PROJECT SUMMARY

Low red blood cell counts, also known as anemia, is the most common symptom among patients with myelodysplastic syndromes (MDS). Anemia leads to fatigue, decreased quality of life and eventually the need for blood transfusions in some patients. Understanding the causes of anemia in MDS is important to develop better therapies for patients.

In this project, we will evaluate momelotinib for the treatment of anemia in MDS. Momelotinib is an oral medication that has been approved by the FDA for the treatment of anemia due to another blood cancer called myelofibrosis. We believe that momelotinib can be an effective treatment for patients with MDS as it enhances the development of red blood cells in the bone marrow.

To test the safety and efficacy of momelotinib in MDS we plan a clinical trial in which patients with MDS will initially receive momelotinib tablets for 12 weeks. If the anemia does not improve with momelotinib, luspatercept, an injection approved for MDS, will be added to momelotinib as we think that momelotinib and luspatercept can enhance each other’s effect. We will perform additional experiments in the laboratory to evaluate how exactly these medications improve anemia and if there are factors that predict a higher likelihood of response.

This project could be the first step on the way to the development of the first oral medication for anemia in MDS (i.e., momelotinib), which which would eliminate the need for injections and clinic visits every 2-3 weeks.