Angioma Alliance

1. Fund, facilitate, and participate in cavernous malformation research to achieve a complete understanding of the disease and facilitate clinical drug trials and other treatment improvements.

2. Provide disease and resource information to educate and improve the lives of people affected by cavernous malformation, caregivers, health professionals, researchers, policymakers, the media, and the general public.

3. Foster and promote a caring community to provide support.

4. Get people involved in advocacy and active participation toward a cure.

5. Build and sustain a broad base of funding sources to adequately support our mission and goals.

To drive research, the Alliance to Cure Cavernous Malformation specializes in funding strategic research grants, bringing experts together, and creating shared resources. Our International Scientific Meeting, DNA/Tissue Bank, patient registry, and free genetic testing program are examples of ways in which we uniquely help basic and clinical researchers as well as clinical trials. Alliance to Cure has been the impetus for many of the research collaborations that exist today, including those between pharmaceutical companies working to develop better treatments and academics who can help them with this effort. We serve as the primary recruiters for all clinical research - there is no clinical study or trial that launches, particularly in the United States, that doesn't involve us as advisors.

To inform, support, and empower those affected, we create structures that bring together patients and families and that offer options for care. Our website, online and video-conference support groups, patient conferences, online Patient-Expert Course, and publications give families what they need to make good decisions about care and to reduce their sense of isolation with this rare disease. Our free genetic testing, peer-reviewed clinical care consensus guidelines, and Center of Excellence network facilitate expert multi-disciplinary care options.

Given our focus on facilitating research and providing patient support, our nine-member staff includes seven who have advanced degrees, with four holding doctorates in relevant fields such as scientific research, molecular genetics, and psychology. In addition to six full-time staff positions, there are three part-time staff members, 35 active ongoing volunteers, and more than 100 additional volunteers who participate in one-off events or activities.

We remain a virtual organization, which allows us both to keep overhead low and also to select the best people for the job, regardless of geography. We have steadily increased our funding which has kept pace with our programmatic needs.

Before Alliance to Cure was founded in 2002, cavernous malformation was a barely-diagnosed disease. Today, there are 14 treatments in the research pipeline, including three in clinical trials, and an active patient community of 10,000.

We drive research progress by:

-Providing strategic seed funding yielding significant returns on investment, filling knowledge gaps, and pointing the way toward promising treatments. For example, $120,000 in two Alliance to Cure-funded grants leveraged a combined $6.1 million in government grants to continue research on CCM3, an aggressive cavernous malformation mutation that often affects children, and focused ultrasound, a noninvasive treatment technology.

-Convening 20 annual international science conferences for CCM experts, leading to transformations in research and care.

-Mobilizing patients to join clinical trials by creating a registry of nearly 4000.

We offer direct, life-changing support to cavernous malformation patients by:

-Establishing 12 Centers of Excellence and 7 Clinical Centers across the U.S., placing most US patients within driving distance of expert cavernous malformation care.

-Educating and supporting 10,000 patients every year through virtual support groups, webinars, newsletters, a Patient-Expert Certification program, and Facebook communities.

-Addressing the diagnosis, treatment, and research gap for Black patients suffering rare diseases through the Breaking Barriers for Black Health Empowerment initiative, which has increased the number of Black patients on the Cavernous Malformation Registry by 500% since the initiative began in 2021.

-Designing and implementing a decade-long patient support and education program in New Mexico that culminated in the New Mexico state legislature appropriating $300,000 in 2023 for a statewide health program to support New Mexico's cavernous malformation patient population. New Mexico has the most concentrated cavernous malformation population in the world due to the Common Hispanic Mutation, a specific genetic mutation passed down from the original Spanish settlers in the 1600s. Alliance to Cure's New Mexico program included free genetic testing for 200 families in New Mexico.

What's next:

-We have launched a research project aiming to screen and test at least 20 promising drug compounds and combinations in labs so that industry partners can make informed decisions about which drugs to adopt for clinical trials. These 20 possible treatments are FDA-approved for indications other than cavernous malformation, but Alliance to Cure has identified their potential effectiveness as cavernous malformation treatments through consultations with the research community, literature reviews, and AI.

-We will be working with our partners to explore the promise of focused ultrasound as a treatment for cavernous malformation.

-Other research projects we are supporting are expected to yield results by 2026.