FDA Approval of Gleevec "Leukemia Pill" Fastest Ever for Anti-Cancer Therapy

The Food and Drug Administration today approved Gleevec for sale in the United States for the treatment of chronic- and acute-phase chronic myelogenous leukemia (CML). The bright orange pill, formerly known as STI571, was developed by Novartis Pharmaceuticals in collaboration with Brian Druker, M.D., director of the Oregon Cancer Institute's Leukemia Center at Oregon Health & Science University.

"This truly is the dawn of a new era for medical research," said Peter Kohler, M.D., president of OHSU. "This is the first drug that demonstrates a therapy targeted to the specific molecular defect can be effective in real patients."

Gleevec targets the activity of CML cells while leaving normal cells alone, resulting in a treatment that is remarkably effective and produces little or no side effects. An oncologist who has studied the activity of tyrosine kinase proteins, Druker helped to develop Gleevec to inhibit the activity of BCR-ABL, an abnormal protein found in CML patients.

After beginning clinical trials of Gleevec in June 1998, Druker found that 31 of 31 patients saw their white blood counts return to normal within weeks, and four saw a complete disappearance of the leukemia from their bone marrow. Further testing has produced even better results in patients in the early stage of the disease, while many patients in the acute phase also have experienced recovery. CML affects about 5,000 people in the U.S. each year.

"The drug has proven to be safe and effective and is living up to our greatest hopes and expectations," said Druker. "Obviously, the FDA felt this was an important treatment to bring to patients as quickly as possible, and I commend the agency for its speed in reviewing the data." A typical cancer therapy can take three to five years for FDA approval, but Gleevec was approved less than three years after testing began.

While the leukemia story is remarkable on its own, Gleevec also is being tested on several other cancer types that are driven by proteins similar to BCR-ABL. In a disease called gastrointestinal stromal tumors (GIST), patients at OHSU and three other sites have experienced tremendous recovery taking Gleevec where no other therapy has worked. Results of the GIST clinical trials are being presented this weekend at the American Society of Clinical Oncology meeting in San Francisco by Charles Blanke, M.D., director of the Oregon Cancer Institute's Gastrointestinal Oncology Program at OHSU.

Clinical trials with Gleevec now are under way in a rare brain cancer called glioblastoma, small-cell lung cancer and a form of prostate cancer. Because Gleevec was already determined to be safe in the trials for CML, these studies will focus on determining its effectiveness in treating the other cancers.

Patients seeking information on Gleevec for CML or GIST can call the Oregon Cancer Institute at 503 494-1080 or visit www.ohsu.edu.

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