BCB-276 for DIPG brain cancer named FDA breakthrough therapy
Developer planning launch of Phase 2 trial to test CAR T-cell therapy
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to the cell therapy BCB-276 as a potential treatment for diffuse intrinsic pontine glioma (DIPG), an aggressive type of brain cancer that mostly affects children.
The FDA awards such status to investigational therapies that are designed to treat serious diseases and that show early clinical evidence of potentially being better than available treatments. The designation aims to speed the development of important new therapies.
With breakthrough therapy status, BCB-276’s developer Brainchild Bio will get access to perks like more frequent communication with the FDA during the development 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 DIPG,” Michael Jensen, MD, founder and chief scientific officer of Brainchild Bio, said in a company press release.
Jensen called the designation “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.”
Phase 1 study found BCB-276 safe for children, young adults with DIPG cancer
The FDA’s award was based on data from a Phase 1 trial, detailed in a study published earlier this year, that showed that BCB-276 could be safely given to children and young adults with DIPG. That first-in-human Phase 1 study was conducted by Seattle Children’s.
Jeff Sperring, MD, CEO of Seattle Children’s, said the hospital is “excited by the early promise shown by our work with Brainchild Bio to advance a potential CAR-T therapy.”
“This designation is an important milestone for Seattle Children’s and demonstrates our continued momentum in pediatric brain cancer research,” Sperring said.
[Seattle Children’s hospital is] excited by the early promise shown by our work with Brainchild Bio to advance a potential CAR-T therapy.
BCB-276 is an autologous CAR T-cell therapy. In this type of therapy, T-cells, which are a type of immune cell, are first collected from a patient. Those cells are then engineered in a lab to equip them with a chimeric antigen receptor, or CAR — a human-made protein that directs the cells to attack a specific molecular target. The modified cells are then infused back into the patient.
This therapy specifically uses a CAR that targets a protein called B7-H3, which is found at high levels in DIPG cells. The treatment is designed to be infused directly into fluid-filled cavities inside of the brain.
Brainchild is planning the launch of a pivotal Phase 2 clinical trial to explore the efficacy of BCB-276 in DIPG. If positive, the company hopes to use the results as a basis to seek FDA approval for the therapy.
About the Author
