January 31, 2019
3 min read

ASCO names progress in rare cancer treatment 'Advance of the Year'

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Monica M. Bertagnolli, MD, FACS, FASCO
Monica Bertagnolli

ASCO has named progress in treating rare cancers as the cancer Advance of the Year, recognizing major research advances that generated new treatments for patients with rare, difficult-to-treat cancers, such as desmoid tumors and uterine serous carcinoma, the society announced in a press release.

ASCO also revealed its list of research priorities and highlighted other achievements in cancer research in the society’s annual “Clinical Cancer Advances” report, 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,” ASCO President Monica Bertagnolli, MD, FACS, FASCO, said in the press release. “Federal investment plays a key role in continuing success — in rare and common cancers alike. We need to prioritize federal funding of cancer research in the years to come.”

Rare but impactful

Rare cancers account for about 20% of all malignancies diagnosed annually in the U.S., but treatment progress has been slow compared with that of more common cancers.

However, research and regulatory achievements have resulted in significant progress against five rare cancers, which together encompass ASCO’s Advance of the Year:

  • Anaplastic thyroid carcinoma: For the first time in nearly 50 years, FDA approved a new treatment for this form of thyroid cancer, a combination of dabrafenib (Tafinlar, Novartis) plus trametinib (Mekinist, Novartis) that is targeted to patients with BRAF-mutated anaplastic thyroid carcinoma. This treatment has been shown to shrink tumors in more than two-thirds of study participants;
  • Desmoid tumors: PFS among patients with this rare form of sarcoma improved with sorafenib (Nexavar; Bayer, Onyx Pharmaceuticals), the first treatment to achieve this outcome;
  • Midgut neuroendocrine tumors: FDA approved 177Lu-Dotatate (Lutathera, Advanced Accelerator Applications), which targets tumor cells with radiation and has been shown to lower the risk for disease progression or death by 79% among patients with advanced disease;
  • Uterine serous carcinoma: The monoclonal antibody trastuzumab (Herceptin, Genentech) delayed progression of the HER2-positive form of this aggressive endometrial cancer; and
  • Tenosynovial giant cell tumor: Pexidartinib became the first promising treatment for this rare cancer of the joints, inducing responses in almost 40% of study patients.

ASCO cited decades of sustained federal support for ongoing clinical research projects that made the progress possible. Several research initiatives funded by NIH produced important insights into rare cancers, and three of the five studies cited as part of the Advance of the Year received funding from the U.S. government, according to the press release.

Future priorities

ASCO also identified nine research priorities, which cover areas of unmet need or knowledge gaps that could guide clinical decision-making.

The highlighted areas call on the research community to:

  • identify ways to more accurately predict response to immunotherapies;
  • better understand and delineate the populations that benefit from adjuvant therapy;
  • apply advances in cellular therapies to solid tumor treatment;
  • bolster research into precision medicine and therapies for pediatric cancers;
  • improve care for older adults with cancer;
  • work to ensure equal access to cancer clinical trials;
  • reduce obesity, a risk factor for various diseases, and its influence on cancer incidence and outcomes; and
  • identify approaches 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,” Richard L. Schilsky, MD, FACP FASCO, ASCO senior vice president and chief medical officer, said in the release. “From prevention through survivorship, these priorities are intended to identify areas where progress is most needed and most promising.”

Federal funding key

Federal investments in cancer research have led to some of the most significant advances in cancer prevention and treatment in the past half century, the ASCO press release stated.

ASCO reported that nearly one-third of the advances included in this year’s report were funded by NIH and other federal agencies. Additionally, research funded by the U.S. government produces billions of dollars in new economic activity and sustains hundreds of thousands of jobs.

ASCO’s National Cancer Opinion Survey reported that 67% of Americans believe the U.S. government should spend more money on discovering new cancer treatments and cures, even if this leads to higher taxes or an increased deficit. NCI’s budget is returning to prerecession levels after years of stagnant funding, but the institute can only fund a small portion of new research projects, the press release stated. – by Jen Byrne

Disclosures: HemOnc Today could not confirm Bertagnolli’s relevant financial disclosures at the time of reporting. Schilsky reports research funding to his institution from AstraZeneca, Bayer, Bristol-Myers Squibb, Genentech, Eli Lilly, Merck and Pfizer. Please see the report for a list of all other authors’ relevant financial disclosures.