Lucio Castilla receives NIH grant to explore therapeutic approach to delay onset of blood cancer

Lucio Castilla, PhD, has received NIH funding from the National Cancer Institute (NCI) to study tyrosine kinase inhibitors as preventive therapy in RUNX1-familial platelet disorder (RUNX1-FPD)—a type of hereditary blood disease, caused by mutations in the RUNX1 gene, that increases the lifetime risk of developing blood cancer. The grant, carried out in collaboration with Paul Liu, MD, PhD, at the NIH, will explore whether imatinib, a drug that is used to treat chronic myelogenous leukemia, can prevent or delay the development of blood cancer in a mouse model of RUNX1-FPD. The results of the study may support the use of imatinib in a phase-II clinical trial as preventive therapy in patients with RUNX1-FPD.