And the Cancer Advance of the Year Is … Progress in Treating Rare Cancers

This progress could not have come about without decades of sustained federal support for clinical cancer research.

Advance of the Year: Progress in Treating Rare Cancers

Although rare cancers account for about 20 percent of all cancers diagnosed in the United States each year, treatment progress has lagged behind that of more common forms of the disease. In the past year, however, research and regulatory achievements in five rare cancers were particularly significant and together comprise ASCO’s Advance of the Year:

• Anaplastic Thyroid Carcinoma: The U.S. Food and Drug Administration approved the first treatment for this form of thyroid cancer in nearly 50 years, a targeted therapy combination of Tafinlar (dabrafenib) plus Mekinist (trametinib).

• Desmoid Tumors: Nexavar (sorafenib) became the first treatment to improve progression-free survival for people with this rare form of sarcoma.

• Midgut Neuroendocrine Tumors: The FDA approved Lutathera (177Lu-Dotatate), which delivers targeted radiation to tumor cells, based on research showing it lowers the risk of disease progression or death by 79 percent for people with advanced disease.

• Uterine Serous Carcinoma: Herceptin (trastuzumab) was shown to slow progression of HER2-positive uterine serous carcinoma, one of the most aggressive forms of endometrial cancer.

• Tenosynovial Giant Cell Tumor: Research identified the first promising therapy, pexidartinib, for this rare cancer of the joints, producing responses in nearly 40 percent of those affected.

This progress could not have come about without decades of sustained federal support for clinical cancer research. 

Federal research investments have driven many of the most important cancer prevention and treatment advances of the last half century. Several ongoing research initiatives sponsored by the National Institutes of Health have yielded key insights for rare cancers, and three of the five studies featured as part of the Advance of the Year received funding from the U.S. government. However, despite Congress’ funding increases for the NIH and the National Cancer Institute over the past three years, NCI’s budget is only now reaching pre-recession levels due to years of stagnant funding, and NCI can only fund a small fraction of new research proposals.

Nine Research Priorities to Advance Progress Against Cancer

For the first time, ASCO has identified specific areas to focus future cancer research efforts. These priorities, listed in no particular order, represent areas of vital unmet need or knowledge gaps that could significantly improve clinical decision-making. ASCO’s Research Priorities include:

• Identify strategies that better predict response to immunotherapies
• Better define the patient populations that benefit from post-operative (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 priorities represent our vision for finding the next generation of cancer cures and reducing cancer’s impact on patients’ lives,” says Richard L. Schilsky, MD, FACP, FSCT, FASCO, ASCO Senior Vice President and Chief Medical Officer. “From prevention through survivorship, these priorities are intended to identify areas where progress is most needed and most promising.”