The 10th Annual Angioma Alliance Cerebral Cavernous Malformations (CCM) Scientific Meeting

第十届血管瘤联盟脑海绵状血管瘤 (CCM) 年度科学会议

• 批准号: 8837838
• 负责人: Amy Lee Akers
• 金额: $ 1万
• 依托单位: ANGIOMA ALLIANCE
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-30 至 2015-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8837838
• 关键词: Academia; Address; Advocacy; Affect; Animal Experimentation; Anniversary; Area; Biological; Blood Vessels; Brain; Brain Diseases; Brain hemorrhage; CCM1 gene; Caenorhabditis elegans; Cavernous Hemangioma; Cavernous Malformation; Cerebrum; Cessation of life; Characteristics; Clinical; Clinical Research; Clinical Trials; Collaborations; Commit; Communities; Consultations; DNA; DNA Library; Data; Development; Disease; Drosophila genus; Educational workshop; Etiology; Excision; Financial Support; Funding; Future; Generations; Genetic; Government Agencies; Headache; Hemangioma; Hemorrhage; Individual; Industry; Institutional Review Boards; Interdisciplinary Study; International; Internet; Lesion; Medicine; Mission; Modeling; Molecular; Motivation; Mus; Mutation; Nature; Neuraxis; Neurologic; Operative Surgical Procedures; Participant; Pathway interactions; Patients; Pharmaceutical Preparations; Pharmacological Treatment; Physicians; Recording of previous events; Research; Research Personnel; Research Project Grants; Rho-associated kinase; Sampling; Schedule; Science; Seizures; Series; Signal Transduction; Site; Spinal Cord; Structure; Surgeon; Symptoms; Tight Junctions; Tissue Banking; Tissue Banks; Tissue Sample; Training; Translating; Vascular Diseases; Writing; Zebrafish; abstracting; base; biobank; brain surgery; cohort; data sharing; empowered; field study; flexibility; innovation; meetings; multidisciplinary; novel; patient advocacy group; patient registry; pre-clinical; public health relevance; success; symposium; unpublished works

 DESCRIPTION (provided by applicant): Angioma Alliance is the Patient Advocacy Group for and by those affected by Cerebral Cavernous Malformations (CCM) - a vascular disorder with unknown disease etiology and no available pharmacological treatment. Our mission is two-fold: to support and educate the patient community and to drive research for a cure. CCM is a brain disease typified by development of vascular lesions that are multi- cavernous, thin-walled structures with inadequate tight junctions and a propensity for hemorrhage. Lesion hemorrhage can result in a variety of clinical symptoms including, most severely, hemorrhagic stroke and/or death. While the biological mechanisms for lesion development and progression continue to be elucidated and no pharmacological treatment is yet available for the patient community. The only treatment remains surgical removal of the lesion; however, a surgical option may not be suitable for all patients, particularly those with multiple and/or deep brain lesions. Facilitating research for the advancement and rapid development of a pharmacological treatment for CCM is the prime motivation for the efforts of the CCM patient community. In order to facilitate novel and collaborative research, Angioma Alliance is committed to hosting the annual CCM Scientific Meeting. This meeting brings together stakeholders from research, advocacy, and government agencies to share and discuss unpublished research in an effort to drive innovation and generate unique and multidisciplinary hypotheses. No other meeting in the world draws together the same cohort of investigators - last year's meeting included surgeons and physicians, geneticists and microbiologists, as well as animal research teams focusing on murine, zebrafish, C. elegans and Drosophila models. The strength of this meeting, and the reason attendees commit to annual attendance, is the opportunity for interactive discussions, data sharing, hypothesis generation, and collaboration. Importantly, the meeting organizers insist on the presentation of unpublished work; therefore, the CCM Scientific Meeting is often the site for first public presentation of important findings that are critical for advancing the field. The primary objective of this proposal is to host the 10th anniversary CCM Scientific Meeting. The nine-year history for this meeting series is one of success and progress in the field of CCM research, much of which can be attributed to the new collaborations initiated at the meeting series. The inaugural CCM meeting was held as an informal single-day workshop for investigators to meet face-to-face and discuss their unpublished data - the agenda was flexible and the discussions were lively. This small workshop has grown to become the premier international scientific conference for those studying any and all aspects of CCM research. The primary objectives of the 2014 CCM Scientific Meeting are: 1. To expand and continue to build upon past meeting success by focusing on new research findings from diverse investigators and facilitating interdisciplinary interactions between investigators who otherwise would not have the opportunity to meet face-to-face; and 2. To translate basic and preclinical findings to develop an inclusive, collaborative and global action plan for clinical trial efforts using a moderated exper panel discussion; and 3. To provide educational and training opportunity for junior investigators.

 描述（由申请人提供）：血管瘤联盟是受脑海绵状血管瘤 (CCM) 影响的患者权益组织，也是受其影响的患者权益组织。CCM 是一种病因不明且无可用药物治疗的血管疾病。我们的使命有两个：支持和教育患者社区并推动治疗研究。 CCM 是一种脑部疾病，其特征为血管病变的发展，该病变为多海绵状、薄壁结构，紧密连接不充分且有出血倾向。病变出血可导致多种临床症状，包括最严重的出血性中风和/或死亡。虽然病变发生和进展的生物学机制仍在不断阐明，但患者群体尚无可用的药物治疗方法。唯一的治疗方法仍然是手术切除病变；然而，手术选择可能并不适合所有患者，特别是那些患有多个和/或深部脑损伤的患者。促进 CCM 药物治疗的进步和快速发展的研究是 CCM 患者群体努力的主要动机。为了促进新颖的协作研究，血管瘤联盟致力于主办年度 CCM 科学会议。这次会议汇集了来自研究、倡导和政府机构的利益相关者，分享和讨论未发表的研究，以推动创新并产生独特的多学科假设。世界上没有其他会议聚集了同样一批研究人员 - 去年的会议包括外科医生和内科医生、遗传学家和微生物学家，以及专注于鼠类、斑马鱼、线虫和果蝇模型的动物研究团队。这次会议的优势以及与会者承诺每年出席的原因是互动讨论、数据共享、假设生成和协作的机会。重要的是，会议组织者坚持展示未发表的作品；因此，CCM 科学会议通常是第一次会议的举办地 公开展示对于推进该领域至关重要的重要发现。该提案的主要目标是举办 CCM 十周年科学会议。该系列会议九年的历史是 CCM 研究领域的成功和进步之一，其中很大一部分可以归功于该系列会议发起的新合作。首次 CCM 会议以非正式的单日研讨会形式举行，研究人员可以面对面讨论他们未发表的数据 - 议程灵活，讨论活跃。这个小型研讨会已发展成为研究 CCM 研究各个方面的人士的首要国际科学会议。 2014 年 CCM 科学会议的主要目标是： 1. 通过关注不同研究人员的新研究成果并促进没有机会面对面交流的研究人员之间的跨学科互动，扩大并继续巩固过去会议的成功； 2. 通过主持的专家小组讨论，转化基本和临床前研究结果，为临床试验工作制定包容性、协作性和全球性的行动计划； 3. 为初级研究者提供教育和培训机会。