Children With Leukemia Have New Rx

Gleevec approved for children with acute lymphoblastic leukemia

(RxWiki News) Acute lymphoblastic leukemia (ALL) is the most common blood cancer found in children. It’s diagnosed in nearly 3,000 children in the United States every year and can progress quickly if left untreated. These kids now have a new treatment option.

The US Food and Drug Administration has approved Gleevec to treat children newly diagnosed with Philadelphia chromosome positive (Ph+) ALL.

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Kids with Ph+ ALL have a genetic mutation that causes the bone marrow to make too many immature infection-fighting white blood cells. Proteins called tyrosine kinases are to blame and the result is a crowding out of healthy white blood cells which fight off infection.

Gleevec blocks tyrosine kinases, which in turn keeps cancerous cells from developing. The medication should be used in combination with chemotherapy to treat children with Ph+ ALL.

“We are pleased that the number of cancer medications for children is on the rise,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval is the result of continuous interactions among the FDA, the Children’s Oncology Group and the National Cancer Institute to provide new and better treatments to American children with cancer.”

This approval is based on a clinical trial conducted by the Children’s Oncology Group and sponsored by the National Cancer Institute. The trial involved 92 patients with very high-risk ALL, defined as having a greater than 45 percent chance of experiencing disease complications within five years of treatment.

The children were divided into five treatment groups, with each group receiving longer treatment with Gleevec in combination with chemotherapy. A total of 50 children received Gleevec for the longest duration, and 70 percent of these patients did not experience either disease relapse (return) or death within four years.

The trial also found that deaths decreased the longer a child was treated with Gleevec and chemotherapy.

Common side effects of this combination therapy included decreased levels of infection-fighting blood cells called neutrophils and decreased blood platelets, which assist in blood clotting, along with liver toxicity and infection.

Gleevec has been approved since 2001 to treat Ph+ chronic myeloid leukemia (CML). Since then, the FDA has approved the medication to treat children with Ph+ CML and adults with Kit (CD117)-positive gastrointestinal stromal tumors (GIST).

Gleevec is marketed by Novartis.

Review Date: 
January 25, 2013