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FDA APPROVES GLEEVEC TO TREAT GASTROINTESTINAL STROMAL CANCER

FDA today approved the cancer drug Gleevec (imatinib mesylate) for treatment of gastrointestinal stromal tumor (GIST). GIST is a relatively uncommon tumor, affecting about 5,000 people in the United States. It is a tumor that generally arises within the stomach or intestinal tract and metastasizes within the abdomen or the pelvis.

First approved in May 2001 for treatment of chronic myeloid leukemia (CML), Gleevec works by blocking enzymes that play a role in cancer growth. In CML, Gleevec blocks the abnormal tyrosine kinase enzyme that plays a major role in that disease. In GIST, Gleevec blocks a different abnormal enzyme found on the tumor cells. As these abnormal enzymes are largely confined to cancer cells, there is relatively little damage to normal cells while cancer cells are killed.

In the study that was the basis for Gleevec's approval for treatment of GIST, 147 patients with unresectable (i.e., cannot be surgically removed) or metastatic GIST received daily oral Gleevec treatment. While no patient had complete disappearance of cancer, 56 patients (38 percent) had reduction in tumor size by 50 percent or greater (partial response). Patients have not been followed long enough to determine the duration of the responses.

Gleevec has been approved under accelerated approval regulations and under the orphan drug program which provides financial incentives for drugs developed to treat rare diseases (diseases that affect fewer than 200,000 patients). Accelerated approval requires continued patient follow-up and information from additional studies to evaluate whether Gleevec provides an actual clinical benefit such as improved survival.

Commonly reported side effects include edema (fluid retention), nausea, vomiting, diarrhea, skin rash, muscle cramps, liver toxicity, and lower blood cell counts. Side effects are generally mild to moderate in severity and rarely require that Gleevec doses be decreased or interrupted for prolonged periods of time. Seven GIST patients had hemorrhage into the tumor or gastrointestinal tract that required red blood cell transfusions.

The drug is manufactured by Novartis AG for Novartis Pharmaceuticals Corp., East Hanover, New Jersey.

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