International Symposium on Juvenile Myelomonocytic Leukemia (JMML)

幼年型粒单核细胞白血病国际研讨会（JMML）

基本信息

批准号：
8986009
负责人：
REBECCA J. CHAN
金额：
$ 0.4万
依托单位：
INDIANA UNIV-PURDUE UNIV AT INDIANAPOLIS
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
起止时间：
2007-09-24 至 2016-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8986009
关键词：
4 year old Advocate Affect Alabama Algorithms Allogenic American Society of Hematology Applications Grants Authorship Biology Blast Phase Bone Marrow Purging CSF1 gene Caring Child Childhood Clinical Clinical Research Clinical Treatment Clinical Trials Clinical Trials Design Collaborations Communication Communities Comprehensive Cancer Center Consensus Development Diagnostic Discrimination Disease Doctor of Medicine Doctor of Philosophy Etiology Europe Extramedullary Face Family Fathers Fostering Foundations Functional disorder Future Genetic Models Goals Grant Granulocyte-Macrophage Colony-Stimulating Factor HRAS gene Hematopoietic Stem Cell Transplantation Hematopoietic stem cells Hypersensitivity Induced Mutation Infant International Intervention Japan Juvenile Myelomonocytic Leukemia Lead Liver Locales Lung Lymphoproliferative Disorders MEK inhibition MEKs Malignant Neoplasms Malignant neoplasm of lung Malignant neoplasm of pancreas Molecular Morbidity - disease rate Mus Mutate Mutation Myeloid Cells Organ failure Outcome Patient observation Patients Pediatric Oncology Pharmacologic Substance Physicians Publications Rare Diseases Recurrent disease Relapse Reporting Request for Applications Research Research Personnel Risk Sampling Scientist Signal Pathway Signaling Protein Somatic Mutation Spleen Stem cell transplant Syndrome Time Tissues Toxic effect Transplantation United States Universities Ursidae Family Work aggressive therapy autoimmune lymphoproliferative syndrome base design disease classification gain of function improved inhibitor/antagonist innovation interest leukemia meetings member mortality mouse model novel preclinical study professor public health relevance response small molecule symposium targeted treatment working group

项目摘要

DESCRIPTION (provided by applicant): Juvenile myelomonocytic leukemia (JMML) is a rare, fatal childhood disease that affects infants and young children less than 4 years of age. JMML is characterized by aberrant myeloid cell overproduction and hypersensitivity to GM-CSF. The only curative therapy is allogeneic hematopoietic stem cell transplantation; however, approximately half of children relapse after this aggressive therapy. Thus, while great advances have been made in understanding the molecular basis for JMML (caused by mutations inducing gain-of- function of the RAS-ERK signaling pathway), unfortunately, improved therapies for this disease remain to be defined and are urgently needed. Over several years, an international community of families, scientists, and clinicians have merged their efforts to advance the research and improved treatment for JMML. A key unifying aspect of this collaborative effort has been the bi-annual International JMML Symposia alternating with bi-annual JMML Working Groups which precede the annual American Society of Hematology (ASH) meetings. These pre-ASH meetings (supported by the NCI via this R13 conference grant since 2007) provide the perfect locale for gathering of JMML clinicians and scientists from Europe, Japan, and the United States, and afford an efficient means of stimulating open communication and collaboration between the various groups. An international approach to this disease is particularly imperative since JMML is a rare disease. The annual symposium organized and sponsored by 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 the 2015 JMML International Symposium, which will be held in conjunction with the American Society of Hematology annual meeting in Orlando, FL. The PI for this meeting, Rebecca J. Chan, M.D., Ph.D., is a member of the JMML Foundation Board of Directors and researcher in the field. Additionally, Mignon L. Loh, M.D., a JMML researcher and clinician, Mr. Fred Dini, President of the JMML Foundation, and Dr. Sandra Thomas, will also be part of the conference planning team.

描述（由申请人提供）：幼年型粒单核细胞白血病 (JMML) 是一种罕见的致命性儿童疾病，影响 4 岁以下的婴儿和幼儿。 JMML 的特征是骨髓细胞过度生成和对 GM-CSF 过敏。治愈性治疗是同种异体造血干细胞移植；然而，尽管取得了巨大进展，但大约一半的儿童在这种积极治疗后复发。不幸的是，在了解 JMML 的分子基础（由诱导 RAS-ERK 信号通路功能获得的突变引起）的过程中，该疾病的改进疗法仍有待确定，并且迫切需要国际社会在几年内进行改进。家庭、科学家和爱好者齐心协力推进 JMML 的研究并改善 JMML 的治疗，这一合作努力的一个关键统一方面是每年两次的国际 JMML 研讨会与每年两次的 JMML 工作组交替举行。这些 ASH 前会议（自 2007 年起由 NCI 通过 R13 会议拨款支持）为来自欧洲、日本和美国的 JMML 最爱和科学家提供了完美的聚会场所。鉴于 JMML 是一种罕见疾病，JMML 基金会组织和赞助的年度研讨会有四个主要内容。目标： 1. 通过促进该领域领先的国内和国际领导者的高水平演讲，分享 JMML 科学和临床研究的最新进展； 2. 为该疾病患者规划和实施创新临床试验提供场所； . 促进 JMML 研究者和患者倡导者之间的沟通；以及 4. 提供一个论坛，让 JMML 的初级研究者能够展示他们的工作并相互交流，并与该领域的高级研究者进行互动。研讨会将与在佛罗里达州奥兰多举行的美国血液学会年会同时举行。本次会议的首席研究员 Rebecca J. Chan 医学博士、博士是 JMML 基金会董事会成员， JMML 研究员和临床医生 Mignon L. Loh 医学博士、JMML 基金会主席 Fred Dini 先生和 Sandra Thomas 博士也将参加会议策划团队。