Acute Lymphoblastic Leukemia (ALL) is the most common blood cancer in children. Although we have seen dramatic improvements in the outcome for patients with this cancer, the current treatments do not cure everyone and are associated with many side effects, including the potential for development of other tumors years after therapy has been completed. Children with Down Syndrome (DS), who have an extra copy of chromosome 21 in all of their cells, are at a 20-fold increased risk of developing ALL. Moreover, these children tend to develop more severe side effects from methotrexate, one of the current standard therapies. Thus, there is an urgent need to develop new, less toxic therapies that specifically target the abnormal ALL cells in these children. In our research, we are studying the role of a protein named DYRK1A in ALL. We hypothesize that this protein plays a critical role in the growth of B cells, and that inhibiting its function will block the growth of ALL cells. Our mission is to better understand how DYRK1A works and to develop new treatments for ALL in both children with and without DS, with an emphasis on the DS group.
|Effective start/end date||7/30/14 → 7/29/15|
- Rally Foundation, Inc. (Agmt 5/16/14)