CAR T Cell Therapy for Pediatric Cancer

This trial tests a new treatment for children and young adults with solid tumors that have returned or are not responding to other treatments. It uses a special kind of immunotherapy, called B7H3 CAR T Cell Immunotherapy, where a participant's own immune cells are modified to better find and fight cancer cells. The trial includes two treatment plans: one uses modified cells to target a single cancer marker, and the other targets two markers, to determine which is more effective. Children or young adults with a relapsed or stubborn solid tumor, who have recovered from past treatments, might be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

The trial protocol does not specify if you need to stop all current medications, but it does require stopping certain treatments like chemotherapy, biologic therapy, and corticosteroids at least 7 days before enrollment if no apheresis product is available. It's best to discuss your specific medications with the trial team.

Research has shown that B7H3 CAR T cells are generally safe for people. These cells target a specific marker on cancer cells and are well-tolerated. Tests in patients with solid tumors revealed only mild side effects and no serious safety issues. Some patients experienced cytokine release syndrome, a manageable reaction where the immune system becomes very active.

For B7H3xCD19 CAR T cells, research also indicates safety. These cells target two markers on cancer cells, enhancing the immune system's ability to fight cancer. Studies found that they can multiply and remain in the body without causing serious problems.

Sometimes, B7H3xCD19 CAR T cells are used with pembrolizumab, a drug that aids the immune system in fighting cancer. Early research shows this combination is safe but requires close monitoring.

Unlike the standard chemotherapy and radiation treatments for pediatric cancer, B7H3 CAR T Cell Immunotherapy uses a cutting-edge approach that harnesses the body's own immune system. This treatment works by genetically modifying T-cells to specifically target and attack cancer cells expressing the B7H3 protein. Researchers are excited because this approach can offer a more precise attack on cancer cells with potentially fewer side effects. Additionally, one arm of the treatment combines this with pembrolizumab, a drug that helps enhance the immune response, potentially boosting the treatment's effectiveness even further.

Research has shown that CAR T cells targeting B7-H3 are highly effective against solid tumors in children. Studies have found that these CAR T cells can significantly slow tumor growth in early tests. In mice, treatment with B7-H3 CAR T cells improved survival rates, with some mice living over 130 days. In this trial, participants may receive B7H3xCD19 CAR T cells, which research indicates grow well in patients and could help fight tumors. Another arm of this trial will test the combination of B7H3xCD19 CAR T cells with pembrolizumab, an immune therapy, which might enhance their effectiveness. Some studies have shown promising results with similar combinations.¹³⁶⁷⁸