Pediatric Studies Explore Drug and Molecular Therapies
When pediatric neuro-oncologist Amar Gajjar, M.D., joined the CERN Foundation leadership team at its inception, he took on a daunting challenge to find new and effective drugs to treat ependymoma.
The disease is typically treated with surgery and radiation because most existing chemotherapy agents are not very effective, explains Dr. Gajjar, also the director of the Neuro-Oncology Division at St. Jude’s Children Research Hospital. Ependymoma is the third most common brain tumor in children.
In collaboration with an international team of investigators, including CERN investigator Richard Gilbertson, M.D., Dr. Gajjar co-led a trial that discovered the drug 5-fluorouracil (5-FU), which is widely used for colon cancer, could have an impact in treating ependymoma if administered rapidly. This discovery in mice led to a
phase I clinical trial supported by the CERN Foundation that determined the drug is safe to administer to children. The trial included 26 patients who were 22 years old and younger with ependymoma that recurred after treatment.
“The drug showed modest activity against the tumors that were tested, but it has led to subsequent research that has tested chemotherapy drugs in combination with 5-FU,” Dr. Gajjar says. Such studies are still in development.
