Cerebral Cavernous Malformations (CCM) is a vascular disorder of the central nervous system. Symptoms of this disorder include neurological deficit, seizures and hemorrhagic stroke as a result from the development of vascular lesions within the brain and spinal cord. Currently there is no pharmacological treatment option for patients with this disorder. Surgical resection of the lesions is the only option, though not all patients are candidates for surgery. CCM may occur sporadically, or due to mutations in one of three genes (CCM1, CCM2, and CCM3). The function of these gene products was entirely unknown when each gene was identified 1999, 2001, and 2005, respectively. Since the identification of these genes there has been increasing interest in studying the pathogenesis and determining the etiology of CCM. Clinicians and researchers from distinct fields have investigated questions relating to gene function, signaling mechanisms, vascular biology and how the structure of the normal vasculature is lost in the disease state. Establishing a comprehensive model for CCM pathogenesis and ultimately conducting the studies necessary to develop a non-invasive treatment and cure requires cooperative input from the entire field of CCM disease research. Prior to 2005, there were no scientific conferences, meetings, or workshops focused specifically on the biology of CCM. Therefore, Angioma Alliance, the advocacy group for those affected by CCM, established the first workshop in November of 2005 and has continued to host and support this annual meeting for the past five years. The Pathobiology of CCM Scientific Workshop is a two-day meeting consisting of oral presentations and discussion of the clinical, basic and translations science associated with CCM biology. Without the CCM workshop, these researchers would not have the opportunity to interact with one another - scientifically, their individual fields generally do not overlap at other larger national meetings. Because of the increasing interest in the pathobiology of CCM, this proposal is designed to request partial funding to expand the scope of the meeting to include representation of new scientific fields and to provide additional support and training opportunities for young investigators, postdoctoral fellows, and PhD candidates.
Three specific aims were developed for this proposal and for the continued success of the CCM workshop: 1. to foster communication and collaboration between distinct scientific disciplines and trainees. 2. To share recent research and clinical findings with members of the CCM research community. 3. To discuss the biological implications of the presented data and the impact on the future of developing a pharmacological treatment for CCM and initiation of clinical trials. !

Public Health Relevance

Cerebral Cavernous Malformations (CCM) is a vascular disorder of the central nervous system that causes a high probability of serious neurological morbidity including, seizures and hemorrhagic stroke. Specific medical therapies for this disorder are not available. The Pathobiology of CCM Scientific Workshop is a forum for clinicians and scientists to disseminate research findings and to discuss the biology of CCM and the potential for development of pharmacological treatment options to improve patient care and public health.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Conference (R13)
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Special Emphasis Panel (ZNS1-SRB-W (38))
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Jacobs, Tom P
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Angioma Alliance
United States
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