Transforming Health Care through Innovative and Impactful Research

February 27, 2026

The Congressionally Directed Medical Research Programs fund research to accelerate therapeutic development for rare diseases affecting Service Members' health and readiness. In fiscal year 2026, the CDMRP received congressional appropriations to address rare cancers and several rare diseases and conditions, some of which the U.S. Department of Veterans Affairs designates as presumptive conditions of military service. Other diseases and conditions may develop without a known cause during service, limiting fitness for duty and reducing manpower, or may affect military Family members, impacting Warfighter deployability.

Researchers report that patients with rare diseases receive a confirmed diagnosis in five years, on average, after symptoms begin. Developing treatments for rare diseases means working with small and globally dispersed patient populations and limited data, requiring highly creative and collaborative teams to identify and test new interventions. The CDMRP supports research in unique treatment approaches and innovative clinical trial designs for rare diseases, overcoming these challenges and accelerating treatment development for patients with few options.

According to the National Institutes of Health, a rare disease affects fewer than 200,000 people in the United States; however, with more than 10,000 conditions identified, rare diseases affect millions of people. Drugs approved by the U.S. Food and Drug Administration are available for approximately five percent of rare diseases.

Direct-to-Patient Tissue Donations Establish a Publicly Available Rare Cancer Registry

In fiscal year 2020, the Rare Cancers Research Program funded a Resource and Community Development Award, led by Jesse Boehm, Ph.D., at Massachusetts Institute of Technology, to expand access to rare soft tissue sarcoma patient samples by creating a publicly available resource for the scientific community.

Boehm's team formed partnerships with the rare cancer community and developed an outreach strategy incorporating social media and distributed printed materials to reach as many patients recently diagnosed with sarcoma in the United States and Canada as possible. Patients volunteer to participate in the ongoing clinical research study by enrolling online. The researchers then send a sample kit to the participant's clinician for direct-to-patient collection of tumor tissue. The clinician sends the sample to Boehm's laboratory, where the research team grows cells and organoids from the tissue and conducts sequencing and genomic analyses to characterize mutations and tumor composition and identify potential novel drug targets.

"By giving the power to patients, we've received samples from dozens of unique states and hospitals, created numerous models that can be shared with the international community, and discovered novel drug targets by performing large scale drug repurposing screens," Boehm said.

As of June 2025, 95 patients spanning at least four different subtypes of soft tissue sarcomas enrolled in the study, exceeding the study's proposed target of 74.

"We believe this [study] has created a framework that can now be scaled to many other rare cancers, resources that empower labs everywhere, and the starting points for therapeutic trials that civilians, Service Members and Veterans can participate in," Boehm said.

Home-Based Strategies Increase Patient Participation in Epidermolysis Bullosa Clinical Trials

In fiscal year 2021, the Peer Reviewed Medical Research Program funded a Focused Program Award, led by Anthony Oro, M.D., Ph.D., at The Leland Stanford Junior University, to create a universal manufacturing platform to serve as the foundation for personalized wound tissue regeneration therapies for epidermolysis bullosa.

Epidermolysis bullosa involves fragile skin that easily tears, blisters, and scars, often caused by inherited mutations in the gene encoding collagen. Disease symptoms vary from minor wounds on the skin surface to severe blisters and sores affecting internal organs, such as the esophagus, with links to increased risk of skin cancer and early death. The platform developed by Oro and team produced personalized tissue stem cells to treat these wounds.

"The CDMRP [award] allowed us to de-risk many aspects of the therapy and to start the conversation with the FDA regulatory agency," Oro said. "This has been invaluable to bring the therapy closer to clinic."

Oro's work also demonstrates potential for translation to other types of wounds, including combat wounds experienced on the battlefield.

"While the therapy focuses on helping the rare disease patients, it has broader impacts to heal poorly closing wounds for civilians, Service Members and Veterans due to injury or degeneration," Oro said.

In addition to using a platform for developing novel wound therapies, Oro and team are working to expand an existing epidermolysis bullosa patient registry. By adding a tissue bank of skin biopsies from wounds and including more well-characterized patient samples, the registry will accelerate development of Oro's therapies and recruitment of patients for clinical trials.

Oro plans to incorporate social media outreach and relationships with national organizations to connect with as many patients as possible. Patients who enroll in the registry will submit samples for genetic testing through the mail. The patient or their caregiver will also provide wound photos taken using a mobile phone, and complete secure online surveys to document symptoms, such as pain, itch and wound size, over time. The remote, home-based approaches will decrease burden of travel for patients and allow Oro's team to reach patients who might not otherwise be able to participate in the registry.

Repurposing Existing Drugs for Treatment of ALS

According to the U.S. Department of Veterans Affairs, amyotrophic lateral sclerosis, or ALS, affects individuals who served in the military 1.5 times more frequently than those with no service history. In 2008, the VA established ALS as a presumptive condition of military service.

In fiscal year 2018, the Amyotrophic Lateral Sclerosis Research Program funded a Therapeutic Development Award, led by Laura Ranum, Ph.D., at the University of Florida, to test metformin, an FDA-approved drug for type 2 diabetes, as a potential therapeutic for ALS. Repurposing existing drugs can shorten development timelines, moving new treatments to patients faster.

"The support provided by the CDMRP's ALSRP has been critical in advancing our work on ALS." Ranum said. "ALS is a rare neurodegenerative disease that affects nerve cells in the brain and spinal cord that control muscle movement, leading to paralysis and death. There are no effective treatments for most forms of ALS."

Using preclinical models with mutations in the C9orf72 gene, the most common genetic cause of ALS, the study found that metformin improved function by significantly reducing harmful protein aggregates that accumulate in the brain and spinal cord and contribute to disease.

With follow-on funding from a fiscal year 2022 ALSRP Pilot Clinical Trial Award, Ranum and her team are translating their preclinical findings closer to patients. The phase 2 clinical trial, which included 23 patients with the C9orf72 mutation, evaluates safety and efficacy of metformin as a treatment for ALS by measuring changes in toxic protein aggregates and other ALS biomarkers in patient bodily fluids over 24 weeks.

"Disease mechanisms and biomarker work associated with the [ALSRP-funded] project may reveal new clues to how ALS and similar diseases develop and how they can be tracked and monitored during clinical trials," Ranum said. "Success of our project would lead to a safe, affordable and readily available treatment for ALS and potentially other diseases."

Advancing Personalized Treatment of Duchenne Muscular Dystrophy

In fiscal year 2021, the Duchenne Muscular Dystrophy Research Program funded an Idea Development Award, led by Andrew Fisher, Ph.D., at the University of California, Davis, to develop a new approach for correcting mutations in the DMD gene that cause absent or defective dystrophin protein.

"Duchenne muscular dystrophy can be caused by mutations in the DMD gene, which codes for the dystrophin protein that connects the muscle cell's internal scaffolding, known as cytoskeleton, to the outside matrix, providing structural stability and protecting muscle fibers from contraction-induced damage," Fisher said.

The team used a human RNA-editing enzyme to target and correct regions of the DMD gene with single-base precision, such that the corrected gene could produce full-length dystrophin protein.

"This [approach] can potentially lead to a one-time treatment to alleviate the disorder," Fisher said.

Promising preliminary findings of this early research to selectively repair disease-causing mutations in the DMD gene lay the groundwork for potential applications to address other rare diseases caused by single gene mutations.

"This method, when fully developed, would also be able to treat many genetic diseases beyond Duchenne muscular dystrophy, which would benefit Veterans and civilians," Fisher said.

FY26 Funding Opportunities Forthcoming

For fiscal year 2026, the U.S. Congress appropriated $1.27 billion in funding for 34 research programs, of which six programs and multiple topics within the Peer Reviewed Medical Research Program address rare diseases and conditions. The CDMRP will release funding opportunities as soon as available for each program.

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.

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Last updated Thursday, February 26, 2026