ASCO Names "Progress in Treating Rare Cancers" as the Advance of the Year

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Reflecting on a year of breakthrough advancements in the treatment of rare, difficult to treat cancers, the American Society of Clinical Oncology (ASCO) has named “Progress in Treating Rare Cancers” as the Advance of the Year.

The American Society of Clinical Oncology (ASCO) has named “Progress in Treating Rare Cancers” as the Advance of the Year in recognition of breakthrough research advances in the treatment of patients with rare and difficult-to-treat cancers.

Although uncommon on their own, rare cancers collectively account for approximately 20% of all cancers; however, treatment progress for rare cancers has traditionally lagged behind other, more common cancers. Due to diversity in clinical presentation and underlying biology, there’s variation in the ability and effectiveness of treatment options. Clinical trials also pose a barrier, as it often takes longer to recruit enough participants for trials assessing new treatments for these cancers.

However, in the past year, there have been various notable research and regulatory advancements. These advancements have had a significant impact on 5 rare cancers, in particular, according to ASCO.


In January 2018, 177Lu-Doctate (Luthara) gained FDA approval for midgut neuroendocrine tumors after the treatment demonstrated that it lowers the risk of disease progression or death by 79% for patients with advanced disease. In May, the FDA approved a combination of targeted therapies dabrafenib (Tafinlar) and trametinib (Mekinist) for patients with BRAF-mutated anaplastic thyroid carcinoma, a rare form of thyroid cancer that hadn’t seen an approved treatment in nearly 50 years.

Also during 2018, it became evident that sorafenib (Nexavar) was the first treatment to improve progression-free survival in patients with desmoid tumors, a rare form of sarcoma; trastuzumab (Herceptin) showed that it slowed progression of HER2-positive uterine serous carcinoma, one of the most aggressive types of endometrial cancer; and pexidartinib was shown to be the first promising treatment for tenosynovial giant cell tumor, with 40% of patients having a response.

These breakthroughs, as well as others, are attributable to decades of sustained federal support for clinical cancer research, which highlights the importance of federal funding, according to ASCO.

“It’s exciting to see such substantial progress over the course of a single year, particularly against rare cancers. With US cancer cases set to rise roughly a third over the next decade, we must continue to advance research that saves lives,” ASCO President Monica Bertagnolli, MD, FACS, FASCO, said in a statement. “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.”

Looking forward, ASCO, for the first time, outlined areas of focus for future cancer research that represent areas of vital unmet need or knowledge gaps, including identify strategies that better predict response to immunotherapies, better define the patient populations that benefit from postoperative therapy, translate innovation in cellular therapies to solid tumors, optimize care for older adults with cancer, and reduce obesity and its impact on cancer incidence and outcomes.