“Drug screen for FPD/AML therapeutics.”

Dr. Poncz and his team propose to enhance RUNX1 levels with the target of correcting the platelet problem, expecting that correction will also correct the risk of getting leukemia. The group specializes in studies on how to make platelets from megakaryocytes and has already identified one drug called RepSox that can correct many of the megakaryocyte defects seen in FPD cells. 

They will continue studies with RepSox and also do a screening of drugs looking for other compounds that correct FPD megakaryocytes at doses that an individual might be able to take on a long-term basis. Drugs will also be tested in RUNX1-deficient mice, adjusting candidate drugs to the lowest level that corrects the platelet counts in these mice. 

The longer-term goal is to test whether that dose protects the mice from getting leukemia. The lab envisions applying this strategy to affected individuals who will take the drug at the lowest dose that corrects their platelet count, with the expectation that the dose will also protect the individual from AML with little side effects. They believe that the proposed strategy offers a short track for drug identification that both reduces the risk of bleeding and of developing AML.