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.)

Public Health Relevance

This grant application requests support for an annual scientific meeting focused on the biology of Juvenile Myelomonocytic Leukemia for four consecutive years. The meetings will be held in conjunction with annual American Society of Hematology meetings. This symposium will focus on recent advances in the pathogenesis of JMML, with invited scientists and clinicians who have demonstrated expertise in Ras pathway signaling, pre-clinical therapeutics, and the conduct of human clinical trials. The overall goal is to provide an opportunity for researchers, physicians, and physician-scientists to share data and implement innovative therapies for children afflicted with this aggressive myeloproliferative/myelodysplastic disorder.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Conference (R13)
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Special Emphasis Panel (ZCA1-PCRB-G (P3))
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Merritt, William D
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Jmml Foundation
San Diego
United States
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Niemeyer, Charlotte M; Loh, Mignon L; Cseh, Annamaria et al. (2015) Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. Haematologica 100:17-22
Chan, Rebecca J; Cooper, Todd; Kratz, Christian P et al. (2009) Juvenile myelomonocytic leukemia: a report from the 2nd International JMML Symposium. Leuk Res 33:355-62