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New Treatment Approved for Certain Patients Living With Myelodysplastic Syndromes

June 07, 2024
by Michael Masciadrelli

The U.S. Food and Drug Administration has approved imetelstat (known commercially as RYTELO) to treat some patients with the rare blood cancer, myelodysplastic syndromes (MDS).

Amer Zeidan, MBBS, chief of hematologic malignancies at Yale Cancer Center and associate professor of internal medicine (Hematology) at Yale School of Medicine, was one of the investigators on the IMerge phase III trial that evaluated imetelstat and the senior author of the research that was published in the Lancet. The trial showed that imetelstat, a first-in-class telomerase inhibitor, leads to durable independence from red blood cell transfusions and a significant improvement in anemia (a condition in which the blood doesn’t have enough healthy blood cells and hemoglobin) for heavily transfused lower-risk MDS patients.

“Few things in a clinical investigator’s career matter as much as touching the lives of many patients he will never see by getting a good drug approved,” Dr. Zeidan said.

MDS is characterized by blood cells in the bone marrow failing to mature into healthy blood cells, causing low blood counts, and health complications that reduce quality of life and shorten survival. RYTELO is for adult patients with low- to intermediate-1 risk MDS, who are transfusion-dependent and require four or more red blood cell units over eight weeks. The new treatment aims to impede cancer growth for these patients with MDS, which affects mostly adults over 60 who are male.

To learn more about Dr. Zeidan’s work and this new MDS treatment, see this article that includes an interview with Dr. Zeidan or click here to read about the findings of the trial that was published in the Lancet.

Submitted by Michael Masciadrelli on June 07, 2024