NM Delegation Introduces Bill to Expand Research on Rare Genetic Disease Prevalent Among New Mexico Hispanics

WASHINGTON D.C. - Today, U.S. Senators Tom Udall (D-N.M.) and Martin Heinrich (D-N.M.) and U.S. Representatives Ben Ray Luján (D-N.M.), Steve Pearce (R-N.M.) and Michelle Lujan Grisham (D-N.M.) announced they have introduced a bill to increase research, education and treatment for Cavernous Cerebral Malformations (CCM), an uncommon genetic blood vessel disease that impacts the brain and spinal cord and is more common among New Mexico Hispanics than any other group. Due to limited research, there is no cure for CCM.  The Cerebral Cavernous Malformations Clinical Awareness, Research and Education Act (CCM-CARE), introduced today on National Rare Disease Day, would expand National Institute of Health (NIH) research to increase awareness, treatment and prevention of CCM, and support the development of tele-health infrastructure to reach rural, underserved communities. 

The disease, also known as cavernous angioma, or Cavernoma, is a devastating blood vessel disease that affects about 600,000 people in the United States. It is characterized by vascular lesions that develop and grow within the brain and spinal cord. It was common among Spanish settlers in the Southwest, and the hereditary mutation continues to cause CCM today. Most people with the disease don't realize they have it until they experience symptoms, which include neurological deficits, seizures, stroke, or sudden death. The only treatment available is brain and spinal surgery, and that can help only certain patients.

"This tragic and rare disease disproportionately affects New Mexicans, and it is imperative that we raise awareness and increase research to find a cure and improve treatment options for those suffering from Cerebral Cavernous Malformations," said Udall, who has worked to raise awareness about CCM since he was a member of the House. "By expanding CCM research through interagency collaboration, and supporting tele-health infrastructure to help medical professionals reach rural communities, this bill will bring us one step closer to preventing, treating and ultimately curing this devastating disease."

“Cavernous Angioma affects New Mexico Hispanics at a dramatically higher rate than other populations," said Heinrich. “Expanding research and treatment centers across the country to identify, monitor, and treat individuals afflicted with this illness is an important step in improving the livelihoods of many New Mexicans and developing a cure."

“As we continue the fight to ensure everyone in this country has access to quality and affordable health care, it’s so important that we remember those among us who suffer from rare and debilitating diseases like the Cerebral Cavernous Malformations," Luján said. “Evidence based treatment, and ultimately cures, both start with well-funded research. I’m proud to support this legislation.” 

“This rare, genetic disease affecting many Hispanic New Mexican communities is too often overlooked," said Pearce. "I am proud to join my colleagues in the New Mexico Delegation to bring CCM to the forefront. Together we can find a successful treatment, ensuring New Mexicans affected by this grave disease have access to vital health care services they need.” 

“Given the disproportionate effect Cavernous Cerebral Malformations has on New Mexicans, it is important that we stand together as a delegation to support efforts to find a cure for this disease,” Lujan Grisham said. “I will continue working hard with my colleagues to raise awareness and increase research on CCM.”

Specifically, CCM-CARE would direct the heads of NIH, Centers for Disease Control and Prevention and the U.S. Food and Drug Administration (FDA) to implement the following measures to further CCM research: 

- Conduct basic, clinical and translational research on CCM; identify and support three national research centers' collaboration on multi-site clinical drug trials; and integrate CCM within relevant existing clinical research networks to expand access to clinical drug trials to CCM patients. 

- Establish a CCM education and information program under the U.S. Department of Health and Human Services (HHS) to expand training for clinicians and scientists; establish a CCM Coordinating Committee at HHS to coordinate all federal CCM health efforts, facilitating the exchange of information and the effectiveness of research efforts; and award grants for CCM surveillance and epidemiology. 

- Support appropriate investigational new drug applications and streamline the process from clinical drug trials to review for CCM treatment; and review appropriate request for designations for orphan drugs for CCM. 

The bill is supported by Angioma Alliance Scientific Advisory Board and Board of Directors.