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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.