NCI Funding Cut Forces Pediatric Brain Tumor Consortium to Halt Clinical Trial Enrollment
Key Insights
The National Cancer Institute has paused funding for the Pediatric Brain Tumor Consortium, forcing children's hospitals and cancer centers to halt enrollment in six active pediatric brain tumor trials.
The PBTC, which includes more than a dozen children's hospitals across the U.S. and Canada, will lose its $4 million annual funding when the current award expires in March 2026.
Five trials evaluating novel therapies for pediatric brain cancers, including a CAR T-cell therapy for ependymoma, are now suspended despite brain tumors being the leading cause of cancer-related death in children.
Children's hospitals and cancer centers across North America have been forced to suspend enrollment in critical pediatric brain tumor clinical trials after the National Cancer Institute paused funding for the Pediatric Brain Tumor Consortium. The decision affects six active studies, including five trials evaluating novel therapies for pediatric brain cancers and a CAR T-cell therapy for ependymoma, a condition with no effective treatments for children.
The Pediatric Brain Tumor Consortium, established by the NCI in 1999, includes more than a dozen children's hospitals and academic centers across the United States and Canada. The network conducts novel phase 1 and 2 clinical trials for individuals from infancy to 21 years old with primary central nervous system tumors.
Funding Transition Creates Treatment Access Concerns
The NCI announced it is increasing support for the Pediatric Early Phase Clinical Trials Network and stated that "the PBTC will not be able to apply for another 5-year funding award to continue its funding beyond March 2026." The consortium currently receives approximately $4 million annually from the NCI, according to Dr. Ira Dunkel, a pediatric oncologist who chairs the consortium.
The Pediatric Early Phase Clinical Trials Network comprises 42 children's hospitals and cancer centers in the United States, Canada, and Australia, with a mission to support early phase trials of pediatric anti-cancer agents. The NCI has committed to "ensuring that ongoing PBTC trials are completed wherever feasible and appropriate, either through direct close-out support to the PBTC after its current funding ends or by transferring studies to the PEP-CTN for completion."
Clinical Impact on Patient Access
Dr. Dunkel expressed concerns about the transition process, stating that some patients would likely lose access to trials and the process would not "be a seamless transition." He is currently exploring whether to transfer PBTC trials to the Pediatric Early Phase Clinical Trials Network.
The impact extends beyond U.S. borders, with particular concerns for Canadian patients. Dr. Daniel Morgenstern, Associate Professor at the University of Toronto, noted that the change is "particularly unfortunate for patients in Canada who may now completely lose access despite The Hospital for Sick Children and Ste Justine in Montreal only recently having joined PBTC."
Advocacy Response Highlights Clinical Urgency
Patient advocates have expressed alarm over the funding decision's implications for families facing pediatric brain tumors. Parvati Tiwari, Chief Medical Officer at SURYA (search) and a patient family advocate, emphasized that brain tumors remain the leading cause of cancer-related death in children. She described the PBTC as "a cornerstone of early-phase clinical research in pediatric brain tumors, giving families access to innovative, life-extending trials, often the only options when standard treatments fail."
Tiwari warned that the consortium's closure represents more than an administrative change, stating that "without it, progress stalls and children pay the price." She highlighted concerns that families will be "left with fewer options, fewer trials, and fewer chances for better outcomes."
Research Infrastructure at Risk
The PBTC has served as what advocates describe as "a vital bridge between researchers and clinicians" and "a catalyst for the rapid translation of scientific discovery into treatment." The consortium's role in facilitating early-phase clinical research has been particularly important given the limited treatment options available for pediatric brain tumor patients when standard therapies fail.
Dr. Morgenstern anticipates that while some studies may be incorporated into the PEP-CTN framework, "overall it will reduce the number of trials being done and slow progress towards new treatment options." This concern reflects broader worries about maintaining the momentum of pediatric brain tumor research during the transition period.