Thanks to the generosity of donors, the Alliance To Cure is able to attract the philanthropy needed to move the multi-million-dollar clinical trial effort from concept to reality, and accelerate a cure.
Specific giving levels enable specific, targeted research:
$20,000– One day of the Alliance To Cure annual international scientific meeting. This meeting is the incubator of the Cure. It offers university researchers and companies from around the world the opportunity to meet, exchange ideas, and form partnerships. This is where hope is born, yielding discoveries throughout the year.
The Alliance To Cure meeting is truly international, attracting researchers from 5 continents. Uniquely, researchers are required to present unpublished work – this keeps the discussion fresh and cutting edge. They are not allowed to share, copy or distribute data they see at the meeting…this makes attendance “can’t miss.” Finally, the trust involved in revealing secrets leads to a cohesive, collaborative community.
Alliance To Cure scientific meetings create breakthroughs. The meeting has nurtured the collaborations that have led to major advances in the understanding and treatment of the illness, including identifying and testing treatment and discovering how patients might be able to reduce the impact of the illness with lifestyle changes.
$15,000 – Four months of the Alliance To Cure DNA/Tissue Bank. The DNA and Tissue Bank currently holds surgically removed cavernous angioma tissue and accompanying DNA samples from members. This is a resource used by researchers around the world. In 2022, the biobank is expanding dramatically to become even more valuable. It will include other biological samples, like plasma and serum – resources that are being used to find ways to diagnose and predict hemorrhage.
$10,000 – Two months of the Alliance To Cure International patient registry – the most critical resource Alliance To Cure offers to research. Over 3000 patients have shared their experiences in our registry, allowing us to paint a picture of the treatments that are needed and what a cure would mean. The registry also gives us a way to reach our patients to let them know when and where they are most needed.
The registry is fueling recruitment for drug trials. It has sparked research regarding the relationship between hereditary CCM and other illnesses. It has been essential for the development of the CCM Health Index – a tool that will be used in clinical drug trials to determine whether a medicine is having an effect.
$5,000 – Supports the AAlliance To Cure website, the first place a newly diagnosed family learns about the illness. It is the central repository of CCM knowledge for everyone affected by the illness. Most families find themselves going back to it frequently when they reach turning points in the illness. Many members have let us know that their doctor directed them to the Alliance To Cure site as the only trusted, comprehensive source of cavernous angioma information.
The website includes sections that help families make care decisions, that show where the nearest Center of Excellence can be found, that connect patients to local support, and that provide opportunities for research participation.
$2,500 – Development of one CCM Center of Excellence.
The growing Alliance To Cure Center of Excellence network is currently 10 Centers in the United States with at least 4 more Centers coming online in 2022. Patients who use a Center of Excellence can expect that they will be scheduled for appointments with specialists in all disciplines – neurology, neurosurgery, and genetics – who have expertise in CCM. They can be confident that their imaging will meet the standards set by Alliance To Cure expert-written Clinical Care Consensus Guidelines. They know that each institution is engaging in cutting edge research for the cure. If they should run into an issue with a Center of Excellence, Alliance To Cure can advocate for them personally.
Because cavernous angioma is a rare disease, getting informed care for CCM has historically been difficult. Without expertise, a clinician may recommend treatments that are harmful to our patients. Alliance To Cure Centers of Excellence take the worry out of finding care so that patients and their families can focus on healing.
$1,000 – Genetic testing and counseling to an Alliance To Cure family: free genetic testing to any US or Canadian resident who meets clinical criteria.
Patients, clinicians, and researchers consider this one of the organization’s most important services. It helps adult patients and parents of at-risk or diagnosed children make informed decisions about care. It also prepares these families to participate in research. Alliance To Cure employs a licensed professional who can order testing, and the test results are transmitted to the patient’s physician. Alliance To Cure has tested hundreds of families since the beginning of the program, and they will continue to offer this service to families so they can protect their health and the health of their children.
Scientific Research that Angioma Alliance is seeking to
fund at amounts $20,000
$50,000 (initial investment to pilot)
Developing a human cavernous angioma model (organoid)
At this time, drugs are tested in mice that are genetically engineered to have cavernous angioma mutations that cause the development of lesions. Researchers have excellent cavernous angioma mouse models but they are still mice, not humans.
Other diseases, like many cancers, have models that allow them to test drugs on human disease tissue without exposing a human directly. Pharmaceutical companies expect to see this evidence before deciding to move forward with a medicine. Angioma Alliance would like to fund the development of such a model. A proof of concept to study, to see whether this is possible, has been proposed and an expert researcher has been selected. Now, it’s time to move forward with funding.
$100,000 (Angioma Alliance contribution)
Testing Focused Ultrasound
Focused ultrasound is a non-invasive technique in which a focused beam of ultrasound targets a lesion. The skull is not opened and there is no radiation involved. Having this technology available for cavernous angioma would be game changing. Research is currently underway but has had difficulty finding and maintaining funding because the National Institutes of Health (NIH) is reluctant to fund experiments using new technology. We do not want this project to stall and are examining ways that we can offer bridge funding to keep it going.
Head-to-Head Mouse Drug Trials
There are many excellent mouse models of cavernous angioma illness. There is also a drug pipeline that is full of medicines that could be repurposed. One goal of Angioma Alliance is to test the best drug candidates in the best mouse models.
As a start, Angioma Alliance has organized a consortium of 13 laboratories from around the world who are documenting their mice and the treatments they’ve tested into a single database. They will then assess which treatments should be tested first and which mice would be most appropriate. Doing such a systematic test will save years.
Once there is a plan, funding will be needed. Researchers estimate it will take $500,000 to test the top candidates in one mouse model. Testing in multiple models would be preferred.
For more information: https://www.angioma.org/progress-toward-the-cure-a-letter-from-our-ceo/
Rapamycin Clinical Trial (4 years for Phase 2 study)
Research published over the last 2 years indicates that a medicine called Rapamycin could be very effective in treating cavernous angioma, particularly in preventing lesions from becoming aggressive and causing problems. This medicine is already approved for other illnesses and could be repurposed for cavernous angioma.
It will take years to obtain funding for such a trial from the National Institutes of Health (NIH). Patients need to know whether this medicine can help them as soon as possible. Angioma Alliance is seeking funding for a small trial of 60 patients in an interested, expert research network.
$500,000 (initial to pilot)
Gene therapy – Gene therapy could be a cure for those with hereditary cavernous angioma. Amazing progress is being made in other diseases but there is tremendous competition for available research funds. Angioma Alliance would like to fund a pilot study with mice to determine whether gene therapy may become a viable option for our patients.