A startup spawned from Seattle Children’s Hospital has raised a $116 million series A round to advance cell therapies for devastating childhood brain tumors.
BrainChild Bio secured the funds from Seattle Children’s and new investor WRF Capital—the investment vehicle for Washington Research Foundation—which BrainChild CEO Steven Brugger described as a “unique and committed” syndicate that highlights the challenge of corralling capital for pediatric brain cancers.
Just three years after pediatric oncologist Michael Jensen, M.D., spun his work on CAR-T therapies out of Seattle Children’s to form BrainChild, the startup now holds enough cash to finish a pivotal phase 2 trial of lead candidate BCB-276 in a rare childhood brainstem cancer called diffuse intrinsic pontine glioma (DIPG).
“DIPG is a fairly common brain tumor in children, and brain tumors are the leading cause of cancer death in kids now,” Jensen explained to Fierce in an interview. Because the tumors grow on the base of the brain shielded by the blood-brain barrier, surgery and existing therapeutics aren’t options for these kids.
Radiation is the most common treatment, Jensen said, but it really only adds a few months of life. This approach is “entirely palliative,” and there's no other approved drug specifically for DIPG, the CEO said.
With 250 to 300 new cases each year, DIPG represents an urgent but manageable initial indication for BrainChild’s approach, which takes T cells from patients and genetically modifies them to attack cancer before reinfusing them into the central nervous system. The biotech’s second program is in the works for glioblastoma, an aggressive brain cancer with a much larger patient pool that also includes adults. The company is planning to launch a phase 1 glioblastoma trial next year.
“We're kids first, but not kids only,” Brugger said of Brianchild's strategy. In conversations with investors, he added, ears often perk up at the mention of glioblastoma, which has 15,000 new cases per year and also a severe unmet need.
For glioblastoma, the cells are engineered to hit three common tumor targets alongside EGFR and IL-13, Jensen said.
Unlike other approved CAR-Ts, which are typically only dosed once, BrainChild’s cells are meant to be given in multiple-dose regimens, Brugger said. So far, all FDA-approved CAR-T therapies are used for blood cancers, while efforts to harness the potent approach for solid tumors have been slow but steady. The first solid tumor CAR-T was only approved a few months ago in China, courtesy of Shanghai-based CARsgen Therapeutics.
A major challenge facing would-be tumor-destroying CAR-T cells is the tumor’s tough defenses, which the cells need to penetrate. On this front, BrainChild actually has things a bit easier than its peers working in other solid tumor types.
Glioblastomas lack the tough collagen matrix of tumors made from skin cells, Jensen said, and are more easily tracked down by CAR-T cells because the chemicals they exude are contained behind the blood-brain barrier.
Studies conducted by BrainChild have shown that when cells are injected into the opposite half of the brain, they move through the corpus callosum—a thick band of nerve fibers in the center of the brain—to find the tumor on the other side, Jensen explained. “These cells are very good, and they look like they eat the tumor from the outside in,” he added.
BrainChild has enjoyed the cooperation of the FDA as it has advanced its DIPG program, Jensen said, with the agency allowing the company to compare BCB-276 to natural history data rather than requiring a placebo arm.
“I think this is going to be one of the first times that the FDA has gotten behind that strategy, which is critically important for a patient population like this,” Jensen said. Otherwise, in a control arm with no CAR-T cells, “100% of kids will die,” he said.