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#Patients looking for #clinicaltrials
I just hung up with a friend I haven’t seen in decades. Her father has advanced cancer, and while she does not have formal medical training, a passerby wouldn’t know it. Her questions are spot on, her resources are peer reviewed and validated, and her questions I’d more likely expect from trainees in a formal oncology training program than from the director of an elementary level tutoring service.
Her father is fortunately doing well, but she’s searching for the next plan for when the standard drugs ultimately fail. We know they will fail. She’s connected to patient advocacy groups, emailing physicians across the country, and looking into clinical trials with their exhaustive lists of exclusion criteria. She sees the logistic difficulties with trials far from home. She’s hit the key issues we face every day in clinical research, and she’s never stepped foot in a medical school lecture hall.
Amazingly her story is not unique. When cancer hits close to home is when these problems become very clear. This same story could easily have been retold as the narrative of former Vice President Joe Biden and his care for his son. Both my friend and Mr. Biden, in fact, asked me the same question: How do we get the cutting-edge science from major research centers out to the rest of the country?
The Cancer Moonshot initiative has done much to promote collaboration, but one major success has been in the Count Me In initiative, a partnership between the Biden Cancer Initiative, Emerson Collective, the Broad Institute, and the Dana-Farber Cancer Institute. Their goal is to gain access to thousands of patients, collect data on treatment and outcomes, and collect biological specimens. They are not alone, the MSK-IMPACT initiative – led by David B. Solit, MD, at my institution – aims to sequence rare cancers. Both programs have heavily leveraged social media to access and engage patients.
There are of course concerns. Coming from hundreds or thousands of different sites will mean the data will likely be heterogeneous in formatting and quality. How do we ensure the security of patient data? Can we rely on patients and family members to report accurately and without bias? We know there are challenges and upside to crowdsourced patient recruitment.
David Ginsburg, MD, Karl Desch, MD, and colleagues enrolled more than 1,000 students from the University of Michigan to participate in a study on blood clotting factors. This led to many important findings on the genetic basis for coagulopathies, but also was instructive in uncovering a worrisome aspect of online patient registration. The group recorded the time taken for registrants to read the consent form – including whether the participant clicked a hyperlink that was embedded. Nearly a quarter of participants accepted the terms of the 2,833-word document in less than 10 seconds, and less than 3% clicked the hyperlink (Ann Intern Med. 2011 Sep 6;155[5]:316-22).
Are these patients, who we are asking for their partnership and trust, really understanding to what they are agreeing?
Surely there is tremendous altruism on the part of these patients. Their hopes of helping the future of cancer care does have a real track record. Crowdsourcing efforts that were less far reaching in scope made substantial impact in discovering the genetic basis for polycythemia vera. The patients, contacted largely through printed newspaper ads, have helped millions of others. What will happen when we add in the power of social media will be exciting to see – and there is something else that comes with great power, but since I can’t seem to remember what that is, I’ll just search online.
Dr. Viny is with the Memorial Sloan-Kettering Cancer Center, N.Y., where he is an assistant attending physician on the leukemia service and is a clinical researcher in the Ross Levine Lab. Follow him on Twitter @TheDoctorIsVin.
I just hung up with a friend I haven’t seen in decades. Her father has advanced cancer, and while she does not have formal medical training, a passerby wouldn’t know it. Her questions are spot on, her resources are peer reviewed and validated, and her questions I’d more likely expect from trainees in a formal oncology training program than from the director of an elementary level tutoring service.
Her father is fortunately doing well, but she’s searching for the next plan for when the standard drugs ultimately fail. We know they will fail. She’s connected to patient advocacy groups, emailing physicians across the country, and looking into clinical trials with their exhaustive lists of exclusion criteria. She sees the logistic difficulties with trials far from home. She’s hit the key issues we face every day in clinical research, and she’s never stepped foot in a medical school lecture hall.
Amazingly her story is not unique. When cancer hits close to home is when these problems become very clear. This same story could easily have been retold as the narrative of former Vice President Joe Biden and his care for his son. Both my friend and Mr. Biden, in fact, asked me the same question: How do we get the cutting-edge science from major research centers out to the rest of the country?
The Cancer Moonshot initiative has done much to promote collaboration, but one major success has been in the Count Me In initiative, a partnership between the Biden Cancer Initiative, Emerson Collective, the Broad Institute, and the Dana-Farber Cancer Institute. Their goal is to gain access to thousands of patients, collect data on treatment and outcomes, and collect biological specimens. They are not alone, the MSK-IMPACT initiative – led by David B. Solit, MD, at my institution – aims to sequence rare cancers. Both programs have heavily leveraged social media to access and engage patients.
There are of course concerns. Coming from hundreds or thousands of different sites will mean the data will likely be heterogeneous in formatting and quality. How do we ensure the security of patient data? Can we rely on patients and family members to report accurately and without bias? We know there are challenges and upside to crowdsourced patient recruitment.
David Ginsburg, MD, Karl Desch, MD, and colleagues enrolled more than 1,000 students from the University of Michigan to participate in a study on blood clotting factors. This led to many important findings on the genetic basis for coagulopathies, but also was instructive in uncovering a worrisome aspect of online patient registration. The group recorded the time taken for registrants to read the consent form – including whether the participant clicked a hyperlink that was embedded. Nearly a quarter of participants accepted the terms of the 2,833-word document in less than 10 seconds, and less than 3% clicked the hyperlink (Ann Intern Med. 2011 Sep 6;155[5]:316-22).
Are these patients, who we are asking for their partnership and trust, really understanding to what they are agreeing?
Surely there is tremendous altruism on the part of these patients. Their hopes of helping the future of cancer care does have a real track record. Crowdsourcing efforts that were less far reaching in scope made substantial impact in discovering the genetic basis for polycythemia vera. The patients, contacted largely through printed newspaper ads, have helped millions of others. What will happen when we add in the power of social media will be exciting to see – and there is something else that comes with great power, but since I can’t seem to remember what that is, I’ll just search online.
Dr. Viny is with the Memorial Sloan-Kettering Cancer Center, N.Y., where he is an assistant attending physician on the leukemia service and is a clinical researcher in the Ross Levine Lab. Follow him on Twitter @TheDoctorIsVin.
I just hung up with a friend I haven’t seen in decades. Her father has advanced cancer, and while she does not have formal medical training, a passerby wouldn’t know it. Her questions are spot on, her resources are peer reviewed and validated, and her questions I’d more likely expect from trainees in a formal oncology training program than from the director of an elementary level tutoring service.
Her father is fortunately doing well, but she’s searching for the next plan for when the standard drugs ultimately fail. We know they will fail. She’s connected to patient advocacy groups, emailing physicians across the country, and looking into clinical trials with their exhaustive lists of exclusion criteria. She sees the logistic difficulties with trials far from home. She’s hit the key issues we face every day in clinical research, and she’s never stepped foot in a medical school lecture hall.
Amazingly her story is not unique. When cancer hits close to home is when these problems become very clear. This same story could easily have been retold as the narrative of former Vice President Joe Biden and his care for his son. Both my friend and Mr. Biden, in fact, asked me the same question: How do we get the cutting-edge science from major research centers out to the rest of the country?
The Cancer Moonshot initiative has done much to promote collaboration, but one major success has been in the Count Me In initiative, a partnership between the Biden Cancer Initiative, Emerson Collective, the Broad Institute, and the Dana-Farber Cancer Institute. Their goal is to gain access to thousands of patients, collect data on treatment and outcomes, and collect biological specimens. They are not alone, the MSK-IMPACT initiative – led by David B. Solit, MD, at my institution – aims to sequence rare cancers. Both programs have heavily leveraged social media to access and engage patients.
There are of course concerns. Coming from hundreds or thousands of different sites will mean the data will likely be heterogeneous in formatting and quality. How do we ensure the security of patient data? Can we rely on patients and family members to report accurately and without bias? We know there are challenges and upside to crowdsourced patient recruitment.
David Ginsburg, MD, Karl Desch, MD, and colleagues enrolled more than 1,000 students from the University of Michigan to participate in a study on blood clotting factors. This led to many important findings on the genetic basis for coagulopathies, but also was instructive in uncovering a worrisome aspect of online patient registration. The group recorded the time taken for registrants to read the consent form – including whether the participant clicked a hyperlink that was embedded. Nearly a quarter of participants accepted the terms of the 2,833-word document in less than 10 seconds, and less than 3% clicked the hyperlink (Ann Intern Med. 2011 Sep 6;155[5]:316-22).
Are these patients, who we are asking for their partnership and trust, really understanding to what they are agreeing?
Surely there is tremendous altruism on the part of these patients. Their hopes of helping the future of cancer care does have a real track record. Crowdsourcing efforts that were less far reaching in scope made substantial impact in discovering the genetic basis for polycythemia vera. The patients, contacted largely through printed newspaper ads, have helped millions of others. What will happen when we add in the power of social media will be exciting to see – and there is something else that comes with great power, but since I can’t seem to remember what that is, I’ll just search online.
Dr. Viny is with the Memorial Sloan-Kettering Cancer Center, N.Y., where he is an assistant attending physician on the leukemia service and is a clinical researcher in the Ross Levine Lab. Follow him on Twitter @TheDoctorIsVin.
Under his IDH2: Epigenetic drivers of clonal hematopoiesis
The world has surely changed in recent years, leading television and Hollywood to recreate many former works of postapocalyptic fiction. Watching the recent Hulu drama “The Handmaid’s Tale” leads many to make modern-day comparisons, but to this hematologist the connection is bone deep.
Hematopoietic stem cells face a century of carefully regulated symmetric division. They are tasked with the mission to generate the heterogeneous and environmentally responsive progenitor populations that will undergo coordinated differentiation and maturation.1
Within this epic battle of Gilead, much like hematopoiesis, there is a hierarchy within the Sons of Jacob. At the top there are few who drive leukemogenicity and require no other co-conspirators. MLL fusions and core binding factor are the Commanders that can overthrow the regulated governance of normal marrow homeostasis. Other myeloid disease alleles are soldiers of Gilead (i.e. FLT3-ITD, IDH1, IDH2, NPM1c, Spliceosome/Cohesin). Their individual characteristics can influence the natural history of the disease and define its strengths and weaknesses, particularly as new targeted therapies are developed.
Recently, Jongen-Lavrencic et al. reported that all disease alleles that persist as minimal residual disease after induction have a negative influence on outcome, except for three – DNMT3A, TET2, and ASXL1.2 What do we make of the “DTA” mutations? Are they the “Eye” – spies embedded in the polyclonal background of the marrow – intrinsically wired with malevolent intent? Are they the innocent, abused Handmaids – reprogrammed by inflammatory “Aunts” at the marrow’s Red Center and forced to clonally procreate? Previous works have suggested the latter, that persistence of mutations in genes found in clonal hematopoiesis (CH) do not portend an equivalent risk of relapse.3,4
A more encompassing question remains as to the equivalency of CH in the absence of a hematopoietic tumor versus CH postleukemia therapy. Comparisons to small cell lymphomas after successful treatment of transformed disease are disingenuous; CH is not itself a malignant state.5 Forty months of median follow-up for post-AML CH is simply not enough time. Work presented at the 2017 annual meeting of the American Society of Hematology by Jaiswal et al. showed that the approximately 1% risk of transformation per year was consistent over a 20-year period of follow up in the Swedish Nurse’s Study.
The recent work in the New England Journal of Medicine adds to the growing understanding of CH, but requires the test of time to know if these cells are truly innocent Handmaids or the Eye in a red cloak. I’ll be exploring these issues and taking your questions during a live Twitter Q&A on June 14 at noon ET. Follow me at @TheDoctorIsVin and @HematologyNews1 for more details. Blessed be the fruit.
Dr. Viny is with the Memorial Sloan-Kettering Cancer Center, N.Y., where he is a clinical instructor, is on the staff of the leukemia service, and is a clinical researcher in the Ross Levine Lab.
References
1. Orkin SH and Zon L. Cell. 2008 Feb 22;132(4):631-44.
2. Jongen-Lavrencic M et al. N Engl J Med 2018; 378:1189-99.
3. Bhatnagar B et al. Br J Haematol. 2016 Oct;175(2):226-36.
4. Shlush LI et al. Nature. 2017;547:104-8.
5. Bowman RL et al. Cell Stem Cell. 2018 Feb 1;22(2):157-70.
The world has surely changed in recent years, leading television and Hollywood to recreate many former works of postapocalyptic fiction. Watching the recent Hulu drama “The Handmaid’s Tale” leads many to make modern-day comparisons, but to this hematologist the connection is bone deep.
Hematopoietic stem cells face a century of carefully regulated symmetric division. They are tasked with the mission to generate the heterogeneous and environmentally responsive progenitor populations that will undergo coordinated differentiation and maturation.1
Within this epic battle of Gilead, much like hematopoiesis, there is a hierarchy within the Sons of Jacob. At the top there are few who drive leukemogenicity and require no other co-conspirators. MLL fusions and core binding factor are the Commanders that can overthrow the regulated governance of normal marrow homeostasis. Other myeloid disease alleles are soldiers of Gilead (i.e. FLT3-ITD, IDH1, IDH2, NPM1c, Spliceosome/Cohesin). Their individual characteristics can influence the natural history of the disease and define its strengths and weaknesses, particularly as new targeted therapies are developed.
Recently, Jongen-Lavrencic et al. reported that all disease alleles that persist as minimal residual disease after induction have a negative influence on outcome, except for three – DNMT3A, TET2, and ASXL1.2 What do we make of the “DTA” mutations? Are they the “Eye” – spies embedded in the polyclonal background of the marrow – intrinsically wired with malevolent intent? Are they the innocent, abused Handmaids – reprogrammed by inflammatory “Aunts” at the marrow’s Red Center and forced to clonally procreate? Previous works have suggested the latter, that persistence of mutations in genes found in clonal hematopoiesis (CH) do not portend an equivalent risk of relapse.3,4
A more encompassing question remains as to the equivalency of CH in the absence of a hematopoietic tumor versus CH postleukemia therapy. Comparisons to small cell lymphomas after successful treatment of transformed disease are disingenuous; CH is not itself a malignant state.5 Forty months of median follow-up for post-AML CH is simply not enough time. Work presented at the 2017 annual meeting of the American Society of Hematology by Jaiswal et al. showed that the approximately 1% risk of transformation per year was consistent over a 20-year period of follow up in the Swedish Nurse’s Study.
The recent work in the New England Journal of Medicine adds to the growing understanding of CH, but requires the test of time to know if these cells are truly innocent Handmaids or the Eye in a red cloak. I’ll be exploring these issues and taking your questions during a live Twitter Q&A on June 14 at noon ET. Follow me at @TheDoctorIsVin and @HematologyNews1 for more details. Blessed be the fruit.
Dr. Viny is with the Memorial Sloan-Kettering Cancer Center, N.Y., where he is a clinical instructor, is on the staff of the leukemia service, and is a clinical researcher in the Ross Levine Lab.
References
1. Orkin SH and Zon L. Cell. 2008 Feb 22;132(4):631-44.
2. Jongen-Lavrencic M et al. N Engl J Med 2018; 378:1189-99.
3. Bhatnagar B et al. Br J Haematol. 2016 Oct;175(2):226-36.
4. Shlush LI et al. Nature. 2017;547:104-8.
5. Bowman RL et al. Cell Stem Cell. 2018 Feb 1;22(2):157-70.
The world has surely changed in recent years, leading television and Hollywood to recreate many former works of postapocalyptic fiction. Watching the recent Hulu drama “The Handmaid’s Tale” leads many to make modern-day comparisons, but to this hematologist the connection is bone deep.
Hematopoietic stem cells face a century of carefully regulated symmetric division. They are tasked with the mission to generate the heterogeneous and environmentally responsive progenitor populations that will undergo coordinated differentiation and maturation.1
Within this epic battle of Gilead, much like hematopoiesis, there is a hierarchy within the Sons of Jacob. At the top there are few who drive leukemogenicity and require no other co-conspirators. MLL fusions and core binding factor are the Commanders that can overthrow the regulated governance of normal marrow homeostasis. Other myeloid disease alleles are soldiers of Gilead (i.e. FLT3-ITD, IDH1, IDH2, NPM1c, Spliceosome/Cohesin). Their individual characteristics can influence the natural history of the disease and define its strengths and weaknesses, particularly as new targeted therapies are developed.
Recently, Jongen-Lavrencic et al. reported that all disease alleles that persist as minimal residual disease after induction have a negative influence on outcome, except for three – DNMT3A, TET2, and ASXL1.2 What do we make of the “DTA” mutations? Are they the “Eye” – spies embedded in the polyclonal background of the marrow – intrinsically wired with malevolent intent? Are they the innocent, abused Handmaids – reprogrammed by inflammatory “Aunts” at the marrow’s Red Center and forced to clonally procreate? Previous works have suggested the latter, that persistence of mutations in genes found in clonal hematopoiesis (CH) do not portend an equivalent risk of relapse.3,4
A more encompassing question remains as to the equivalency of CH in the absence of a hematopoietic tumor versus CH postleukemia therapy. Comparisons to small cell lymphomas after successful treatment of transformed disease are disingenuous; CH is not itself a malignant state.5 Forty months of median follow-up for post-AML CH is simply not enough time. Work presented at the 2017 annual meeting of the American Society of Hematology by Jaiswal et al. showed that the approximately 1% risk of transformation per year was consistent over a 20-year period of follow up in the Swedish Nurse’s Study.
The recent work in the New England Journal of Medicine adds to the growing understanding of CH, but requires the test of time to know if these cells are truly innocent Handmaids or the Eye in a red cloak. I’ll be exploring these issues and taking your questions during a live Twitter Q&A on June 14 at noon ET. Follow me at @TheDoctorIsVin and @HematologyNews1 for more details. Blessed be the fruit.
Dr. Viny is with the Memorial Sloan-Kettering Cancer Center, N.Y., where he is a clinical instructor, is on the staff of the leukemia service, and is a clinical researcher in the Ross Levine Lab.
References
1. Orkin SH and Zon L. Cell. 2008 Feb 22;132(4):631-44.
2. Jongen-Lavrencic M et al. N Engl J Med 2018; 378:1189-99.
3. Bhatnagar B et al. Br J Haematol. 2016 Oct;175(2):226-36.
4. Shlush LI et al. Nature. 2017;547:104-8.
5. Bowman RL et al. Cell Stem Cell. 2018 Feb 1;22(2):157-70.