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 shortlived. 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 neoplasia 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 thus 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. Support is sought for five 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. The 2007 meeting will be held December 6-7 in Atlanta, Georgia. The PI of this meeting is Mignon Loh, MD, who is a member of the board of directors of The JMML Foundation. She and her colleagues, Drs. Christian Kratz and Kevin Shannon will plan the meeting with organizational support from The JMML Foundation. A total of 50-60 participants are anticipated to attend. Special attention will be made to include junior investigators, post-doctoral fellows, and clinicians and scientists from underrepresented communities. This grant application requests support for an annual scientific meeting focused on the biology of Juvenile Myelomonocytic Leukemia for five consecutive years. The meetings will be held in conjunction with the 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 disorder.
|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|