International Symposium on Juvenile Myelomonocytic Leukemia (JMML)

幼年型粒单核细胞白血病国际研讨会（JMML）

• 批准号: 8706070
• 负责人: REBECCA J. CHAN
• 金额: $ 0.5万
• 依托单位: JMML FOUNDATION
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-09-24 至 2015-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8706070
• 关键词: Adopted; Advertising; Advisory Committees; Advocate; Affect; Allogenic; American Society of Hematology; Applications Grants; Area; Attention; Basic Science; Biology; CBL gene; California; Cells; Child; Clinical; Clinical Research; Clinical Trials; Collaborations; Collection; Communication; Communities; Consensus; Cytotoxic Chemotherapy; Data; Data Reporting; Development; Diagnostic; Disease; Disease remission; Disease-Free Survival; Doctor of Philosophy; Enrollment; Ethnic group; Etiology; Event; Family Physicians; Family member; Foundations; Funding; Future; Genotype; Goals; Granulocyte-Macrophage Colony-Stimulating Factor; Hematopoietic stem cells; Human; Human Resources; Hypersensitivity; Individual; Infant; Innovative Therapy; International; Investigation; Juvenile Myelomonocytic Leukemia; KRAS2 Gene Mutation; Knowledge; Laboratory Markers; Laboratory Research; Life; Loss of Heterozygosity; MAP Kinase Gene; Macrophage Colony-Stimulating Factor; Malignant Neoplasms; Manuscripts; Measurement; Medical; Meta-Analysis; Mutation; Myelodysplastic/Myeloproliferative Disease; NF1 gene; Newsletter; Oncogenic; Outcome; PTPN11 gene; Participant; Pathogenesis; Pathway interactions; Patients; Pediatrics; Phenotype; Physicians; Postdoctoral Fellow; Principal Investigator; Probability; Production; Productivity; Publishing; Ras Signaling Pathway; Recurrent disease; Relapse; Request for Applications; Research; Research Personnel; Risk; Role; Sampling; San Francisco; Scientist; Stem cell transplant; Stratification; Therapeutic; Therapeutic Agents; Transplantation; Tumor Suppressor Genes; Underrepresented Minority; Universities; Update; base; beneficiary; chemotherapy; data sharing; experience; improved; innovation; interest; leukemia; meetings; member; pre-clinical; professor; research study; response; symposium; working group

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.)

描述（由申请人提供）：我们请求支持为研究幼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，还将邀请几位治疗医生和患有这种疾病的儿童家属参加。 JMML 是一种侵袭性白血病，影响婴儿和幼儿。它很快就会致命，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来实现，这可能对这些幼儿的发育产生毁灭性的影响。过去几年中已经取得了几项重要的科学突破，这些突破明确表明 Ras/MAPK 通路的过度激活是这种疾病的始发事件。鉴于对致癌 Ras 在癌症中的作用的积极研究，以及对这种致命疾病改进疗法的需求，本次会议为基础科学研究人员提供了一个重要的场所；临床科学家、监管科学家和受影响者的家人可以互动和协作。 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进展，以加强和延续该领域研究人员之间的国际合作； o 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； 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具有重叠兴趣的研究人员参与 JMML 研究，吸引新的研究人员参与；和 o 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，并与美国血液学会联合举行会议。通过这种方式，我们将利用并继续之前会议期间建立的成功的国际合作。为了最大限度地提高生产力，我们建议采用交替的年度会议形式，奇数年举行正式研讨会，偶数年举行非正式工作组。 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。本次拨款申请的首席研究员 (PI) 包括 Mignon Loh 医学博士（旧金山加利福尼亚大学临床儿科副教授、基金会医学咨询委员会成员）和 Sandra Thomas 博士（官员兼主任） JMML 基金会的。博士。 Kevin Shannon、Charlotte Niemeyer、Stephan Grupp、Atsushi Manabe、Christian Flotho 和 Rebecca Chan 以及 Fred Dini 先生将成为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 名参与者参加，2013 年将有 75 名参与者。将特别关注包括初级研究人员、博士后研究员、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。）