Stem Cell Transplants May Show Promise for Multiple Sclerosis

By Denise Mann WebMD Health News

Reviewed by Louise Chang, MD

Oct. 10, 2012 -- New research suggests that stem cell transplants to treat certain brain and nervous system diseases such as multiple sclerosis may be moving closer to reality.

One study found that experimental stem cell transplants are safe and possibly effective in children with a rare genetic brain disease. Another study in mice showed that these cells are capable of transforming into, and functioning as, the healthy cell type. The stem cells used in the two studies were developed by study sponsor StemCells, Inc.

Both papers appear online in Science Translational Research.

The work, while still in its infancy, may have far-reaching implications for the treatment of many more common diseases that affect the brain and nervous system.

Researchers out of the University of California, San Francisco (UCSF), looked at the how neural stem cells behaved when transplanted into the brains of four young children with an early-onset, fatal form of Pelizaeus-Merzbacher disease (PMD).

PMD is a very rare genetic disorder in which brain cells called oligodendrocytes can't make myelin. Myelin is a fatty substance that insulates the nerve fibers of the brain, spinal cord, and optic nerves (central nervous system), and is essential for transmission of nerve signals so that the nervous system can function properly.

In multiple sclerosis, the myelin surrounding the nerve is targeted and damaged by the body's immune system.

The new study found that the neural stem cell transplants were safe. What's more, brain scans showed that the implanted cells seem to be doing what is expected of them -- i.e. making myelin.

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Stem Cell Transplants May Show Promise for Multiple Sclerosis

Early Results Show Promise for Stem Cells in Treating Chronic Liver Failure

Stem cell transfusions may someday replace the need for transplants in patients who suffer from liver failure caused by hepatitis B, according to a new study coming out of Beijing. . The results are published in the October issue of STEM CELLS Translational Medicine. Worldwide more than 500,000 people die each year from this condition.

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In China, hepatitis B virus (HBV) infection accounts for the highest proportion of liver failure cases. While liver transplantation is considered the standard treatment, it has several drawbacks including a limited number of donors, long waiting lists, high cost and multiple complications. Our study shows that mesenchymal stem cell (MSCs) transfusions might be a good, safe alternative, said Fu-Sheng Wang, Ph.D., M.D., the studys lead author and director of the Research Center for Biological Therapy (RCBT) in Beijing.

Wang along with RCBT colleague, Drs. Ming Shi and Zheng Zhang of the Research Center for Biological Therapy, The Institute of Translational Hepatology led the group of physician-scientists from the centers and Beijing 302 Hospital who conducted the study.

MSC transfusions had already been shown to improve liver function in patients with end-stage liver diseases. This time, the researchers wanted to gauge the safety and initial efficacy of treating acute-on-chronic liver failure (ACLF) with MSCs. The American Association for the Study of Liver Diseases and the European Association for the Study of the Liver define ACLF as an acute deterioration of pre-existing chronic liver disease usually related to a precipitating event and associated with increased mortality at three months due to multisystem organ failure. The short-term mortality rate for this condition is more than 50 percent.

MSCs have self-renewing abilities and the potential to differentiate into various types of cells. More importantly, they can interact with immune cells and cause the immune system to adjust to the desired level.

Of the 43 patients in this pilot study each of whom had liver failure resulting from chronic HBV infection 24 were treated with MSCs taken from donated umbilical cords and 19 were treated with saline as the control group. All received conventional therapy as well. The liver function, adverse events and survival rates were then evaluated during the 48-week or 72-week follow-up period.

Along with increased survival rates, the patients liver function improved and platelet count increased. No significant side effects were observed throughout the treatment and follow-up period.

While the results are preliminary and this pilot study includes a small number of patients, MSC transfusions appear to be safe and may serve as a novel therapeutic approach for HBV-associated ACLF patients, Dr. Shi said.

The study also highlights several key issues that will need to be considered in the design of future clinical studies, such as the optimal type of stem cells that will be infused, the minimum effective number of the cells and the best route of administration, Dr. Wang added.

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Early Results Show Promise for Stem Cells in Treating Chronic Liver Failure

Safety results of intra-arterial stem cell clinical trial for stroke presented

ScienceDaily (Oct. 11, 2012) 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.

The results were presented October 11 by Sean Savitz, M.D., professor of neurology and director of the Stroke Program at The University of Texas Health Science Center at Houston (UTHealth), at the 8th World Stroke Congress in Brasilia, Brazil.

The trial is the only randomized, double-blind, placebo-controlled intra-arterial clinical trial in the world for ischemic stroke. It is studying the safety and efficacy of a regenerative therapy developed by Aldagen Inc., a wholly-owned subsidiary of Cytomedix, Inc., that uses a patient's own bone marrow stem cells, which can be administered between 13 and 19 days post-stroke.

The therapy, called ALD-401, consists of stem cells that are identified using Aldagen's proprietary technology to isolate cells that express high levels of an enzyme that serves as a marker of stem cells. Pre-clinical studies found that these cells enhance recovery after stroke in mice. The cells are administered into the carotid artery. Patients are followed for 12 months to monitor safety and to assess mental and physical function.

"We have been approved by the Data Safety Monitoring Board (DSMB) to move the study into the next phase, which will allow us to expand the number of sites in order to complete enrollment," said Savitz, senior investigator for the multi-center study. As per the protocol for the trial, the Food and Drug Administration required a review by the DSMB prior to advancing to the next phase.

Preclinical research, including research at the UTHealth Medical School, has suggested that stem cells can promote the repair of the brain after an ischemic stroke, which is caused by a blood clot in the brain. Stroke is a leading cause of disability and the fourth-leading cause of death in the United States, according to 2008 statistics reported by the Centers for Disease Control and Prevention.

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The above story is reprinted from materials provided by University of Texas Health Science Center at Houston.

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Safety results of intra-arterial stem cell clinical trial for stroke presented

Scientists discuss stem cell discoveries at New York Stem Cell Foundation Conference

Public release date: 11-Oct-2012 [ | E-mail | Share ]

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (October 11, 2012) For the second day, The New York Stem Cell Foundation (NYSCF) Seventh Annual Translational Stem Cell Research Conference hosts the world's most preeminent stem cell scientists to present their findings on how advances in stem cell science lead to better treatments and cures for disease and injury. The conference is held at The Rockefeller University in Manhattan on October 10-11.

Today, in disease-specific sessions, scientists will share their latest finds in moving stem cell research to treatments in the following areas: cancer and blood disease; diabetes and autoimmunity; heart and muscles; neurodegeneration and spinal cord injury.

In Cancer and Blood Disease, Elaine Fuchs, PhD, The Rockefeller University, will share findings on identification of skin cancer stem cells, which have implications in understanding other cancers as well as stem cells. Joanne Kurtzberg, MD, Duke University, will discuss her work developing therapies for disease with autologous cord blood transplants. Ravi Majeti, PhD, Stanford University, will describe his recent insights into acute myeloid leukemia and how stem cell technologies can lead to new cancer treatments.

Dieter Egli, PhD, The New York Stem Cell Foundation (NYSCF), will open the session on Diabetes and Autoimmunity by detailing his group's development of stem cell-derived models of pancreatic beta cells for the study of diabetes. Yuval Dor, PhD, Hebrew University, Israel, will discuss experiments with pancreatic beta cells with the goal to understand the regenerative potential of these cells. Matthias von Herrath, MD, Novo Nordisk, will delve into another aspect of Type 1 diabetes, the problem of autoimmunity. He will close the session by sharing insights into the need for an immune modulated therapy to diabetes.

Before the afternoon sessions, Shahin Rafii, MD, Weill Medical College of Cornell University will deliver the first of two keynote addresses of the conference. He will describe his recent successes in deriving vascular cells from amniotic cells.

In the afternoon session on Heart and Muscle Diseases, Amy Wagers, PhD, Harvard University, will focus on advances in treatments and explain how studies into the mechanisms of tissue stem cell renewal may have relevant therapeutic implications. Gordon Keller, PhD, McEwen Centre for Regenerative Medicine, Canada, will describe modeling cardiac cell development from human pluripotent cells for use in toxicology and electrophysiology studies. Helen Blau, PhD, Stanford University, will describe her research to improve stem cell culture in the direction of stem cell fate and for drug screens.

In Neurodegeneration and Spinal Cord Injury, Paola Arlotta, PhD, Harvard University and a NYSCF-Robertson Stem Cell Investigator, will address the application of stem cells to understanding and possibly treating these debilitating diseases and conditions, and will describe investigations to direct reprogramming of neurons into different neuronal lineages. Lorenz Studer, MD, Memorial Sloan-Kettering Cancer Center, will discuss the potential stem cell technology holds in the treatment of Parkinson's disease. Despite past failures in the replacement of lost dopamine neurons, Dr. Studer will describe his novel protocols for the generation of these neurons for eventual use in clinical trials.

Rudolf Jaenisch, MD, The Whitehead Institute, will deliver the second keynote address of the day. Building on Shinya Yamanaka's paradigm-changing work in induced pluripotent stem (iPS) cell reprogramming, Dr. Jaenisch will discuss new methods to counter the generally low successful output of these cells. He will also summarize how targeted genome editing may help unleash the potential of iPS cells and embryonic stem cells for both the study of and therapy for disease.

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Scientists discuss stem cell discoveries at New York Stem Cell Foundation Conference

Brain Stem-Cell Implants Help Children With Rare Illness

Four boys with a rare and often fatal brain disease were implanted with stem cells that began fixing damage that impeded their ability to walk, talk and eat, a trial found.

The findings, published today in the journal Science Translational Medicine, are from the first stage of human tests funded by StemCells Inc. (STEM), a Newark, California-based company.

The children have a genetic disorder called Pelizaeus- Merzbacher, in which the brain cant make myelin, the fatty insulation for nerve cells that helps conduct brain signals. The children all had evidence of myelin growth a year later. The increased abilities shown by three of the boys in the University of California San Francisco study may bode well for other diseases caused by a lack of myelin insulation, including multiple sclerosis and cerebral palsy, the authors wrote.

Those were severely impaired children, said Stephen Back, a professor of pediatrics and neurology at Portlands Oregon Health & Science University, in a telephone interview. The fact that they showed any neurological improvement is very encouraging.

Back did work in mice that preceded todays work in humans, which he wasnt directly involved in. His study, published simultaneously, showed that the animals with no myelin at all grew some after being implanted with human stem cells.

Pelizaeus-Merzbacher disease causes the degeneration of the nervous system, and there is no cure or standard treatment. People with the illness experience a loss of coordination, thinking and motor abilities. Its one of several disorders linked to genes that control myelin production.

The incidence of the disease is 1 in 200,000 to 500,000 people, according to todays study of the boys.

The boys were between the ages of 1 and 6. They were given purified neural stem cells from a fetal brain, which was then grown in culture. The stem cells were inserted into the frontal lobe, using brain imaging as a guide. The boys brains were scanned 24 to 48 hours after surgery to assess safety.

The children were on drugs to suppress their immune systems and prevent their bodies from rejecting the stem cells for nine months. Side effects included rashes, diarrhea and fever. One boy had fluid collect under his scalp, which later vanished on its own. A second subject had some bleeding in the brain after the surgery, which was without clinical consequence, according to the paper.

One of the boys developed the ability to take steps with assistance and began to speak single words. Another started eating solid food on his own. A third began to walk without the assistance of a walker and began eating on his own.

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Brain Stem-Cell Implants Help Children With Rare Illness

StemGenex™ on Adult Stem Cell-Based Therapy for Multiple Sclerosis

LA JOLLA, Calif., Oct. 10, 2012 /PRNewswire/ --New research directions are being explored to find therapies for hard to treat diseases. One exciting new approach is the use of autologous Adult Stem Cells. Multiple Sclerosis (MS) is one of the many notable diseasesadult stem cell therapycould potentially impact. Multiple Sclerosis (MS) is a disorder in which an individual's own immune system attacks the 'myelin sheath'. The myelin sheath serves to protect the nerve cells within the body's central nervous system (CNS). The damage caused by MS may result in many types of symptoms including:

(Photo: http://photos.prnewswire.com/prnh/20121010/LA89802-INFO)

Currently there is no cure for MS, but MS stem cell therapiesattempt to slow the disease's progression and limit symptoms. Since adult stem cells have the ability to differentiate into many different types of cells, such as those required for proper functioning and protection of nerve cells, the use of adult stem cells for MS therapy could be of substantial value. Adult stem cells can be isolated with relative ease from an individual's own 'adipose' (fat) tissue. As a result, adult stem cell therapy is not subject to the ethical or religious issues troubling embryonic methods.

Encouragingly for MS treatment potential, scientific researchers have been studying the properties of adipose-derived stem cells. Their results from canine and equine studies suggest anti-inflammatory and regenerative roles for these stem cells. Also, further research findings suggest these adipose-derived stem cells can have specific immune-regulating properties. Markedly, clinical-based work conducted overseas has indicated that individuals suffering from MS could respond well to adipose-derived stem cell treatment, with a substantially improved quality of life.

The US based company, StemGenex, is pioneering new methods for using adipose derived adult stem cells to help in diseases with limited treatment options like MS. StemGenex has been conducting research with physicians over the last 5 years to advance adult stem cell treatment protocols for alleviating MS symptoms. StemGenex's proprietary protocol includes the use of a double activation process, which increases both the viability and the quantity of stem cells that are received in a single application.

To find out more about stem cell treatments contact StemGenex either by phone at 800.609.7795 or email at Contact@StemGenex.com.

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StemGenex™ on Adult Stem Cell-Based Therapy for Multiple Sclerosis

The New York Stem Cell Foundation announces $9 million to 6 new NYSCF-Robertson Investigators

Public release date: 10-Oct-2012 [ | E-mail | Share ]

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (October 10, 2012) The New York Stem Cell Foundation (NYSCF) named six of the most promising scientists as its 2012 NYSCF Robertson Investigators.

Each Investigator will receive a $1.5 million award disbursed over the next five years to foster his or her innovative research by expanding laboratories and by training other scientists. Three of the scientists were named NYSCF Robertson Stem Cell Investigators, a program in its third year, and three were named NYSCF Robertson Neuroscience Investigators, a program in its second year.

"These young Investigators truly undertake the most daring yet rewarding stem cell research. We are all honored to support the future investigations of these promising talents," said Susan L. Solomon, Chief Executive Officer of NYSCF.

The Investigators were announced at NYSCF's Seventh Annual Translational Stem Cell Research Conference, held at The Rockefeller University in Manhattan.

Designed to support scientists engaged in novel neuroscience and cutting-edge translational stem cell research, the two Investigator programs aid these researchers as they move beyond postdoctoral work and establish their own laboratories.

The Investigator award builds on the previous success of NYSCF's Postdoctoral Fellowship program, which is the largest program of postdoctoral support for stem cell researchers in the United States, and has provided funding for 35 postdoctoral researchers to date.

Marc Tessier-Lavigne, President of The Rockefeller University, chaired the NYSCF Robertson Neuroscience Investigator program's selection committee.

"With such an outstanding group of young scientists, we had an exceptionally difficult decision to make. We are thrilled to be able to give these awards to such a talented group of scientists," remarked Tessier-Lavigne. "We are confident they will become leaders in the field of neuroscience."

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The New York Stem Cell Foundation announces $9 million to 6 new NYSCF-Robertson Investigators