BrainChild Bio launches pivotal trial of CAR T therapy for aggressive pediatric brain cancer

BrainChild Bio has begun a pivotal Phase 2 trial testing BCB-276, its experimental CAR T cell therapy targeting the B7-H3 protein, in children with diffuse intrinsic pontine glioma, a rare and aggressive brainstem tumor with few treatment options. The company develops CAR T therapies aimed at central nervous system tumors.

The trial, called ILLUMINATE, is an open label, single arm study looking at whether BCB-276 is effective and confirming its safety in children and young adults newly diagnosed with DIPG who have already completed standard focal radiation therapy. BrainChild designed it as a single arm, pivotal registrational study meant to speed the path toward filing a biologics license application for BCB-276, based on discussions with the FDA. The therapy also holds FDA Fast Track designation, adding another layer of support for expedited development and review. The study is running across six pediatric neuro-oncology centers in the US, all of which have either completed or are in the process of activation.

Michael Jensen, BrainChild’s founder and chief scientific officer, said advancing this pivotal study for children with DIPG has been the company’s central goal and reflects the team’s sustained effort toward its mission of fighting pediatric brain cancer. He said the company’s aim is to deliver breakthrough treatments that give these children a real chance at a cure without sacrificing quality of life.

BCB-276 is made from each patient’s own immune cells and delivered directly into the cerebrospinal fluid through an implanted reservoir-catheter device, rather than through the bloodstream. Patients receive doses roughly every two weeks, for up to 15 doses total over about seven to eight months. The trial’s primary endpoint is overall survival, with safety, tolerability, progression-free survival and radiographic response tracked as secondary measures.

Cori Abikoff, BrainChild’s vice president of clinical development, said families dealing with a DIPG diagnosis need new options urgently. She acknowledged the substantial difficulty of developing therapies for the disease but said the company is hopeful ILLUMINATE will move the field closer to a meaningful treatment advance for these children. She described the study as an important step in testing what BCB-276 can offer newly diagnosed DIPG patients, and said BrainChild is working closely with leading pediatric neuro-oncology investigators and study sites to run the trial with the urgency and rigor the disease demands.

The therapy grew out of work Jensen and his team originally did at Seattle Children’s, which BrainChild later licensed exclusively. In an earlier FDA-authorized study, the Phase 1 BrainChild-03 trial, the same CAR T cell therapy showed a manageable safety profile when given on an outpatient basis and produced early signs of encouraging survival outcomes, results that were published in Nature Medicine.

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Related Topics and Keywords

brain cancer, BrainChild Bio, CAR-T therapy, pediatric brain cancer