Critical Side Effect Study to Assess the Combination of Trametinib and Everolimus

Treatment offers hope for most common type of brain tumor in children

Hackensack Meridian Children’s Health is participating in a trial studying the side effects of combining two drugs, trametinib and everolimus, in treating pediatric and young adult patients with high-grade gliomas. Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center — a member of the global Pacific Pediatric Neuro-Oncology Consortium (PNOC) research group — is one of only 11 sites participating in this PNOC clinical trial for children and young adults. Derek Hanson, M.D., chief, Pediatric Neurology-Oncology, Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, serves as PNOC principal investigator.

“High-grade gliomas account for 15-20 percent of all brain tumors in children, and more research is needed to identify treatments that may improve long-term survival rates,” said Dr. Hanson. “This study will provide essential data to guide the use of this combination of drugs in pediatric patients with recurrent high-grade gliomas and may also support renewed interest in further development in other clinical settings.”

High-grade gliomas, brain tumors that originate from glial cells, which support and nourish neurons in the brain, are the most common type of brain tumor occurring in children. These two medications have previously been used in adults, but this is the first time researchers have sought to use this pairing of medications in children. In this phase I trial, investigators are studying the side effects and optimal doses for these medications.

Trametinib is a drug that acts by targeting a protein in cells called MEK and disrupting tumor growth. Combining it with everolimus, a drug that may block another pathway in a tumor’s growth, may be effective in disrupting the replication of tumor cells. Patients will receive customized dosages with treatment repeated for 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity.

Joseph M. Sanzari Children’s Hospital is also participating in a related PNOC study which will test the use of immunotherapy treatments nivolumab and ipilimumab in treating recurrent high-grade gliomas in children. Derek Hanson, M.D., and Katharine Offer, M.D., are recruiting for that study.

Learn more about pediatric oncology advancements at Hackensack Meridian Children’s Health.