PEP-CTN Chair Elizabeth Fox, MD
|
Pediatric Brain Tumor Consortium Funding Changes: Your Questions AnsweredIn the Fall of 2025, the National Cancer Institute (NCI) announced that the Pediatric Brain Tumor Consortium (PBTC) will no longer receive NCI funding after March 2026. Essentially, this means the PBTC will cease to exist.
PBTC’s work in early phase trials for children with brain tumors will move to the Pediatric Early Phase Clinical Trials Network (PEP-CTN), a broader network of early phase pediatric cancer clinical trials that operates under the Children's Oncology Group (COG). |
The NCI explained that the goal for this transition was to improve clinical trial development and expand access to pediatric brain tumor trials at more sites (PBTC operated across about 15 sites, whereas PEP-CTN operates across more than 40). But for many families, this shift felt jarring and raised questions about how the change would impact access to clinical trials.
“These trials mean hope for kids and families impacted by pediatric brain cancer. So the news that the NCI would no longer fund PBTC felt devastating,” said Lia Gore, M.D., Vice Chair of COG. “We want those families to know: PEP-CTN is doing everything possible to make this transition smooth, limit delays, and to continue to offer trials of innovative therapies with the potential to save lives.”
Dr. Gore, COG Chair Doug Hawkins, M.D., and PEP-CTN Chair Elizabeth Fox, M.D., address questions about this transition and share how PEP-CTN is working to make it as efficient as possible.
Can you tell us a bit about PBTC and their work?
Dr. Hawkins: PBTC was started in 1999 with the understanding that pediatric brain tumors are some of the most difficult cancers to treat. Brain tumors are:
- Collectively, the most common solid tumor in children under age 14.
- One of the deadliest forms of pediatric cancer.
- A type of cancer where standard treatments can cause devastating side effects. Surgery and radiation to treat brain tumors can impact a child’s ability to speak, see, learn, move and grow.
Research has led to the understanding that pediatric brain tumors are made up of many different molecular subtypes of cancer. This means there is no one-size-fits-all treatment, and there's a critical need for more targeted treatments with fewer side effects.
PBTC was launched with a focus on early phase trials (trials that examine new potential treatments to study their safety, dosage, and efficacy). The idea was that creating a collaborative network to test new ideas could lead to significant improvements in outcomes for these kids.
Since its inception, PBTC has designed and conducted more than 40 clinical trials for pediatric brain tumors. For example, PBTC-029 was a phase 1/2 trial that established the dosing, safety, and efficacy of selumetinib in recurrent pediatric low-grade glioma, the most common childhood brain tumor. It resulted in broad use in the community, further frontline COG studies, and investigation of other related new agents.
Will PBTC’s funding be transferred to PEP-CTN?
Dr. Hawkins: No. PEP-CTN will request supplemental funding from NCI to support the transition, but it will also need to revise its budget to take on additional trials. Any changes would be subject to NCI approval for additional funding.
Does PEP-CTN have experience and expertise in early-phase clinical trials for pediatric brain tumors?
Dr. Fox: Yes. At each hospital that offers PEP-CTN clinical trials, pediatric neuro-oncologists — specialists who treat these tumors every day — are closely involved in both caring for patients and running the trials. Fourteen of the fifteen PBTC sites are also PEP-CTN sites and PEP-CTN can add that site for specific trials. This means that when studies move into PEP-CTN, the same research teams and investigators who originally developed and led the trials and cared for the children continue their work within the network. Families can be confident that early-phase trials are guided by experts and the experience of a national clinical trials network.
How have PBTC and PEP-CTN managed this transition?
Dr. Fox: The transition is going very well. PBTC and PEP-CTN leadership are meeting frequently to:
- Ensure patients who are currently enrolled do not experience pauses or disruptions in care.
- Transition administrative and technical aspects (like imaging studies and data storage) from PBTC to PEP-CTN.
- Meet with patient advocacy groups to determine how we can best meet the needs of children with brain tumors.
- Build trial-specific teams for children with brain tumors. Increase the operational support team to manage the increase in trials and expand the neuro-oncology expertise within the PEP-CTN leadership team.
|
What’s happening to PBTC’s active studies during this transition?
Dr. Fox: PBTC stopped enrolling new patients in trials shortly after the transition was announced. This was a very difficult decision, but it was the safest decision. PBTC needed to ensure that no child would start the study and then experience pauses in treatment or issues with safety monitoring because of the transition. We are working hard to: |
Lia Gore, MD
|
"A few strong voices can truly make a difference — and we need those voices now more than ever. " |
- Reopen trials for new enrollment this summer.
- Ensure that children currently enrolled in studies complete them according to plan.
- Make sure that all patients who could be eligible for a trial are tracked during the transition, so no one misses the opportunity to receive additional therapy when trials reopen.
- Avoid major changes (scientific amendments) to studies that delay reopening because the science is already very strong.
- Activate a new trial. PBTC was close to opening a new trial before the transition was announced. PEP-CTN has taken on that study and the NCI has agreed that no further NCI reviews are needed, so we hope to activate that trial through the PEP-CTN soon.
Dr. Hawkins: As pediatric oncologists, we know that brain cancers remain some of the most difficult to treat — and COG has a vision to change that. We know this work is strongest when we do it together. This transition may come with challenges, but we will overcome them. Our focus remains on what matters most: running impactful trials that can lead to better treatments with fewer side effects and, ultimately, better outcomes for kids.
Dr. Gore: We hear your concerns, and we know that learning that any trial is pausing enrollment can feel like a loss of hope. We are doing everything we can to re-open trials under PEP-CTN.
It's also important for families to know that this isn’t the first time PEP-CTN and COG have taken on trials that started with other organizations. We’ve done this before, and we’re working hard to make the transition as efficient as possible.
Will this transition ultimately achieve the NCI’s vision to improve clinical trial development and expand access to pediatric brain tumor trials?
Dr. Hawkins: Once the transition is complete, we will be able to offer these studies at more institutions. Bringing these studies to more sites will take time, but they will ultimately be available at more institutions — and many families won’t have to travel as far for care.
Dr. Fox: We are working together to achieve better outcomes for children with brain tumors. We must all do our part, and ask what else we can do. The NCI’s vision cannot be achieved if federal funding for childhood cancer clinical research is cut. Federal funding is critical to support practice-changing clinical trials that improve the outcomes and decrease the side effects of cancer therapy.
For families who can’t enroll in PBTC trials right now, are there other options?
Dr. Fox: Yes, there are a number of organizations offering pediatric brain tumor clinical trials right now, including:
- The Collaborative Network for Neuro-oncology Clinical Trials (CONNECT), which conducts clinical trials in high-risk pediatric brain tumors such as DIPG, other high-grade gliomas, and embryonal tumors.
- Pediatric Neuro-Oncology Consortium (PNOC), dedicated to bringing new therapies to children and young adults with brain tumors.
- Many large pediatric hospitals offer studies led within their institution.
- The PEP-CTN looks forward to cooperation with these groups and the Children’s Oncology Group CNS Committee to provide access to trials of the most promising innovative therapies for children with brain tumors.
What can I do to support ongoing funding for pediatric cancer research?
Dr. Hawkins: Philanthropy always has been and continues to be vital for progress against pediatric cancer. Gifts of any size make a difference. They support the collecting and storing of samples, designing and conducting clinical trials, data analysis, and many other things. Donations to COG don’t just support kids in one community, but at many institutions around the world.
Dr. Gore: You can also use your voice. Share your story and encourage others in your community to do the same. Reach out to your elected leaders and tell them why funding for pediatric cancer is important to you. It’s like the Margaret Mead quote: “Never doubt that a small group of thoughtful, committed citizens can change the world. Indeed, it is the only thing that ever has.” A few strong voices can truly make a difference — and we need those voices now more than ever.
Clinical trials for children with brain tumors must continue without interruption. Your gifts help COG design, launch, and sustain the early-phase studies that give families hope when options are limited.