Biotech firm to advance novel CAR T-cell therapy for rare, fatal pediatric brain tumor

A clinical-stage biotechnology company will advance a novel autologous chimeric antigen receptor T-cell therapy that targets the immune checkpoint B7-H3 for diffuse intrinsic pontine glioma, or DIPG, a rare incurable pediatric brain tumor.

In a press release, manufacturer BrainChild Bio, based in Seattle, said it plans to investigate BCB-276 in a pending multicenter, phase 2 clinical trial to be conducted by Seattle Children’s, with the goal of accelerating a path toward submission of a Biologics License Application to be granted by the FDA.

DIPG, a tumor that arises in the pons, affects approximately 200 to 300 children per year in the United States. There are no current treatments beyond palliative radiation, with overall life expectancy post-diagnosis estimated at 8 to 11 months.

Evidence for the advancement of BCB-276 arose from preliminary data within a phase 1 study initiated and conducted by Seattle Children’s that examined SCRI-CARB7H3(s), the research cell product from which BCB-276 was derived.

BrainChild-03 was a single-center, dose-escalation phase 1 clinical trial of repetitive

intracerebroventricular (ICV) dosing of SCRI-CARB7h3(s), in children with recurrent/refractory central nervous system tumors and DIPG.

Data from a subset of 21 enrollees included patients who began their CAR T treatment after disease progression (n = 12) those who began their CAR T treatment before disease progression (n = 9).

Preliminary safety analyses showed that repetitive ICV dosing in up to 10x10 cells was well-tolerated as an outpatient regimen, while preliminary efficacy analyses found the median time from diagnosis to death for all 21 patients treated was 19.8 months.

Additionally, per data cited in the release, three patients who commenced treatment prior to disease progression remained alive at 44.6 months, 45.6 months and 52.5 months, respectively, from their time of diagnosis.

“We are very pleased to have a solid path forward for our clinical development of BCB-276 in [diffuse intrinsic pontine glioma], enabling us to continue our progress for children and families struggling with this devastating brain cancer that currently has no approved treatments,” Michael Jensen, MD, founder and chief scientific officer of BrainChild Bio, stated in the release. “We look forward to continuing to work with the FDA and to generate the additional data required to support a successful [investigational new drug] submission.”