It is our mission to inform, support, and mobilize those affected by cavernous malformations and drive research for better treatments and a cure. We do this by developing and executing strategic, creative, high-return interventions as a model for rare diseases.
A permanent cure for cavernous malformations
1) Facilitate and participate in cavernous malformation research to achieve a complete understanding of the disease and facilitate clinical drug trials and other treatment improvements.
- Initiate and deepen relationships with biotech/pharmaceutical companies and developers of other potential treatments to introduce cavernous malformations as a potential target.
- Introduce academic and industry partners and encourage collaboration.
- Remain conversant and share information regarding the progress of non-pharmaceutical, non-invasive treatment development, such as microbiome, focused ultrasound, and hypoxia treatment. Create educational campaigns.
- Create a path to increasing researcher and industry access to additional research resources such as plasma, other biosamples, and animal models.
- Fully enroll clinical drug trials
- Promote and expand our International Cavernous Malformation Patient Registry.
- Work closely with FDA, NIH, academic researchers, and the pharmaceutical industry to develop realistic clinical outcomes and trial design, including assisting in the creation of guidelines for the conduct of clinical trials which can be used as an introduction and map for sponsors, serving on Clinical Advisory Boards, and organizing and/or attending FDA meetings as possible.
- Partner as the participant recruiter in the development of a patient-reported burden of disease measure.
- Identify, communicate, and address (where possible) barriers to clinical drug trial participation and retention.
- Create a multimedia educational campaign
- Address logistical barriers, possibly including small travel stipends for non-industry trials, etc.
- Establish a network of clinical research centers in preparation for clinical drug trials and to facilitate consistent treatment.
- Support dissemination of clinical treatment guidelines. Plan to update guidelines as needed.
- Extend invitations to potential clinical centers and assess interest and readiness.
- Assist in correcting disease knowledge deficits at Centers.
- Expand the clinical component of the annual Scientific Meeting, led by Center of Excellence key personnel.
- Expand our patient identification, support, and clinical center program for special populations and their providers, including Hispanic patients, Black patients, those with founder mutations, and CCM3 mutations.
- Develop collaborative relationships among researchers and institutions by connecting them through projects and conferences. Place special emphasis on attracting and developing the next generation of investigators.
- Maintain a genotyping campaign to increase the number of patients eligible for clinical drug trial participation
- Continue to identify barriers to genotyping
- Continue to fund and create a genotyping subsidy program for those unable to afford genetic testing.
- Target those believed to be at risk for founder mutations on the basis of genealogy, offering genealogy services, community-building, and testing in order to expand our patient base and increase awareness of the illness in specific geographies and groups.
- Participate in consortia and as an IRB-approved enrollment site for translational and clinical research, leveraging our unique position with the patient community.
2) Provide disease and resource information to educate and improve the lives of people affected by cavernous malformations, caregivers, health professionals, researchers, policymakers, the media, and the general public.
- Maintain a website and printed materials that provide up-to-date information.
- Organize and host regional educational events.
- Place a specific focus on increasing health professionals’ knowledge of the condition.
- Create a webinar series to provide information and progress updates.
- Launch and promote a Patient-Expert Certification Program to develop and recognize mentors.
3) Foster and promote a caring community to provide support.
- Provide multiple avenues of online community support internationally, nationally, and regionally.
- Develop programs to support members which may include local face-to-face support, videoconferencing, and other avenues of outreach.
- Develop programming for those who are living with CCM-related chronic deficits.
- Foster international sister organizations that can assist in locating and organizing patients and researchers, including assisting with the formation of international consortia.
4) Get people involved in advocacy and active participation toward a cure.
- Inform and encourage every individual to actively participate to their full potential.
- Encourage people to share their experiences and provide a public social media platform to raise public awareness.
- Encourage and facilitate research participation.
- Provide tools for more successful fundraising events.
- Encourage and train people to contact their legislators on both the federal and state level to promote increased funding of cavernous malformation-related research and services, particularly leveraging information gained from founder mutation outreach.
- Encourage people to educate and engage with their medical providers to expand the Alliance to Cure Cavernous Malformation umbrella.
- Establish and nurture local affiliate groups to promote grassroots growth that includes mutual support, public awareness, Center of Excellence identification and development, and fundraising.
- Mentor a new generation of volunteer leadership to sustain and develop the Board of Directors and Board activities.
5) Build and sustain a broad base of funding sources to support our mission and goals.
- Develop and execute an efficient, effective, long-term fundraising strategy that includes peer-to-peer events, major and individual donors, and corporate and foundation support.
- Leverage local affiliate groups as potential fundraising event organizers.
- Schedule and integrate major donor development into executive-level organizational activities.
- Offer training to Board members to facilitate their role as community stewards.
- Explore mechanisms for planned giving.
- Use fundraising activities to increase awareness and understanding of cavernous malformations and the work of Alliance to Cure Cavernous Malformation.
Last updated 4.14.22