Supporting research initiatives aimed at treating, preventing and curing a diverse range of brain disorders.

Based on their inside knowledge of the CCM field and their understanding of the greater research landscape, the Alliance to Cure Cavernous Malformation identifies high-impact, generally high-risk/high-reward opportunities for funding. They search for potential research partners, focusing on programs with the best potential to move the work forward towards a cure. Once a research partner has been selected, collaboration begins on a budget and a plan. 



In 2022, with your support, the Alliance to Cure Cavernous Malformation awarded $87,000 to the University of Virginia to fund research using focused ultrasound as a treatment for CCM.

The results have been nothing short of astounding. First, in a soon-to-be-published paper, UVA researchers demonstrated that focused ultrasound has the potential to be a cure for people with one cavernous malformation and a near cure for those with more than one. In mouse models, a combination of low-intensity focused ultrasound and microbubbles healed existing CCMs. When used in very young mice, it kept them from forming in the first place. The Alliance to Cure is now actively pursuing a human clinical safety trial for focused ultrasound with the intent to enroll the first patients in 2025.

Second, UVA was able to leverage the funding and advice they received from the Alliance into a $3.1 million NIH grant.  The NIH grant will continue UVA’s exciting laboratory work, including exploring whether monoclonal antibody treatments and gene therapy can be delivered to lesions with focused ultrasound.

Your investment yielded a potential cure

and a 3500% return!

New! Pre-Clinical Treatment Testing Program

The Alliance to Cure is developing a pre-clinical treatment testing program in which the Alliance selects compounds to be tested in cell lines and in a mouse colony that is owned and directed by the Alliance. There are too many potential treatments sitting on shelves because researchers don’t have the time or space to test them. Drug companies also need quick access to testing resources to decide whether their new drugs are worth moving forward for CCM.

The Alliance staff and scientific advisors have the expertise to select compounds and design these experiments. Currently, the first batch of 16 potential treatments is being tested in cell lines at a commissioned university, and mice will soon be arriving at a scientific research organization – a non-profit expert research facility – where the colony will be developed and housed.

This program is a major investment with a minimum budget of $200,000/year. The potential reward is equally high. A pre-clinical treatment testing program allows the Alliance to quickly evaluate promising treatments and gives this CCM disease a competitive advantage over other diseases, as drug companies consider where they will focus their efforts.  This testing program will save years and countless lives.


Clinical Trials

There are currently four clinical drug trials underway or soon to launch! The Alliance to Cure Cavernous Malformation is central to each of them. Because the Alliance to Cure staff are CCM experts, the moment a drug company announces its intention to work on CCM as a treatment target, they call the Alliance to get advice on pre-clinical testing and trial design. The Alliance also serves as the primary patient recruiter once trials are underway.

The Atorvastatin trial will announce results in Fall, 2024. The Recursion safety trial, the first industry-sponsored trial for CCM, completed enrollment ahead of schedule last summer (2023) and will also announce results in early Fall. With Alliance assistance, trials sponsored by two other drug companies, Neurelis and Ovid, are scheduled to begin in late 2024 or early 2025. Treatments are becoming a reality.


Targeted Drug Delivery Using Nanoparticles

With your help, we are funding the exploration of advanced drug delivery methods to get treatments directly to tumors. This work goes hand-in-hand with the recent discovery of the cancer-like biology of CCM. If we need to resort to cancer drugs to shrink a CCM, we want to isolate the drugs’ activity to the tumor itself to prevent side effects. Focused ultrasound may be curative for those with one CCM, but we also need a mechanism to deliver medicines on a daily or weekly basis for those with multiple CCM’s, like our sweet Elle.

In 2023, the Alliance to Cure awarded the University of Pennsylvania’s nanoparticle drug delivery research program $75,000 in seed funding. We have already received promising news from their work, and the UPenn research team is leveraging this funding into a much larger NIH grant request. 


Gene Therapy

In 2023, the Alliance to Cure awarded $18,400 to Dr. Angela Glading at the University of Rochester to support her work developing gene therapies for CCM.

Treatment for CCM is currently limited to surgery, and though several drugs currently in pre-clinical development or clinical trials give hope that we will eventually be able to manage the disease, they do not represent a cure.  Pre-clinical studies of the drugs currently in development have, at best, demonstrated a 50% reduction in lesion burden. Combination therapy (treatment with more than one drug at once) could be more effective, but this type of therapy is likely to have significant side effects, which may limit how long the drugs can be used and their use in children. These limitations have led Dr. Glading’s lab to look at gene therapy as a treatment/cure for CCM. Their project aims to develop novel tools that could be used to rectify the mutations that underlie CCM development in patients of any age.

This grant funds efforts to target gene therapy to brain endothelial cells directly rather than to every cell in the body. This would make gene therapy, once it’s developed, a safer option for our patients.  Our funding covers a small but critical portion of this work.