ASCO Clinical Cancer Advances 2019 Names Advance of the Year: Progress in Treating Rare Cancers


Over the past year, major research advances provided new treatment options for patients with rare, difficult-to-treat cancers. In recognition of these achievements, ASCO named “Progress in Treating Rare Cancers” as the Advance of the Year. To continue the forward momentum, ASCO also debuted its list of research priorities to accelerate progress against cancer. These and additional milestones in cancer research are featured in Clinical Cancer Advances 2019: ASCO’s Annual Report on Progress Against Cancer, released today. 

“It’s exciting to see such substantial progress over the course of a single year, particularly against rare cancers. With U.S. cancer cases set to rise by roughly a third over the next decade, we must continue to advance research that saves lives,” said ASCO President Monica Bertagnolli, MD, FACS, FASCO. “Federal investment plays a key role in continuing progress—in rare and common cancers alike. We need to prioritize federal funding of cancer research in the years to come. Americans are counting on it.” 

Advance of the Year: Progress in Treating Rare Cancers

Although rare cancers account for about 20% 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: The 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 anaplastic thyroid cancer. This approach produced response in over two-thirds of study participants.
  • Desmoid Tumors: Sorafenib became the first treatment to improve progression-free survival for patients with this rare form of sarcoma.
  • Foregut, Midgut, and Hindgut Neuroendocrine Tumors: The FDA approved Lu-177 dotatate (Lutathera), which delivers targeted radiation to tumor cells, based on research in patients with somatostatin receptor–positive midgut tumors showing it lowers the risk of disease progession or death by 79%.
  • Uterine Serous Carcinoma: 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% 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 (NIH) 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:

  1. Identify strategies that better predict response to immunotherapies;
  2. Better define the patient populations that benefit from adjuvant therapy;
  3. Translate innovations in cellular therapies to solid tumors;
  4. Increase precision medicine research and treatment approaches in pediatric cancers;
  5. Optimize care for older adults with cancer;
  6. Increase equitable access to cancer clinical trials;
  7. Reduce the long-term consequences of cancer treatment;
  8. Reduce obesity and its impact on cancer incidence and outcomes;
  9. 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,” said 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.” 

Why Federal Funding for Research Matters

Federal research investments have driven many of the most important cancer prevention and treatment advances of the last half century. Nearly one-third of the advances featured in the Clinical Cancer Advances 2019 report received funding from the NIH and other federal agencies. Research funded by the U.S. government also generates billions of dollars in new economic activity and supports hundreds of thousands of jobs. According to ASCO’s 2018 National Cancer Opinion Survey, 67% of Americans say the U.S. government should spend more money on finding treatments and cures for cancer, even if it means higher taxes or adding to the deficit.

Despite Congress’ funding increases for the NIH and the National Cancer Institute (NCI) over the past 3 years, the NCI’s budget is only now reaching prerecession levels due to years of stagnant funding, and NCI can only fund a small fraction of new research proposals.

About Clinical Cancer Advances

Clinical Cancer Advances, now in its 14th edition, is published online at and in the Journal of Clinical Oncology at Several of the top advances featured in this year’s report (about 20%) were made possible by funding from Conquer Cancer, ASCO’s Foundation, or were led by past grantees who have continued their careers in oncology research.

The report editors' full disclosures can be found at

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.