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Entrectinib, Roche’s New Precision Medicine Therapy, Shows Promise for Treating Pediatric Brain Tumors


For many years, clinical trials of experimental cancer drugs have primarily focused on treating adults. However, a new trial from Roche Holding AG demonstrates how the landscape is changing and opening the door to promising new treatments for kids with brain cancer.

Ahead of their annual meeting starting May 31, the American Society of Clinical Oncology has released results of an early phase clinical trial of Roche’s entrectinib. This drug compound targets mutations in the NTRK, ROS1 and ALK genes, which have been found to drive tumor growth in a small percentage of children with brain cancer and other types of solid tumors.

Although the trial results are preliminary, every patient whose tumor had these mutations has responded well to the treatment.

“It’s stunning,” Giles Robinson, lead author of the study and a pediatric brain tumor oncologist at St. Jude Children’s Research Hospital, told Bloomberg. “It speaks of the potency of targeted therapy when you have the target and the right drug.”

When the RACE Act goes into effect next year, more pharmaceutical companies will be joining Roche in including pediatric patients in their clinical trials. This new federal law will require pharmaceutical companies to test new cancer drugs in children if the molecular target it’s aimed at it is found in both adults and children.

“Clinical drug development in pediatric oncology has long been an afterthought, despite medical rationale,” says Dr. Joanne Salcido, vice president of research and advocacy at the PBTF. “Typically, it doesn’t happen until testing in adults is completed, if at all. Thanks to the RACE Act, regulatory incentives are in place to change that practice and move the timeline up -- so that more of these pediatric trials not only happen, they happen sooner as a forethought for children battling cancer.”

The Roche drug is currently under review by the U.S. Food and Drug Administration for both pediatric and adult use, with a decision expected by August.

Read more of Bloomberg’s coverage of the trial here.