Olutasidenib offers new hope for myelodysplastic syndrome patients
A trial has demonstrated that olutasidenib is highly effective in treating intermediate- to very high-risk mutant IDH1 myelodysplastic syndrome (MDS), previously thought to be incurable without transplantation.
Research from the University of Miami (FL, USA) has revealed that olutasidenib, a targeted drug already indicated for mIDH1 relapsed/refractory acute myeloid leukemia (AML), is also highly effective against mIDH1 MDS. Current care for MDS is limited to blood transfusions, traditional chemotherapy, such as azacitidine, and stem cell transplants; high- and very high-risk MDS is considered incurable.
Justin Watts and his team administered olutasidenib to 22 MDS patients who had tumors with IDH1 mutations, of which 86% were classified as having high or very high risk disease. Participants received olutasidenib or olutasidenib with azacitidine at their physician’s discretion.
Overall, 59% of the study population responded to the treatment with high rates of conversion from transfusion-dependence to independence. The median overall survival rate of 27.2 months across the study population.
“We saw really quite remarkable outcomes in a very high-risk MDS population, not only in response rates, but also in blood count improvement, long duration of response and improved overall survival,” explained Watts. He also noted that these results aligned with those reported for another inhibitor of mIDH1, ivosidenib, commenting, “Both are exceptional drugs.”
Insights from 5-year data on olutasidenib for mutated IDH1 acute myeloid leukemia
We sat down with Justin Watts to discuss data from the Phase II trial that led to the US FDA approval of olutasidenib for IDH1-mutated AML.
This work is part of a larger project at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, to study other tools to tackle blood cancer. Watts plans to look further into the potential of olutasidenib to elicit long-term responses in AML and MDS patients.
“We’re trying to get a more precise signature of who these patients are. How can we tell which patients are going to do well with this therapy and have longer-term remissions?” considered Watts. “That’s the next paper.”