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The JAK2 gene is part of a signaling system that is crucial for hematopoiesis and that is inappropriately activated in the myeloproliferative diseases. Three years ago, a Harvard Medical School team identified a single point mutation in the JAK2 gene that is present in 99% of patients with polycythemia vera (PV) and in roughly half of patients with myelofibrosis and essential thrombocythemia. The team then created a mouse model with the same gene defect: Such mice developed PV.
The team next developed a small molecule that inhibited JAK2. Mice with PV that were treated with this small molecule had dramatic reductions in hematocrit and leukocyte counts, dose-dependent reductions in extramedullary hematopoiesis, and reductions in activity of t…