FDA Approves Gleevec for Type of Childhood Leukemia

The US Food and Drug Administration (FDA) has approved Gleevec (imatinib) to treat children who are newly diagnosed with a form of acute lymphoblastic leukemia (ALL). The treatment is for Philadelphia chromosome (Ph) positive ALL, a type of leukemia caused by a specific genetic change. The drug is used in combination with chemotherapy. It works by killing leukemia cells that contain the damaged chromosome.

The drug was approved following a clinical trial of 92 children and young adults with Ph-positive ALL. The patients were divided into 5 treatment groups, with each group receiving imatinib plus chemotherapy for a different period of time. Of the 50 patients who received imatinib for the longest period, 70% lived for at least 4 years without a relapse. In addition, deaths decreased with the increasing duration of imatinib and chemotherapy treatment.

“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 at the FDA Center for Drug Evaluation and Research, in a statement.

According to the Children’s Oncology Group, the standard treatment for Ph-positive ALL has been a stem cell transplant followed by 3 to 6 months of chemotherapy. However, cure rates were less than 50% with this approach. Gleevec in combination with chemotherapy has doubled cure rates, and stem cell transplants are no longer automatically considered to be the best way to treat children with Ph-positive ALL.

The most common side effects in children with Ph-positive ALL treated with imatinib and chemotherapy included decreased levels of infection-fighting blood cells called neutrophils; decreased levels of blood platelets, which assist in blood clotting; liver toxicity; and infection.

Imatinib was previously approved to treat children with another type of leukemia, Ph-positive chronic myeloid leukemia (CML), as well as several cancerous and non-cancerous conditions in adults.

Gleevec is marketed by Novartis.

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