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 microenvironment that controls hematopoietic stem cells can be targeted for the treatment of a set of disorders called myeloproliferative neoplasias, the most prominent of which are chronic myelomonocytic leukemia (CMML), juvenile myelomonocytic leukemia (JMML), and atypical chronic myelogenous leukemia (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 stem cells—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 cells 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: One cell's meat is another cell's poison
More information: Paper: Neuropathy of haematopoietic stem cell niche is essential for myeloproliferative neoplasms, DOI: 10.1038/nature13383
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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 microenvironment that controls hematopoietic stem cells can be targeted for the treatment of a set of disorders called myeloproliferative neoplasias, the most prominent of which are chronic myelomonocytic leukemia (CMML), juvenile myelomonocytic leukemia (JMML), and atypical chronic myelogenous leukemia (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 stem cells—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 cells 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: One cell's meat is another cell's poison
More information: Paper: Neuropathy of haematopoietic stem cell niche is essential for myeloproliferative neoplasms, DOI: 10.1038/nature13383
Medical Xpress on facebook
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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 ...
KIT researchers develop artificial bone marrow
Jan 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 ...
Transfer of a few immune cells can protect immunodeficient patients
Jun 17, 2014
When patients have to undergo a bone marrow transplant, the procedure weakens their immune system. Viruses that are usually kept in check in a healthy immune system may then cause potentially fatal infections. ...
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 ...
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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 ...
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