Imetelstat, a Telomerase Inhibitor, Induces Morphologic and Molecular Remissions In Myelofibrosis
Geron is studying Imetelstat as a treatment for Myelofibrosis, a disease in which abnormal blood cells build up in the bone marrow, forming thick scar tissues that slows the production of healthy blood cells. Other MF drugs treat the symptoms of the disease (Jakafi).
“Background: JAK inhibitors, including ruxolitinib, are to date incapable of inducing complete (CR) or partial (PR) remissions, reversal of bone marrow (BM) fibrosis or molecular responses in myelofibrosis (MF). This is consistent with the fact that JAK2 mutations are neither specific nor pathogenetically essential for the disease. Other currently available drugs in MF are equally ineffective in terms of disease-modifying activity.”
“Conclusions: The current study signifies the potential value of telomerase-based treatment strategies in MF and identifies imetelstat as an active drug in that regard. The observed morphologic and molecular remissions confirm selective anti-clonal activity, which has thus far eluded other drugs in MF, including JAK inhibitors. The association between response and spliceosome mutations suggests a broader application for the drug in myeloid malignancies.” Perhaps this broader application may apply to PV and ET?
Click on the picture below that links to the American Society of Hematology abstract: