Top cancer advance: Treatment of rare diseases

The American Society of Clinical Oncology (ASCO) named “Progress in Treating Rare Cancers” as the Advance of the Year for 2018, citing five major studies as examples of significant breakthroughs.

In an ASCO Special Article published in the Journal of Clinical Oncology, Sumanta K. Pal, MD, of City of Hope Comprehensive Cancer Center, Duarte, Calif., and colleagues, identified five studies that notably advanced cancer research.

Each study “reflects the impressive gains we have made in understanding these so-called orphan diseases and in tailoring treatments to target their unique characteristics,” wrote ASCO president Monica M. Bertagnolli, MD, in an introduction to the report.

One of the significant advances included use of a new combination of targeted therapies for a rare thyroid cancer that elicited responses in more than two-thirds of patients. A second study showed sorafenib improving progression-free survival for patients with desmoid tumors. In addition, patients with advanced midgut neuroendocrine tumors had a 79% lower risk of disease progression or death when treated with a new therapy of targeted radiation to tumor cells, lutetium-177 (177Lu)–Dotatate, compared with standard therapy; and trastuzumab, a standard treatment for human epidermal growth factor receptor 2 (HER2)–positive breast cancer, expanded its reach and significantly slowed progression of HER2-positive uterine serous carcinoma, the authors wrote. Finally, the “first promising therapy – the colony-stimulating factor-1 (CSF-1) inhibitor pexidartinib – for a rare cancer of the joints known as tenosynovial giant cell tumor, showed an overall response rate of 39.3% in patients taking pexidartinib versus 0% in patients taking a placebo,” they said.

For the first time, the ASCO progress report included a list of priorities to guide future research efforts, stated as follows:

• Identify strategies that better predict response to immunotherapies.
• Better define the patient populations that benefit from postoperative (adjuvant) therapy.
• Translate innovations in cellular therapies to solid tumors.
• Increase precision medicine research and treatment approaches in pediatric cancers.
• Optimize care for older adults with cancer.
• Increase equitable access to cancer clinical trials.
• Reduce the long-term consequences of cancer treatment.
• Reduce obesity and its impact on cancer incidence and outcomes.
• Identify strategies to detect and treat premalignant lesions.

“These priority areas, listed in no particular order, address an unmet need or help fill a knowledge gap in areas critical to improving patient care and outcomes,” the authors wrote.

The report acknowledged the value of federally funded research and the importance of ongoing federal investment in cancer research.

Dr. Pal disclosed relationships with Pfizer, Novartis, Aveo, Myriad Pharmaceuticals, Genentech, Exelixis, Bristol-Myers Squibb, Astellas Pharma, Ipsen, Eisai, and Medivation. Coauthors disclosed relationships with these and other companies.

SOURCE: Pal SK et al. J Clin Oncol. 2019 Jan 31. doi: 10.1200/JCO.18.02037.

The American Society of Clinical Oncology (ASCO) named “Progress in Treating Rare Cancers” as the Advance of the Year for 2018, citing five major studies as examples of significant breakthroughs.

In an ASCO Special Article published in the Journal of Clinical Oncology, Sumanta K. Pal, MD, of City of Hope Comprehensive Cancer Center, Duarte, Calif., and colleagues, identified five studies that notably advanced cancer research.

Each study “reflects the impressive gains we have made in understanding these so-called orphan diseases and in tailoring treatments to target their unique characteristics,” wrote ASCO president Monica M. Bertagnolli, MD, in an introduction to the report.

One of the significant advances included use of a new combination of targeted therapies for a rare thyroid cancer that elicited responses in more than two-thirds of patients. A second study showed sorafenib improving progression-free survival for patients with desmoid tumors. In addition, patients with advanced midgut neuroendocrine tumors had a 79% lower risk of disease progression or death when treated with a new therapy of targeted radiation to tumor cells, lutetium-177 (177Lu)–Dotatate, compared with standard therapy; and trastuzumab, a standard treatment for human epidermal growth factor receptor 2 (HER2)–positive breast cancer, expanded its reach and significantly slowed progression of HER2-positive uterine serous carcinoma, the authors wrote. Finally, the “first promising therapy – the colony-stimulating factor-1 (CSF-1) inhibitor pexidartinib – for a rare cancer of the joints known as tenosynovial giant cell tumor, showed an overall response rate of 39.3% in patients taking pexidartinib versus 0% in patients taking a placebo,” they said.

For the first time, the ASCO progress report included a list of priorities to guide future research efforts, stated as follows:

• Identify strategies that better predict response to immunotherapies.
• Better define the patient populations that benefit from postoperative (adjuvant) therapy.
• Translate innovations in cellular therapies to solid tumors.
• Increase precision medicine research and treatment approaches in pediatric cancers.
• Optimize care for older adults with cancer.
• Increase equitable access to cancer clinical trials.
• Reduce the long-term consequences of cancer treatment.
• Reduce obesity and its impact on cancer incidence and outcomes.
• Identify strategies to detect and treat premalignant lesions.

“These priority areas, listed in no particular order, address an unmet need or help fill a knowledge gap in areas critical to improving patient care and outcomes,” the authors wrote.

The report acknowledged the value of federally funded research and the importance of ongoing federal investment in cancer research.

Dr. Pal disclosed relationships with Pfizer, Novartis, Aveo, Myriad Pharmaceuticals, Genentech, Exelixis, Bristol-Myers Squibb, Astellas Pharma, Ipsen, Eisai, and Medivation. Coauthors disclosed relationships with these and other companies.

SOURCE: Pal SK et al. J Clin Oncol. 2019 Jan 31. doi: 10.1200/JCO.18.02037.

The American Society of Clinical Oncology (ASCO) named “Progress in Treating Rare Cancers” as the Advance of the Year for 2018, citing five major studies as examples of significant breakthroughs.

In an ASCO Special Article published in the Journal of Clinical Oncology, Sumanta K. Pal, MD, of City of Hope Comprehensive Cancer Center, Duarte, Calif., and colleagues, identified five studies that notably advanced cancer research.

Each study “reflects the impressive gains we have made in understanding these so-called orphan diseases and in tailoring treatments to target their unique characteristics,” wrote ASCO president Monica M. Bertagnolli, MD, in an introduction to the report.

One of the significant advances included use of a new combination of targeted therapies for a rare thyroid cancer that elicited responses in more than two-thirds of patients. A second study showed sorafenib improving progression-free survival for patients with desmoid tumors. In addition, patients with advanced midgut neuroendocrine tumors had a 79% lower risk of disease progression or death when treated with a new therapy of targeted radiation to tumor cells, lutetium-177 (177Lu)–Dotatate, compared with standard therapy; and trastuzumab, a standard treatment for human epidermal growth factor receptor 2 (HER2)–positive breast cancer, expanded its reach and significantly slowed progression of HER2-positive uterine serous carcinoma, the authors wrote. Finally, the “first promising therapy – the colony-stimulating factor-1 (CSF-1) inhibitor pexidartinib – for a rare cancer of the joints known as tenosynovial giant cell tumor, showed an overall response rate of 39.3% in patients taking pexidartinib versus 0% in patients taking a placebo,” they said.

For the first time, the ASCO progress report included a list of priorities to guide future research efforts, stated as follows:

• Identify strategies that better predict response to immunotherapies.
• Better define the patient populations that benefit from postoperative (adjuvant) therapy.
• Translate innovations in cellular therapies to solid tumors.
• Increase precision medicine research and treatment approaches in pediatric cancers.
• Optimize care for older adults with cancer.
• Increase equitable access to cancer clinical trials.
• Reduce the long-term consequences of cancer treatment.
• Reduce obesity and its impact on cancer incidence and outcomes.
• Identify strategies to detect and treat premalignant lesions.

“These priority areas, listed in no particular order, address an unmet need or help fill a knowledge gap in areas critical to improving patient care and outcomes,” the authors wrote.

The report acknowledged the value of federally funded research and the importance of ongoing federal investment in cancer research.

Dr. Pal disclosed relationships with Pfizer, Novartis, Aveo, Myriad Pharmaceuticals, Genentech, Exelixis, Bristol-Myers Squibb, Astellas Pharma, Ipsen, Eisai, and Medivation. Coauthors disclosed relationships with these and other companies.

SOURCE: Pal SK et al. J Clin Oncol. 2019 Jan 31. doi: 10.1200/JCO.18.02037.