Dr Mascarenhas on the Role of Imetelstat in the Myelofibrosis Treatment Paradigm

“One application of [imetelstat treatment] is as a bridge to transplant for some patients, but not every patient is a transplant candidate. Many patients are not. For many patients, this drug could be used outside of the usual world of JAK inhibition, for patients who are looking to achieve disease course modification.”

John O. Mascarenhas, MD, a professor of medicine at the Icahn School of Medicine at Mount Sinai; as well as director of the Center of Excellence for Blood Cancers and Myeloid Disorders, director of the Adult Leukemia Program, and a member of The Tisch Cancer Institute, discussed the potential role of imetelstat in the treatment of patients with myelofibrosis.

During his interview, Mascarenhas emphasized that one application of imetelstat may be as a bridge to allogeneic stem cell transplantation for select patients. Although transplantation remains the only curative option for myelofibrosis, only a minority of patients are eligible due to the high prevalence of advanced age, comorbidities, and adverse disease biology among patients with myelofibrosis. However, for patients proceeding to transplant, imetelstat may help control disease prior to transplantation, thereby optimizing outcomes, he said.

Mascarenhas also noted that since most patients with myelofibrosis are not transplant candidates, the therapeutic potential of imetelstat beyond the transplant setting is significant. In these patients, imetelstat may provide an opportunity for disease modification by improving progression-free and overall survival, he explained. Unlike JAK inhibitors, imetelstat may alter the natural course of disease for patients with limited treatment options, according to Mascarenhas.

Importantly, Mascarenhas highlighted that imetelstat use may also expand to the transplant-eligible population. Patients initially considered ineligible for transplant could, in some cases, achieve responses that subsequently allow them to proceed with curative therapy, he said. This dual role positions imetelstat as both a bridge to transplant for some patients and a disease-modifying option for those unlikely to undergo transplant.

For patients not pursuing transplantation, Mascarenhas added that imetelstat could still represent a meaningful therapeutic option, particularly for those with high-risk disease or poor outcomes following JAK inhibitor therapy. By addressing an unmet need in this population, imetelstat may help change the disease trajectory in select patients, he said.