FDA Approves Gleevec for Treatment of Pediatric Leukemia

05/20/03    Portland, Ore.

OHSU scientist Brian Druker helped develop drug for treatment of CML

The Food and Drug Administration has approved the anticancer drug Gleevec to treat children with chronic myeloid leukemia (CML), a rare, life-threatening form of cancer that accounts for about 2 percent of all leukemias in children.

The FDA approved Gleevec for the treatment of children with chronic-phase Philadelphia chromosome positive CML whose disease has recurred after a stem cell transplant or who are resistant to interferon alpha-based therapy.

"Gleevec has been as effective, if not more so, in children with CML as it has been in adults with CML," said Brian Druker, M.D., JELD-WEN Chair of Leukemia Research at Oregon Health & Science University Cancer Institute and a Howard Hughes Medical Investigator. "This offers great hope for children with CML in its chronic phase."

Druker, in collaboration with scientists at Novartis, helped develop Gleevec to target the molecular cause of CML. Studies have shown Gleevec to be much more effective and substantially less toxic than interferon for adult patients with newly diagnosed CML.

Gleevec was approved for the treatment of pediatric CML under the FDA's accelerated approval program. The program helps make products for serious or life-threatening diseases available earlier in the development process by allowing approval to be based on a promising effect of the drug.

Today's accelerated approval of Gleevec for use in children is based on extrapolation of results from Gleevec-treated adults with CML together with good responses in a small number of children.

In 2001 the Food and Drug Administration broke a record for cancer therapy approval by fast-tracking Gleevec, approving it in less than three months for patients who failed interferon treatment. In 2002 the FDA approved Gleevec as an effective treatment for gastrointestinal stromal tumors, a deadly form of intestinal cancer that, until then, had been difficult to treat.

Earlier this year, an editorial published in the New England Journal of Medicine dubbed Gleevec the "gold standard" treatment for CML. Recent studies also have found Gleevec to be effective in treating hypereosinophilic syndrome, a rare and often fatal blood disorder.

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