International Symposium on Juvenile Myelomonocytic Leukeumia (JMML)

描述（由申请人提供）：我们正在请求支持为研究青少年粒单核细胞白血病（JMML）的研究人员举办年度科学会议。此外，还将邀请几位主治医生和患有这种疾病的儿童的家属参加。JMML是一种侵袭性白血病，影响婴儿和幼儿。它是迅速致命的，对化疗的反应通常是短暂的。目前，唯一已知的治愈方法是通过造血干细胞移植，这可能对这些幼儿的发育产生毁灭性的影响。在过去的几年里，已经取得了一些关键和重要的科学发现，部分是通过R13会议资助的可用性，这进一步加深了我们对JMML作为一种由Ras/MAPK通路过度激活引发的疾病的理解。鉴于致癌Ras在癌症中的作用的积极调查，以及对这种致命疾病的改进疗法的需求，本次会议提供了一个关键的场所，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庭成员可以进行互动和合作。JMML基金会的年度研讨会有四个主要目标：1。通过培养该领域的国内和国际领导人的高水平演讲，分享JMML科学和临床研究的最新进展; 2.为规划和实施针对该疾病患者的创新临床试验提供场所; 3.促进JMML研究人员和患者权益倡导者之间的沟通;以及4.提供一个论坛，让司法监测和监察研究所的初级调查员介绍他们的工作，相互交流，并与外地的高级调查员交流。我们连续五年寻求支持，会议与美国血液学会年会一起举行。通过这种方式，我们将利用并继续在以前的会议期间成功开展的国际合作。为了最大限度地提高生产力，我们建议每年交替举行会议，奇数年举行正式专题讨论会，偶数年举行非正式工作组会议。2010年会议将于1- 2月1日至2日在佛罗里达的奥兰多举行。本次会议的PI是Sandra托马斯博士，JMML基金会的官员和Mignon洛博士，基金会医疗顾问委员会的成员。Christian Flotho博士和Fred Dini先生也将成为会议规划团队的一员，并得到JMML基金会的额外组织支持。预计2010年将有40人参加，2011年将有75人参加。将特别注意包括初级研究人员，博士后研究员，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：项目叙述/公共卫生相关性该资助申请要求支持连续五年关注青少年粒单核细胞白血病（JMML）的生物学和临床治疗的年度科学会议。本次研讨会将重点关注JMML起源和发展的最新进展，邀请在Ras通路信号传导，治疗方案的实验室测试和人体临床试验方面具有专业知识的科学家和临床医生。总体目标是为研究人员，医生和医生科学家提供一个机会，分享数据并为患有这种侵袭性血液癌症的儿童实施创新疗法。

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 critical and important scientific findings have been made over the past several years, in part through the availability of this R13 conference grant, that have furthered our understanding of JMML as a disease initiated by hyperactivation of the Ras/MAPK pathway. 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 major goals: 1. to share the latest developments in JMML scientific and clinical research by fostering high level presentations from leading national and international leaders in the field; 2. to provide a venue for planning and implementing innovative clinical trials for patients with this disease; 3. to facilitate communication between JMML investigators and patient advocates; and 4. to provide a forum in which junior investigators in JMML can present their work and interact with each other and with senior investigators in the field. We seek support for five consecutive years, with meetings being held in conjunction with the American Society of Hematology annual meeting. 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 2010 meeting will be held December 1-2 in Orlando, Florida. The PIs of this meeting are Sandra Thomas, PhD, an officer of The JMML Foundation and Mignon Loh, MD, a member of the foundation's Medical Advisory Board. Dr. Christian Flotho and Mr. Fred Dini will also be part of the conference planning team with additional organizational support from The JMML Foundation. A total of 40 participants are anticipated to attend in 2010 and 75 in 2011. Special attention will be made to include junior investigators, post-doctoral fellows, and clinicians and scientists from underrepresented communities.) PUBLIC HEALTH RELEVANCE: PROJECT NARRATIVE/PUBLIC HEALTH RELEVANCE This grant application requests support for an annual scientific meeting focused on the biology and clinical treatment of Juvenile Myelomonocytic Leukemia (JMML) for five consecutive years. This symposium will focus on recent advances in the understanding of the origination and development of JMML, with invited scientists and clinicians who have demonstrated expertise in Ras pathway signaling, laboratory testing of treatment options, 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 cancer of the blood.