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: 8546684
• 负责人: Mignon Lee-Cheun Loh
• 金额: $ 1万
• 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/8546684
• 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）：我们正在申请支持一个青少年髓细胞白血病（JMML）研究人员的年度科学会议。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。jml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，唯一已知的治疗方法是通过造血干细胞移植实现的，这可能对这些幼儿的发育产生破坏性影响。在过去的几年中，已经取得了一些重要的科学突破，这些突破坚定地表明Ras/MAPK通路的过度激活是该疾病的起始事件。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的改进治疗的需要，本次会议为基础科学研究者提供了一个重要的场所；临床科学家，监管科学家和受影响的家庭成员可以相互作用和合作。JMML基金会的年度研讨会有四个具体目标：