Microenvironment of hematopoietic stem cells can be a target for myeloproliferative disorders

June 22, 2014
This image depicts the authors of the study, including Dr. Mendez and others. Credit: CNIC

The protective microenvironment of the hematopoietic stem cell niche, which produces cells of the blood and the immune system, also protects against myeloproliferative neoplasia.

The discovery of a new therapeutic target for certain kinds of myeloproliferative disease is, without doubt, good news. This is precisely the discovery made by the Stem Cell Physiopathology group at the CNIC (the Spanish National Cardiovascular Research Center), led by Dr. Simón Méndez–Ferrer. The team has shown that the that controls can be targeted for the of a set of disorders called myeloproliferative neoplasias, the most prominent of which are chronic myelomonocytic leukemia (CMML), juvenile myelomonocytic leukemia (JMML), and atypical (CML).

The findings, published today in Nature, demonstrate that these myeloproliferative neoplasias only appear after damage to the microenvironment that sustains and controls the hematopoietic —the cells that produce the cells of the blood and the immune system. Protecting this microenvironment, or niche, has thus emerged as a new route for the treatment of these diseases, for which there is currently no fully effective treatment.

"In normal conditions, the microenvironment is able to control the proliferation, differentiation and migration of the hematopoietic stem cell. A specific genetic mutation in these results in inflammatory injury to the microenvironment and this control breaks down. What our work shows is that this damage can be prevented or reversed by treatments that target the niche," explained Dr. Méndez-Ferrer.

Indeed, the same team of researchers has demonstrated the efficacy of a possible new treatment, which has been patented through the CNIC. The treatment involves an innovative use of clinically approved treatments for other diseases, so that, according to the authors, "it shouldn't be associated with adverse side effects". The new treatment route has been tested in animals and has received financial backing for a multicenter phase II clinical trial. "This study has a very strong translational and clinical potential", emphasized study first author Dr. Lorena Arranz, who added that "current treatment for myeloproliferative neoplasias is largely symptomatic and directed at preventing thrombosis and fatal cardiovascular events".

The only real cure available today is a bone marrow transplant, which is not advisable in patients over 50 years old. "This makes it important to identify new therapeutic targets for the development of effective treatments," the investigators conclude.

Explore further: New findings may help overcome hurdle to successful bone marrow transplantation

More information: Paper: Neuropathy of haematopoietic stem cell niche is essential for myeloproliferative neoplasms, DOI: 10.1038/nature13383

Related Stories

KIT researchers develop artificial bone marrow

January 10, 2014

Artificial bone marrow may be used to reproduce hematopoietic stem cells. A prototype has now been developed by scientists of KIT, the Max Planck Institute for Intelligent Systems, Stuttgart, and Tübingen University. The ...

Pushing the boundaries of stem cells

May 7, 2014

Adults suffering from diseases such as leukemia, lymphoma, and other blood-related disorders may benefit from life-saving treatment commonly used in pediatric patients. Researchers at the Icahn School of Medicine at Mount ...

One cell's meat is another cell's poison

May 30, 2014

As a new therapeutic approach, Janus kinases are currently in the limelight of cancer research. The focus of interest is the protein JAK2. By inhibiting this protein one tries to cure chronic bone marrow diseases, such as ...

Recommended for you

Researchers grow retinal nerve cells in the lab

November 30, 2015

Johns Hopkins researchers have developed a method to efficiently turn human stem cells into retinal ganglion cells, the type of nerve cells located within the retina that transmit visual signals from the eye to the brain. ...

Shining light on microbial growth and death inside our guts

November 30, 2015

For the first time, scientists can accurately measure population growth rates of the microbes that live inside mammalian gastrointestinal tracts, according to a new method reported in Nature Communications by a team at the ...

Functional human liver cells grown in the lab

November 26, 2015

In new research appearing in the prestigious journal Nature Biotechnology, an international research team led by The Hebrew University of Jerusalem describes a new technique for growing human hepatocytes in the laboratory. ...


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.