Study Finds Promising Treatment for Myelofibrosis

Myelofibrosis treatment with imetelstat led to remission for some patients in small study

/ Author:  / Reviewed by: Robert Carlson, M.D Beth Bolt, RPh

(RxWiki News) Myelofibrosis is a type of chronic leukemia that attacks bone marrow. Researchers at Mayo Clinic showed promising study results for one treatment for this disease.

These researchers found that some patients treated with imetelstat had complete bone marrow recovery, allowing them to once again make their own red blood cells.

This study also showed a promising rate of partial remission, a period where symptoms improve or the affected area becomes smaller.

"Consult with your doctor about treatment options."

This study was led by Ayalew Tefferi, MD, from the Division of Hematology at the Mayo Clinic in Rochester, MN.

Dr. Tefferi and his team studied 33 myelofibrosis patients being treated with imetelstat, a telomerase inhibitor that works by killing the cells within a tumor.

These researchers were able to follow 18 of the original study group for at least three months. Of the 22 patients, five had complete or partial remissions.

The researchers also reported that two of the five patients with complete or partial remission also experienced complete molecular remission as the bone marrow cells returned to normal.

"Imetelstat, a new drug, is shown to have some activity towards myleofibrosis. This is important as this class of drugs, telomerase inhibitors, might be effective in treating bone marrow diseases," said Subhakar "Sub" Mutyala, MD, Associate Director of the Baylor Scott & White Cancer Institute and Associate Professor at Texas A&M College of Medicine in Temple, Texas.

"These drugs will be studied more to know which diseases and how effective they will be in the future," Dr. Mutyala told dailyRx News.

According to the Mayo Clinic, myelofibrosis is a form of chronic leukemia that leaves bone marrow scarred and unable to perform normal blood cell production. The recovery of cancerous bone marrow cells is rare under the best situation.

This study, while promising, was performed on a small group of patients over a short period. A larger, long-term study is required to validate the performance of imetelstat.

This study was presented on December 9 at the American Society of Hematology's annual meeting.

Study co-author Monic J. Stuart, MD, MPH, disclosed a consultancy with Geron Corporation.

Financial support for this study was provided by Geron Corporation.

Review Date: 
December 10, 2013
Last Updated:
February 19, 2014