Sangeetha Venugopal, MD, of the University of Miami Sylvester Comprehensive Cancer Center, offers her thoughts on the investigational drug KER-050 in lower-risk myelodysplastic syndromes (MDS), the COMMANDS trial, and whether she believes MDS is a distinct disease.
KER-050 is being evaluated in an ongoing phase II trial in patients with transfusion-dependent lower-risk MDS. KER-050 targets specific proteins (activins A, B, and growth differentiation factors 8, 11) that are part of the TGF-β superfamily. It works by helping the development and maturation of cells responsible for producing red blood cells and platelets. This drug could potentially reduce the need for red blood cell transfusions and help the body use iron more effectively in making new red blood cells, which might also reduce iron overload directly at its source, according to the authors of the abstract presented at the 65th ASH Annual Meeting.
“The one thing that caught my attention was this drug was particularly active even in those patients without ring sideroblasts,” she said.
She also highlighted the latest research in stem cell transplant in MDS from the meeting. One abstract in particular introduced a clinical- and genomic-based decision support system to determine the optimal timing for allogeneic hematopoietic stem cell transplantation in patients with MDS. This system uses the Molecular International Prognostic Scoring System (IPSS-M) and Revised IPSS (IPSS-R) for post-transplant outcome stratification, she explained.
“What they found was very interesting,” Dr. Venugopal said. “When they did categorize based on IPSS-M, patients with either low or moderate low-risk benefit did benefit from a delay [in] transplant, and those belonging to moderate-high and very high-risk categories, immediate transplant was associated with improved outcomes regardless of age, and these results were confirmed with the validation cohort attesting to the reliability and generalizability of this decision model.”
In terms of the question of if she believes MDS is a distinct disease, she gave an unequivocal yes.
“I come from the stable of MD Anderson Cancer Center and [Guillermo] Garcia-Manero, MD, so this is entirely a different disease.”