Thursday, October 18, 2012

University of Maryland School of Medicine scientists develop stem cell model for hereditary disease



    October 16, 2012
Discovery could allow faster testing of drugs for Gaucher disease and neurodegenerative conditions such as Parkinson's
A new method of using adult stem cells as a model for the hereditary condition Gaucher disease could help accelerate the discovery of new, more effective therapies for this and other conditions such as Parkinson's, according to new research from the University of Maryland School of Medicine.
Scientists at the University of Maryland School of Medicine reprogrammed stem cells to develop into cells that are genetically similar to and react to drugs in a similar way as cells from patients with Gaucher disease. The stem cells will allow the scientists to test potential new therapies in a dish, accelerating the process toward drug discovery, according to the paper published online in the journal the Proceedings of the National Academy of Sciences (PNAS) on Oct. 15 (Panicker et.al.).
The study was funded with $1.7 million in grants from the Maryland Stem Cell Research Fund; researchers received a start-up grant for $200,000 in 2007 and a larger, five-year grant for $1.5 million in 2009.
"We have created a model for all three types of Gaucher disease, and used stem cell-based tests to evaluate the effectiveness of therapies," says senior author Ricardo Feldman, Ph.D., associate professor of microbiology and immunology at the University of Maryland School of Medicine, and a research scientist at the University of Maryland Center for Stem Cell Biology and Regenerative Medicine. "We are confident that this will allow us to test more drugs faster, more accurately and more safely, bringing us closer to new treatments for patients suffering from Gaucher disease. Our findings have potential to help patients with other neurodegenerative diseases as well. For example, about 10 percent of Parkinson's disease patients carry mutations in the recessive gene for Gaucher disease, making our research possibly significant for Parkinson's disease as well."
Gaucher disease is the most frequent lipid-storage disease. It affects 1 in 50,000 people in the general population. It is most common in Ashkenazi Jews, affecting 1 in 1,000 among that specific population. The disease occurs in three subtypes - Type 1 is the mildest and most common form of the disease, causing symptoms such as enlarged livers and spleens, anemia and bone disease. Type 2 causes very serious brain abnormalities and is usually fatal before the age of two, while Type 3 affects children and adolescents.
The condition is a recessive genetic disorder, meaning that both parents must be carriers for a child to suffer from Gaucher. However, said Dr. Feldman, studies have found that people with only one copy of a mutated Gaucher gene - those known as carriers - are at an increased risk of developing Parkinson's disease.
"This science is a reflection of the mission of the University of Maryland School of Medicine - to take new treatments from bench to bedside, from the laboratory to patients, as quickly as possible," says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and John Z. and Akiko K. Bowers Distinguished Professor and dean of the University of Maryland School of Medicine. "We are excited to see where this research goes next, bringing new hope to Gaucher patients and their families."
Dr. Feldman and his colleagues used the new reprogramming technology developed by Shinja Yamanaka in Japan, who was recognized with this year's Nobel Prize for Medicine or Physiology. Scientists engineered cells taken from the skin of Gaucher patients, creating human induced pluripotent stem cells, known as hiPSC - stem cells that are theoretically capable of forming any type of cell in the body. Scientists differentiated the cells to form white blood cells known as macrophages and neuronal cells.
A key function of macrophages in the body is to ingest and eliminate damaged or aged red blood cells. In Gaucher disease, the macrophages are unable to do so - they can't digest a lipid present in the red blood cell membrane. The macrophages become engorged with lipid and cannot completely clear the ingested red blood cells. This results in blockage of membrane transport pathways in the macrophages lodged in the bone marrow, spleen and liver. The macrophages that the scientists created from the reprogrammed stem cells exhibited this characteristic hallmark of the macrophages taken from Gaucher patients.
To further test the stem cells, the scientists administered a recombinant enzyme that is effective in treating Gaucher patients with Type 1 disease. When the cells were treated with the enzyme, the function of the macrophages was restored - they completely cleared the red blood cells.
"The creation of these stem cell lines is a lovely piece of stem cell research," said Curt Civin, M.D., professor of pediatrics and physiology, associate dean for research and founding director of the Center for Stem Cell Biology & Regenerative Medicine at the University of Maryland School of Medicine. "Dr. Feldman is already using these Gaucher patient-derived macrophages to better understand the disease fundamentals and to find novel medicines for Gaucher disease treatment. A major goal of our Center for Stem Cell Biology & Regenerative Medicine is to translate our fundamental discoveries into innovative and practical clinical applications that will enhance the understanding, diagnosis, treatment, and prevention of many human diseases. Clinical applications include not only transplantation of stem cells, but also the use of stem cells for drug discovery as Dr. Feldman's studies so beautifully illustrate."
"We are looking forward to testing new drugs on these cells, getting new therapies to patients," says Dr. Feldman.
University of Maryland Medical Center


Protein could be key for drugs that promote bone growth
Georgia Health Sciences University researchers have developed a mouse that errs on the side of making bone rather than fat, which could eventually lead to better drugs to treat inflammatory diseases such as rheumatoid arthritis.
Study suggests how expanding waistlines may contribute to cancer
Fat progenitor cells may contribute to cancer growth by fortifying the vessels that provide needed blood to tumors, according to preclinical research findings by investigators at The University of Texas Health Science Center at Houston (UTHealth).
Realizing the potential of stem cell therapy
New animal studies provide additional support for investigating stem cell treatments for Parkinson's disease, head trauma, and dangerous heart problems that accompany spinal cord injury, according to research findings released today.
Scientists identify mammal model of bladder regeneration
While it is well known that starfish, zebrafish and salamanders can re-grow damaged limbs, scientists understand very little about the regenerative capabilities of mammals.
Stem cells from muscle tissue may hold key to cell therapies for neurodegenerative diseases
Scientists at Wake Forest Baptist Medical Center have taken the first steps to create neural-like stem cells from muscle tissue in animals.
Safety results of intra-arterial stem cell clinical trial for stroke presented
Early results of a Phase II intra-arterial stem cell trial for ischemic stroke showed no adverse events associated with the first 10 patients, allowing investigators to expand the study to a targeted total of 100 patients.
Human neural stem cells study offers new hope for children with fatal brain diseases
Physician-scientists at Oregon Health & Science University Doernbecher Children's Hospital have demonstrated for the first time that banked human neural stem cells - HuCNS-SCs, a proprietary product of StemCells Inc. - can survive and make functional myelin in mice with severe symptoms of myelin loss.
UCSF study shows evidence that transplanted neural stem cells produced myelin
A Phase I clinical trial led by investigators from the University of California, San Francisco and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.
New function of a protein involved in colon cancer is identified
Researchers from IMIM, Hospital del Mar Medical Research Institute, have succeeded in determining the function of a new variant of enzyme IKKalpha (IKKα) to activate some of the genes taking part in the tumor progressions of colorectal cancer.
Artificial cornea gives the gift of vision
Blindness is often caused by corneal diseases. The established treatment is a corneal transplant, but in many cases this is not possible and donor corneas are often hard to come by. In the future, an artificial cornea could make up for this deficiency and save the vision of those affected. More Stem Cells Current Events and Stem Cells News Articles
Stem Cells: A Very Short Introduction Stem Cells: A Very Short Introduction
by Jonathan Slack (Author)

Embryonic stem cells have been hot-button topics in recent years, generating intense public interest as well as much confusion and misinformation. In this Very Short Introduction, leading authority Jonathan Slack offers a clear and informative overview of stem cells--what they are, what scientists do with them, what stem cell therapies are available today, and how they might be used in the future. Slack explains the difference between embryonic stem cells, which exist only in laboratory cultures, and tissue-specific stem cells, which exist in our bodies, and he discusses how embryonic stem cells may be used in the future to treat such illnesses as diabetes, Parkinson's disease, heart disease, spinal trauma, and retinal degeneration. But he stresses that, despite important advances, the...
Stem Cells For Dummies Stem Cells For Dummies
by Lawrence S.B. Goldstein (Author), Meg Schneider (Author)
The first authoritative yet accessible guide to this controversial topicStem Cell Research For Dummies offers a balanced, plain-English look at this politically charged topic, cutting away the hype and presenting the facts clearly for you, free from debate. It explains what stem cells are and what they do, the legalities of harvesting them and using them in research, the latest research findings from the U.S. and abroad, and the prospects for medical stem cell therapies in the short and long term.Explains the differences between adult stem cells and embryonic/umbilical cord stem cellsProvides both sides of the political debate and the pros and cons of each side's opinionsIncludes medical success stories using stem cell therapy and its promise for the futureComprehensive and unbiased, Stem...
Essentials of Stem Cell Biology, Second Edition Essentials of Stem Cell Biology, Second Edition
by Robert Lanza (Editor), John Gearhart (Editor), Brigid Hogan (Editor), Douglas Melton (Editor), Roger Pederson (Editor), E. Donnall Thomas (Editor), James Thomson (Editor), Sir Ian Wilmut (Editor)
First developed as an accessible abridgement of the successful Handbook of Stem Cells, Essentials of Stem Cell Biology serves the needs of the evolving population of scientists, researchers, practitioners and students that are embracing the latest advances in stem cells. Representing the combined effort of seven editors and more than 200 scholars and scientists whose pioneering work has defined our understanding of stem cells, this book combines the prerequisites for a general understanding of adult and embryonic stem cells with a presentation by the world's experts of the latest research information about specific organ systems. From basic biology/mechanisms, early development, ectoderm, mesoderm, endoderm, methods to application of stem cells to specific human diseases, regulation and...
The Stem Cell Hope: How Stem Cell Medicine Can Change Our Lives The Stem Cell Hope: How Stem Cell Medicine Can Change Our Lives
by Alice Park (Author)
A landmark book by the senior science writer at Time magazine introduces us to a medical breakthrough that can save our lives.
Few people know much about stem cell research beyond the ethical questions raised by using embryos. But in the last decade, stem cell research has made huge advances toward eliminating some of our most intractable diseases. Now this sweeping and accessible book introduces us to this cutting-edge science that will revolutionize medicine and change the way we think about and treat disease.
Alice Park takes us from stem cell's controversial beginnings to the recent electrifying promise of being able to create the versatile cells without using embryos at all. She shows us how stem cells give researchers an unprecedented ability to study disease while...
Human Stem Cell Technology and Biology: A Research Guide and Laboratory Manual Human Stem Cell Technology and Biology: A Research Guide and Laboratory Manual
by Gary S. Stein (Editor), Maria Borowski (Editor), Mai X. Luong (Editor), Meng-Jiao Shi (Editor), Kelly P. Smith (Editor), Priscilla Vazquez (Editor)
Human Stem Cell Technology & Biology: A Research Guide and Laboratory Manual integrates readily accessible text, electronic and video components with the aim of effectively communicating the critical information needed to understand and culture human embryonic stem cells.Key Features:An authoritative, comprehensive, multimedia training manual for stem cell researchersEasy to follow step-by-step laboratory protocols and instructional videos provide a valuable resourceA must-have for developing laboratory course curriculums, training courses, and workshops in stem cell biologyPerspectives written by the world leaders in the fieldIntroductory chapters will provide background informationThe volume will be a valuable reference resource for both experienced investigators pursuing stem cell and...
Stem Cell Now Stem Cell Now
by Christopher Thomas Scott (Author)
While many believe stem cell research holds the key to curing a wide range of ailments, others see this research as opening a Pandora’s box that will devalue human life. In Stem Cell Now, Christopher Scott—executive director of Stanford University’s Stem Cells and Society Program—lays out the scientific and ethical issues surrounding this national dilemma. Scott guides readers through the latest advances in stem cell research in clear, accessible language, telling the stories of the researchers who are exploring the potential of stem cells to cure cancer, grow new organs, and repair the immune system. He also leads readers through a discussion of the question at the heart of the explosive ethical debate: How, as a society, do we balance our responsibilities to the unborn and the...
Stem Cells Stem Cells
by Eapen Cherian (Author), G. Nandhini (Author), Anil Kurian (Author), K. Rajkumar (Foreword) Stem Cells: Scientific Facts and Fiction Stem Cells: Scientific Facts and Fiction
by Christine Mummery (Author), Ian Sir Wilmut (Author), Anja Van De Stolpe (Author), Bernard Roelen (Author)
In the past decades our understanding of stem cell biology has increased tremendously. Many types of stem cells have been discovered in tissues of which everyone presumed were unable to regenerate in adults; these include particularly the heart and the brain. There is vast interest in stem cells from biologists and clinicians who see the potential for regenerative medicine and future treatments for chronic diseases like Parkinson, diabetes and spinal cord lesions based on the use of stem cells and entrepreneurs in biotechnology who expect new commercial applications ranging from drug discovery to transplantation therapies. As is often the case in science, many early claims turned out to be different from those expected. Embryonic stem cell therapies have not moved rapidly into clinical...
Essential Stem Cell Methods (Reliable Lab Solutions) Essential Stem Cell Methods (Reliable Lab Solutions)
by Robert Lanza (Editor), Irina Klimanskaya (Editor)
As part of the Reliable Lab Solutions series, this volume offers an abridged and comprehensive update of selected chapters that first appeared in the three-volume stem cell series published in Methods in Enzymology. Currently, stem cells are of great interest to scientists and clinicians due to their unique ability to differentiate into various tissues of the body, making them a promising source of cells for regenerative medicine and drug discovery as well as an excellent model of vertebrate development. Essential Stem Cell Methods features a detailed set of protocols written by experts in the field and hand-selected by the editors to help researchers drive advances in this fast-moving field. The result is a clear set of step-by-step methods which steer the reader through the...
Cells That Heal Us From Cradle To Grave: A Quantum Leap in Medical Science Cells That Heal Us From Cradle To Grave: A Quantum Leap in Medical Science
Roger M. Nocera, M.D., a world leader in stem cell therapy research, announces a breakthrough medical science discovery that will revolutionize health care as we know it.
In his new book "Cells That Heal Us From Cradle To Grave: A Quantum Leap in Medical Science," Dr. Nocera reveals that a medical science discovery made in 2003 has been developed in medical clinics around the world and proven to be effective in the treatment of many heretofore incurable diseases.
Cells That Heal Us From Cradle To Grave explains how this medical science discovery is on a par with Immunology discovered two centuries ago with the smallpox vaccine, and with the discovery of antibiotics a century later.
Dr. Nocera’s book is a primer on how this amazing new medical...

View the original article here

No comments:

Post a Comment