Experimental MDS Drug Appears Beneficial for Some

A clinical trial featuring an experimental drug may offer benefits for some patients with a deadly cancer of the blood-producing bone marrow cells, according to a researcher at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine.

“Many patients with myelodysplastic syndromes (MDS) require frequent red blood cell transfusions, resulting in a lower quality of life, as well as shorter lifespans from the disease,” said Mikkael Sekeres, MD, MS, Sylvester chief of the Division of Hematology Leukemia Section and professor of medicine at the Miller School. “In a new phase 3 clinical trial, the drug Imetelstat demonstrated clear advantages for these patients for whom other therapies either didn’t work or aren’t likely to work.”

A Global Cancer Collaboration

Dr. Sekeres serves on the international steering committee for the clinical trial and is co-author of a new Lancet study that involves researchers at 118 sites in 17 countries.

The Geron Corporation funded the study, which has obtained a standard review and has applied for Food and Drug Administration approval. A decision is expected by mid-2024.

“It is very gratifying to be part of research that could lead to the next approved drug for MDS patients,” said Dr. Sekeres.

Sylvester is one of the world’s leading MDS centers, added Dr. Sekeres, who chairs the Aplastic Anemia and MDS International Foundation.

A Deadly Cancer

About 20,000 new cases of MDS are diagnosed in the U.S. each year, and between 50,000 and 100,000 people currently are living with the disease, which has an average survival of three to five years. Most MDS patients develop anemia and typically require frequent transfusions, according to Dr. Sekeres, because the bone marrow does not produce enough red blood cells.

Lower-risk MDS patients with anemia are typically treated with medications that stimulate the hormone erythropoietin to make red blood cells.

“However, this only works in 25 to 40 percent and is only effective for 12 to 18 months,” said Dr. Sekeres. “That’s why the clinical trial of Imetelstat is so important for these patients.”

The Lancet study involved 178 MDS patients, with two-thirds receiving the drug and one-third given a placebo. Forty percent of the participants who were treated with Imetelstat were able to go without a blood transfusion for eight weeks or longer.

Those who responded, on average, remained transfusion-free for one year. Other benefits included an increase in hemoglobin levels and a reduction in patient-reported fatigue.

“This is the first trial showing statistically significant improvements in these MDS patients,” said Dr. Sekeres. “We look forward to the FDA’s review of Imetelstat as we continue to seek safe and effective treatments for this deadly cancer.”