Each year, 3,000 to 6,000 people are diagnosed with gastrointestinal stromal tumor (GIST)—the most common sarcoma of the gastrointestinal tract. Imatinib myeslate, or Gleevec®, has proved to be a highly effective therapy for many patients with GIST, although long-term survival is poor due to the development of imatinib-resistant GIST mutation types.
Research recently published in Science Translational Medicine demonstrated promising results for a new treatment, BLU-285, which specifically targets two of the most common imatinib-resistant GIST mutations: KIT and PDGFRA. With an international team of researchers, co-author Michael Heinrich, M.D., professor of hematology and medical oncology in the OHSU Knight Cancer Institute, School of Medicine, and practicing physician at the VA Portland Health System, showed that the highly selective compound BLU-285 inhibits well-characterized disease-driving KIT mutants both in vitro and in vivo in preclinical models.
The data show 67 percent of patients with heavily pretreated KIT-driven GIST treated with BLU-285 had radiographic tumor reductions. Based on this data, the current Phase I trial remains ongoing and Blueprint Medicines is planning for a Phase III randomized study.
See OHSU News for more about the BLU-285 clinical trial
Disclosures: Heinrich has worked with Blueprint Medicines, the company that developed BLU-285, as a consultant and is an investigator for Blueprint Medicines’ ongoing Phase 1 GIST study. He has received research funding from Blueprint Medicines and has provided expert testimony for Novartis. Brian Druker, M.D., director of the OHSU Knight Cancer Institute, is a scientific co-founder of Blueprint Medicines. He was not involved in this clinical trial.
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