Illustration of CAR T-cell immunotherapy.

Research hospital Seattle Children’s announced today the spinout of its for-profit company BrainChild Bio, Inc., which will develop CAR T-cell therapies targeting pediatric central nervous system (CNS) cancers. Underpinning the new company will be a license to the CAR T-cell technology that has been developed at Seattle Children’s and will look to accelerate the CAR T-cell therapy and clinical translational efforts of Michael Jensen, MD, and others at Seattle Children’s Therapeutics.

Seattle Children’s Therapeutics, launched in 2012, has managed clinical trials of advanced immunotherapies for the treatment of leukemia, lymphoma, brain tumors, and other solid tumors enrolling more than 500 patients over that time. BrainChild Bio will look to build upon this base to broaden access to these therapies via collaborations with biotech companies. Initial funding for BrainChild Bio is being provided by Seattle Children’s.

“As one of the largest dedicated pediatric institutes in the country, we are extremely proud of the discoveries, clinical trials, and cures that have come from inside our own walls,” said Jeff Sperring, MD, CEO of Seattle Children’s. “We also know there are kids around the globe that cannot come to Seattle to be treated. We believe this gives us the best opportunity to accelerate this technology bringing potential cures to kids faster.”

As its name implies, BrainChild Bio will focus on pediatric brain tumors with its CAR T-cell therapy program focusing on diffuse intrinsic pontine glioma (DIPG), an incurable type of childhood cancer that forms in the brainstem. This program will be enhanced and supported by four ongoing CAR T clinical studies—called BrainChild-04—being conducted by Seattle Children’s Therapeutics which are seeking to validate safety and confirm early efficacy of CAR T-cell therapy against four targets in pediatric CNS tumors.

Once the company has demonstrated clinical proof-of-concept against DIPG, it will seek both a pediatric registration for this indication and extend the application of the program to include additional hard-to-treat brain tumors including glioblastoma and brain metastases.

“As a physician, I work with children and their families who have limited therapeutic choices to treat their tumors—and it is devastating that there are few safe and curative options for them,” said Nicholas Vitanza, MD, CNS CAR T-cell lead and DIPG research lead at Seattle Children’s. “Developing therapies that are tailored specifically for pediatric patients will generate better medicines to address CNS tumors while protecting these young patients and their developing bodies and minds.”

According to BrainChild Bio, its CAR T-cell therapy approach will leverage synthetic technologies that will include multiplex targeting and enhanced potency control. Key aspects of this approach include:

  • Multiple targets in a single CAR T-cell therapy to prevent tumor escape
  • Novel transgenes to increase potency that engage only when within the direct tumor environment
  • Switching technologies to control the CAR T cells directly within the tumor
  • CAR T design and manufacturing processes which have been proven over many years at Seattle Children’s Therapeutics
  • Novel CAR T-cell administration directly into the brain minimizing systemic toxicities and enabling regular repeat dosing to ensure prolonged presence of CAR T-cells and durable efficacy

“BrainChild Bio is founded with a mission to bring the best ideas forward to push the bounds of scientific discovery in service of children with cancer,” said Jensen, founder of BrainChild Bio who will also serve as its CSO. “For far too long, children have been deprioritized for commercialized medicines, and families have been left without options. We are steadfast in our commitment to cracking the code of harnessing CAR T-cell technology in CNS tumors and we are uniquely positioned to do so.”

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