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（由申请方提供）：脑海绵状血管畸形（CCM）是一种中枢神经系统血管疾病。这种疾病的症状包括神经功能缺损，癫痫发作和出血性中风，这是由于脑和脊髓内血管病变的发展。目前，对于患有这种疾病的患者没有药物治疗选择。手术切除病变是唯一的选择，但并非所有患者都适合手术。CCM可能偶尔发生，或由于三种基因（CCM 1，CCM 2和CCM 3）之一的突变。这些基因产物的功能在1999年、2001年和2005年分别被鉴定时是完全未知的。自从这些基因被发现以来，人们对研究CCM的发病机制和确定病因的兴趣越来越大。来自不同领域的临床医生和研究人员已经研究了与基因功能，信号传导机制，血管生物学以及正常血管结构如何在疾病状态下丢失有关的问题。建立CCM发病机制的综合模型并最终进行开发非侵入性治疗和治愈所需的研究需要CCM疾病研究整个领域的合作投入。在2005年之前，没有专门针对CCM生物学的科学会议或讲习班。因此，血管瘤联盟，CCM患者的倡导团体，于2005年11月设立了第一个研讨会，并在过去五年中继续主办和支持这一年度会议。CCM科学研讨会的病理生物学是一个为期两天的会议，包括口头报告和与CCM生物学相关的临床，基础和翻译科学的讨论。如果没有CCM研讨会，这些研究人员将没有机会相互交流-科学上，他们的个人领域通常不会在其他更大的国家会议上重叠。由于对CCM病理生物学的兴趣日益增加，本提案旨在申请部分资金以扩大会议范围，以包括新科学领域的代表，并为年轻研究人员，博士后研究员和博士候选人提供额外的支持和培训机会。为使《集束弹药公约》研讨会继续取得成功，提出了三个具体目标：1.促进不同科学学科与受训人员之间的交流与合作。 2.与CCM研究社区的成员分享最新的研究和临床发现。 3.讨论所提供数据的生物学意义以及对CCM药物治疗和临床试验启动的未来影响。! 公共卫生关系：脑海绵状血管畸形（CCM）是一种中枢神经系统血管疾病，可导致严重神经系统疾病，包括癫痫发作和出血性卒中。没有针对这种疾病的特定药物治疗。CCM科学研讨会的病理生物学是临床医生和科学家传播研究成果的论坛，并讨论CCM的生物学以及开发药物治疗方案以改善患者护理和公共卫生的潜力。