Archive for May, 2012

ANN ARBOR, Mich.--(BUSINESS WIRE)--

RetroSense Therapeutics, a gene therapy company dedicated to vision restoration, announced completion of a pre-IND meeting with the Center for Biological Evaluation and Research (CBER) division of the FDA that took place on May 22nd 2012 regarding RetroSenses lead biologic, RST-001 for vision restoration in retinal degenerative conditions.

The purpose of the meeting was to obtain CBERs guidance for the clinical path to a Biologics License Application (BLA) for RST-001 in the US, and clarity on the steps required for Investigational New Drug (IND) submission. The discussions included manufacturing criteria, the scope and design of the preclinical studies, and the scope and design of Phase I and IIa clinical trials.

Sean Ainsworth, CEO, noted,"We were quite encouraged by the FDA's feedback and comments and identified nothing that will hinder our path to the clinic. RetroSense remains on schedule to bring RST-001 to the clinic safely and expeditiously.

Peter Francis, MD, PhD, Clinical Director, led the meeting. Dr. Francis stated, It was a successful, productive interaction with the FDA. We were thankful to have representation by Foundation Fighting Blindness, who have been supportive of our novel approach to vision restoration.

We are very excited about the potential for this innovative treatment to restore vision in people who are blind from retinal degenerations, says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. It is an elegant and powerful approach to overcoming devastating eye diseases.

About RetroSense Therapeutics

RetroSense Therapeutics is a biotechnology company developing a game-changing gene therapy to restore vision in patients suffering from blindness due to retinitis pigmentosa (RP) and advanced dry age-related macular degeneration (advanced dry-AMD). There are currently no FDA approved therapies to improve or restore vision in patients with these retinal degenerative conditions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information about RetroSense, visit http://www.retro-sense.com/.

About Foundation Fighting Blindness

The Foundation Fighting Blindness is a national non-profit organization driving research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect more than 10 million Americans. Since 1971, the Foundation has raised over $450 million as the leading non-governmental funder of retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a variety of retinal degenerative diseases, and proving a cure is in sight. With a network of 50 chapters, the Foundation also provides support, education and resources to affected individuals and their families in communities across the country.

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RetroSense Therapeutics Completes pre-IND Meeting for RST-001

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Sarah Jackson press_releases@the-jci.org Journal of Clinical Investigation

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response. A recent clinical trial indicated that gene therapy to insert the correct ADA gene in the patient's own bone marrow cells can also lead to a good response.

However, patients were noted to have defects in B cell tolerance, meaning that some B cells that react to antigens from the body fail to be eliminated, leading to an autoimmune response. Dr. Eric Meffre and colleages at Yale University in New Haven, Connecticut and Alessandro Aiuti in Milan, Italy joined together to better understand why patients developed B cell tolerance problems. They found that loss of the ADA gene directly contributes to B cell tolerance problems and that these defects are mostly corrected after gene therapy. Their results point to a previously unknown role for ADA in B cell response and support the use of gene therapy as an effective treatment option for ADA-deficient severe combined immunodeficiency patients.

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TITLE:

Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy

AUTHOR CONTACT:

Eric Meffre Yale University School of Medicine, New Haven, CT, USA Phone: 1-203-737-4535; Fax: 1-203-785-7903; E-mail: eric.meffre@yale.edu

ACCOMPANYING COMMENTARY

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Gene therapy can correct forms of severe combined immunodeficiency

Scientists on Wednesday reported that they have for the first time taken skin cells from heart attacks patients and turned them into healthy heart tissue that could hopefully be used to one day repair damaged heart muscle.

The healthy, beating heart tissue was grown successfully in the lab from human-induced pluripotent stem cells (hiPSCs), and while scientists said they were not safe enough to put back into human patients, they appeared to work well with other cells when implanted into rats. HiPSCs are a recently discovered source far less controversial than use of embryonic stem cells. And, because the transplanted hiPSCs come from the individual, it could resolve the problems seen with tissue and organ rejection.

While the technique has shown promise in rats, the scientists say there are numerous obstacles to overcome and it could take up to ten years or longer before clinical trials could be available for humans. Even so, it is a significant advance in the quest for replacement cell therapy for heart failure patients.

More people are surviving following a heart attack than ever before and therefore the number of people living with a damaged heart and heart failure is increasing, Nicholas Mills, a consultant cardiologist at Edinburgh University, told The Guardian. Unfortunately, the body has only very limited capacity to repair the heart following a heart attack. There is therefore an urgent need to develop effective and safe treatments to regenerate the heart.

Recent research has shown that hiPSCs could be derived from young and healthy people and are capable of transforming into heart cells. However, researchers have not been able to obtain those cells from elderly and diseased patients. And until now, researchers have not been able to show that heart cells created from hiPSCs could integrate with existing heart tissue.

What is new and exciting about our research is that we have shown that its possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young the equivalent to the stage of his heart cells when he was just born, said lead researcher Professor Lior Gepstein, of Sohnis Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine, Technion-Israel Institute of Technology and Rambam Medical Center in Haifa, Israel.

For their study, published in the European Heart Journal, Limor Zwi-Dantsis, a PhD student in the Sohnis Research Laboratory, Gepstein and colleagues took skin cells from two male heart failure patients (ages 51 and 61) and reprogrammed them with three genes (Sox2, Klf4 and Oct4), followed by a small molecule (valproic acid) to the cell nucleus.

The team also used an alternative strategy that involved a virus that delivered reprogramming information to the cell nucleus but which was capable of being removed afterward to avoid insertional oncogenesis.

Using these methods, the hiPSCs were able to differentiate to become cardiomyocytes (heart muscle cells) just as effectively as hiPSCs that had been developed from healthy, young volunteers. The researchers were then able to make cardiomyocytes develop into heart muscle tissue, which they cultured together with pre-existing cardiac tissue. The tissues were beating together within 48 hours, said the researchers.

The researchers transplanted the new tissue into the hearts of healthy rats and found that the grafted tissue started to establish connections with the cells in the host tissue.

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Human Skin Cells Turned Into Healthy Heart Muscle

On a special surface that could help advance stem cell therapies, UM researchers have turned human skin cells into adult-derived stem cells, coaxed them into bone cells and then transplanted them into holes in the skulls of mice. The cells produced four times as much new bone growth as in the mice without the extra bone cells. In this pink-stained image, the black outline partially encloses the new bone growth in the skull. Image credit: Villa-Diaz, L.G., Brown, S.E., Liu, Y. Ross, A.M., Lahann, J.M., Krebsbach, P.H., University of Michigan

ANN ARBOR University of Michigan researchers have proven that a special surface, free of biological contaminants, allows adult-derived stem cells to thrive and transform into multiple cell types. Their success brings stem cell therapies another step closer.

To prove the cells regenerative powers, bone cells grown on this surface were then transplanted into holes in the skulls of mice, producing four times as much new bone growth as in the mice without the extra bone cells.

An embryos cells really can be anything they want to be when they grow up: organs, nerves, skin, bone, any type of human cell. Adult-derived induced stem cells can do this and better. Because the source cells can come from the patient, they are perfectly compatible for medical treatments.

In order to make them, Paul Krebsbach, professor of biological and materials sciences at the UM School of Dentistry, said, We turn back the clock, in a way. Were taking a specialized adult cell and genetically reprogramming it, so it behaves like a more primitive cell.

Specifically, they turn human skin cells into stem cells. Less than five years after the discovery of this method, researchers still dont know precisely how it works, but the process involves adding proteins that can turn genes on and off to the adult cells.

Before stem cells can be used to make repairs in the body, they must be grown and directed into becoming the desired cell type. Researchers typically use surfaces of animal cells and proteins for stem cell habitats, but these gels are expensive to make, and batches vary depending on the individual animal.

You dont really know whats in there, said Joerg Lahann associate professor of chemical engineering and biomedical engineering.

For example, he said that human cells are often grown over mouse cells, but they can go a little native, beginning to produce some mouse proteins that may invite an attack by a patients immune system.

The polymer gel created by Lahann and his colleagues in 2010 avoids these problems because researchers are able to control all of the gels ingredients and how they combine.

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UM: Stem-Cell-Growing Surface Enables Bone Repair

A growth factor isolated from human stem cells shows promising results in a mouse model of multiple sclerosis.

Human mesenchymal stem cells (hMSCs) have become a popular potential therapy for numerous autoimmune and neurological disorders. But while these bone marrow-derived stem cells have been studied in great detail in the dish, scientists know little about how they modulate the immune system and promote tissue repair in living organisms.

Now, one research team has uncovered a molecular mechanism by which hMSCs promote recovery in a mouse model of multiple sclerosis (MS).

According to research, published online Sunday (May 20) in Nature Neuroscience, a growth factor produced by hMSCs fights MS in two ways: blocking a destructive autoimmune response and repairing neuronal damage. The finding could help advance ongoing clinical trials testing hMSCs as a therapy for MS.

The researchers have identified a unique factor that has surprisingly potent activity mediating neuron repair, said Jacques Galipeau, a cell therapy researcher at Emory University in Atlanta, Georgia, who was not involved in the research. The magnitude of the effect on a mouse model of MS is a big deal.

MS is an autoimmune disease in which the immune system attacks myelin sheaths that surround and protect nerve cells. The attack leaves nerves exposed and unable to send signals to the brain and back, resulting in the loss of motor skills, coordination, vision, and cognitive abilities. There is no cure for MS, and most current therapies work to simply suppress the immune system, preventing further neuronal damage. None have demonstrated an ability to also repair damaged myelin and promote recovery.

In 2009, Robert Miller and colleagues at Case Western Reserve University in Cleveland, Ohio, demonstrated that hMSCs dramatically reversed the symptoms of multiple sclerosis in a mouse model of the disorder. The animals got better, recalled Miller. The team hypothesized that the stem cells suppress the immune response and promote remyelination.

But Miller wanted to know exactly what the cells were doing. To find out, his team isolated the medium on which the hMSCs were grown to determine if the cells or something they secreted was responsible for the observed recovery. The medium alone was enough to induce recovery in mice, pointing to the latter.

To find out exactly which molecule or molecules in the medium were responsible, the researchers separated the proteins in the fluid based on the molecular weight and injected each isolate into mice exhibiting symptoms of MS. The mid-weight solution, of proteins with masses between 50 and 100 kilodaltons (kDa), caused recovery. That eliminated a huge number of potential candidates, said Miller.

The researchers then narrowed the field again with a literature search for a molecule that fit their criteria: secreted by hMSCs, 50-100 kDa in size, and involved in tissue repair. They identified hepatocyte growth factor (HGF), a cytokine made by mesenchymal cells that has been shown to promote tissue regeneration and cell survival in numerous experiments. Sure enough, HGF alone was enough to promote recovery in the MS mouse models, and blocking the receptor for HGF in those mice blocked recovery. The team also demonstrated that HGF suppresses immune responses in vivo and accelerates remyelination of neurons in vitro. Finally, they saw that HGF causes remyelination in rats with a lesion on their spinal cord.

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Could Stem Cells Cure MS?

Editor's Choice Main Category: Multiple Sclerosis Article Date: 24 May 2012 - 14:00 PDT

Current ratings for: 'Recovery From Multiple Sclerosis By Growth Factor In Stem Cells'

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Animals that were injected with hepatocyte growth factor were noted to have grown new neural cells and lower levels of inflammation. Most significantly, the researchers noted that the protective envelope of myelin, the myelin sheath, which surrounds the core of a nerve fiber and facilitates the transmission of nerve impulses, re-grew and covered lesions that were caused by MS.

Robert H. Miller, professor of neurosciences at the School of Medicine and vice president for research at Case Western Reserve University declared: "The importance of this work is we think we've identified the driver of the recovery."

MS is caused by damage to the myelin sheath, the protective covering that surrounds nerve cells. The nerve damage is caused by inflammation, which occurs when the body's own immune cells attacks the nervous systems located in areas of the brain, the optic nerve, and spinal cord. This damage can cause an interruption of the nerve signals, which results in loss of balance and coordination, cognitive ability, as well as in other functions and in time, these intermittent losses may become permanent. In 2009, Caplan and Miller discovered that mice with MS injected with human mesenchymal stem cells recovered from the type of damage that was brought on by MS. A clinical trial is currently underway based on their research, whereby patients with MS are injected with their own stems cells.

During this trial, the team decided to first establish whether the presence of stem cells or other cells induce recovery. They injected a total of 11 animals with MS with the medium, in which mesenchymal stem cells that were taken from bone marrow grew, discovering that all animals displayed a rapid reduction in functional deficits. An analysis demonstrated that unless the injected molecules had a certain size or weight, i.e. between 50 and 100 kiloDaltons, the course of the disease remained unchanged.

Other research, as well as the team's own studies, suggested that this was likely to be instigated by the hepatocyte growth factor, which is secreted by mesenchymal stem cells.

The team then injected the animals with either 50 or 100 nanograms of the growth factor on alternate days for a 5-day period and observed a decrease in the level of signaling molecules that promote inflammation, whilst the level of signaling molecules that oppose inflammation increased. The researchers noted a growth of neural cells, whilst nerves that were exposed because of MS were rewrapped with myelin. Recovery was marginally better in those mice that received the 100-nanogram injections compared with those receiving the 50-nanogram injections.

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Recovery From Multiple Sclerosis By Growth Factor In Stem Cells

May 24, 2012

Connie K. Ho for RedOrbit.com

Technology is rapidly progressing and so is research related to stem cells.

Researchers from the University of Michigan recently announced that they found a special surface without biological contaminants that can help adult-derived stem cells to grow and change into different cell types. The findings, published in the journal Stem Cells, are considered a breakthrough in stem cell research.

In the study, scientists grew bone cells on the surface and then transplanted the cells to the skulls of mice to look at the cells regenerative powers. The results showed that the cells produced four times as much new bone growth in mice without the help of extra bone cells. The importance of these adult-derived induced stem cells is that they come from the patient and these cells are compatible for medical treatments.

We turn back the clock, in a way. Were taking a specialized adult cell and genetically reprogramming it, so it behaves like a more primitive cell, commented Paul Krebsbach, professor of biological and materials sciences at the U-M School of Dentistry, on the process of stem cell creation.

In the project, researchers examined how human skin cells are turned into stem cells and, even though they are not exactly sure as to how the process works, how it involves the addition of proteins that can signal the genes to turn on and off to the adult cells. Prior to being used to repair parts of the body, the stem cells are grown and directed to become a specific cell type. Researchers were able to use the surface of the animal cells and proteins for stem cell habitats, but saw that the amount of cells produced could vary by animal.

You dont really know whats in there, noted Joerg Lahann, associate professor of chemical engineering and biomedical engineering.

One difficulty researchers have encountered in the past is the fact that human cells and animals cells can sometimes mix. However, the polymer gel made by Lahann and his fellow researchers helped avoid this problem. Researchers were able to gain better control over the gels ingredients and how they were combined.

Its basically the ease of a plastic dish, Lahann said. There is no biological contamination that could potentially influence your human stem cells.

Continued here:
Bone Repair Via Stem-cell-growing Surface

Dr. John D. Cunningham / Getty Images

In a medical first, scientists in Haifa, Israel, took skin cells from two heart failure patients and reprogrammed them into stem cells that generated healthy, beating heart muscle cells in the lab. Though human testing is likely a decade off, the hope is that such cells can be used to help people with heart failure repair their damaged hearts with their own skin cells.

In the current study, scientists first mixed the newly developed heart cells with pre-existing heart tissue within days, the cells were beating together. The heart tissue was then transplanted into rats, where it integrated with the rats healthy heart cells.

What is new and exciting about our research is that we have shown that its possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young the equivalent to the stage of his heart cells when he was just born, says lead researcherDr. Lior Gepstein, a senior clinical electrophysiologist at Rambam Medical Center in Israel, said in a statement.

The researchers were pleased to find that the cells made from the two heart failure patients, ages 51 and 61, generated heart muscle cells that were just as effective as those developed from healthy, young controls.

(MORE: Study During Beijing Olympics Shows How Pollution Harms the Heart)

If the technology works in human hearts, it could potentially prevent problems of immune rejection, since the cells would be the patients own. It would also avoid the moral issues surrounding the use of embryonic stem cells, since such reprogrammed stem cells or human induced pluripotent stem (iPS) cells do not use embryos.

But its still too early to predict whether the procedure could be successful humans. The new study involved cells from only two patients and were transplanted only into healthy animals. The authors note that human clinical trials are likely at least five or 10 years away. Further, creating iPS cells is not an easy or efficient process; its not clear whether enough cells could be made quickly enough to repair the broad-scale damage that occurs after a heart attack.

Reprogramming skin cells to become stem cells also introduces the potential for the cells to grow out of control and become cancerous. The Israeli researchers took additional steps removing certain transcription factors and viral factors to reduce the risk of cancer. But these hurdles would have to be revisited if the technique is tested in human patients.

This is an interesting paper, but very early and its really important for patients that the promise of such a technique is not oversold, John Martin, a professor of cardiovascular medicine at University College London, told Reuters.The chances of translation are slim and if it does work it would take around 15 years to come to clinic.

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Scientists Turn Skin Cells into Healthy Heart Cells

LONDON (May 23): Scientists have for the first time succeeded in taking skin cells from patients with heart failure and transforming them into healthy, beating heart tissue that could one day be used to treat the condition.

The researchers, based in Haifa, Israel, said there were still many years of testing and refining ahead. But the results meant they might eventually be able to reprogram patients' cells to repair their own damaged hearts.

"We have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young - the equivalent to the stage of his heart cells when he was just born," said Lior Gepstein from the Technion-Israel Institute of Technology, who led the work.

The researchers, whose study was published in the European Heart Journal on Wednesday, said clinical trials of the technique could begin within 10 years.

Heart failure is a debilitating condition in which the heart is unable to pump enough blood around the body. It has become more prevalent in recent decades as advances medical science mean many more people survive heart attacks.

At the moment, people with severe heart failure have to rely on mechanical devices or hope for a transplant.

Researchers have been studying stem cells from various sources for more than a decade, hoping to capitalise on their ability to transform into a wide variety of other kinds of cell to treat a range of health conditions.

There are two main forms of stem cells - embryonic stem cells, which are harvested from embryos, and reprogrammed "human induced pluripotent stem cells" (hiPSCs), often originally from skin or blood.

TISSUES BEATING TOGETHER

Gepstein's team took skin cells from two men with heart failure - aged 51 and 61 - and transformed them by adding three genes and then a small molecule called valproic acid to the cell nucleus.

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Scientists turn skin cells into beating heart muscle

In the first procedure of its kind, skin cells taken from patients suffering from heart failure were reprogrammed and changed into heart muscle cells. Not only were the transformed cells healthy, but they were also transplanted into the hearts of rats and were able to integrate with the existing heart tissue.

Published in the European Heart Journal, the research examined the use of human-induced pluripotent stem cells (hiPSCs) to treat damaged hearts. HiPSCs are cells that are derived from other cells in a persons body.

We were able to show [in earlier studies] that you can take these hiPSCS from healthy heart patients and coax them into bonafide heart cells, lead author Lior Gepstein, professor of medicine (cardiology) and physiology at the Technion-Israel Institute of Technology and Rambam Medical Center in Haifa, Israel, told FoxNews.com. The question we asked in this study was whether you can do the same from an elderly individual that had suffered from advance heart failure.

Because hiPSCs are derived from the person in need of the stem cells, they could potentially help to bypass the painful process of rejection that many transplant patients go through. According to Gepstein, if this process is perfected, it could lead to much more localized treatments.

When there is significant damage from a heart attack, or with heart failure, where the heart doesnt pump enough blood into circulation, patients usually need a heart transplant, Gepstein said. But perhaps in the future, we can take a small sample of skin and convert them into stem cells specific to that patient. Then we can only replace the area with scar tissue rather than replace the dying heart.

In order to transform the skin cells into hiPSCs, Gepstein and his colleagues gave them a reprogramming cocktail, which involved delivering three genes (Sox2, Klf4 and Oct4), followed by a small molecule called valproic acid, to the nucleus of the cell.

This process turned the skin cells into heart muscle cells, or cardiomyocytes, which the researchers were able to subsequently turn into heart muscle tissue by culturing them together with cardiac tissue.

We converted the cells back into a state that resembles their early state in the embryo, Gepstein said. So they highly resemble the patients cells at the time they were born. When you give them proper conditions, they can become any type of cell in the body.

This area of study has advanced very rapidly, Gepstein added. You can take almost any type of adult cells - hair follicles, blood cells, etc. - and reprogram them to make hiPSCS cells. Skin cells are the easiest way to do it, and you dont need a lot of them.

Once the tissue had formed, it was transplanted into the hearts of healthy rats, where it successfully grafted and integrated with the existing tissue.

Originally posted here:
Scientists convert skin cells into full functioning heart cells

WEDNESDAY, May 23 (HealthDay News) -- In a medical science first, researchers turned skin cells from heart failure patients into heart muscle cells that may then be used to fix damaged cardiac tissue.

The researchers said the achievement -- done initially with rats -- opens up the prospect of using heart failure patients' own stem cells -- a form of cell called human-induced pluripotent stem cells (hiPSCs) -- to repair damaged hearts. And since the reprogrammed stem cells would originate with the patient, their immune systems would not reject the cells as foreign, the researchers explained.

They added, however, that many obstacles must be overcome before it would be possible to use hiPSCs in humans this way, and any clinical trial would be at least five to 10 years away.

"We have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young -- the equivalent to the stage of his heart cells when he was just born," study leader Lior Gepstein said in a European Heart Journal news release. The study's findings are scheduled for online publication in the journal May 23.

Gepstein is professor of medicine (cardiology) and physiology at the Sohnis Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine at the Technion Israel Institute of Technology and Rambam Medical Center in Haifa, Israel.

One expert in the United States applauded the achievement.

"The ability to source a patient's own skin cells and transform them into heart muscle is truly revolutionary," said Dr. Gregory Fontana, chairman of cardiothoracic surgery at Lenox Hill Hospital in New York City.

The results are "another step toward the treatment of heart failure with stem cells," he said. "Although further work is needed, this work represents another step closer to the clinic."

In the study, the researchers retrieved skin cells from two male heart failure patients, ages 51 and 61, and then reprogrammed them in the lab to develop into heart muscle tissue, which was then blended with pre-existing heart tissue. Within 24 to 48 hours, the tissues were beating together.

The new tissue was transplanted into healthy rat hearts and started to establish connections with the cells of the rat hearts. Success in animal experiments does not necessarily translate to success in humans, however.

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Scientists Turn Skin Cells Into Cardiac Cells to Help Failing Hearts

People who suffer from heart failure could someday be able to use their own skin stem cells to regenerate their damaged heart tissue, according to a new Israeli study.

Researchers took stem cells from the skin of two patients with heart failure and genetically programmed them to become new heart muscle cells. They then transplanted the new cells into healthy rats and found that the cells integrated with cardiac tissue that already existed.

The study, published in European Heart Journal, marks the first time ever that scientists could use skin cells from people with heart failure and transform damaged heart tissue this way.

The newly generated cells turned out to be similar to embryonic stem cells, which can potentially be programmed to grow into any type of cell.

"What is new and exciting about our research is that we have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young the equivalent to the stage of his heart cells when he was just born," Dr. Lior Gepstein, lead researcher and a senior clinical electrophysiologist at Rambam Medical Center in Haifa, Israel, said in a news release.

The findings open up the possibility, the authors wrote, that people can use their own skin cells to repair their damaged hearts, which could prevent the problems associated with using embryonic stem cells.

"This approach has a number of attractive features," said Dr. Tom Povsic, an interventional cardiologist at Duke University Medical Center. "We can get the cells that you start with from the patient himself or herself. It avoids the ethical dilemma associated with embryonic stem cells and it removes the possibility of rejection of foreign stem cells by the immune system." Povsic was not involved with the Israeli study.

Another advantage of using skin cells is that other types of cells taken from patients themselves, such as bone marrow cells, could potentially lead to the development of unhealthy tissue.

"If a patient is already sick with heart disease, one of the reasons it may develop is that stem cells weren't able to repair the heart the way they should," Povsic added. Skin cells, he explained, are generally healthy.

"It is very exciting and very interesting, but we are far away from taking this to patients," said Dr. Marrick Kukin, director of the Heart Failure Program at St. Luke's-Roosevelt Hospital who was also not involved in the Israeli study.

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Can Stem Cells Repair Heart Tissue?

Editor's Choice Main Category: Cardiovascular / Cardiology Article Date: 25 May 2012 - 0:00 PDT

Current ratings for: 'Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells'

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This achievement is significant, as it opens up the prospect of treating heart failure patients with their own, human-induced pluripotent stem cells (hiPSCs) to fix their damaged hearts.

Furthermore, the cells would avoid being rejected as foreign as they would be derived from the patients themselves. The study is published in the European Heart Journal. However, the researchers state that it could take a minimum of 5 to 10 years before clinical trials could start due to the many obstacles that must be overcome before using hiPSCs in humans is possible.

Although there has been advances in stem cell biology and tissue engineering, one of the major problems scientists have faced has been lack of good sources of human heart muscle cells and rejection by the immune system. Furthermore, until now, scientific have been unable to demonstrate that heart cells created from hiPSCs could integrate with existing cardiovascular tissue.

"What is new and exciting about our research is that we have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are health and young - the equivalent to the stage of his heart cells when he was just born," said Professor Lior Gepstein, Professor of Medicine (Cardiology) and Physiology at the Sohnis Research Laboratory for Cardiac Electrophysiology and Regenerative Medicine, Technion-Israel Institute of Technology and Rambam Medical Center in Haifa, Israel, who led the study.

In the study, Professor Gepstein, Ms Limor Zwi-Dantsis, and their colleagues retrieved skin cells from two male heart failure patients, aged 51 and 61 years, and reprogrammed the cells by delivering 3 transcription factors (Sox2, Oct4, and Klf4) in addition to a small molecule called valproic acid, to the cell nucleus. The team did not include a transcription factor called c-Myc as it is a known cancer-causing gene.

Professor Gepstein said:

In addition, the team used an alternative strategy involving a virus transferred reprogramming data to the cell nucleus. However, the team removed the virus after the information had been transferred in order to avoid insertional oncogenesis.

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Skin Cells From Heart Failure Patients Made Into Healthy New Heart Muscle Cells

(CBS News) A new study of patients with heart failure found a novel treatment approach might reverse the damage that has long been considered irreversible: Fixing their damaged hearts using stem cells derived by their own skin cells.

Stem cells heal heart attack scars, regrow healthy muscle Stem cells cure heart failure? What "breakthrough" study shows

In what scientists are calling a first, skin cells were taken from heart failure patients and transformed into stem cells, which were then turned into heart muscle cells capable of beating - albeit in a petri dish.

The treatment approach has scientists buzzing because it avoids the risk of possible immune system rejection from transplanting "foreign" stem cells, since the cells came from patients' own bodies.

"What is new and exciting about our research is that we have shown that it's possible to take skin cells from an elderly patient with advanced heart failure and end up with his own beating cells in a laboratory dish that are healthy and young - the equivalent to the stage of his heart cells when he was just born," the study's author Professor Lior Gepstein, professor of cardiology and physiology at the Technion-Israel Institute of Technology in Haifa, said in a news release.

Just how do skin cells become heart cells? Researchers took skin cells from two male patients with heart failure, a 51 and 61-year-old, and genetically reprogrammed them by injecting a cocktail of "transcription factors" and a virus into the nucleus of the skin cell, followed by removing the virus and transcription factors that have been linked to cancerous tumor growth. The goal was to reprogram the cells into human-induced pluripotent stem cells (hiPSCs) that could help repair hearts.

"One of the obstacles to using hiPSCs clinically in humans is the potential for the cells to develop out of control and become tumours," explained Prof Gepstein in using the technique.

Once in stem cell-form, the cells differentiated in a petri dish to become heart muscle cells called cardiomyocytes, which the researchers then combined with heart tissue and cultured them into healthy heart muscle tissue. Within 48 hours, the tissues were beating together.

"The tissue was behaving like a tiny microscopic cardiac tissue comprised of approximately 1000 cells in each beating area," Gepstein said in a statement.

The researchers then transplanted the new human tissue into rats, finding it grafted to the rat's host cardiac tissues. Their research is published in the May 22 issue of the European Heart Journal.

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Skin cells transformed into beating heart tissue, fueling heart failure treatment hopes

Teva Pharmaceutical Industries Ltd. (Nasdaq: TEVA; TASE: TEVA) will enter the personalized medicine field, which will have high efficiency to cost ratios, albeit limited market segments, says Harel Finance analyst Steven Tepper following yesterday's speech by new Teva president and CEO Dr. Jeremy Levin at the ILSI Biomed 2012 National Life Sciences and Technology Week conference. Tepper says that the appointment of Dr. Michal Hayden, an expert in personalized medicine, as president of Global R&D and Chief Scientific Officer hints at this direction.

Tepper says, "Teva will continue to be a leader in generics, and will presumably continue to develop non-prescription drugs and biosimilars, which are essential in a world of escalating health expenses." He adds, "Teva is not abandoning the innovative field, but will presumably try to focus on the diseases of tomorrow in an attempt to reach drugs with high cost-efficiency ratios."

Finally, "Teva, which already has geographical diversity, will continue to expand geographically, especially in emerging markets, such as China and India, as well as in South America."

Tepper gives Teva a "Buy" recommendation with a target price of $55/NIS 212. Teva's share price rose 1.5% by early afternoon on the TASE today to NIS 150.20, after rising 0.5% on Nasdaq yesterday to $39.18, giving a market cap of $36.9 billion.

Published by Globes [online], Israel business news - http://www.globes-online.com - on May 23, 2012

Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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Harel: Teva to enter personalized medicine field

MONTREAL - Custom-made therapy rather than a one-size-fits-all approach is being hailed as the new medical reality, but theres a gap between great expectations and what current technology and limited means can offer, especially when it comes to treatment for cancer patients, experts say.

A provincial cancer advocacy group will be looking at the promise of personalized medicine at a symposium in Montreal Wednesday featuring top-notch researchers in cancer and novel treatments.

The conference coincides with a series of public hearings by the Quebec commission on ethics in science and technology, held in Montreal, Quebec and Rimouski, on personalized medicine: Who? How? And at what price?

What does personalized medicine actually mean to the patient, the researcher, the commercial developer and to society, which may or may not be able to pay the costs generated by new technologies, said Nathalie Rodrigue of the Coalition Priorit Cancer, whose advocacy group supports patients timely access to advanced treatments.

Personalized medicine is a proactive approach to health care based on technological advances that enable the use of genetic or molecular information in treating and preventing disease, according to the definition by CEPMED, the Montreal-based centre for excellence in personalized medicine.

That innovation includes whole genome sequencing or spelling out a persons entire DNA genetic code, as an option for personalized prevention programs and follow-up medical treatment.

Some scientists are going beyond genome analysis and using cutting-edge sequencing to identify mutations at the root of a patients tumour. Cancer treatment is then determined by various tests during a patients treatment to see how the tumour evolves and reacts to medication.

Science can do a lot to map the genetic evolution of disease and monitor response to treatment, said research oncologist Mark Basik of the Lady Davis Institute for Medical Research at the Montreal Jewish General Hospital.

But we have to go very slowly and carefully adapt all these discoveries to the clinic, Basik said. Some renowned clinics in the United States started tests for personalized treatment of certain tumours only to discover that the therapy was not hitting its target because the tumour had evolved again, he added.

Or, they were not confirmed in larger trials so those tests were offered prematurely, he said. Its a shortcut we cannot take. We have to make sure repeatedly that these things do what they are meant to do.

Original post:
Symposium to explore personalized medicine

Public release date: 23-May-2012 [ | E-mail | Share ]

Contact: Kim Menard kim.menard@uphs.upenn.edu 215-662-6183 University of Pennsylvania School of Medicine

PHILADELPHIA - While researchers are busy identifying new biomarkers to detect disease and tailor treatments to individual needs, legal battles have been waged all the way up to the Supreme Court, trying to sort out whether a private company can own the rights to a particular biomarker.

In a new Perspective piece published today in the New England Journal of Medicine, Jason Karlawish, MD, professor of Medicine, Medical Ethics and Health Policy in the Perelman School of Medicine at the University of Pennsylvania, and co-author Aaron S. Kesselheim, MD, JD, MPH, from Brigham and Women's Hospital and Harvard Medical School, delve into a series of high profile court cases testing the limits of patent protection.

In the months since a US Supreme Court ruling unanimously "rendered invalid two patents covering a method for determining proper drug dosage," as Nature reports, discussions have swirled about how to pay for personalized medicine. The NEJM co-authors report that "a patentable process now needs to involve an inventive and novel application of a law of nature beyond well-understood, routine, conventional activity, previously engaged in by those in the field."

Without patents protecting such medical discoveries, some have argued that there is no way to recoup the costs of biomarker innovation. To that end, Supreme Court Justice Breyer suggested whether special market-exclusivity protection was warranted.

Instead, the authors suggest that enhanced public funding, public-private partnerships, and open-source consortia may improve biomarker discovery and development, more than a private model. According to the NEJM piece, "the Supreme Court's move to free the fundamental processes of medical diagnosis from private ownershipcould ultimately enhance the public health."

As biomarkers become more and more prevalent -- helping diagnose diseases, and pairing with treatments targeted to individual needs -- there will need to be solutions to balance the needs of ensuring access to this useful information and paying for personalized medicine.

###

Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $4.3 billion enterprise.

Originally posted here:
Who pays for personalized medicine?

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Gary Mans gary.mans@louisville.edu 502-852-7504 University of Louisville

LOUISVILLE, Ky. The University of Louisville has received $6.3 million from the Leona M. and Harry B. Helmsley Charitable Trust to support research in the Department of Neurological Surgery and the Kentucky Spinal Cord Injury Research Center at UofL developing the next generation of technology to help paralyzed people regain movement in their limbs and enhance their quality of life.

In May 2011, Susan Harkema, Ph.D., professor of neurological surgery, and Jonathan Hodes, M.D., chair of neurological surgery, and their colleagues published a study in "The Lancet" demonstrating that the use of continual direct electrical stimulation of a patient's lower spinal cord using "off the shelf" technology designed for pain relief can allow a person to go from being wheelchair-bound to being able to stand, remain standing and bear weight. Researchers at the University of California, Los Angeles (UCLA) and California Institute of Technology (Cal Tech) collaborated on the study.

Since that time, the team has replicated its findings in at least two more patients, and the Helmsley Charitable Trust grant will help the team develop the technology needed to advance the research. Researchers from UofL, UCLA, Cal Tech and Case Western Reserve are involved in the development of the new technology.

"One of the biggest issues we face is the limitations imposed by the technology," Harkema said. "We need to develop the next generation of electrical stimulator containing the best possible circuitry and a new control system so that patients can have the ability to take advantage of this therapy at home and in their communities. Currently, it is limited to use in the laboratory here in Louisville."

"This most generous grant from the Helmsley Charitable Trust enables our researchers within the Department of Neurological Surgery and the Speed School of Engineering, and their colleagues at prestigious institutions across the country to move forward with the goal of improving the lives of millions of people," said UofL President James R. Ramsey. "This grant recognizes the innovative work taking place at UofL. Through this support and the work of our researchers and their subjects, we will continue to translate science into applications that transform people's lives."

"We're excited that this work has already proven what many thought was impossible: patients with absolutely no motor function can stand and step with assistance," said John Codey, trustee of the Helmsley Trust. "We hope the innovative work conducted by the faculty and staff of the University of Louisville and its partners continues to advance the technology and research base needed to treat more patients, resulting in improved outcomes."

In the paper from May, Harkema and her colleagues demonstrated that continual direct epidural electrical stimulation of the subject's lower spinal cord mimics signals the brain normally transmits to initiate movement. Once that signal is given, the research shows, the spinal cord's own neural network combined with the sensory input derived from the legs to the spinal cord is able to direct the muscle and joint movements required to stand and step with assistance on a treadmill.

The other crucial component of the potential therapy is an extensive regime of intensive physical therapy training called Locomotor Training while the spinal cord is being stimulated and the subject suspended over the treadmill. Assisted by rehabilitation specialists, an individual's spinal cord neural network is retrained to produce the muscle movements necessary to stand and to take assisted steps.

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Helmsley Charitable Trust grants $6.3 million to University of Louisville for neurosurgery

CLEARWATER, FL--(Marketwire -05/24/12)- Biostem U.S., Corporation, (HAIR.PK) (HAIR.PK) (Biostem, the Company), a fully reporting public company in the stem cell regenerative medicine sciences sector, announces a $5,000,000 financing agreement through private placement of stock.

CEO, Dwight Brunoehler, announced today that the company has signed an agreement with a funder to issue 20,000,000 shares of the company's common stock in exchange for $5,000,000 in cash or 25 cents ($.25) per share. No other considerations will be granted to the funder in exchange for the cash payment.

In announcing the funding agreement, Mr. Brunoehler commented, "We consider the eagerness of the funder to acquire Biostem shares at a price above the current market to be a tribute to our proven proprietary technology to enhance hair re-growth using human stem cells. Although we anticipated funding the company through the sale of a convertible debenture, the funder insisted on being able to acquire stock at a set price now, rather than risk having to convert at higher prices later. Although Rule 144 sale restrictions usually cause private placements of stock to be executed at a discount to the market, Biostem feels that its current share price is not truly reflective of the value of its proprietary technology; as well as the fact that the technology is already being employed, and the overall size of the hair replacement marketplace. It was for this reason that the company and the funder were able to come to an agreement to price the private placement above the current share price."

About Biostem U.S., Corporation

Biostem U.S., Corporation is a fully reporting Nevada corporation with offices in Clearwater, Florida. Biostem is a technology licensing company with proprietary technology centered on providing hair re-growth using human stem cells. The company also intends to train and license selected physicians to provide Regenerative Cellular Therapy treatments to assist the body's natural approach to healing tendons, ligaments, joints and muscle injuries by using the patient's own stem cells. Biostem U.S. is seeking to expand its operations worldwide through licensing of its proprietary technology and acquisition of existing stem cell related facilities. The company's goal is to operate in the international biotech market, focusing on the rapidly growing regenerative medicine field, using ethically sourced adult stem cells to improve the quality and longevity of life for all mankind.

More information on Biostem U.S., Corporation can be obtained through http://www.biostemus.com, or by calling Fox Communications Group 310-974-6821.

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Biostem U.S., Corporation Announces $5,000,000 Financing Agreement Through Private Placement of Stock

ROCKVILLE, Md., May 21, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that Richard Garr, CEO and President, will present at the World Stem Cells & Regenerative Medicine Congress in London (http://www.terrapinn.com/2012/stemcells/index.stm) on Tuesday, May 22nd at 12:30 PM. Mr. Garr's presentation, "Stem Cell Applications for Neurodegenerative Disorders," will review Neuralstem's cellular therapy trial in ALS, its neurogenic small molecule trial in major depressive disorder (MDD), and provide an overview on plans to expand the cellular therapy program.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

About Neuralstem

Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem is in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its cell therapy platform, including spinal cord injury, ischemic spastic paraplegia and chronic stroke. The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in chronic spinal cord injury.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company has received approval from the FDA to conduct a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include CTE (chronic traumatic encephalopathy), Alzheimer's disease, anxiety, and memory disorders.

For more information, please visit http://www.neuralstem.com or connect with us on Twitter and Facebook.

Cautionary Statement Regarding Forward Looking Information

This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10-K for the year ended December 31, 2011 and the Form 10-Q for the period ended March 30, 2012.

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Neuralstem CEO to Present at the World Stem Cells and Regenerative Medicine Congress in London

Tim and Padma Vellaichamy of Parramatta have had their new born child's umbilical cord stored cryogenically for future treatment. Pictured with their as yet unnamed three week old daughter. Picture: Adam Ward Source: The Daily Telegraph

IT'S current preservation for future regeneration - and now umbilical cord tissue is going on ice in Australia for the first time.

Usually discarded after birth, umbilical tissue from newborn babies is being collected and cryogenically frozen to be used one day for regenerative and stem cell medicine. And it doesn't just have potential for the babies involved, either. Experts say stem cells could also be used for family members who are genetically compatible.

It is hoped the cells will eventually be able to be used to repair damaged tissues and organs, with researchers investigating its uses for treating diseases like multiple sclerosis, cerebral palsy and diabetes, as well as for bone and cartilage repair.

Although cord blood storage has been available for many years, Cell Care Australia has added cord tissue storage in anticipation of new discoveries in the regenerative medicine field.

Cell Care Australia medical director associate professor Mark Kirkland said the storage process - already popular in the US, Europe and Southeast Asia - was long overdue for Australian shores.

"The science is developing around the world and we're really behind the rest of the world in providing parents the option to store these cells and we thought it was about time it was brought here," he said.

"It's finding a way to take what would otherwise be waste tissue and turning it into something of potential future value for not only your child but also potentially for other family members.'

Parramatta couple Tim and Padma Vellaichamy are among the first to use the service in Australia.

Mr Vellaichamy, 31, said he heard of the technology while working as a dentist in India and decided to store their daughter's cord cell tissue after birth three weeks ago.

Read the rest here:
Frozen cord could save a life

CLEARWATER, FL--(Marketwire -05/21/12)- Biostem U.S., Corporation, (HAIR.PK) (HAIR.PK) (Biostem, the Company), a fully reporting public company in the stem cell regenerative medicine sciences sector, today announced that Jeanne Ann Lumadue, MD, PhD, MBA, has been appointed to its Scientific and Medical Board of Advisors (SAMBA).

Dr. Lumadue currently is Medical Director at the Mount Nittany Physician Group Laboratory in State College, PA. She also serves as Medical Director of the Central Pennsylvania Blood Bank and is a member of the medical staff of the Mount Nittany Medical Center, all in State College.

Dr. Lumadue stated, "Biostem's international technology development and licensing approach is well planned. Stem cell regenerative medicine is a rapidly expanding field that has the potential to affect every human being in a positive way. I am delighted to be part of this highly promising company."

Biostem CEO Dwight Brunoehler said, "I am thrilled for the opportunity to work with Jeanne again. She is an innovative thinker, a tireless contributor, and a great team player."

Dr. Lumadue received her undergraduate degree magna cum laude from the Pennsylvania State University and her PhD in Genetics from Yale University. She received an MD degree from the Johns Hopkins University in Baltimore, MD, where she also did residency and fellowship training in anatomic and clinical pathology. She has served as Pathologist and Assistant Medical Director of Transfusion Medicine at the Johns Hopkins Hospital, the Medical Director of Laboratory Hematology and Stem Cell Processing at Children's National Medical Center in Washington, DC, and the Medical Director of Transfusion Services and Stem Cell Processing at the Inova Fairfax Hospital in Falls Church, Virginia.

She is a member of the American Society of Hematology, the College of American Pathologists, the American Society of Clinical Pathologists and the AABB, for which she serves as a facility assessor.

About Biostem U.S., Corporation

Biostem U.S., Corporation is a fully reporting Nevada corporation with offices in Clearwater, Florida. Biostem is a technology licensing company with proprietary technology centered on providing hair re-growth using human stem cells. The company also intends to train and license selected physicians to provide Regenerative Cellular Therapy treatments to assist the body's natural approach to healing tendons, ligaments, joints and muscle injuries by using the patient's own stem cells. Biostem U.S. is seeking to expand its operations worldwide through licensing of its proprietary technology and acquisition of existing stem cell related facilities. The company's goal is to operate in the international biotech market, focusing on the rapidly growing regenerative medicine field, using ethically sourced adult stem cells to improve the quality and longevity of life for all mankind.

More information on Biostem U.S., Corporation can be obtained through http://www.biostemus.com, or by calling Fox Communications Group 310-974-6821.

Excerpt from:
Biostem U.S., Corporation Adds Jeanne Ann Lumadue, MD, PhD, MBA to Its Scientific and Medical Board of Advisors

Tim and Padma Vellaichamy of Parramatta have had their new born child's umbilical cord stored cryogenically for future treatment. Pictured with their as yet unnamed three week old daughter. Picture: Adam Ward Source: The Daily Telegraph

IT'S current preservation for the future regeneration - and now umbilical cord tissue is going on ice in Australia for the first time.

Usually discarded after birth, umbilical tissue from newborn babies is being collected and cryogenically frozen to be used one day for regenerative and stem cell medicine. And it doesn't just have potential for the babies involved, either. Experts say stem cells could also be used for family members who are genetically compatible.

It is hoped the cells will eventually be able to be used to repair damaged tissues and organs, with researchers investigating its uses for treating diseases like multiple sclerosis, cerebral palsy and diabetes, as well as for bone and cartilage repair.

Although cord blood storage has been available for many years, Cell Care Australia has added cord tissue storage in anticipation of new discoveries in the regenerative medicine field.

Cell Care Australia medical director associate professor Mark Kirkland said the storage process - already popular in the US, Europe and Southeast Asia - was long overdue for Australian shores.

"The science is developing around the world and we're really behind the rest of the world in providing parents the option to store these cells and we thought it was about time it was brought here," he said.

"It's finding a way to take what would otherwise be waste tissue and turning it into something of potential future value for not only your child but also potentially for other family members.'

Parramatta couple Tim and Padma Vellaichamy are among the first to use the service in Australia.

Mr Vellaichamy, 31, said he heard of the technology while working as a dentist in India and decided to store their daughter's cord cell tissue after birth three weeks ago.

Originally posted here:
Stem cell medicine thrown umbilical rope

Public release date: 20-May-2012 [ | E-mail | Share ]

Contact: Jessica Studeny jessica.studeny@case.edu 216-368-4692 Case Western Reserve University

A substance in human mesenchymal stem cells that promotes growth appears to spur restoration of nerves and their function in rodent models of multiple sclerosis (MS), researchers at Case Western Reserve University School of Medicine have found.

Their study is embargoed until published in the online version of Nature Neuroscience at 1 p.m. U.S. Eastern Standard Time on Sunday, May 20.

In animals injected with hepatocyte growth factor, inflammation declined and neural cells grew. Perhaps most important, the myelin sheath, which protects nerves and their ability to gather and send information, regrew, covering lesions caused by the disease.

"The importance of this work is we think we've identified the driver of the recovery," said Robert H. Miller, professor of neurosciences at the School of Medicine and vice president for research at Case Western Reserve University.

Miller, neurosciences instructor Lianhua Bai and biology professor Arnold I. Caplan, designed the study. They worked with Project Manager Anne DeChant, and research assistants Jordan Hecker, Janet Kranso and Anita Zaremba, from the School of Medicine; and Donald P. Lennon, a research assistant from the university's Skeletal Research Center.

In MS, the immune system attacks myelin, risking injury to exposed nerves' intricate wiring. When damaged, nerve signals can be interrupted, causing loss of balance and coordination, cognitive ability and other functions. Over time, intermittent losses may become permanent.

Miller and Caplan reported in 2009 that when they injected human mesenchymal stem cells into rodent models of MS, the animals recovered from the damage wrought by the disease. Based on their work, a clinical trial is underway in which MS patients are injected with their own stem cells.

In this study, the researchers first wanted to test whether the presence of stem cells or something cells produce promotes recovery. They injected mice with the medium in which mesenchymal stem cells, culled from bone marrow, grew.

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Growth factor in stem cells may spur recovery from MS

SARATOGA, Calif.--(BUSINESS WIRE)--

The Myelin Repair Foundation (MRF) today announced the results of a new peer-reviewed research study published in Nature Neuroscience that demonstrates functional improvement in immune response modulation and myelin repair with factors derived from mesenchymal stem cell (MSC) treatment in animal models of multiple sclerosis (MS). Funded by the Myelin Repair Foundation, this research conducted by Case Western Reserve University scientists showed positive results with human mesenchymal stem cells in animal models of MS by not only successfully blocking the autoimmune MS response, but also repairing myelin, demonstrating an innovative potential myelin repair treatment for MS.

Multiple sclerosis is a disease of the immune system that attacks the myelin, causing exposed nerves or lesions which block brain signals, causing loss of motor skills, coordination and cognitive ability. Compared to the controls, this research study showed fewer and smaller lesions found on the nerves in the MSC treatment group. MSCs were found to block the formation of scar tissue by suppressing the autoimmune response, which would otherwise cause permanent damage to the nerves. Furthermore, the research showed that MSC treatment also repaired myelin, enhancing myelin regeneration of the damaged axon and the rewrapping of the myelin around the axon in animal models of MS. One treatment of MSCs provided long-term protection of the recurring disease.

Led by Myelin Repair Foundation Principal Investigator and Vice President for Research & Technology Management at Case Western Reserve Universitys Dr. Robert Miller, this study documents a new promising pathway for treating multiple sclerosis that blocks the autoimmune response and reverses the myelin damage in animal models of MS. The human MSCs used in this study were culled from adult stem cells derived from the bone marrow.

We are thrilled with the publication of this important research study that examines a new pathway to treat multiple sclerosis, one that reverses the damage of the disease, said Dr. Robert Miller. Since we were just beginning to understand how MSCs provide myelin repair for lesions, with the Myelin Repair Foundations support, we continue to deepen our knowledge of exploring the next generation of MS treatments that stimulate healing, rather than symptom suppression of the disease.

We pride ourselves on supporting best-in-class scientists devoted to find new ways to treat multiple sclerosis, advancing highly innovative research projects that otherwise would not have moved forward, said Scott Johnson, president of the Myelin Repair Foundation. The success of Case Western Reserve Universitys study and recognition in this prestigious journal furthers our goal to identify new pathways to treat multiple sclerosis by supporting a multi-disciplinary team of the best researchers in the field.

About the Myelin Repair Foundation

The Myelin Repair Foundation (MRF) (http://www.myelinrepair.org) is a Silicon Valley-based, non-profit research organization focused on accelerating the discovery and development of myelin repair therapeutics for multiple sclerosis. Its Accelerated Research Collaboration (ARC) model is designed to optimize the entire process of medical research, drug development and the delivery of patient treatments.

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Nature Neuroscience Study Shows Unique Scientific Support for Potential New Myelin Repair Treatment for Multiple ...