Our impact, together
Thank you.
You most likely know someone who has cavernous malformation (cavernoma, CCM). You know how life-changing better treatments and a cure would be.
As you’ll see in this impact report, with your support in FY2024-2025, we are closer to better treatments and a cure for CCM than ever before.
Thank you for your commitment to drive a cure and improve lives for all affected by CCM. Thank you for being part of our alliance. Together, we will create a permanent cure for CCM – available, accessible, and affordable for all.
FY2024-FY2025 Impact at a Glance
Driving Research for a Cure
- Led planning of a clinical trial for low-intensity focused ultrasound, a noninvasive treatment that halted the growth of 94% of CCMs in mice (Nature Biomedical Engineering, 2025)
- Launched the first coordinated research program dedicated solely to accelerating CCM drug discovery (CCM CureDriver™ Lab)
- Began negotiations for transfer of assets from Recursion Pharmaceuticals following SYCAMORE — CCM’s first industry-developed drug trial — which showed promising results in a subset of patients
- Convened 100+ CCM scientists at our 20th International CCM Scientific Meeting, the largest and longest-running conference dedicated to advancing CCM research
- 5 new treatments entered the global CCM clinical pipeline
Improving Care
- 3,000 patients seen at our network of recognized CCM Centers of Excellence and Clinical Centers
- 60 updated and expanded recommendations for CCM diagnosis, monitoring, and treatment published in Guidelines for the Diagnosis and Clinical Management of Cavernous Malformation (Neurosurgery, 2025).
Empowering Patients
- 75,000 CCM patients reached through education programs, support groups, community events, and online resources
Learn more about OUR VISION and MISSION, OUR VALUES, and OUR PROGRAMS
Advancing promising noninvasive CCM treatments and cures
A non-invasive potential treatment for CCM families: Focused Ultrasound
We accelerated the development of low-intensity focused ultrasound (LIFU) for CCM, a potentially groundbreaking noninvasive treatment. Preliminary
data presented at our 2023 International CCM Science Meeting showed that LIFU halted the growth of 94% of CCM lesions in a CCM mouse model. In 2024, we began planning a clinical trial of LIFU to determine if LIFU could one day be an effective treatment — or even a cure — for our CCM families, especially those with currently inoperable CCM lesions.
YOUR 2024-2025 IMPACT:
- In early 2024, we organized the stakeholders — the University of Virginia pre-clinical and clinical trials teams, the manufacturer NaviFUS, and the Focused Ultrasound Foundation — to introduce the idea of a clinical trial and create a plan.
- We initiated a meeting with the Food and Drug Administration (FDA) – approver of the trial – to educate them about CCM and the need to test LIFU. Only the Alliance to Cure could do this.
- The University of Virginia and the Alliance to Cure developed and revised the LIFU study protocol (the design of the clinical trial) to ensure FDA permission to move forward with a trial. Pending FDA approval, we will enroll CCM patients in a safety trial of LIFU by early 2027.
CCM CureDriverTM Lab: Expediting preclinical discovery and de-risking industry investment
CCM drug development has traditionally faced two systemic barriers: 1) access to CCM mouse models and 2) lack of incentives to test repurposed drugs for CCM. We launched our CCM CureDriverTM Lab in 2024 to bypass these roadblocks by developing a CCM mouse colony and conducting preclinical testing on repurposed drugs. Such programs are rare for patient-advocacy organizations to undertake because of the scientific expertise and expense involved, and we experienced setbacks in our first year of the program (2024). In 2025, however, we made progress in our program to expedite preclinical research.
FY2025 HIGHLIGHTS:
- We laid the groundwork for developing a CCM mouse colony in 2026 at a leading nonprofit biomedical research organization so that drug companies can more easily test their compounds without undertaking the time-consuming process of developing their own CCM mice. Our previous attempt in 2024 to establish a mouse colony at a different scientific research organization was unsuccessful. The colony is expected to be fully developed by the end of 2026.
- We contracted with an experienced CCM researcher to develop a 10-arm study protocol for testing five repurposed drugs and drug combinations in CCM mouse models to generate data for advancing these drugs through the clinical trial pipeline. (These drugs are known to interact with proteins and pathways involved with CCM.) Results are expected by the end of 2026.
A deep pipeline of potential treatments, built with our guidance and research tools
The CCM Clinical Trial Pipeline shows all known CCM treatments under development worldwide. Since 2023, five new treatments have entered the pipeline.
In 2024-2025, we drove treatment development through ongoing technical guidance and three critical resources: our International CCM Scientific Meeting, Patient Registry, and Biobank.
International CCM Patient Registry
Our Patient Registry is overseen by an external International Review Board. It is at ccmregistry.org
IN 2024-2025:
- 3,352+ surveys completed by patients for researchers
- 826 patients enrolled in our Registry, representing a 150% increase since 2023
CCM Biobank
Patients can consent to donate biosamples like blood, saliva, and resected cavernous malformation lesions for research when they join the Patient Registry. For scientists, the biobank is a powerful resource – a treasure.
IN 2024-2025:
- 158 patients consented to donate biosamples, growing by 24% since 2023
- 44 patients donated samples to our biobank, bringing the total to 1173
International CCM Science Meeting
In July 2025, we held our 20th International CCM Science Meeting in Toronto, convening more than 100 leading CCM scientists to share preliminary data, form collaborations, and deepen CCM understanding. Most major CCM research breakthroughs from the last 20 years have grown out of these meetings.
Finding a path forward after the REC-994 trial
In 2024, Recursion Pharmaceuticals shared results from their Phase 2 SYCAMORE trial, the first industry- sponsored clinical trial of a drug developed for CCM. The drug, REC-994, was found to be safe and well-tolerated, and there were encouraging exploratory efficacy trends, including decreases in lesion volume and improvement in overall function and CCM-associated symptoms. We had worked alongside Recursion for a decade to make this trial happen, from advising on trial design and educating the FDA, to mobilizing patients to enroll. However, in the spring of 2025, Recursion discontinued the REC-994 trial. Intent on understanding why encouraging trends were observed in some patients, we immediately began negotiations with Recursion to inherit assets from the trial. We finalized the negotiations in 2026 and will begin conducting analyses as to why a portion of trial volunteers responded well to REC-994. Our hope is that further analysis could provide a rationale for re-evaluating REC-994 as a viable treatment for some groups of patients.
More, better care options for CCM families
A network of experienced, multidisciplinary CCM care teams
Our CCM Center program recognizes 22 U.S. hospitals that we’ve designated as CCM Centers of Excellence or CCM Clinical Centers. CCM Centers offer experienced, multidisciplinary care for adults and children affected by CCM. The program makes it easier for families to locate specialists with a deep understanding of CCM.
YOUR 2024-2025 IMPACT:
- 3,000+ CCM patients served at CCM Centers annually (alliancetocure.org/ccmcenters).
- We recognized three more hospitals as CCM Centers: Texas Children’s, Seattle Children’s, and Mayo Clinic in Florida.
AJ, 6, three months after his brain surgery at a CCM Center of Excellence.
AJ’s family traveled 1,750 miles so thatAJ could get treatment for his CCM seizures at a CCM Center in 2025. AJ’s parents learned about the experienced, multi-disciplinary CCM care at CCM Centers after connecting with the Alliance.
__________________
“We know how lucky he is to have had such a great medical team, surgeon, and advocates at the Alliance.” – Jennifer, AJ’s mom
Lives Changed in 2024-2025
We provided support, information, and community to approximately 75,000 individuals affected by CCM in 2024-2025 through support groups, informational webinars, our comprehensive website (alliancetocure.org), online communities, and in-person events.
Key Achievements
- Our first CCM Family Weekend: In the summer of 2024, we hosted 70+children and adults living with CCM at a lodge in the Adirondacks for our inaugural CCM Family Weekend. Participants met others with CCM for the first time and left with lasting friendships.
- 1:1 Patient Navigation launched in April 2025: We have already connected nearly 100 individuals with complex cases to necessary care and resources.
- Key support groups added: In addition to our long-standing twice-weekly patient support groups, we began holding regular groups specifically for individuals affected by spinal cord CCM, individuals affected by brainstem CCM, young adults, women, men, and caregivers.
Awareness-Raising & Advocacy
State and Federal Advocacy Highlights (2024-2025)
- Secured an additional $300,000 in New Mexico state funding to improve care for CCM patients in New Mexico, leading to a total of $1 million allocated to CCM care since 2023. New Mexico has the highest concentration of CCM patients in the world.
- Organized CCM families to share their stories with U.S. Senate offices, leading to the correction of a critical mistake in the 2022 Inflation Reduction Act that had discouraged rare disease drug development.
- Successfully advocated the U.S. Department of Labor to include clinical trial participation in the Family and Medical Leave Act (FMLA).
Proud to be members of: American Brain Coalition, Coalition to Transform Clinical Trial Engagement, CZI Rare as One, Global Genes Leadership Council, NORD, Rare Disease Diversity Coalition, Rare Disease Legislative Advocates, Rare Diseases Clinical Research Network, Rare Epilepsy Network, Save Rare Treatments Task Force
Leadership & Financials
Ways to give
By check, mail to this address:
Alliance to Cure Cavernous Malformation
977 Seminole Trail Box 367
Charlottesville VA 22901
Through a donor advised fund (DAF)
Our EIN: 02-0600697
Our legal name: Angioma Alliance d/b/a Alliance to
Cure Cavernous Malformation
The address associated with our account:
Alliance to Cure Cavernous Malformation
977 Seminole Trail PMB 367
Charlottesville VA 22901
Stock
Our brokerage firm: Fidelity
Our account number: Z40135019
DTC number: 0226
Our account title: Alliance to Cure Cavernous
Malformation
The address associated with our account:
Alliance to Cure Cavernous Malformation
977 Seminole Trail PMB 367
Charlottesville VA 22901
Please email dwinchester@alliancetocure.org
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