The Cerebral Cavernous Malformations Clinical Awareness, Research, and Education (CCM-CARE) Act of 2019 would authorize and prioritize funding to support awareness, research, and education programs focusing on cerebral cavernous malformations (CCM).
The bill would require the following actions from these organizations:
The National Institutes of Health would:
- Be authorized to award grants to both public and non-profit private organizations to support research on CCM, including topics such as the safety and effectiveness of treatments, gene therapy, early diagnostic measures, patient quality of life, and an overall search for new treatments, biomarkers, animal models, etc.;
- Support the expansion or establishment of a network comprising two major CCM clinical research centers, as well as six to ten centers for patients to receive treatment and participate in research;
- Create a CCM Consortium composed of representatives from patient advocacy groups and the centers mentioned above for the purpose of developing educational programs and materials on CCM;
- Establish or expand a CCM training program to educate scientists and healthcare professionals (including physicians, medical students, and care coordinators) on CCM diagnosis and treatment; and
- Provide grants and stipends to individuals and small biotechnology entities for various endeavors, such as pursuing CCM physician training and developing novel treatments.
The Centers for Disease Control and Prevention would:
- Appropriate funds and be authorized to award grants to both public and non-profit private organizations to collect, analyze, and report data related to CCM, including CCM incidence, symptoms, and treatment efficacy.
The Food and Drug Administration (FDA) would:
- Support current research on the identification of biological targets and markers that would meet FDA standards for testing future treatments;
- Coordinate with researchers to verify the above biomarkers and validate outcome assessments for CCM, allowing researchers to conduct clinical trials at a faster pace; and
- Work with researchers to move appropriate applications for CCM clinical trials and investigational new drugs through expedited FDA review pathways, including the Orphan Drug Designation, Fast Track, and Priority Review pathways.
CCM is a relatively common condition (affecting about one in 100 people) in which tiny blood vessels become enlarged and misshapen, forming clusters that are prone to bursting. Although a quarter of those with CCM never experience any symptoms or problems, CCM can be a life-threatening condition, especially when the clusters form in the brain or spinal cord.
This bill estimates that there are 600,000 people in the United States affected by CCM. The sponsors of this bill are the entire Congressional delegation from New Mexico, the state where CCM has the largest impact: There is an inherited form of the disorder that is common among Hispanic populations, resulting in New Mexico having the highest population density of CCM worldwide.