method for extended RBC typing for patients with sickle cell disease at the Children’s Hospital of Philadelphia.

About 65%-70% of antibodies drop to levels that are not detectable by routine assays, further demonstrating the need for DNA-based methods. The drugs used to dampen the immune response in many of these patients may also hinder or impact detection of antibodies that remain at levels that continue to cause in vivo hemolysis, Dr. Westhoff said in an interview.

For patients with sickle cell disease in many Western countries, antigen matching for CEK, at a minimum, is routine. The United States is now moving in this direction.

“Modern transfusion practice is moving to knowing what antigens the patient is at risk to become immunized against. ... What antigens does the patient lack and what antibodies could the patient make,” Dr. Westhoff said in an interview. “It’s a major advantage in your transfusion service to expedite work-ups and patient care by having that extended antigen profile.”

Dr. Westhoff reported no relevant financial disclosures.

BETHESDA, MD. – Obtaining an extended red cell antigen profile before a patient’s first transfusion can help improve outcomes and avoid complications in highly transfused patients, according to one researcher.

“We strongly feel that you should get an extended red cell type on the first encounter” for patients facing long-term transfusion support, Connie Westhoff, PhD, of the New York Blood Center, said at Sickle Cell in Focus, a conference held by the National Institutes of Health. “This can be a one-time test. It doesn’t have to be repeated.”

She also stressed the importance of ensuring that this information travels with patients, who may be seen at various hospitals. “One of the challenges here is making this part of the patient’s electronic medical record.”

Alloimmunization has been a major concern for chronically transfused patients, so there’s a real advantage to knowing a patient’s extended red cell antigen profile before the patient develops alloantibodies following transfusion, Dr. Westhoff said. Hemolytic transfusion reactions can sometimes destroy patients’ own RBCs in addition to the transfused RBCs. Having the patient’s profile to identify the potential cause of the incompatibility and guide transfusion support in an emergency can be lifesaving.

The implementation of genotyping by DNA-based methods has brought down the cost of this screening, making it no longer a barrier in care.


Dr. Westhoff cited a study she and her colleagues published on a study typing patients who have sickle cell disease (Transfusion. 2015 Jun;55[6 Pt 2]:1388-93). In that paper, they found that DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens, compared with hemagglutination methods. This led to its implementation as the primary method for extended RBC typing for patients with sickle cell disease at the Children’s Hospital of Philadelphia.

About 65%-70% of antibodies drop to levels that are not detectable by routine assays, further demonstrating the need for DNA-based methods. The drugs used to dampen the immune response in many of these patients may also hinder or impact detection of antibodies that remain at levels that continue to cause in vivo hemolysis, Dr. Westhoff said in an interview.

For patients with sickle cell disease in many Western countries, antigen matching for CEK, at a minimum, is routine. The United States is now moving in this direction.

“Modern transfusion practice is moving to knowing what antigens the patient is at risk to become immunized against. ... What antigens does the patient lack and what antibodies could the patient make,” Dr. Westhoff said in an interview. “It’s a major advantage in your transfusion service to expedite work-ups and patient care by having that extended antigen profile.”

Dr. Westhoff reported no relevant financial disclosures.

BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.

bubaone/DigitalVision Vectors

People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.

“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.

People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.

Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.

She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).

It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.

“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.

One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.

“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”

Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).

Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.

“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.

Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.

BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.

bubaone/DigitalVision Vectors

People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.

“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.

People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.

Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.

She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).

It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.

“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.

One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.

“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”

Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).

Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.

“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.

Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.

BETHESDA, MD. – With many people surviving well into adulthood with sickle cell disease (SCD) because of advances in treatment, there’s a strong need for more primary care to address chronic conditions, such as obesity and the complications of the blood disorder, researchers said.

bubaone/DigitalVision Vectors

People who have lived for decades with SCD may be at higher risk for renal disease while still needing the same routine vaccinations and screening for colon, prostate, and lung cancer that the general population receives, Sophie Lanzkron, MD, of Johns Hopkins University, Baltimore, said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

And obesity is an additional a concern in treating people with SCD, Dr. Lanzkron said.

“It is really hard to have a conversation for 20 minutes with a patient about pain, talk about what you’re going to do about their sickle cell disease, and then address all of” their routine health needs, she said.

People with SCD seem less likely to get renal transplants, but those with end-stage kidney disease should be encouraged to be evaluated for them, Dr. Lanzkron advised.

Older data had suggested that patients with SCD who underwent renal transplant didn’t do as well as everyone else who underwent the procedure, but new data have changed that approach. “There’s some additional data in the modern era suggesting that the outcomes for people who undergo transplant with sickle cell disease are the same as for those who undergo it with diabetes,” Dr. Lanzkron said.

She highlighted one newer study in which the kidney transplant survival rate was 73.1% among individuals with SCD, compared with 74.1% for those with diabetes (Nephrol Dial Transplant. 2013 Apr;28[4]:1039-46).

It’s unclear what the average life expectancy is at this time for someone with SCD, Dr. Lanzkron said. Research looking at death certificate data suggests a median age of death in the mid-40s, but there are limitations to this work given it may exclude many older people with SCD, she said.

“We’re hopeful that people are living into their 50s and 60s, but we don’t have a lot of great data,” she said.

One of the organizers of the NIH conference said she hoped that Dr. Lanzkron’s presentation would draw attention to the need for primary care for people with SCD. Maintaining a healthy lifestyle is particularly important for this group because they likely have had complications from the disease, as well as issues seen with normal aging, Swee Lay Thein, MBBS, of the National Heart, Lung, and Blood Institute, said in an interview.

“This is a key message for many patients with sickle cell disease,” Dr. Thein said. “It’s important to hook up with a primary care physician.”

Dr. Thein cited a recent paper, which reported on four people who had lived into their 80s with sickle cell disease. The paper said their longevity was aided by factors such as being nonsmokers, abstaining from alcohol or drinking it only on occasionally, and maintaining a normal body mass index (Blood. 2016 Nov 10;128[19]:2367-9).

Additionally, the patients had close ties with relatives. The paper said that one patient was married with a helpful husband. Others in this octogenarian set had maintained close ties with their children.

“A common factor for all of the four patients in their 80s was that they had a healthy lifestyle and very strong family support,” Dr. Thein said.

Dr. Lanzkron has been an investigator for trials sponsored by Pfizer, Global Blood Therapeutics, and Ironwood.

BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.

Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).

Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.

“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.

Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.

The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.

In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.

The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.

Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”

Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.

“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.

She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”

The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.

Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.

BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.

Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).

Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.

“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.

Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.

The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.

In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.

The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.

Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”

Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.

“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.

She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”

The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.

Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.

BETHESDA, MD. – Experimental gene therapies for sickle cell disease and thalassemia appear to be advancing, with BCL11A among the most promising targets in this field, researchers said at Sickle Cell in Focus, a conference held by the National Institutes of Health.

Several highly anticipated presentations on the topic are expected for December meeting of the American Society of Hematology.

Alexis A. Thompson, MD, of Northwestern University, Chicago, reviewed highlights from a study in which the majority of patients given a gene therapy for transfusion dependent beta-thalassemia didn’t need subsequent blood transfusions. The New England Journal of Medicine in April published the results of this work done with Bluebird Bio’s LentiGlobin gene therapy (N Engl J Med. 2018; 378:1479-93).

Of the 22 patients in this trial, 15 have become transfusion independent, Dr. Thompson said in her presentation. Those patients that did not have this positive outcome still appear to have been helped by the gene therapy, she said. They had a median of 60% reduction in their transfusion volumes and nearly 60% in their number of transfusions.

“Whether it was transfusion independence or reduction in their transfusion volume or number, the vast majority of individuals in this first large-scale study had clinical benefit” from the therapy, said Dr. Thompson, who was the lead author of the study.

Dr. Thompson, the current president of the American Society of Hematology (ASH), said she’s looking forward to presentations on some of the most advanced gene therapies for sickle cell disease and thalassemia at the group’s annual meeting in December. The ASH presentations include those of John F. Tisdale, MD, who will report the latest data on LentiGlobin gene therapy in sickle cell disease, and Punam Malik, MD, of Cincinnati Children’s Hospital, who has developed a gamma globin lentivirus vector. There also will be a first readout on a particularly novel approach taken by researchers at Boston Children’s Hospital, led by David Williams, MD.

The development of CRISPR-Cas9 “has really opened up the field” of gene therapy, aiding researchers at Boston Children’s in their efforts to develop a treatment to maintain fetal hemoglobin production, Daniel E. Bauer, MD, PhD, of Boston Children’s Hospital, said during his presentation at the NIH conference.

In a related clinical trial, using lentiviral gene therapy rather than gene editing, researchers at Boston Children’s began an open-label, nonrandomized, single-center pilot study that involves a single infusion of autologous bone marrow derived CD34+ HSC cells transduced by a vector containing a short-hairpin segment of RNA targeting the gene BCL11A.

The study has a maximum accrual of seven evaluable patients, according to the NIH’s clinical trials website. The protocol is similar to bone marrow transplant, in that native blood stem cells are eliminated by myeloablative conditioning therapy. In this gene therapy, the patient’s own blood stem cells are then infused after the new genetic material has been added to counter the normal BCL11A off switch.

Swee Lay Thein, MBBS, an organizer of the NIH’s sickle cell conference, said in an interview that the “gene therapy side is really looking very optimistic.”

Dr. Thein, a senior investigator for sickle-cell genetics at the National Heart, Lung, and Blood Institute, earlier in her career discovered segments of DNA, including the BCL11A gene. She said that gaining greater understanding about genomic variation might someday aid in determining which people need more intense intervention for their sickle cell disease.

“You would be able to predict who will have more severe disease; we could monitor them more closely and perhaps even advocate for gene therapy or bone marrow transplant before complications have occurred rather than waiting for them to occur,” Dr. Thein said.

She referred to this as her “dream” for care of people with sickle cell disease. “This is still far off in the horizon.”

The NHLBI in September 2018 launched its Cure Sickle Cell Initiative. The agency estimates that it spends about $100 million on sickle cell disease research each year. The inherited blood disorder affects about 100,000 people in the United States and 20 million individuals worldwide. In sickle cell disease, a single genetic mutation causes red blood cells to form abnormal, sickle shapes that can clog the blood vessels and deprive cells of oxygen.

Dr. Thompson reported research funding and consulting agreements with Biomarin, Bluebird Bio, Celgene, Novartis, and Shire. Dr. Bauer reported patents related to BCL11A enhancer editing, consulting agreements with Merck and Pfizer, and research support from Bioverativ.

ROCKVILLE, MD. – Using a digital application could allow more adults to try cognitive-behavioral therapy (CBT) to combat insomnia and expand access to a technique that’s been shown to ward off depression, a researcher said at a National Institute of Mental Health conference on mental health services research.

ROCKVILLE, MD. – Using a digital application could allow more adults to try cognitive-behavioral therapy (CBT) to combat insomnia and expand access to a technique that’s been shown to ward off depression, a researcher said at a National Institute of Mental Health conference on mental health services research.

ROCKVILLE, MD. – Using a digital application could allow more adults to try cognitive-behavioral therapy (CBT) to combat insomnia and expand access to a technique that’s been shown to ward off depression, a researcher said at a National Institute of Mental Health conference on mental health services research.

ROCKVILLE, MD. – The problem of medical comorbidities in people with serious mental illness (SMI) persists and must be addressed, researchers said at a National Institute on Mental Health conference on mental health services research. Part of that effort, they said, is a more careful consideration of risks tied to the off-label use of second-generation antipsychotics.

Courtesy Dr. Joshua Breslau

Risks tied to antipsychotics

People with SMI face cardiovascular risks not only from unhealthy behaviors but also from the medications used to treat their psychiatric conditions. The American Diabetes Association and American Psychiatric Association in 2004 released a consensus statement on the impact of antipsychotics such as clozapine, olanzapine, and risperidone on obesity and diabetes. It included guidelines for monitoring the metabolic status of patients both at baseline and after initiating treatment, including checking body mass index, waist circumference, blood pressure, fasting glucose, and fasting lipids.

Yet, substantial evidence suggests that the medical community still has not paid enough attention to the health risks of those medications, said Alisa Busch, MD, of Harvard Medical School, Boston.

“A slew of research has shown since then that we have done a very poor job in adhering to those monitoring guidelines,” Dr. Busch said.

A fellow panelist, Marcela Horvitz-Lennon, MD, MPH, of the Rand Corp., presented results from her study showing continued common use of second-generation antipsychotics for off-label use for treatment of anxiety, posttraumatic stress disorder, and dementia in people of all ages.

Consistent with previous research, Dr. Horvitz-Lennon and her colleagues found that off-label use of second-generation antipsychotics was common during 2008-2012 in the four states they studied. They looked at available data from fee-for-service Medicare, Medicaid, and dually (Medicaid-Medicare) covered adult beneficiaries in California, Georgia, Mississippi, and Oklahoma.

Throughout the study period, California had the highest rate of fee-for-service beneficiaries whose SGA use was consistently off label (44.6%). Georgia had the lowest rate of always off-label use (35.1%), while Mississippi (42%) and Oklahoma (36.3%) fell somewhere in the middle.

When second-generation antipsychotics have approved uses such as schizophrenia, the known profile of a medication gives some assurance that the benefit of the medications will exceed the risk for that patient, she said. “When the medication is used off label, the implication is that there is no good evidence that the benefits are there,” Dr. Horvitz-Lennon said. “Hence, the potential for harm is most likely exceeding that likely or unlikely benefit.”

Dr. Daumit, Dr. Breslau, and Dr. Horvitz-Lennon said they had no financial disclosures.

ROCKVILLE, MD. – The problem of medical comorbidities in people with serious mental illness (SMI) persists and must be addressed, researchers said at a National Institute on Mental Health conference on mental health services research. Part of that effort, they said, is a more careful consideration of risks tied to the off-label use of second-generation antipsychotics.

Courtesy Dr. Joshua Breslau

Risks tied to antipsychotics

People with SMI face cardiovascular risks not only from unhealthy behaviors but also from the medications used to treat their psychiatric conditions. The American Diabetes Association and American Psychiatric Association in 2004 released a consensus statement on the impact of antipsychotics such as clozapine, olanzapine, and risperidone on obesity and diabetes. It included guidelines for monitoring the metabolic status of patients both at baseline and after initiating treatment, including checking body mass index, waist circumference, blood pressure, fasting glucose, and fasting lipids.

Yet, substantial evidence suggests that the medical community still has not paid enough attention to the health risks of those medications, said Alisa Busch, MD, of Harvard Medical School, Boston.

“A slew of research has shown since then that we have done a very poor job in adhering to those monitoring guidelines,” Dr. Busch said.

A fellow panelist, Marcela Horvitz-Lennon, MD, MPH, of the Rand Corp., presented results from her study showing continued common use of second-generation antipsychotics for off-label use for treatment of anxiety, posttraumatic stress disorder, and dementia in people of all ages.

Consistent with previous research, Dr. Horvitz-Lennon and her colleagues found that off-label use of second-generation antipsychotics was common during 2008-2012 in the four states they studied. They looked at available data from fee-for-service Medicare, Medicaid, and dually (Medicaid-Medicare) covered adult beneficiaries in California, Georgia, Mississippi, and Oklahoma.

Throughout the study period, California had the highest rate of fee-for-service beneficiaries whose SGA use was consistently off label (44.6%). Georgia had the lowest rate of always off-label use (35.1%), while Mississippi (42%) and Oklahoma (36.3%) fell somewhere in the middle.

When second-generation antipsychotics have approved uses such as schizophrenia, the known profile of a medication gives some assurance that the benefit of the medications will exceed the risk for that patient, she said. “When the medication is used off label, the implication is that there is no good evidence that the benefits are there,” Dr. Horvitz-Lennon said. “Hence, the potential for harm is most likely exceeding that likely or unlikely benefit.”

Dr. Daumit, Dr. Breslau, and Dr. Horvitz-Lennon said they had no financial disclosures.

ROCKVILLE, MD. – The problem of medical comorbidities in people with serious mental illness (SMI) persists and must be addressed, researchers said at a National Institute on Mental Health conference on mental health services research. Part of that effort, they said, is a more careful consideration of risks tied to the off-label use of second-generation antipsychotics.

Courtesy Dr. Joshua Breslau

Risks tied to antipsychotics

People with SMI face cardiovascular risks not only from unhealthy behaviors but also from the medications used to treat their psychiatric conditions. The American Diabetes Association and American Psychiatric Association in 2004 released a consensus statement on the impact of antipsychotics such as clozapine, olanzapine, and risperidone on obesity and diabetes. It included guidelines for monitoring the metabolic status of patients both at baseline and after initiating treatment, including checking body mass index, waist circumference, blood pressure, fasting glucose, and fasting lipids.

Yet, substantial evidence suggests that the medical community still has not paid enough attention to the health risks of those medications, said Alisa Busch, MD, of Harvard Medical School, Boston.

“A slew of research has shown since then that we have done a very poor job in adhering to those monitoring guidelines,” Dr. Busch said.

A fellow panelist, Marcela Horvitz-Lennon, MD, MPH, of the Rand Corp., presented results from her study showing continued common use of second-generation antipsychotics for off-label use for treatment of anxiety, posttraumatic stress disorder, and dementia in people of all ages.

Consistent with previous research, Dr. Horvitz-Lennon and her colleagues found that off-label use of second-generation antipsychotics was common during 2008-2012 in the four states they studied. They looked at available data from fee-for-service Medicare, Medicaid, and dually (Medicaid-Medicare) covered adult beneficiaries in California, Georgia, Mississippi, and Oklahoma.

Throughout the study period, California had the highest rate of fee-for-service beneficiaries whose SGA use was consistently off label (44.6%). Georgia had the lowest rate of always off-label use (35.1%), while Mississippi (42%) and Oklahoma (36.3%) fell somewhere in the middle.

When second-generation antipsychotics have approved uses such as schizophrenia, the known profile of a medication gives some assurance that the benefit of the medications will exceed the risk for that patient, she said. “When the medication is used off label, the implication is that there is no good evidence that the benefits are there,” Dr. Horvitz-Lennon said. “Hence, the potential for harm is most likely exceeding that likely or unlikely benefit.”

Dr. Daumit, Dr. Breslau, and Dr. Horvitz-Lennon said they had no financial disclosures.

ROCKVILLE, MD. – People working in the mental health field are more likely to disclose their past or present treatment for psychosis than are professionals in other fields, a researcher said at a National Institute of Mental Health conference on mental health services research.

The researcher, Nev Jones, PhD, presented the findings of a small survey she conducted in 2014 and 2015 of adults with current or past experiences of psychotic disorder who described themselves as having had or having a successful career. The research was not conducted for publication purposes but as part of an effort to develop tools for students with psychosis as they continued in higher education, said Dr. Jones, of the department of mental health law and policy at the University of South Florida, Tampa.

One of those tools was a closed program that was akin to Facebook; people with early psychosis could use this to “look at successful adults across a wide range of careers and how they had navigated accommodations, disclosure, education as well as vocational choice,” she said.

Dr. Jones did not ask participants about their gender and race, but she did query them on highest degree earned. The poll was disseminated with the assistance of the National Alliance on Mental Illness and that of Stanford (Calif.) University, where Dr. Jones was a postdoctoral fellow. Of the sample presented, 33% had a masters degree (MSW, MBA), and 15% had a doctoral level degree (JD, MD, PhD).

People who worked in fields outside of mental health care were far less likely to have revealed their conditions to colleagues or employers, with 14 of 67 participants having made no disclosure. Of 14 who had made no disclosure, 12 were in fields such as banking, economics, secondary education, nursing, pediatrics, and computer programming.

Dr. Jones said she received several calls from students and staff at Stanford who were unwilling to fill out the survey.

“They were very concerned about the risks of inadvertent disclosure, even though it was anonymous, because they had unique, potentially identifiable career paths that they could not lay out in their responses without the fear that that would disclose [identify] them,” Dr. Jones said.

An additional 17 of the 67 participants made what Dr. Jones termed “selective disclosures,” such as telling a coworker who was considered a friend or a supportive boss. The majority of the respondents to Jones’s survey – 36 of the 67 participants – were open about their conditions. All but one of the respondents in this broad-disclosure group worked in mental health fields.

Dr. Jones described the broad-disclosure designation as “meaning that there is nobody in their life who doesn’t know.”

“They’re out professionally. They’ve published a book. They speak,” Dr. Jones said. “If you Google them on the Internet, you would quickly learn that they had a psychiatric disability or psychosis.”

Dr. Jones herself falls into that camp. She’s told media outlets, including the online newspaper MinnPost, about her own experience being diagnosed with schizophrenia while a PhD student. The online magazine Pacific Standard ran a full-length feature about her return to academia.

About 100,000 adolescents and young adults in the United States experience first-episode psychosis each year, and the peak onset hits between 15 and 25 years of age, according to the NIMH. About a decade ago, the NIMH launched its Recovery After an Initial Schizophrenia Episode (RAISE) initiative to examine use of coordinated specialty care treatments for people who were experiencing a first episode of psychosis. Congress in 2014 moved to provide a stream of federal funding for those kinds of efforts.

“We’re going to be soon starting to discharge, on an annual basis, potentially tens of thousands of young people from these specialized early intervention programs,” Dr. Jones said. “So it becomes really pressing to understand what’s happening to them in the context of reintegration.”

Another presenter at the panel, Marjorie L. Baldwin, PhD, of Arizona State University, Tempe, is an economist who has published a book based, in part, on her son’s struggles, “Beyond Schizophrenia: Living and Working With a Serious Mental Illness” (Lanham, Md.: Rowman & Littlefield Publishers, 2016).

She presented findings from a pilot study for a larger project looking at the issue of disclosures of serious mental illness in the workplace. She and her colleague in this work, Steven C. Marcus, PhD, of the University of Pennsylvania, Philadelphia, separately spoke about the difficulties in securing funding for the project, including five failed R01 grant applications.

“The 6th time was the charm with NIH,” Dr. Baldwin said.

An initial hurdle was finding a cost-effective way to identify workers with serious mental illness who hold or have held what she termed “competitive jobs,” which Dr. Baldwin described as those that paid at least minimum wage and are not subsidized for people with disabilities.

“You cannot do this kind of a study with random dialing because it would be way too expensive,” she said. “Schizophrenia and serious mental illnesses are not rare, but they are fairly uncommon.”

Several years ago, though, she learned of a long-running health survey into which she could “piggyback” questions on mental health status. She presented results of a pilot study with about 230 people with serious mental illness who had held or had competitive jobs. Of this group, 52% had left their most recent job for reasons other than mental illness, while those conditions had caused an additional 21% to leave. But Dr. Baldwin and her colleagues found 27% still working.

Like Dr. Jones, Dr. Baldwin said some of those workers were in professional fields, such as accounting, law, education; others worked in the service and construction industries.

“Contrary to the stereotypes, people with serious mental illness whose symptoms are reasonably well controlled can work, and many are capable of supporting themselves in mainstream competitive jobs,” Dr. Baldwin said.

Dr. Jones had no disclosures tied to her survey. Dr. Baldwin and Dr. Marcus had no disclosures other than the NIH R01 grant.

ROCKVILLE, MD. – People working in the mental health field are more likely to disclose their past or present treatment for psychosis than are professionals in other fields, a researcher said at a National Institute of Mental Health conference on mental health services research.

The researcher, Nev Jones, PhD, presented the findings of a small survey she conducted in 2014 and 2015 of adults with current or past experiences of psychotic disorder who described themselves as having had or having a successful career. The research was not conducted for publication purposes but as part of an effort to develop tools for students with psychosis as they continued in higher education, said Dr. Jones, of the department of mental health law and policy at the University of South Florida, Tampa.

One of those tools was a closed program that was akin to Facebook; people with early psychosis could use this to “look at successful adults across a wide range of careers and how they had navigated accommodations, disclosure, education as well as vocational choice,” she said.

Dr. Jones did not ask participants about their gender and race, but she did query them on highest degree earned. The poll was disseminated with the assistance of the National Alliance on Mental Illness and that of Stanford (Calif.) University, where Dr. Jones was a postdoctoral fellow. Of the sample presented, 33% had a masters degree (MSW, MBA), and 15% had a doctoral level degree (JD, MD, PhD).

People who worked in fields outside of mental health care were far less likely to have revealed their conditions to colleagues or employers, with 14 of 67 participants having made no disclosure. Of 14 who had made no disclosure, 12 were in fields such as banking, economics, secondary education, nursing, pediatrics, and computer programming.

Dr. Jones said she received several calls from students and staff at Stanford who were unwilling to fill out the survey.

“They were very concerned about the risks of inadvertent disclosure, even though it was anonymous, because they had unique, potentially identifiable career paths that they could not lay out in their responses without the fear that that would disclose [identify] them,” Dr. Jones said.

An additional 17 of the 67 participants made what Dr. Jones termed “selective disclosures,” such as telling a coworker who was considered a friend or a supportive boss. The majority of the respondents to Jones’s survey – 36 of the 67 participants – were open about their conditions. All but one of the respondents in this broad-disclosure group worked in mental health fields.

Dr. Jones described the broad-disclosure designation as “meaning that there is nobody in their life who doesn’t know.”

“They’re out professionally. They’ve published a book. They speak,” Dr. Jones said. “If you Google them on the Internet, you would quickly learn that they had a psychiatric disability or psychosis.”

Dr. Jones herself falls into that camp. She’s told media outlets, including the online newspaper MinnPost, about her own experience being diagnosed with schizophrenia while a PhD student. The online magazine Pacific Standard ran a full-length feature about her return to academia.

About 100,000 adolescents and young adults in the United States experience first-episode psychosis each year, and the peak onset hits between 15 and 25 years of age, according to the NIMH. About a decade ago, the NIMH launched its Recovery After an Initial Schizophrenia Episode (RAISE) initiative to examine use of coordinated specialty care treatments for people who were experiencing a first episode of psychosis. Congress in 2014 moved to provide a stream of federal funding for those kinds of efforts.

“We’re going to be soon starting to discharge, on an annual basis, potentially tens of thousands of young people from these specialized early intervention programs,” Dr. Jones said. “So it becomes really pressing to understand what’s happening to them in the context of reintegration.”

Another presenter at the panel, Marjorie L. Baldwin, PhD, of Arizona State University, Tempe, is an economist who has published a book based, in part, on her son’s struggles, “Beyond Schizophrenia: Living and Working With a Serious Mental Illness” (Lanham, Md.: Rowman & Littlefield Publishers, 2016).

She presented findings from a pilot study for a larger project looking at the issue of disclosures of serious mental illness in the workplace. She and her colleague in this work, Steven C. Marcus, PhD, of the University of Pennsylvania, Philadelphia, separately spoke about the difficulties in securing funding for the project, including five failed R01 grant applications.

“The 6th time was the charm with NIH,” Dr. Baldwin said.

An initial hurdle was finding a cost-effective way to identify workers with serious mental illness who hold or have held what she termed “competitive jobs,” which Dr. Baldwin described as those that paid at least minimum wage and are not subsidized for people with disabilities.

“You cannot do this kind of a study with random dialing because it would be way too expensive,” she said. “Schizophrenia and serious mental illnesses are not rare, but they are fairly uncommon.”

Several years ago, though, she learned of a long-running health survey into which she could “piggyback” questions on mental health status. She presented results of a pilot study with about 230 people with serious mental illness who had held or had competitive jobs. Of this group, 52% had left their most recent job for reasons other than mental illness, while those conditions had caused an additional 21% to leave. But Dr. Baldwin and her colleagues found 27% still working.

Like Dr. Jones, Dr. Baldwin said some of those workers were in professional fields, such as accounting, law, education; others worked in the service and construction industries.

“Contrary to the stereotypes, people with serious mental illness whose symptoms are reasonably well controlled can work, and many are capable of supporting themselves in mainstream competitive jobs,” Dr. Baldwin said.

Dr. Jones had no disclosures tied to her survey. Dr. Baldwin and Dr. Marcus had no disclosures other than the NIH R01 grant.

ROCKVILLE, MD. – People working in the mental health field are more likely to disclose their past or present treatment for psychosis than are professionals in other fields, a researcher said at a National Institute of Mental Health conference on mental health services research.

The researcher, Nev Jones, PhD, presented the findings of a small survey she conducted in 2014 and 2015 of adults with current or past experiences of psychotic disorder who described themselves as having had or having a successful career. The research was not conducted for publication purposes but as part of an effort to develop tools for students with psychosis as they continued in higher education, said Dr. Jones, of the department of mental health law and policy at the University of South Florida, Tampa.

One of those tools was a closed program that was akin to Facebook; people with early psychosis could use this to “look at successful adults across a wide range of careers and how they had navigated accommodations, disclosure, education as well as vocational choice,” she said.

Dr. Jones did not ask participants about their gender and race, but she did query them on highest degree earned. The poll was disseminated with the assistance of the National Alliance on Mental Illness and that of Stanford (Calif.) University, where Dr. Jones was a postdoctoral fellow. Of the sample presented, 33% had a masters degree (MSW, MBA), and 15% had a doctoral level degree (JD, MD, PhD).

People who worked in fields outside of mental health care were far less likely to have revealed their conditions to colleagues or employers, with 14 of 67 participants having made no disclosure. Of 14 who had made no disclosure, 12 were in fields such as banking, economics, secondary education, nursing, pediatrics, and computer programming.

Dr. Jones said she received several calls from students and staff at Stanford who were unwilling to fill out the survey.

“They were very concerned about the risks of inadvertent disclosure, even though it was anonymous, because they had unique, potentially identifiable career paths that they could not lay out in their responses without the fear that that would disclose [identify] them,” Dr. Jones said.

An additional 17 of the 67 participants made what Dr. Jones termed “selective disclosures,” such as telling a coworker who was considered a friend or a supportive boss. The majority of the respondents to Jones’s survey – 36 of the 67 participants – were open about their conditions. All but one of the respondents in this broad-disclosure group worked in mental health fields.

Dr. Jones described the broad-disclosure designation as “meaning that there is nobody in their life who doesn’t know.”

“They’re out professionally. They’ve published a book. They speak,” Dr. Jones said. “If you Google them on the Internet, you would quickly learn that they had a psychiatric disability or psychosis.”

Dr. Jones herself falls into that camp. She’s told media outlets, including the online newspaper MinnPost, about her own experience being diagnosed with schizophrenia while a PhD student. The online magazine Pacific Standard ran a full-length feature about her return to academia.

About 100,000 adolescents and young adults in the United States experience first-episode psychosis each year, and the peak onset hits between 15 and 25 years of age, according to the NIMH. About a decade ago, the NIMH launched its Recovery After an Initial Schizophrenia Episode (RAISE) initiative to examine use of coordinated specialty care treatments for people who were experiencing a first episode of psychosis. Congress in 2014 moved to provide a stream of federal funding for those kinds of efforts.

“We’re going to be soon starting to discharge, on an annual basis, potentially tens of thousands of young people from these specialized early intervention programs,” Dr. Jones said. “So it becomes really pressing to understand what’s happening to them in the context of reintegration.”

Another presenter at the panel, Marjorie L. Baldwin, PhD, of Arizona State University, Tempe, is an economist who has published a book based, in part, on her son’s struggles, “Beyond Schizophrenia: Living and Working With a Serious Mental Illness” (Lanham, Md.: Rowman & Littlefield Publishers, 2016).

She presented findings from a pilot study for a larger project looking at the issue of disclosures of serious mental illness in the workplace. She and her colleague in this work, Steven C. Marcus, PhD, of the University of Pennsylvania, Philadelphia, separately spoke about the difficulties in securing funding for the project, including five failed R01 grant applications.

“The 6th time was the charm with NIH,” Dr. Baldwin said.

An initial hurdle was finding a cost-effective way to identify workers with serious mental illness who hold or have held what she termed “competitive jobs,” which Dr. Baldwin described as those that paid at least minimum wage and are not subsidized for people with disabilities.

“You cannot do this kind of a study with random dialing because it would be way too expensive,” she said. “Schizophrenia and serious mental illnesses are not rare, but they are fairly uncommon.”

Several years ago, though, she learned of a long-running health survey into which she could “piggyback” questions on mental health status. She presented results of a pilot study with about 230 people with serious mental illness who had held or had competitive jobs. Of this group, 52% had left their most recent job for reasons other than mental illness, while those conditions had caused an additional 21% to leave. But Dr. Baldwin and her colleagues found 27% still working.

Like Dr. Jones, Dr. Baldwin said some of those workers were in professional fields, such as accounting, law, education; others worked in the service and construction industries.

“Contrary to the stereotypes, people with serious mental illness whose symptoms are reasonably well controlled can work, and many are capable of supporting themselves in mainstream competitive jobs,” Dr. Baldwin said.

Dr. Jones had no disclosures tied to her survey. Dr. Baldwin and Dr. Marcus had no disclosures other than the NIH R01 grant.