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news.BrainChild Bio's B7 homolog 3 (B7-H3) targeting autologous chimeric antigen receptor (CAR) T-cell therapy, BCB-276, has received the regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for treating diffuse intrinsic pontine glioma (DIPG).
Diffuse intrinsic pontine glioma is stated to be an incurable paediatric brain tumour. Credit: Natasha Connell on Unsplash.
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This marks a step forward in the development of treatments for this incurable paediatric brain tumour.
BrainChild Bio noted that a CAR T-cell therapy overcomes the challenges posed by DIPG, such as its location in the brainstem, the tumour’s infiltrative growth, and the intact blood-brain barrier during the progression of the tumour.
According to the company, BCB-276 is tailored to be administered by a locoregional delivery of targeted CAR T-cells into the cerebrospinal fluid directly via an indwelling reservoir-catheter device, allowing the cells to access the tumour bed.
This method of administration of B7-H3 CAR T-cell therapy was leveraged, and as a result, the outcomes from the BrainChild-03 Phase I trial, carried out by the company’s partner, Seattle Children’s Research Institute in the US, demonstrated an overall survival benefit in individuals with brain tumours.
BrainChild Bio chief scientific officer and founder Michael Jensen said: “We are very pleased to now also receive RMAT designation, less than one month after being granted breakthrough therapy designation from the FDA for our lead CAR T therapy, BCB-276, for the treatment of DIPG.
“Receiving designations from two independent reviews within the FDA further validates the positive CAR-T clinical results achieved by our team to date and the urgent need for a treatment for DIPG.”
The company is currently preparing to progress the therapy into a Phase II study.
This multi-centre, registration trial is intended to provide backing for a potential biologics licence application (BLA) to the US regulator for treating paediatric and young adults with DIPG.
This plan has been formulated following a Type B meeting with the agency, which took place last year.