By Michael Rytting and Sara Farris
Recently, the U.S. Food and Drug Administration (FDA) approved the use of Gleevec in combination with chemotherapy to treat newly diagnosed children with Philadelphia chromosome-positive acute lymphocytic leukemia (Ph+ ALL).
While this is good news, it comes more than 10 years after it was approved in adults and about 8 years after I treated my first pediatric patient with chemotherapy plus Gleevec followed by a stem cell transplant. This young patient came here from the Philippines with Ph+ ALL, and I was able to treat her 'off-label' since Gleevec had already been approved in adults and was well-tolerated. Today she is cured of cancer.
Historically, high-risk Ph+ ALL patients received chemotherapy and a stem cell transplant. However, in 2009, a study in the Journal of Clinical Oncology showed that the addition of Gleevec to chemotherapy increased the 3-year survival without relapse from 35% to 80.5%. In retrospect, perhaps my patient might not have needed the transplant (and its associated risks) to be cured of her cancer.
For the most part, this latest approval doesn't really change the therapy for children with this disease. In fact, we have already moved forward with enrolling patients on similar therapies in clinical trials that involve newer variations of Gleevec. The new drugs may be more effective or have fewer toxicities.








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