CHLA receives $410,000 grant for 'first in childhood leukemia' multi-site study

Childrens Hospital Los AngelesJul 21 2010

J. Eric Bubbers, PhD, of the Saban Research Institute at Childrens Hospital Los Angeles, has received a $410,000 grant from Phase One Foundation. The award will fund a "first in childhood leukemia" multi-site study in children with relapsed or refractory acute lymphocytic leukemia (ALL) or acute myelogenous leukemia (AML). Dr. Bubbers is the administrative director of Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL), an academic research consortium consisting of 33 member institutions in four countries.

"Both ALL and AML are cancers of the blood and bone marrow, with ALL being the most common cancer occurring in children," explains Dr Bubbers. "These diseases begin when a single cell changes from a normal cell to a cancer cell. Patients receive treatment - chemotherapy and/or radiation - with the hope of achieving remission. But sometimes that doesn't happen, or the remission doesn't last."

A child is in remission when he or she is not showing any signs or symptoms of disease. But sometimes the cancer cells return. This condition is called relapse. Other patients undergo treatment but not all the cancer cells are gone from their body. In this case, the patient has refractory disease. When a child has relapsed or refractory disease, different treatments may be required.

The TACL consortium is studying a new alternative treatment for these patients. Gene-targeted therapy, a type of personalized medicine, is based on the specific type of chromosomal changes occurring in a patient's cells. Certain genetic mutations, those of FLT3 and c-Kit, are associated with a particularly poor prognosis. This multi-center study will use a new drug that has been expressly optimized to inhibit FLT3 and c-Kit and has been effective in treating adults with these diseases. The goal of the phase I study is to determine a safe and biologically-active dose in young patients.