What is the CRC-SCA?
The CRC-SCA (Clinical Research Consortium for Spinocerebellar Ataxia) is a well-coordinated network of physician scientists with expertise in clinical ataxia research. All the members of the CRC-SCA belong to a wider investigative network known as the Cooperative Ataxia Group (CAG) which has been involved in clinical research in the field of ataxias.
Spinocerebellar ataxias 1, 2, 3 and 6 are the most common, autosomal dominantly inherited cerebellar degenerations that produce imbalance, poor coordination, speech difficulties and ultimately a wheelchair bound existence. The genetic defects that underlie these diseases have been defined in the last 15 years and significant molecular genetic research has brought us to the threshold of treatment trials. The diseases are overall rare, slowly progressive, cause increasingly severe neurological difficulties and are variable to a significant degree across and within genotypes. The natural history of the SCA’s and factors that may modify them are incompletely understood. There is also limited knowledge on factors that may modify the clinical aspects of the SCA.
Mission
- The CRC-SCA’s primary goal is to provide the infrastructure necessary for future clinical trials to test the safety and efficacy of therapeutic interventions for the Spinocerebellar Ataxia 1, 2, 3 and 6 and share the knowledge gained about the symptoms and course of these rare diseases with other researchers and the public.
- Train neurologists to become specialists who can both treat and carry out research in these conditions.
- Use the information and the help of the trained investigators to find treatments for these diseases.
Goals of the Clinical Research Consortium for Spinocerebellar Ataxia:
- Enroll a large group of patients with Spinocerebellar Ataxia 1, 2, 3 or 6 into an observational cohort study (where subjects are followed over time and clinical data specifically for research are collected at regular intervals).
- Establish a patient contact registry.
- Discover new laboratory markers of disease that will lead to better treatment and deeper scientific understanding of the causes of this disease.
- Develop improved methods for studying Spinocerebellar Ataxia.
- Work with Spinocerebellar patient support groups to help those patients who wish to be involved in research connect with those doctors conducting the research.
- Help other scientists do more research on Ataxia by providing specimens and clinical data from the CRC-SCA for analysis.
- Train new young investigators in the field of Spinocerebellar Ataxia.
- Construct and maintain an electronic website resource with significant information for clinicians, researchers, and patients.
Department of Health and Human Services
National Institutes of Health
The Clinical Research Consortium for Spinocerebellar Ataxias (CRC-SCA) is a member of the Rare Diseases Clinical Research Network, which is funded by the National Institutes of Health.