Abstract
were misidentifi ed as leiomyosarcomas, which don ’ t respond to chemotherapy; survival rates generally didn ’ t exceed 10% by 30 months after diagnosis, according to Heinrich. Scientists now know that roughly 95% of GIST cases involve kit mutations. Connecting GIST to the kit gene was pivotal because imatinib — developed initially against bcr-abl mutations in chronic myelogenous leukemia — also hits kit as an unintended target. Today, 65% – 70% of imatinib-treated GIST patients are alive at 30 months, Heinrich said. The fi rst GIST patient treated with imatinib was a Finnish woman with metastatic disease. She had family connections to Novartis executives, who gave her the drug. Known only as “patient zero,” she achieved a miraculous response, according to Norman Scherzer, executive director of the Life Raft Group, a GIST advocacy group, in Wayne, N.J. So in 2000, Heinrich, together with Jonathan Fletcher, M.D., and George DeMitri, M.D., both from the Dana – Farber Cancer Institute in Boston, followed up with a phase II study of imatinib in 147 patients with metastatic disease. Among them was Scherzer ’ s wife, Anita, who still takes imatinib. Like patient zero, most of these individuals achieved dramatic responses, prompting the FDA to grant accelerated
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