Stem Cell + Nivolumab for Pediatric Brain Tumor

(Peds CHAMP1ON Trial)

This trial explores a new treatment for children with certain aggressive brain tumors that have recurred or resisted standard treatments. The researchers are testing a combination of stem cells and the drug nivolumab (an immunotherapy), administered either before or after surgery, to determine if they can shrink these challenging tumors. Children diagnosed with specific high-grade brain tumors and who have experienced tumor recurrence after initial treatments might be suitable candidates. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative approach.

The trial protocol does not specify if you need to stop taking your current medications. However, you must have recovered from any recent cancer treatments and be on a stable or decreasing dose of corticosteroids for at least 7 days before joining. It's best to discuss your specific medications with the study team.

Research has shown that nivolumab, a key component of the treatment under study, has generally been safe in past studies with children. It was used in various combinations and proved mostly safe in these trials. Some children experienced side effects, but these were usually manageable. Nivolumab has also been used for certain types of childhood cancers before, providing some confidence in its safety.

Researchers are excited about using stem cells combined with Nivolumab for pediatric brain tumors because it represents a novel approach in cancer treatment. Unlike traditional therapies that primarily target the tumor directly, this treatment leverages the body's immune system. Nivolumab is an immunotherapy drug that helps the immune system recognize and attack cancer cells more effectively, while ex vivo hematopoietic stem cells (exHSCs) may enhance this immune response. Additionally, the treatment is being tested in two different approaches: one administered before surgery to shrink the tumor and one after surgery to prevent recurrence, providing flexibility and potentially improving outcomes for young patients.

Research has shown that nivolumab holds promise for treating pediatric cancers, particularly those that are difficult to treat. In previous studies, nivolumab led to lasting improvements and extended the lives of children with tough-to-treat tumors. Nivolumab aids the immune system in identifying and attacking cancer cells. In this trial, researchers are studying two approaches: one arm administers nivolumab and specially prepared stem cells (exHSCs) after surgery or biopsy, and the other arm administers them before surgery or biopsy. While the results of this combination are still under investigation, each component has shown promise in improving outcomes for challenging brain tumors.²³⁵⁶⁷