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PNAS Publishes OncoMed Data Demonstrating Potent Anti-Cancer Activity for Novel Wnt Pathway Antibody OMP-18R5
Posted: July 4, 2012 at 12:18 am
REDWOOD CITY, Calif.--(BUSINESS WIRE)--
OncoMed Pharmaceuticals, Inc., a clinical-stage company developing novel therapeutics that target cancer stem cells (CSCs), or tumor-initiating cells, today announced that Proceedings of the National Academy of Sciences of the United States of America (PNAS) July 2, 2012 PNAS Early Edition has published online OncoMed data demonstrating the potent anti-cancer activity of OMP-18R5, the companys first Wnt pathway product candidate, in multiple preclinical human tumor models. OMP-18R5 is currently in Phase 1 clinical testing.
OMP-18R5 is, we believe, the first therapeutic antibody to enter clinical trials that specifically inhibits the Wnt pathway, an important pathway in cancer. Our research published in PNAS suggests that inhibiting the Wnt pathway within tumors has the potential to improve treatment for multiple cancers, said Austin Gurney, Ph.D., Senior Vice President, Molecular and Cellular Biology of OncoMed Pharmaceuticals and a co-author of the paper. OMP-18R5 has demonstrated the remarkable ability to drive the differentiation of tumor cells in preclinical models. Tumors become less tumorigenic.
The Wnt/beta-catenin pathway, which signals through the Frizzled (Fzd) receptor family and several co-receptors, has long been implicated in cancer. OncoMed researchers identified a novel therapeutic approach to targeting the Wnt pathway with a monoclonal antibody. OMP-18R5, initially identified by binding to Frizzled 7, interacts with key Fzd receptors through a conserved epitope within the extracellular domain and thereby inhibits canonical Wnt signaling induced by multiple Wnt family members. In xenograft studies with minimally passaged human tumors, OMP-18R5 inhibits the growth of a range of tumor types and exhibits synergistic activity with standard-of-care chemotherapeutic agents. These data are published in current edition of PNAS in an article titled Wnt Pathway Inhibition via the Targeting of Frizzled Receptors Results in Decreased Growth and Tumorigenicity of Human Tumors.
Paul Hastings, President and Chief Executive Officer of OncoMed Pharmaceuticals, said, OMP-18R5 is among a rich pipeline of therapeutic candidates discovered by OncoMed that are now in clinical trials. We look forward to reporting results from our initial trials as this product advances through the clinic.
About OMP-18R5
OMP-18R5 is a monoclonal antibody optimized to block a key signaling pathway in cancer. Specifically, OMP-18R5 selectively targets Frizzled receptors, activators of Wnt signaling. OMP-18R5 treatment exhibits broad anti-tumor activity in preclinical models. OMP-18R5 is currently in a Phase 1 clinical trial in patients with advanced solid tumors. This trial will assess safety, pharmacokinetics, and provide initial indications of anti-tumor efficacy and the effects of OMP-18R5 on Wnt pathway biomarkers. OMP-18R5 is part of OncoMeds collaboration with Bayer Pharma AG.
About OncoMed Pharmaceuticals
OncoMed Pharmaceuticals is a clinical-stage company that discovers and develops novel therapeutics targeting cancer stem cells, the cells shown to be capable of driving tumor growth, recurrence and metastasis. OncoMed has advanced three anti-cancer therapeutics into the clinic, demcizumab (OMP-21M18), OMP-59R5, and OMP-18R5, which target key cancer stem cell signaling pathways including Notch and Wnt. In addition, OncoMeds pipeline includes several novel preclinical product candidates targeting multiple validated cancer stem cell pathways, including the RSPO-LGR pathway. OncoMed has formed strategic alliances with Bayer Pharma AG and GlaxoSmithKline. Privately held, OncoMeds investors include: US Venture Partners, Latterell Venture Partners, The Vertical Group, Morgenthaler Ventures, Phase4Ventures, Delphi Ventures, Adams Street Partners, De Novo Ventures, Bay Partners and GlaxoSmithKline. Additional information can be found at the companys website: http://www.oncomed.com.
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PNAS Publishes OncoMed Data Demonstrating Potent Anti-Cancer Activity for Novel Wnt Pathway Antibody OMP-18R5
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Amniotic fluid yields alternatives to embryonic stem cells
Posted: July 4, 2012 at 12:18 am
ScienceDaily (July 3, 2012) Stem cells found in amniotic fluid can be transformed into a more versatile state similar to embryonic stem cells, according to a study published July 3 in the journal Molecular Therapy. Scientists from Imperial College London and the UCL Institute of Child Health succeeded in reprogramming amniotic fluid cells without having to introduce extra genes. The findings raise the possibility that stem cells derived from donated amniotic fluid could be stored in banks and used for therapies and in research, providing a viable alternative to the limited embryonic stem cells currently available.
Amniotic fluid surrounds and nourishes the fetus in the womb. It can be extracted through the mother's abdomen using a needle in a process called amniocentesis, which is sometimes used to test for genetic diseases. The fluid contains stem cells that come from the fetus. These cells have a more limited capacity to develop into different cell types than stem cells in the embryo.
The researchers used stem cells from amniotic fluid donated by mothers undergoing amniocentesis for other purposes during the first trimester of pregnancy. The cells were grown on a gelatinous protein mixture in the lab and reprogrammed into a more primitive state by adding a drug called valproic acid to the culture medium. An extensive set of tests found that these reprogrammed cells have characteristics very similar to embryonic stem cells, which are capable of developing into any cell type in the body -- a property known as pluripotency.
Even after growing in culture for some time, the reprogrammed cells were able to develop into functioning cells of many different types, including liver, bone and nerve cells. They also maintained their pluripotency even after being frozen and rethawed.
The results suggest that stem cells derived from amniotic fluid could be used in treatments for a wide range of diseases. Donated cells could be stored in banks and used in treatments, as well as in disease research and drug screening. A previous study estimated that cells from 150 donors would provide a match for 38% of the population.
Alternatives to embryonic stem cells are keenly sought because of ethical concerns and limited availability of donor embryos. Previous research has shown that it is possible to make adult cells become pluripotent by introducing extra genes into the cells, often using viruses. However, the efficiency of the reprogramming is very low and there is a risk of problems such as tumours caused by disrupting the DNA. The new study is the first to induce pluripotency in human cells without using foreign genetic material. The pluripotent cells derived from amniotic fluid also showed some traits associated with embryonic stem cells that have not been found in induced pluripotent stem cells from other sources.
Amniocentesis is associated with a small risk of causing a miscarriage, estimated to be about one in 100.
Dr Pascale Guillot, from the Department of Surgery and Cancer at Imperial, said: "Amniotic fluid stem cells are intermediate between embryonic stem cells and adult stem cells. They have some potential to develop into different cell types but they are not pluripotent. We've shown that they can revert to being pluripotent just by adding a chemical reagent that modifies the configuration of the DNA so that genes that are expressed in the embryo get switched back on.
"These cells have a wide range of potential applications in treatments and in research. We are particularly interested in exploring their use in genetic diseases diagnosed early in life or other diseases such as cerebral palsy."
Dr Paolo De Coppi, from the UCL Institute of Child Health, who jointly led the study with Dr Guillot, said: "This study confirms that amniotic fluid is a good source of stem cells. The advantages of generating pluripotent cells without any genetic manipulation make them more likely to be used for therapy.
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Adult stem cells from bone marrow: Cell replacement/tissue repair potential in adult bone marrow stem cells in animal …
Posted: July 4, 2012 at 12:18 am
ScienceDaily (July 3, 2012) searchers from the University of Maryland School of Maryland report promising results from using adult stem cells from bone marrow in mice to help create tissue cells of other organs, such as the heart, brain and pancreas -- a scientific step they hope may lead to potential new ways to replace cells lost in diseases such as diabetes, Parkinson's or Alzheimer's.
The research in collaboration with the University of Paris Descartes is published online in the June 29, 2012 edition of Comptes Rendus Biologies, a publication of the French Academy of Sciences.
"Finding stem cells capable of restoring function to different damaged organs would be the Holy Grail of tissue engineering," says lead author David Trisler, PhD, assistant professor of neurology at the University of Maryland School of Medicine.
He adds, "This research takes us another step in that process by identifying the potential of these adult bone marrow cells, or a subset of them known as CD34+ bone marrow cells, to be 'multipotent,' meaning they could transform and function as the normal cells in several different organs."
University of Maryland researchers previously developed a special culturing system to collect a select sample of these adult stem cells in bone marrow, which normally makes red and white blood cells and immune cells. In this project, the team followed a widely recognized study model, used to prove the multipotency of embryonic stem cells, to prove that these bone marrow stem cells could make more than just blood cells. The investigators also found that the CD34+ cells had a limited lifespan and did not produce teratomas, tumors that sometimes form with the use of embryonic stem cells and adult stem cells cultivated from other methods that require some genetic manipulation.
"When taken at an early stage, we found that the CD34+ cells exhibited similar multipotent capabilities as embryonic stem cells, which have been shown to be the most flexible and versatile. Because these CD34+ cells already exist in normal bone marrow, they offer a vast source for potential cell replacement therapy, particularly because they come from a person's own body, eliminating the need to suppress the immune system, which is sometimes required when using adults stem cells derived from other sources," explains Paul Fishman, MD, PhD, professor of neurology at the University of Maryland School of Medicine.
The researchers say that proving the potential of these adult bone marrow stem cells opens new possibilities for scientific exploration, but that more research will be needed to see how this science can be translated to humans.
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Study Results: Adult Stem Cells From Bone Marrow
Posted: July 4, 2012 at 12:18 am
FOR IMMEDIATE RELEASE: July 3, 2012
UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE INVESTIGATORS FIND CELL REPLACEMENT/ TISSUE REPAIR POTENTIAL IN ADULT BONE MARROW STEM CELLS IN ANIMAL MODEL
Scientists Looking for Potential Avenue to Grow Cells of Different Organs
Newswise Baltimore, MD July 3, 2012. Researchers from the University of Maryland School of Maryland report promising results from using adult stem cells from bone marrow in mice to help create tissue cells of other organs, such as the heart, brain and pancreas - a scientific step they hope may lead to potential new ways to replace cells lost in diseases such as diabetes, Parkinsons or Alzheimers. The research in collaboration with the University of Paris Descartes is published online in the June 29, 2012 edition of Comptes Rendus Biologies, a publication of the French Academy of Sciences.
Finding stem cells capable of restoring function to different damaged organs would be the Holy Grail of tissue engineering, says lead author David Trisler, PhD, assistant professor of neurology at the University of Maryland School of Medicine.
He adds, This research takes us another step in that process by identifying the potential of these adult bone marrow cells, or a subset of them known as CD34+ bone marrow cells, to be multipotent, meaning they could transform and function as the normal cells in several different organs.
University of Maryland researchers previously developed a special culturing system to collect a select sample of these adult stem cells in bone marrow, which normally makes red and white blood cells and immune cells. In this project, the team followed a widely recognized study model, used to prove the multipotency of embryonic stem cells, to prove that these bone marrow stem cells could make more than just blood cells. The investigators also found that the CD34+ cells had a limited lifespan and did not produce teratomas, tumors that sometimes form with the use of embryonic stem cells and adult stem cells cultivated from other methods that require some genetic manipulation.
When taken at an early stage, we found that the CD34+ cells exhibited similar multipotent capabilities as embryonic stem cells, which have been shown to be the most flexible and versatile. Because these CD34+ cells already exist in normal bone marrow, they offer a vast source for potential cell replacement therapy, particularly because they come from a persons own body, eliminating the need to suppress the immune system, which is sometimes required when using adults stem cells derived from other sources, explains Paul Fishman, MD, PhD, professor of neurology at the University of Maryland School of Medicine.
The researchers say that proving the potential of these adult bone marrow stem cells opens new possibilities for scientific exploration, but that more research will be needed to see how this science can be translated to humans.
The results of this international collaboration show the important role that University of Maryland School of Medicine researchers play in advancing scientific understanding, investigating new avenues for the development of potentially life-changing treatments, says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and the John Z. and Akiko K. Bowers Distinguished Professor and dean of the University of Maryland School of Medicine.
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Amniotic fluid offers alternative stem cell source
Posted: July 4, 2012 at 12:18 am
Stem cells taken from amniotic fluid can be transformed into a more versatile state similar to embryonic stem cells and may offer an alternative to the medically valuable but controversial cells, scientists said on Tuesday.
British researchers said they had succeeded in reprogramming amniotic fluid cells without having to introduce extra genes.
This suggests the possibility that stem cells derived from donated amniotic fluid could be stored in banks and used for medical therapies and in research, they said, offering a less problematic alternative to embryonic stem cells.
Stem cells are the body's master cells, the source for all other cells. Scientists say that by helping to regenerate tissue, they could offer new ways of treating diseases for which there are currently no treatments - including heart disease, Parkinson's and stroke.
Embryonic stem cells are harvested from embryos and have the potential to become almost any type of tissue. Other types of stem cells, including adult or so-called "induced pluripotent" stem cells, are less controversial, but are also less flexible.
Alternatives to embryonic stem cells are always being keenly sought, partly due to ethical concerns and also due to the limited availability of donor embryos.
In this study, published in the journal Molecular Therapy, scientists from Imperial College London and University College London's (UCL) Institute of Child Health said amniotic fluid stem cells are an intermediate between embryonic and adult stem cells.
Pluripotent
"They have some potential to develop into different cell types but they are not pluripotent," said Pascale Guillot, from the Imperial's department of surgery and cancer.
But he said their study had shown that these cells can revert to being fully flexible, or "pluripotent", by adding a chemical that modifies the configuration of the DNA.
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Cryo-Save´s Cost-Free Donation Programme Serves Families in Need
Posted: July 3, 2012 at 11:19 am
ZUTPHEN, The Netherlands, July 3, 2012 /PRNewswire/ --
Free of charge, Cryo-Saves programme gives the opportunity to treat a family member diagnosed with a life-threatening disease treatable with stem cells.
Cryo-Save, the leading international family stem cell bank, shows continuous commitment to its corporate social responsibility programme. Family and childrens health is the companys number one priority. The Cryo-Save Cost-Free Family Donation Programme is specifically designed to offer families in need the collection and cryopreservation of their newborns umbilical cord blood stem cells. Free of charge, it gives the opportunity to treat a family member diagnosed with a life-threatening disease treatable with stem cells. This includes diseases such as Sickle Cell Anaemia and some forms of Leukaemia.
Arnoud van Tulder, CEO of Cryo-Save: "Our goal is to help as many families as possible with the stem cells they store with Cryo-Save. Through our Cost-Free Donation Programme, we offer direct help to families in need. This is very important for us; being able to apply our knowledge and expertise to help save lives!"
Thanks to Cryo-Saves international reach and more than 40 local offices which are in touch with their communities needs, each country is striving to make a positive difference in their community. The Cost-Free Donation Programme is promoted in each country. Among other activities, the company recently supported two fundraising events: Cryo-Saves Netherlands office sponsored a cycling event organised by the Alpe dHuZes Cancer Rehabilitation (A-CaRe) programme, which aims to develop and implement rehabilitation programmes for specific cancer patients and survivor groups in the Netherlands. Cryo-Save Serbia is supporting the NGO Everything for a Smile where children suffering from renal diseases are invited to enjoy a rare weekend in nature.
Along with its continuous efforts to educate the general public about stem cells, Cryo-Save strives to make a difference in peoples lives and encourages all its employees to consider the impact their work makes on the environment and local communities.
Cryo-Save, the leading international family stem cell bank, stores more than 200,000 samples from umbilical cord blood, cord tissue and adipose tissue. There are already many diseases treatable by the use of stem cells, and the number of treatments will only increase. Driven by its international business strategy, Cryo-Save is now represented in over 40 countries on three continents, with ultra-modern processing and storage facilities in Arabia, Belgium, Germany, India and South Africa.
Cryo-Save: http://www.cryo-save.com/group
Cryo-Save Group N.V.
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Researchers block pathway to cancer cell replication
Posted: July 2, 2012 at 4:22 pm
Public release date: 2-Jul-2012 [ | E-mail | Share ]
Contact: Debra Kain ddkain@ucsd.edu 619-543-6163 University of California - San Diego
Research suggests that patients with leukemia sometimes relapse because standard chemotherapy fails to kill the self-renewing leukemia initiating cells, often referred to as cancer stem cells. In such cancers, the cells lie dormant for a time, only to later begin cloning, resulting in a return and metastasis of the disease.
One such type of cancer is called pediatric T cell acute lymphoblastic leukemia, or T-ALL, often found in children, who have few treatment options beyond chemotherapy.
A team of researchers led by Catriona H. M. Jamieson, MD, PhD, associate professor of medicine at the University of California, San Diego School of Medicine and Director of Stem Cell Research at UC San Diego Moores Cancer Center studied these cells in mouse models that had been transplanted with human leukemia cells. They discovered that the leukemia initiating cells which clone, or replicate, themselves most robustly activate the NOTCH1 pathway, usually in the context of a mutation.
Earlier studies showed that as many as half of patients with T-ALL have mutations in the NOTCH1 pathway an evolutionarily conserved developmental pathway used during differentiation of many cell and tissue types. The new study shows that when NOTCH1 activation was inhibited in animal models using a monoclonal antibody, the leukemia initiating cells did not survive. In addition, the antibody treatment significantly reduced a subset of these cancer stem cells (identified by the presence of specific markers, CD2 and CD7, on the cell surface.)
"We were able to substantially reduce the potential of these cancer stem cells to self-renew," said Jamieson. "So we're not just getting rid of cancerous cells: we're getting to the root of their resistance to treatment leukemic stem cells that lie dormant."
The study results suggest that such therapy would also be effective in other types of cancer stem cells, such as those that cause breast cancer, that also rely on NOTCH1 for self-renewal.
"Therapies based on monoclonal antibodies that inhibit NOTCH 1 are much more selective than using gamma-secretase inhibitors, which also block other essential cellular functions in addition to the NOTCH1 signaling pathway," said contributor A. Thomas Look, MD of Dana-Farber/Children Hospital Cancer Center in Boston. "We are excited about the promise of NOTCH1-specific antibodies to counter resistance to therapy in T-ALL and possibly additional types of cancer."
In investigating the role of NOTCH1 activation in cancer cell cloning, the researchers showed that leukemia initiating cells possess enhanced survival and self-renewal potential in specific blood-cell, or hematopoietic, niches: the microenvironment of the body in which the cells live and self-renew.
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In real time, Yale scientists watch stem cells at work regenerating tissue
Posted: July 1, 2012 at 6:16 pm
Scientists have for the first time watched and manipulated stem cells as they regenerate tissue in an uninjured mammal, Yale researchers report July 1 online in the journal Nature.
Using a sophisticated imaging technique, the researchers also demonstrated that mice lacking a certain type of cell do not regrow hair. The same technique could shed light on how stem cells interact with other cells and trigger repairs in a variety of other organs, including lung and heart tissue.
This tells us a lot about how the tissue regeneration process works, said Valentina Greco, assistant professor of genetics and of dermatology at the Yale Stem Cell Center, researcher for the Yale Cancer Center and senior author of the study.
Greco and her team focused on stem cell behavior in the hair follicle of the mouse. The accessibility of the hair follicle allowed real-time and non-invasive imaging through a technology called 2-photon intravital microscopy.
Using this method, Panteleimon Rompolas, a post-doctoral fellow in Grecos lab and lead author of this paper, was able to study the interaction between stem cells and their progeny, which produce all the different types of cells in the tissue. The interaction of these cells with the immediate environment determines how cells divide, where they migrate and which specialized cells they become.
The technology allowed the team to discover that hair growth in mice cannot take place in the absence of connective tissue called mesenchyme, which appears early in embryonic development.
Stem cells not only spur growth of hair in mammals including humans, but also can serve to regenerate many other types of tissues.
Understanding how stem cell behavior is regulated by the microenvironment can advance our use of stem cells for therapeutic purposes and uncover mechanisms that go wrong in cancer and other diseases, Greco said.
The study was funded by an Alexander Brown Coxe postdoctoral fellowship. This work was supported in part by the American Skin Association and the American Cancer Society and the Yale Rheumatologic Disease Research Core Center and the National Institutes of Health.
Other Yale authors include Elizabeth Deschene, Giovanni Zito, David G. Gonzalez, Ichiko Saotome and Ann M. Haberman.
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Skin Cells Create Stem Cells In Huntington Disease Study
Posted: June 30, 2012 at 4:15 am
June 29, 2012
Connie K. Ho for redOrbit.com Your Universe Online
In 1993, the autosomal dominant gene mutation responsible for Huntingtons Disease (HD) was discovered. However, no treatments are known to slow its progression. New research may pave the way to better understanding of the disease. Researchers at Johns Hopkins recently announced that they were able to produce stem cells from skin cells from a person who had severe, early-onset form of HD; the cells were then changed into neurons that degenerated like the cells affected by HD.
The research was recently published in the journal Cell Stem Cell. The investigators worked with an international consortium in creating HD in a dish. The group was made up of scientists from Johns Hopkins University School of Medicine, Cedars-Sinai Medical Center, the University of California at Irvine, as well as six other groups. The team looked at many other HD cell lines and control cell lines to verify that the results were consistent and reproducible in other labs. The investigators believe that the findings allow them to better understand and eliminate cells in people in with HD. They hope to study the effects of possible drug treatments on cells that would be otherwise found deep in the brain.
Having these cells will allow us to screen for therapeutics in a way we havent been able to before in Huntingtons disease, remarked lead researcher Dr. Christopher A. Ross, a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine, in a prepared statement. For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic.
The team of researchers is studying small molecules for the ability to block HD iPSC degeneration to see if they can be developed into new drugs for HD. As well, the ability to produce from stem cells the same neurons found in HD may have effects for similar research in other neurodegenerative diseases like Alzheimers and Parkinsons. In the experiment, Ross took a skin biopsy from a patient with very early onset HD. The patient was seven years old at the time, with a severe form of disease and a mutation that caused it. By using cells from a patient who had quickly progressing HD, Ross team were able to mimic HD in a way that could be used by patients who had different forms of HD.
The skin cells were grown in culture and reprogrammed to induce stem cells that were pluripotent. Then, another cell line was created in the same way from someone who didnt have HD. The other HD and control iPS cells were produced as part of the NINDS funded HD iPS cell consortium. Investigators from Johns Hopkins and the other consortium labs changed the cells into typical neurons and then into medium spiny neurons. The process took a total of three months and the scientists found the medium spiny neurons from the HD cells acted how the medium spiny neurons form an HD patient would. The cells demonstrated quick degeneration when cultured in the lab with a basic culture medium that didnt include extensive supporting nutrients. On the other hand, control cell lines didnt demonstrate neuronal degeneration.
These HD cells acted just as we were hoping, says Ross, director of the Baltimore Huntingtons Disease Center. A lot of people said, Youll never be able to get a model in a dish of a human neurodegenerative disease like this. Now, we have them where we can really study and manipulate them, and try to cure them of this horrible disease. The fact that we are able to do this at all still amazes us.
Source: Connie K. Ho for redOrbit.com Your Universe Online
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Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice
Posted: June 30, 2012 at 4:15 am
Editor's Choice Main Category: Muscular Dystrophy / ALS Also Included In: Transplants / Organ Donations Article Date: 29 Jun 2012 - 11:00 PDT
Current ratings for: Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice
A new study published in Science Translational Medicine reveals that researchers have, for the first time, managed to turn fibroblast cells, i.e. common cells within connective tissue, from muscular dystrophy patients into stem cells and subsequently changed these cells into muscle precursor cells. After modifying the muscle precursor cells genetically, the researchers transplanted them into mice.
In future, this new technique could be used in order to treat patients with the rare condition of limb-girdle muscular dystrophy, which primarily affects the shoulders and hips, and maybe other types of muscular dystrophies. The method was initially developed in Milan at the San Raffaele Scientific Institute and was completed at UCL.
Muscular dystrophy is a genetic disorder, which typically affects skeletal muscles. The condition leads to severely impaired mobility and can, in severe cases result in respiratory and cardiac dysfunction. At present, there is no effective treatment for the condition. A number of new potential therapies, including cell therapy, are entering clinical trials.
The scientists of this study concentrated their research on genetically modifying mesoangioblasts, i.e. a self-renewing cell that originates from the dorsal aorta and differentiates into most mesodermal tissues, which demonstrated its potential for treating muscular dystrophy in earlier studies.
Given that the muscles of patients with muscular dystrophy are depleted of mesonangioblasts, the researchers were unable to obtain sufficient numbers of these cells from patients with limb-girdle muscular dystrophy, and therefore "reprogrammed" adult cells from these patients into stem cells, which enabled them to prompt them to differentiate into mesoangioblast-like cells.The team then genetically corrected these 'progenitor' cells by using a viral vector, and injected them into mice with muscular dystrophy so that the cells targeted damaged muscle fibers.
In a mice study, the same process demonstrated that dystrophic mice were able to run on a treadmill for longer a longer time than dystrophic mice that did not receive the cells.
Research leader, Dr Francesco Saverio Tedesco, from UCL Cell & Developmental Biology, who led the study, explained:
Professor Giulio Cossu, also an author at UCL, concluded:
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Stem Cells From Muscular Dystrophy Patients Transplanted Into Mice
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