The Promising Treatment Of Imetelstat And Transfusion Independence

Current standard drug therapies are chemotherapy and radiation. But, these options don’t always meet the critical needs in treatment and can have serious side effects. Drug strategies are being developed to precisely target cancer cells while leaving the surrounding normal cells unaffected. 

Telomerase is expressed in cancer cells but rarely noticeable in normal cells and is now a designated drug target. Imetelstat is a first-in-class telomerase inhibitor. It has shown to decrease transfusion dependence (to treat anemia) and increase hemoglobin levels for patients with low-risk MDS that is relapsed or refractory.

Lenalidomide and luspatercept are often used for relapsed or refractory MDS, but they are more effective in specific subpopulations. Imetelstat directly targets “malignant clones with abnormally high telomerase activity, enabling recovery of hematopoiesis (blood cell production).” A recent clinical trial showed a 40% transfusion independence rate for patients treated with Imetelstat.

The Phase 3 portion of the IMerge study (NCT02598661) tested the safety and efficacy of Imetelstat. It’s findings showed:

• 39.8% of patients did not require transfusions for at least an 8 week consecutive period. “At 1 year, patients experienced respective transfusion independence rates of 13.6%.”
• Patients achieved a significant improvement in hemoglobin levels. (An average rise of 3.6 g/dL.)
• The median age was 72 years old. (The range was 44-87.)
• The use of Imetelstat has disease-modifying potential.

“Today is a great day for lower risk MDS patients who are living with the burden of transfusions. The results from the IMerge Phase 3 study were resoundingly positive, presenting compelling durability of transfusion independence, delivering on the promise of Imetelstat and telomerase inhibition for these patients,” said Dr. John A. Scarlett, Geron’s chairman and chief executive officer.

The side effects that a patient can experience with Imetelstat are:

• Weakness and lack of energy.
• Leg swelling.
• Headache and diarrhea.
• Alanine aminotransferase increase, which could be a sign of liver disease.
• Moderate to severe thrombocytopenia (low platelet count).
• Neutropenia (the susceptibility of infection).

“Imetelstat provides a clinically meaningful benefit in patients with MDS who are transfusion dependent.”