Over the past year, major research advances provided new treatment options for patients with rare, difficult-to-treat cancers. In recognition of these achievements, the American Society of Clinical Oncology names “Progress in Treating Rare Cancers” as the Advance of the Year. To continue the forward momentum, ASCO also debuts its list of Research Priorities to accelerate progress against cancer. These and additional milestones in cancer research are featured in ASCO’s annual Clinical Cancer Advances report.
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 impactful and together comprise ASCO’s Advance of the Year:
Anaplastic Thyroid Carcinoma: FDA approved the first treatment for this form of thyroid cancer in nearly 50 years, a targeted therapy combination of dabrafenib (Tafinlar) plus trametinib (Mekinist) for patients with BRAF-mutated ATC. This approach produced tumor shrinkage in over two-thirds of study participants.
Desmoid Tumors: Sorafenib (Nexavar) became the first treatment to improve progression-free survival for patients with this rare form of sarcoma.
Midgut Neuroendocrine Tumors: FDA approved 177Lu-Dotatate (Lutathera), which delivers targeted radiation to tumor cells, based on research showing it lowers the risk of disease progression or death by 79% for patients with advanced disease.
Uterine Serous Carcinoma: Trastuzumab (Herceptin) 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% of patients.
This progress could not have come about without decades of sustained federal support for clinical cancer research. 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.
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.
