Rare Cancers

Rare diseases often come with an invisible burden—years of searching for answers, countless doctors who dismiss or misdiagnose their symptoms, and the emotional toll of navigating a healthcare system unequipped for their needs.

Experts at the Fred Hutchinson Cancer Center have created a drug-matching platform to help narrow down additional uses for drugs that have already been investigated and FDA approved.


Although not the most common of cancers, pediatric and adult sarcomas affect a significant proportion of patients, and managing these malignancies first requires understanding them, according to Paula Sanborn, BSN, RN.

On December 3, 2019, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to the biopharmaceutical company Adaptimmune Therapeutics for ADP-A2M4 for the treatment of synovial sarcoma. Earlier this year, the FDA granted Orphan Drug designation to the agent for the treatment of soft tissue sarcomas.

In the SARC028 phase 2 multicenter trial, reported at the 2017 ASCO Annual Meeting, pembrolizumab achieved encouraging responses in soft tissue sarcoma, especially undifferentiated pleomorphic sarcoma (UPS) and liposarcoma.

Journal of Oncology Navigation & Survivorship
JONS

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