Private not-for-profits are also stepping in to support rare cancer research. Cycle for Survival has raised more than $140 million to fund rare cancer clinical trials, research studies, and technologies led by Memorial Sloan Kettering Cancer Center in New York, New York.

The Rare Cancer Research Foundation is taking a different, innovative, approach: the 2-year-old foundation strives to get patients with a rare cancer to donate excess tissue from their own procedures so that researchers will have additional samples for cell line trials.      

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This research is already delivering remarkable results.

“I really think that is what is also changing is our grouping of cancers not only by disease type but now by molecular phenotype,” Dr Schott said. “So that maybe there’s more in common between breast and ovarian cancer, for example, than what we knew before. Maybe, instead of treating breast cancer one way and ovarian cancer another, you might treat breast and ovarian cancer with a BRCA mutation the same way.”

In October, researchers from the University of California, San Diego Moores Cancer Center reported that they were able to use a precision medicine therapy strategy to identify effective targeted treatments for patients with rare cancer tumors. More than half of the patients “achieved stable disease (SD) ≥6 months (n = 3), partial remission (PR; n = 6), or complete remission (CR; n = 2). Matched therapy resulted in significantly longer progression-free survival compared with last prior unmatched therapy.”5

Still, as more rare cancers are identified, the need for rethinking approaches to research will become greater.

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Failure to do so, the editorial noted, has a direct negative impact on patients: “[w]e should not judge scientific proposals and scientific merit in rare cancer studies in the same way as we do for mainstream malignancies, as this may not only be scientifically flawed, but may also represent a strategic mistake that will deny therapeutic advancement to almost a quarter of all cancer patients.”


  1. Alvi MA, Wilson RH, Salto-Tellez M. Rare cancers: the greatest inequality in cancer research and oncology treatment. Br J Cancer. 2017;117(9):1255-7. doi: 10.1038/bjc.2017.321
  2. Panageas KS. Clinical trial design for rare cancers – why a less conventional route may be required. Expert Rev Clin Pharmacol. 2015;8(6):661-3. doi: 10.1586/17512433.2015.1088382
  3. DeSantis CE, Kramer JL, Jemal A. The burden of rare cancers in the United States. CA Cancer J Clin. 2017;67(4):261-72. doi: 10.3322/caac.21400
  4. Schott AF, Welch JJ, Verschraegen CF, Kurzrock R. The National Clinical Trials Network: conducting successful clinical trials of new therapies for rare cancers. Semin Oncol. 2015;42(5):731-9. doi: 10.1053/j.seminoncol.2015.07.010
  5. Kato S, Kurasaki K, Ikeda S, Kurzrock R. Rare Tumor Clinic: the University of California San Diego Moores Cancer Center experience with a precision therapy approach. Oncologist. 2017 Oct 16. doi: 10.1634/theoncologist.2017-0199 [Epub ahead of print]