International Symposium on Juvenile Myelomonocytic Leukemia (JMML)

描述(由申请人提供)：我们请求支持为研究青少年粒单核细胞白血病(JMML)的研究人员举行的年度科学会议。此外，几名治疗医生和患有这种疾病的儿童的家人将被邀请参加。JMML是一种侵袭性白血病，影响婴儿和幼儿。它是迅速致命的，而且对化疗的反应通常是短暂的。目前，唯一已知的治疗方法是通过造血干细胞移植实现的，这可能会对这些年幼的儿童产生毁灭性的发育影响。在过去的几年里，已经取得了几项重要的科学突破，坚定地表明RAS/MAPK通路的过度激活是这种疾病的始动事件。鉴于对致癌RAS在癌症中的作用的积极调查，以及对这种致命疾病改进治疗的需要，这次会议是基础科学研究人员、临床科学家、监管科学家和受影响者的家人可以互动和合作的关键场所。JMML基金会的年度研讨会有四个具体目标： O目标1.分享JMML实验室和临床研究的最新发展，以加强和保持该领域研究人员之间的国际合作； O目标2.为登记参加国际临床试验的JMML患者提供协调标准化测量的场所； O目标3.通过鼓励初级科学家、代表性不足的少数族裔研究人员和兴趣重叠的研究人员参加，使新的研究人员参与JMML研究；以及 O目的4.促进JMML研究人员和患者权益倡导者之间的沟通，以鼓励JMML患者参与正在进行的基础和临床研究。 我们连续四年寻求支持，会议是与美国血液学会联合举行的。通过这种方式，我们将利用并继续利用前几次会议确定的成功的国际合作。为了最大限度地提高生产率，我们建议轮流举行年度会议，在奇数年举行正式研讨会，在偶数年举行非正式工作组会议。2011年会议将于12月8日至9日在加利福尼亚州圣地亚哥举行。这项赠款申请的首席研究员是Mignon Loh医学博士，他是加州大学旧金山分校的临床儿科学副教授，也是该基金会的医学咨询委员会成员，以及Sandra Thomas博士，他是JMML基金会的官员和董事。凯文·香农博士、夏洛特·尼迈耶博士、斯蒂芬·格鲁普博士、真部敦博士、克里斯蒂安·弗洛托博士、丽贝卡·陈博士以及弗雷德·迪尼先生将是会议策划团队的关键人员，JMML基金会将提供额外的组织支持。预计2011年将有60人参加，2013年将有75人参加。将特别注意初级调查人员、博士后研究员、相关领域的新调查人员以及来自代表性不足社区的临床医生和科学家。) 公共卫生相关性：这项赠款申请要求为连续四年以青少年粒单核细胞白血病生物学为重点的年度科学会议提供支持。这些会议将与美国血液病学会的年度会议一起举行。本次研讨会将聚焦于JMML发病机制的最新进展，邀请在RAS通路信号转导、临床前治疗和人类临床试验方面具有专长的科学家和临床医生参加。总体目标是为研究人员、医生和内科科学家提供机会，为患有这种侵袭性骨髓增生性/骨髓异常增生性疾病的儿童共享数据和实施创新疗法。

DESCRIPTION (provided by applicant): We are requesting support for an annual scientific meeting for investigators studying juvenile myelomonocytic leukemia (JMML). In addition, several treating physicians and families of children afflicted with this disease will be invited to attend. JMML is an aggressive form of leukemia that affects infants and young children. It is rapidly fatal, and responses to chemotherapy are generally short-lived. Currently, the only known cures are achieved through hematopoietic stem cell transplants, which can have devastating developmental effects on these young children. Several important scientific breakthroughs have been made over the past several years that firmly implicate hyperactivation of the Ras/MAPK pathway as the initiating event in this disease. Given the active investigation of the role of oncogenic Ras in cancer, and the need for improved therapies for this fatal disease, this meeting serves a critical venue in which basic science investigators; clinical scientists, regulatory scientists, and family members of those affected can interact and collaborate. The annual symposium of the JMML foundation has four Specific Aims: o Aim 1. Share the latest developments in JMML laboratory and clinical research to strengthen and perpetuate international collaboration among investigators in the field; o Aim 2. Provide a venue to coordinate standardized measurements for patients with JMML enrolled on international clinical trials; o Aim 3. Engage new investigators in JMML research by encouraging attendance by junior scientists, under-represented minority investigators, and researchers with overlapping interests; and o Aim 4. Facilitate communication between JMML investigators and patient advocates to encourage the participation of JMML patients in ongoing basic and clinical research studies. We seek support for four consecutive years, with meetings being held in conjunction with the American Society of Hematology. In this manner, we will capitalize on and continue successful international collaborations set during prior meetings. To maximize productivity, we propose an alternating yearly meeting format, with formal symposia in odd-numbered years and informal working groups in even-numbered years. The 2011 meeting will be held December 8-9 in San Diego, CA. The Principal Investigators (PIs) for this grant application are Mignon Loh, MD, an Associate Professor of Clinical Pediatrics at the University of California, San Francisco and a member of the foundation's Medical Advisory Committee, and Sandra Thomas, PhD, an Officer and Director of The JMML Foundation. Drs. Kevin Shannon, Charlotte Niemeyer, Stephan Grupp, Atsushi Manabe, Christian Flotho, and Rebecca Chan, as well as Mr. Fred Dini, will be key personnel on the conference planning team, and additional organizational support will be provided by The JMML Foundation. A total of 60 participants are anticipated to attend in 2011 and 75 in 2013. Special attention will be made to include junior investigators, post-doctoral fellows, new investigators from related fields and clinicians and scientists from underrepresented communities.) PUBLIC HEALTH RELEVANCE: This grant application requests support for an annual scientific meeting focused on the biology of Juvenile Myelomonocytic Leukemia for four consecutive years. The meetings will be held in conjunction with annual American Society of Hematology meetings. This symposium will focus on recent advances in the pathogenesis of JMML, with invited scientists and clinicians who have demonstrated expertise in Ras pathway signaling, pre-clinical therapeutics, and the conduct of human clinical trials. The overall goal is to provide an opportunity for researchers, physicians, and physician-scientists to share data and implement innovative therapies for children afflicted with this aggressive myeloproliferative/myelodysplastic disorder.