FDA Grants Breakthrough Status to CAR-T Therapy for Pediatric Brain Tumor

The United States Food and Drug Administration (FDA) has granted breakthrough therapy designation to treatment with an investigational B7-H3 targeting autologous CAR-T cell therapy for pediatric patients with diffuse intrinsic pontine glioma, an incurable pediatric brain tumor, according to a news release from BrainChild Bio, Inc.

The FDA grants breakthrough therapy designation to experimental medicines that show early signs that they lead to a major improvement for the treatment of patients with serious or life-threatening conditions. Medicines with this designation receive extra support and faster review from the regulatory agency, including more meetings with the FDA and the ability to submit parts of their application early. In turn, this can lead to a faster development and review process.

“Breakthrough therapy designation gives us the possibility to accelerate the development path for BCB-276 as a CAR-T cell therapy that can potentially transform the treatment of diffuse intrinsic pontine glioma,” stated Dr. Michael Jensen, founder and chief scientific officer of BrainChild Bio, in the news release. “This designation is a major milestone for the children and families afflicted with these devastating brain tumors and represents a new paradigm for treating central nervous system brain tumors in children and adults, including a large number of patients suffering with glioblastomas and brain metastases.”

This regulatory decision was based on overall survival data from the phase 1 BrainChild-03 trial — conducted by BrainChild Bio’s academic partner, Seattle Children’s, and recently published in Nature Medicine — which evaluated the treatment in children and young adults with diffuse intrinsic pontine glioma.

Notably, the company went on to share in the release that they are preparing to advance BCB-276 to a phase 2 multi-center, pivotal registration trial to support a potential biologics license application to the FDA for patients within the population. According to the official FDA website, fda.gov, a biologics license application is a request submitted to the FDA asking for approval to sell and distribute a biologic treatment — such as a vaccine or cell therapy — in the United States.

This clinical plan was created after BrainChild Bio and the FDA agreed on next steps during a scheduled meeting in late 2024, the release notes.

“This designation is an important milestone for Seattle Children’s and demonstrates our continued momentum in pediatric brain cancer research,” Dr. Jeff Sperring, CEO of Seattle Children’s, noted in the release. “We harness the power of research to bring potential cures to kids faster, and we’re excited by the early promise shown by our work with BrainChild Bio to advance a potential CAR-T therapy.”

Diffuse intrinsic pontine glioma is an aggressive brain tumor found in a part of the brainstem called the pons. It affects approximately 300 children each year in the United States, most commonly between ages 5 and 10 years. Moreover, according to the release, the current standard treatment for patients with diffuse intrinsic pontine glioma is radiation, which helps relieve symptoms but does not cure the disease. On average, children survive approximately 11 months after diagnosis.

Treating diffuse intrinsic pontine glioma is difficult due to its sensitive location in the brainstem, its tendency to grow and spread into healthy brain tissue, and the presence of the blood-brain barrier, which blocks many treatments from reaching the tumor.

To address these unmet needs within the population, investigators are evaluating this new approach which involves delivering CAR-T cells directly into the fluid around the brain and spine. Using an implanted device, doctors can give repeated doses of CAR-T cells that travel through the brain’s fluid pathways to reach the tumor. This method allows the treatment to directly target the tumor while avoiding any on-target, off-tumor toxicities, offering more durable and sustained efficacy.

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