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Research achievements

Key research achievements in the history of DF/CHCC

  • The first successful leukemia remission by Sidney Farber, MD, in 1947
  • Introduction of multiple-agent treatment (combination chemotherapy) for leukemia in 1972. With successive improvements, childhood leukemia cure rates now stand at 80 percent or more.
  • Discovery of interleukin-11 (IL-11) and leadership in its development as a drug (Neumega™) to treat chemotherapy-induced thrombocytopemia 
  • Leadership in developing a new drug called defibrotide as a promising new treatment for hepatic veno-occlusive disease, a major complication of stem cell transplantation
  • Leadership in developing national standards of care for pediatric cancer survivors and pediatric palliative care
  • Molecular cloning of the gene for von Willebrand's disease, leading to development of recombinant von Willebrand's factor (vWF) for clinical use (currently in phase III clinical trials)
  • The first published experience with double autologous peripheral blood stem cell transplant for high-risk neuroblastoma
  • Leadership in clinical trials for Ewing sarcoma, which have driven five-year event-free survival rates to at about 70 percent
  • Initiation of the first antiangiogenic treatment trials for pediatric brain tumors
  • Trials in FA and dyskeratosis congenita, a pair of rare, life-threatening genetic blood diseases
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