2020: A Look Back and a Look Ahead

Dear Friend of Alliance to Cure Cavernous Malformation,

COVID-19 has changed the face of this year, but it hasn’t changed our determination to improve the lives of cavernous malformation patients and their families. You make this possible. With your support, we’ve made great progress in 2020, and we’re planning ambitious new initiatives as we enter 2021.  This year, to advance research and care, we:

Began a COVID-19 registry, the only registry in the world tracking CCM patients who test positive for COVID. So far, we’ve learned that our members do not seem to be more seriously affected as a result of COVID infection than the general public, which is heartening. We should have more data in the coming months. In the meantime, we’ve offered our members regular webinars with our scientific advisors to keep them up to date on the latest recommendations for our patients.

Created a strong virtual community with support groups meeting at least weekly and countless webinars and informational meetings. Our community is strong and thriving, even though we can’t be together in person.

Hosted our annual International Scientific Meeting in a unique format, with pre-recorded presentations and live discussion sessions. Research progress continues apace with some COVID impacts, but so far very little momentum has been lost.

Testified in two critical FDA Meetings to advance the development of the CCM-Health Index as a qualified tool for measuring the effectiveness of treatments.

Inaugurated a virtual poster session series. In these quarterly meetings, research trainees present their work to expert scientists for feedback and critique. We are keeping scientific discussions alive around the year.

Recognized a new CCM Clinical Center at the University of Miami/Jackson Health System, offering an opportunity for multi-disciplinary expert care to those who live in the southern US.

Completely updated our website with a new look and even more comprehensive information for patients, their families, medical providers, and researchers.

Continued our New Mexico program, where we have made such a difference and geared up our genealogy work with the CCM2 community, an effort that will help us find patients and raise awareness.

Continued supporting multiple clinical drug trials as well as the development of other forms of treatment and prevention. We are very excited about the possibilities as we move forward to 2021.

Accelerating Cures in 2021

In 2021, in addition to our existing programs, we plan to launch an exciting new initiative to Accelerate Cures. It has taken researchers and the patient community years to develop all the pieces needed for a successful drug trial. Within the next few years, we will have everything in place: a robust and eager patient community, tools like validated surveys and biomarkers that will measure the efficacy of a medicine, a network of Centers of Excellence to host trials, and a large pipeline of potential treatments. We now need to attract the attention of treatment developers (the pharmaceutical industry and others) in what is a very competitive field among rare diseases. We need to convey the message that we are ready, that a drug trial is feasible, and that developing treatments for CCM would bring a good return on investment.

To that end, in 2021, we plan to launch an initiative to accelerate cures. We will be hiring an individual who can coordinate a critical examination of our current resources in order to address any deficiencies, craft a white paper delineating our strong position, and engage in outreach to prospective industry partners. The individual will be familiar with business development in the pharmaceutical sector and will have a mission-driven mindset. We have no doubt this initiative will increase both the number of treatments and speed of development for CCM. We ask for your support to launch this critical initiative.

In 2021, we also will be assisting with existing clinical trials and improving care in a myriad of ways. We are broadening our patient base with a new Breaking Barriers initiative that will offer outreach and connection to Black patients. We will be supporting the start of Phase 2 trials of Recursion’s medicine REC-994 which is being developed specifically for CCM. Our support of this trial includes attending FDA meetings, consulting on trial design, and recruiting. We will finish recruiting for the atorvastatin trial at the University of Chicago. We intend to expand our DNA/Tissue Bank and our patient registry, and we hope to recognize additional Centers of Excellence if travel allows.

During this giving season, please join us in ensuring that our dream of accelerating cures in 2021 comes true by making a tax-deductible year-end donation at Angioma.org/Donate.

Thank you for your support through this unusual year and for the trust that you place in us. We will find a cure together.

With Gratitude,

Connie Lee, Psy.D.
President and CEO

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