Drug induces morphologic, molecular and clinical remissions in myelofibrosis

December 5, 2013

Imetelstat, a novel telomerase inhibiting drug, has been found to induce morphologic, molecular and clinical remissions in some patients with myelofibrosis a Mayo Clinic study has found. The results were presented today at the 2013 American Society of Hematology Annual Meeting in New Orleans.

"These are early results but they are promising, says the study's lead author, Ayalew Tefferi, M.D. a hematologist at Mayo Clinic. "Some in our clinical trial taking imetelstat obtained dramatic responses and there have been some complete responses which is almost unheard of for drug therapy in this disease."

Myelofibrosis is a chronic myeloid cancer in which 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 , including JAK2, MPL, CALR, ASXL1 and spliceosome pathway mutations.

"Typically, 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 in our trial have reverted back to normal bone marrow."

Imetelstat works by inhibiting telomerase activity in tumor cells which leads to cell death.

Researchers studied imetelstat in 33 patients at Mayo Clinic and have now followed the first 22 patients for a minimum of six months. Among the 22, five patients achieved complete or partial remissions, including reversal of fibrosis in four of the five patients. Two of the five patients with complete or partial remission have also experienced complete molecular remissions. The overall response rate was 41 percent.

Explore further: Ruxolinitib better at reducing myelofibrosis symptoms, study shows

Related Stories

Ruxolinitib better at reducing myelofibrosis symptoms, study shows

December 12, 2011
In a major advance in treatment, a multicenter study found that ruxolinitib did a better job than off-label chemotherapy drugs reducing the terrible symptoms associated with myelofibrosis, including pain, enlarged spleen, ...

Combination therapy using JAK2 and HSP90 inhibitors increased efficacy in myelofibrosis in vivo

December 9, 2012
Researchers have demonstrated that combination therapy with PU-H71 and ruxolitinib increases the durability and effectiveness of a treatment that had previously shown limited utility for patients with myelofibrosis.

Therapy using stem cells, bone marrow cells, appears safe for patients with ischemic cardiomyopathy

November 18, 2013
Alan W. Heldman, M.D., of the University of Miami Miller School of Medicine, and colleagues conducted a study to examine the safety of transendocardial stem cell injection (TESI) with autologous mesenchymal stem cells and ...

New findings may help overcome hurdle to successful bone marrow transplantation

May 28, 2013
Blood diseases such as leukemia, multiple myeloma, and myelodysplasia can develop from abnormal bone marrow cells and a dysfunctional bone marrow microenvironment that surrounds these cells. Until now, researchers have been ...

Recommended for you

Study provides insight into link between two rare tumor syndromes

August 22, 2017
UCLA researchers have discovered that timing is everything when it comes to preventing a specific gene mutation in mice from developing rare and fast-growing cancerous tumors, which also affects young children. This mutation ...

Retaining one normal BRCA gene in breast, ovarian cancers influences patient survival

August 22, 2017
Determining which cancer patients are likely to be resistant to initial treatment is a major research effort of oncologists and laboratory scientists. Now, ascertaining who might fall into that category may become a little ...

Clear link between heavy vitamin B intake and lung cancer

August 22, 2017
New research suggests long-term, high-dose supplementation with vitamins B6 and B12—long touted by the vitamin industry for increasing energy and improving metabolism—is associated with a two- to four-fold increased lung ...

Study identifies miR122 target sites in liver cancer and links a gene to patient survival

August 22, 2017
A new study of a molecule that regulates liver-cell metabolism and suppresses liver-cancer development shows that the molecule interacts with thousands of genes in liver cells, and that when levels of the molecule go down, ...

Zebrafish larvae could be used as 'avatars' to optimize personalized treatment of cancer

August 21, 2017
Portuguese scientists have for the first time shown that the larvae of a tiny fish could one day become the preferred model for predicting, in advance, the response of human malignant tumors to the various therapeutic drugs ...

Scientists discover vitamin C regulates stem cell function, curbs leukemia development

August 21, 2017
Not much is known about stem cell metabolism, but a new study from the Children's Medical Center Research Institute at UT Southwestern (CRI) has found that stem cells take up unusually high levels of vitamin C, which then ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.