The Medalist Trial: Luspatercept Reduced Transfusion Need in Some Myelodysplastic Syndromes

MedicalResearch.com Interview with:

Dr. Alan List MD President and Chief Executive Officer Moffitt Cancer Center Tampa, FL

Dr. List

Dr. Alan List MD
President and Chief Executive Officer
Moffitt Cancer Center
Tampa, FL

MedicalResearch.com: What is the background for this study?  

Response: In patients with lower risk Myelodysplastic Syndromes (MDS), which accounts for the vast majority of patients with MDS overall, the most common symptomatic cytopenia is anemia. These patients, overtime, become dependent upon red blood cell transfusions and with that, they face a risk of iron loading as well as complications that occur with it. The standard first line therapy that we consider for these patients is erythropoietin-stimulating agents (ESAs). Patients who are transfusion dependent have a low response rate to ESAs, and responses are of short duration. There limited effective limited treatment options for those patients unresponsive or lose response to ESAs.

For years, we’ve known that the transforming growth factor (TGF)-β pathway play an important pathogenetic role in suppressing red cell maturation and cell survival.

Luspatercept is an agent that acts as an erythroid maturation agent by inhibiting the TGF-β signaling pathway by neutralizing a select group of TGF-β superfamily ligands.  Continue reading