International Symposium on Juvenile Myelomonocytic Leukemia

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在肿瘤形成中的作用的积极研究，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患者的家人可以互动和合作的关键场所。因此，JMML基金会的年度研讨会有四个主要目标：1.通过促进该领域领先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发展；2.为这种疾病的患者提供一个规划和实施创新临床试验的场所；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，JMML的初级研究人员可以在其中介绍他们的工作，并与该领域的彼此和高级调查人员进行互动。连续五年寻求支持，并与美国血液病学会联合举行会议。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。2007年会议将于12月6日至7日在佐治亚州亚特兰大举行。这次会议的PI是JMML基金会董事会成员、医学博士Mignon Loh。她和她的同事克里斯蒂安·克拉茨博士和凯文·香农博士将在JMML基金会的组织支持下计划这次会议。预计将有50-60人参加。将特别注意包括初级调查人员、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。

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 shortlived. 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 neoplasia 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 thus 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. Support is sought for five 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. The 2007 meeting will be held December 6-7 in Atlanta, Georgia. The PI of this meeting is Mignon Loh, MD, who is a member of the board of directors of The JMML Foundation. She and her colleagues, Drs. Christian Kratz and Kevin Shannon will plan the meeting with organizational support from The JMML Foundation. A total of 50-60 participants are anticipated to attend. Special attention will be made to include junior investigators, post-doctoral fellows, and clinicians and scientists from underrepresented communities.