The 6th Annual Pathobiology of Cerebral Cavernous Malformations (CCM) Scientific

第六届年度脑海绵状血管瘤病理学 (CCM) 科学会议

• 批准号: 8061209
• 负责人: Amy Lee Akers
• 金额: $ 1.04万
• 依托单位: ANGIOMA ALLIANCE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-09-27 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8061209
• 关键词: 6th; Annual; Pathobiology; Cerebral; Cavernous

DESCRIPTION (provided by applicant): 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.

描述（由申请人提供）：脑海绵状畸形（CCM）是中枢神经系统的血管疾病。这种疾病的症状包括由于大脑和脊髓内血管病变的发展而导致的神经功能不足，癫痫发作和出血性中风。目前，该疾病患者尚无药理治疗选择。病变的手术切除是唯一的选择，尽管并非所有患者都是手术的候选者。 CCM可能偶有地发生，或者是由于三个基因之一（CCM1，CCM2和CCM3）中的突变引起的。当分别鉴定出1999、2001和2005分别确定每个基因时，这些基因产物的功能是完全未知的。由于鉴定了这些基因，因此对研究发病机理和确定CCM病因的兴趣越来越多。来自不同领域的临床医生和研究人员研究了与基因功能，信号机制，血管生物学以及正常血管结构如何在疾病状态下丢失的问题。建立一个用于CCM发病机理的综合模型，并最终进行开发非侵入性治疗和治疗所必需的研究，这需要从CCM疾病研究的整个领域进行合作输入。在2005年之前，没有专门针对CCM生物学的科学会议，会议或讲习班。因此，受CCM影响者的倡导小组Angioma Alliance于2005年11月建立了第一次研讨会，并在过去五年中继续主持和支持本年会议。 CCM科学研讨会的病理生物学是为期两天的会议，包括口头演示和讨论与CCM生物学相关的临床，基础和翻译科学。没有CCM研讨会，这些研究人员将没有机会彼此互动 - 从科学上讲，他们的各个领域通常不会在其他较大的国家会议上重叠。由于对CCM病理生物学的兴趣越来越大，该提案旨在要求部分资金扩大会议范围，以包括代表新的科学领域，并为年轻的研究人员，博士后研究员和PhD候选人提供更多的支持和培训机会。为此提案开发了三个具体目标，以及CCM研讨会的持续成功：1。促进独特的科学学科和学员之间的沟通和协作。 2。与CCM研究社区的成员分享最近的研究和临床发现。 3。讨论所提出的数据的生物学意义以及对开发用于CCM的药理学治疗的未来和临床试验的启动的影响。呢 公共卫生相关性：脑海绵状畸形（CCM）是中枢神经系统的一种血管疾病，导致严重神经发病的可能性很高，包括癫痫发作和出血性中风。该疾病的特定医疗疗法不可用。 CCM科学研讨会的病理生物学是一个论坛，旨在供临床医生和科学家传播研究结果，并讨论CCM的生物学以及开发药理治疗方案的潜力，以改善患者护理和公共健康。