Courtesy Story, Congressionally Directed Medical Research Programs

The Congressionally Directed Medical Research Programs support research into new treatments for brain cancers affecting Service Members and their Families.

Brain and central nervous system tumors are among the most common cancers for adolescents and young adults, which include individuals between 15 and 39 years of age. The U.S. Department of Veterans Affairs designates brain cancers as presumptive cancers associated with military service.

With current standard of care, the 5-year survival rate for all brain tumor types is 33%, indicating a need for improved treatment options. The CDMRP supports research aimed to develop new treatment strategies for these deadly cancers.

Predicting New Therapeutic Targets for Diffuse Hemispheric Glioma

In fiscal year 2022, the Rare Cancers Research Program supported an Idea Development Award led by Anthony Wang, M.D. at the University of California, Los Angeles to identify new treatment targets for diffuse hemispheric glioma. Diffuse hemispheric glioma is an aggressive cancer occurring in the cerebral hemispheres of the brain and primarily affects adolescents and young adults.

“As a surgeon and a scientist working with one of the world’s deadliest diseases, sticking with our current standard therapies is just not enough for my patients,” Wang said.

Wang developed a computational resource called IRIS-Long to identify tumor-specific markers resulting from RNA dysregulation in cancer cells. As of August 2025, Wang and team compared healthy and cancer tissue samples from 15 patients. They uncovered unique, cancer-specific targets for T cell immunotherapy. This data informed a clinical trial for a new treatment funded by other sources.

“Though this project focuses on a specific subset of brain cancers, the concept is applicable to a wide range of human cancers,” Wang said. “With proof of concept demonstrated through our Rare Cancers Research Program project, we can now begin to think about expanding testing to include other forms of brain cancer, and other forms of cancer throughout the body.”

Engineered Virus Therapy for Diffuse Midline Glioma

In the United States, brain cancers account for 14.3% of all cancer cases in children. Tumor location and treatment may leave children with life-long intellectual and neurological impairments, placing a significant burden on the child and their family.

In fiscal year 2024, the Peer Reviewed Cancer Research Program funded a Clinical Trial Award led by Gregory Friedman, M.D., at the University of Texas MD Anderson Cancer Center to evaluate a new therapy for diffuse midline glioma. Diffuse midline glioma causes tumors to form in the middle structures of the brain, such as the brain stem or spinal cord, and predominantly affects children. Friedman initiated a phase 1 clinical trial to assess the safety and tolerability of oncolytic herpes simplex virus as a cancer treatment. This therapy uses a genetically engineered virus to target and kill specific cancer cells. The therapy also activates the patient’s immune system, leading to further destruction of cancer cells.

“Our project is developing a second-generation engineered herpes simplex virus that selectively infects and destroys tumor cells while sparing normal brain tissue and stimulating a localized anti-tumor immune response,” Friedman said.

As of February 2026, the study team obtained most regulatory approvals and anticipates beginning patient recruitment within the next few months. Positive results from this work would support later stage clinical trials and offer a new treatment option for children impacted by diffuse midline glioma.

“The safety, feasibility and immunological data generated will help shape future immunotherapy trial designs and accelerate development of similar approaches,” Friedman said. “This work also supports military health priorities by addressing brain tumors that can affect Service Members’ readiness and long-term care needs for Service Members, their Families and Veterans, ultimately contributing to better outcomes across civilian and military populations.”

Sharing Resources to Enable Future Discoveries in Brain Cancer Research

Embryonal brain tumors develop from fetal cells remaining in the brain after birth. These rare tumors account for approximately 3.3% of all brain tumors and primarily affect young children.

In fiscal year 2022, the Rare Cancers Research Program awarded a Resource Community Development Award led by Annie Huang, Ph.D., at the Hospital for Sick Children to update and expand the Rare Brain Tumor Consortium, a global resource that collects and stores biological samples and clinical data for future research.

“Support from the DOD has enabled the collection of clinical data, including treatment-related clinical data, from rare tumor patients globally,” Huang said. “This information has been invaluable in informing development of new paradigms of treatment.”

As of October 2025, Huang’s team successfully inventoried and audited 2,251 of the 3,000 existing samples to ensure sample integrity and improve access for investigators. The team also registered 25 new embryonal brain tumor cases in the biobank, which included samples, clinical data and accompanying analyses.

“Unlike cancers in other locations, treatment of a cancer in the brain has distinct implications for survivors,” Huang said. “A majority of children with rare brain cancers are very young. Current maximum therapy approaches leave unacceptable life-long brain and other organ damage for this highly vulnerable population.”

Huang also works closely with patients and their families, seeking their guidance on the consortium’s activities and priorities to increase their impact. A patient advocacy committee provided input for new education modules to inform families of advancements and resources available for children. Huang and team also plan to organize a virtual family conference to connect researchers with patients and their families as the consortium and biobank continue to expand.

Fiscal Year 2026 Opportunities Forthcoming

For fiscal year 2026, the U.S. Congress appropriated $1.27 billion in funding for 34CDMRP research programs, including the Peer Reviewed Cancer and Rare Cancers Research Programs, to support research to discover and develop new brain cancer treatments for Service Members, their Families, Veterans and the American public.

To receive updates regarding fiscal year 2026 funding opportunity announcements, please subscribe to email notifications through the electronic Biomedical Research Application Portal. You can also visit the homepage of the CDMRP website for updates and other organizational publications and news releases.