International Symposium on Juvenile Myelomonocytic Leukemia (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，已经取得了一些重要的科学突破，这些突破坚定地暗示Ras/MAPK通路的过度活化是这种疾病的起始事件。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具体目标： 目标1。分享JMML实验室和临床研究的最新进展，以加强和保持该领域研究人员之间的国际合作; 目标2。为参加国际临床试验的JMML患者提供协调标准化测量的场所; 目标3。通过鼓励初级科学家、代表性不足的少数民族研究人员和兴趣重叠的研究人员参加JMML研究，吸引新的研究人员; 目标4。促进JMML研究者和患者倡导者之间的沟通，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，并与美国血液学学会一起举行会议。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。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。本补助金申请的主要研究者（PI）是Mignon洛，MD，加州大学旧金山分校弗朗西斯科临床儿科学副教授，基金会医学咨询委员会成员，以及Sandra托马斯，PhD，JMML基金会的官员和董事。Kevin Shannon、夏洛特尼迈耶、Stephan Grupp、Atsushi Manabe、Christian Flotho和Rebecca Chan博士以及Fred Dini先生将是会议规划团队的关键人员，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。将特别注意包括初级研究人员，博士后研究员，来自相关领域的新研究人员以及来自代表性不足社区的临床医生和科学家。

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.)