Imetelstat, a novel drug that targets telomerase, has demonstrated potential value in treating patients with myelofibrosis, according to the results of a study published today in the New England Journal of Medicine.
“We observed that Imetelstat was active and induced morphologic and molecular remissions in some patients with myelofibrosis,” says Ayalew Tefferi, M.D., a hematologist at Mayo Clinic and lead author of the study. “We also observed that Imtelstat demonstrated selective anti-clonal activity, inhibiting the growth of cancer cells, which we had not previously documented with other drugs.”
Myelofibrosis is a chronic myeloid cancer in which bone marrow cells that produce blood cells develop and function abnormally. The result is the formation of scar tissue in the bone marrow (fibrosis), severe anemia that often requires transfusion, weakness, fatigue, and an enlarged spleen and liver. Patients with myelofibrosis harbor one of several genetic mutations in their blood stem cells, including JAK2, MPL, CALR, ASXL1 and spliceosome pathway mutations.3317325_0009”Typically, myelofibrosis is characterized by marrow scarring, and, although patients may derive symptomatic relief from other treatments, such as ruxolitinib, they usually do not revert back to normal bone marrow,” Dr. Tefferi says. “Some patients treated with Imetelstat have reverted back to normal bone marrow.” Imetelstat works by inhibiting telomerase activity in tumor cells, which leads to cell death.