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FDA Approves ‘First Drug of its Kind’ for Myelodysplastic Syndrome

The FDA’s approval of imetelstat for certain patients with myelodysplastic syndrome-related anemia is a win for preserving a blood cancer patient’s quality of life. 

Rye Brook, N.Y., June 11, 2024 – The U.S. Food and Drug Administration (FDA) last week approved imetelstat (Rytelo™) to treat adults who have low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia. 

Anemia occurs when there is a low number of red blood cells in the body.

“Severe cases of anemia that require routine red blood cell transfusions are a major concern for patients with MDS,” says Lee Greenberger, Ph.D., Chief Scientific Officer of The Leukemia & Lymphoma Society (LLS). “Imetelstat is the first drug of its kind and offers durable transfusion independence for patients with MDS who do not respond to transfusions or are ineligible for standard treatments that are typically used to treat anemia.” 

Imetelstat is an inhibitor of telomerase. This is an enzyme that regulates the length of telomeres, which are protective caps at the end of DNA that shorten as cells divide. Patients with MDS have shorter telomeres but higher activity of telomerase, which leads to an inability to produce enough red blood cells. 

Imetelstat selective activity on malignant (cancerous) cells enables bone marrow recovery and leads to an increased production of red blood cells. This reduces the need for some patients with MDS to receive repeated blood transfusions who fail to respond to FDA-approved agents that stimulate the production of red blood cells. 

Learn more about anemia and other low blood count-related side effects on 

Reduction in Blood Transfusion Dependence

Imetelstat is approved for use in patients who require four or more transfusions of red blood cell units over eight weeks or who have not responded to, lost response to, or are ineligible for red blood cell-stimulating agents, which are commonly used to increase red blood cell production. 

In clinical trials, imetelstat was associated with both an eight-week and 24-week improvement in red blood cell transfusion independence. 

This means that more patients who received treatment with imetelstat did not need a red blood cell transfusion at any point during a consecutive eight- or 24-week period, compared to those who received a placebo.

“Preserving a blood cancer patient’s quality of life often takes a backseat to killing the cancer itself,” says Dr. Greenberger. “However, it’s important that we don’t overlook someone’s ability to carry out their daily routine while receiving treatment that attacks their blood cancer. Any treatment that helps improve quality of life is a much-needed development.” 

Investing in Research to Find MDS Cures

LLS has invested heavily in MDS research over its 75-year history with a proven track record of awarding research grants to investigators who have made a difference in the treatment of this blood cancer. 

Amer Zeidan, MBBS, who is a recent Career Development Program awardee, was heavily involved in the clinical trial that led to this approval of imetelstat. Dr. Zeidan, the inaugural Chief of the Division of Hematologic Malignancies at Yale Cancer Center, was awarded the LLS grant to investigate ways to harness a patient’s immune system to improve outcomes in patients with myeloid malignancies, such as MDS. 

LLS also currently has almost $30 million invested across multi-year research projects to improve understanding of MDS and myeloproliferative neoplasms (MPNs) and develop the next generation of treatment breakthroughs.

One such active research project is to understand the role our metabolism plays in the progression of MDS. 

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