June 29, 2012

Connie K. Ho for redOrbit.com Your Universe Online

In 1993, the autosomal dominant gene mutation responsible for Huntingtons Disease (HD) was discovered. However, no treatments are known to slow its progression. New research may pave the way to better understanding of the disease. Researchers at Johns Hopkins recently announced that they were able to produce stem cells from skin cells from a person who had severe, early-onset form of HD; the cells were then changed into neurons that degenerated like the cells affected by HD.

The research was recently published in the journal Cell Stem Cell. The investigators worked with an international consortium in creating HD in a dish. The group was made up of scientists from Johns Hopkins University School of Medicine, Cedars-Sinai Medical Center, the University of California at Irvine, as well as six other groups. The team looked at many other HD cell lines and control cell lines to verify that the results were consistent and reproducible in other labs. The investigators believe that the findings allow them to better understand and eliminate cells in people in with HD. They hope to study the effects of possible drug treatments on cells that would be otherwise found deep in the brain.

Having these cells will allow us to screen for therapeutics in a way we havent been able to before in Huntingtons disease, remarked lead researcher Dr. Christopher A. Ross, a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine, in a prepared statement. For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic.

The team of researchers is studying small molecules for the ability to block HD iPSC degeneration to see if they can be developed into new drugs for HD. As well, the ability to produce from stem cells the same neurons found in HD may have effects for similar research in other neurodegenerative diseases like Alzheimers and Parkinsons. In the experiment, Ross took a skin biopsy from a patient with very early onset HD. The patient was seven years old at the time, with a severe form of disease and a mutation that caused it. By using cells from a patient who had quickly progressing HD, Ross team were able to mimic HD in a way that could be used by patients who had different forms of HD.

The skin cells were grown in culture and reprogrammed to induce stem cells that were pluripotent. Then, another cell line was created in the same way from someone who didnt have HD. The other HD and control iPS cells were produced as part of the NINDS funded HD iPS cell consortium. Investigators from Johns Hopkins and the other consortium labs changed the cells into typical neurons and then into medium spiny neurons. The process took a total of three months and the scientists found the medium spiny neurons from the HD cells acted how the medium spiny neurons form an HD patient would. The cells demonstrated quick degeneration when cultured in the lab with a basic culture medium that didnt include extensive supporting nutrients. On the other hand, control cell lines didnt demonstrate neuronal degeneration.

These HD cells acted just as we were hoping, says Ross, director of the Baltimore Huntingtons Disease Center. A lot of people said, Youll never be able to get a model in a dish of a human neurodegenerative disease like this. Now, we have them where we can really study and manipulate them, and try to cure them of this horrible disease. The fact that we are able to do this at all still amazes us.

Source: Connie K. Ho for redOrbit.com Your Universe Online

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Skin Cells Create Stem Cells In Huntington Disease Study

28-06-2012 13:54 About 5 months ago, she came home from the beach with my husband limping on her right back leg. Now 8 weeks later after stem cell therapy... we were happy (well, maybe not so much...) to see her back to her old, wild, hyper self again.

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Ipo 8 weeks after stem cell therapy at Surf Paws Animal Hospital - Video

The laboratory grew and stored human stem cells, which are capable of becoming any cell in the body, and made them available to scientists nationwide for use in experiments to study diseases such as diabetes and spinal cord injuries. When it is dismantled, several thousand vials of stem cellswill be sent back to the research centers where they originated, and the equipment will be given to other UMass labs.

Susan Windham-Bannister, president of the Massachusetts Life Sciences Center, a quasi-public agency that oversees the $1 billion life sciences initiative, defended the decision to initially fund the stem cell bank. She said there are many examples of technology that in hindsight are unnecessary, but at the time it was conceived, when the investment was made, it was absolutely state of the art. The center, she said, was one of them.

Originally, the bank was seen as a repository for embryonic stem cell lines that were being created but were not eligible for federal funding under Bush-era restrictions. The field has evolved significantly since then, with President Obamas loosening of restrictions on federal funding and the development of new technologies for making stem cells.

Still, stem cell banks are seen as useful by some. The California Institute for Regenerative Medicine, for example, is preparing to invest $10 million in its own stem cell banking initiative, and another $20 million to underwrite the creation of stem cells from patients with specific diseases.

Massachusetts Senate minority leader Bruce Tarr, Republican of Gloucester, said he was concerned that lawmakers had not been told the bank would close.

Given the fact that this is a resource that was created by an act of the Legislature, I would hope anyone seeking to change its status would consult with the Legislature, he said. The notion has always been we have been working hard to make Massachusetts a leader in stem cell research, and I dont know how ceasing the operations of the stem cell bank advances that goal.

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UMass stem cell lab to close

28.06.2012 - (idw) Ruhr-Universitt Bochum

RUB biologists have deliberately transformed stem cells from the spinal cord of mice into immature nerve cells. This was achieved by changing the cellular environment, known as the extracellular matrix, using the substance sodium chlorate. Via sugar side chains, the extracellular matrix determines which cell type a stem cell can generate. Influencing precursor cells pharmacologically so that they transform into a particular type of cell can help in cell replacement therapies in future says Prof. Dr. Stefan Wiese, head of the Molecular Cell Biology work group. Matrix modified Taking the fate of stem cells in hand RUB researchers generate immature nerve cells

RUB biologists have deliberately transformed stem cells from the spinal cord of mice into immature nerve cells. This was achieved by changing the cellular environment, known as the extracellular matrix, using the substance sodium chlorate. Via sugar side chains, the extracellular matrix determines which cell type a stem cell can generate. Influencing precursor cells pharmacologically so that they transform into a particular type of cell can help in cell replacement therapies in future says Prof. Dr. Stefan Wiese, head of the Molecular Cell Biology work group. Therapies, for example, for Parkinsons, multiple sclerosis or amyotrophic lateral sclerosis could then become more efficient. The team describes its findings in Neural Development.

Sulphate determines the fate of stem cells

Sodium chlorate acts on metabolism enzymes in the cell which attach sulphate groups to proteins. If these sulphates are not installed, the cell continues to form proteins for the extracellular matrix, but with modified sugar side chains. These chains in turn send out signals that define the fate of the stem cells. Stem cells can not only develop into nerve cells, but also form astrocytes or oligodendrocytes, which are, for instance, responsible for the mineral balance of the nerve cells or which form their insulation layer. What happens to the stem cells if the sulphate pattern is changed by sodium chlorate was examined by Dr. Michael Karus and his colleagues.

The RUB-laboratories of Prof. Dr. Stefan Wiese, Prof. Dr. Andreas Faissner and Prof. Dr. Irmgard Dietzel-Meyer collaborated for the study. Using antibodies, the researchers showed that cells which they had treated with sodium chlorate developed into nerve cells. They also analysed the flow of sodium ions into the cells. The result: treated cells showed a lower sodium current than mature nerve cells. Sodium chlorate thus favours the development of stem cells into nerve cells, but, at the same time, also inhibits the maturation - a positive side effect, as Wiese explains: If sodium chlorate stops the nerve cells in an early developmental phase, this could enable them to integrate into the nervous system following a transplant better than mature nerve cells would do.

Bibliographic record

M. Karus, S. Samtleben, C. Busse, T. Tsai, I.D. Dietzel, A. Faissner, S. Wiese (2012): Normal sulphation levels regulate spinal cord neural precursor cell proliferation and differentiation, Neural Development, doi:10.1186/1749-8104-7-20

Further information

Prof. Dr. Stefan Wiese, Molecular Cell Biology Work Group, Faculty of Biology and Biotechnology at the Ruhr-Universitt, 44780 Bochum, Germany, Tel. +49/234/32-22041 stefan.wiese@rub.de

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Taking the fate of stem cells in hand: RUB researchers generate immature nerve cells

By Daily Mail Reporter

PUBLISHED: 07:58 EST, 28 June 2012 | UPDATED: 08:44 EST, 28 June 2012

Robin Roberts' older sister has spoken out about being her sole bone marrow donor after learning she was a match.

Sally-Ann, who anchors a morning show in New Orleans, will be essential to her GMA host sister's treatments for myelodysplastic syndrome (MDS), a blood and bone marrow disease.

The mother-of-three, 55, told the New York Post yesterday how she had been so desperate to be a match for her sister, she and her friends made a prayer circle around the test kit.

'We prayed, "please let this be a match,"' she admitted.

Perfect match: Sally-Ann Roberts, pictured with Robin earlier this month, has spoken about how she learned she would be her sister's sole bone marrow donor

She admitted: 'Im the big sister. Im the one whos supposed to be suffering because of age. But thats not the way it is.'

To donate her bone marrow, Sally-Ann explained that she will have five days of injections to boost her blood cell count, before her blood is passed through a machine that will extract the stem cells her sister, 51, so desperately needs.

'The way it is explained to me is that they will first have to knock out her immune system in order for my stem cells to be accepted by her body,' she said.

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'We prayed that I'd be a match': Robin Roberts' sister Sally-Ann on learning she was the sole bone marrow donor

An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine and the UCSF Gladstone Institutes, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients.

The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

UCI scientists were part of a consortium that in 1993 identified the autosomal dominant gene mutation responsible for HD, but there is still no cure, and no treatments are available to even slow its onset or progression. The research, published online today in the journal Cell Stem Cell, is the work of the Huntington's Disease iPSC Consortium. Participants examined several other cell lines and control cell lines to ensure that their results were consistent and reproducible in different labs.

"Our discovery will enable us for the first time to test therapies on human Huntington's disease neurons," said Leslie Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior, one of the world's leading HD experts and a senior author of the study. "This has been a remarkable time in HD research, with the advent of stem cell technologies that have allowed these scientific advancements. Also, having a team of scientists working together as a consortium has benefited the research tremendously and accelerated its pace."

Huntington's is such a rare disease, although it is the most common inherited neurodegenerative disorder. It afflicts approximately 30,000 people in the United States-with another 75,000 people carrying the gene that will eventually lead to it.

"An advantage of this human model is that we now have the ability to identify changes in brain cells over time-during the degeneration process and at specific stages of brain-cell development," said Gladstone Senior Investigator Dr. Steve Finkbeiner. "We hope this model will help us more readily uncover relevant factors that contribute to Huntington's disease and especially to find successful therapeutic approaches."

UC Irvine press release

Gladstone Institutes press release

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Huntington’s disease neurons created from stem cells

Newswise Researchers at the Buck Institute have corrected the genetic mutation responsible for Huntingtons Disease (HD) using a human induced pluripotent stem cell (iPSC) that came from a patient suffering from the incurable, inherited neurodegenerative disorder. Scientists took the diseased iPSCs, made the genetic correction, generated neural stem cells and then transplanted the mutation-free cells into a mouse model of HD where they are generating normal neurons in the area of the brain affected by HD. Results of the research are published in the June 28, 2012 online edition of the journal Cell Stem Cell.

iPSCs are reverse-engineered from human cells such as skin, back to a state where they can be coaxed into becoming any type of cell. They can be used to model numerous human diseases and may also serve as sources of transplantable cells that can be used in novel cell therapies. In the latter case, the patient provides a sample of his or her own skin to the laboratory. We believe the ability to make patient-specific, genetically corrected iPSCs from HD patients is a critical step for the eventual use of these cells in cell replacement therapy, said Buck faculty Lisa Ellerby, PhD, lead author of the study. The genetic correction reversed the signs of disease in these cells the neural stem cells were no longer susceptible to cell death and the function of their mitochondria was normal. Ellerby said the corrected cells could populate the area of the mouse brain affected in HD, therefore, the next stage of research involves transplantation of corrected cells to see if the HD-afflicted mice show improved function. Ellerby said these studies are important as now we can deliver patient-specific cells for cell therapy, that no longer have the disease causing mutation.

Huntington's disease (HD) is a devastating, neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems. It typically becomes noticeable in mid-adult life, with symptoms beginning between 35 and 44 years of age. Life expectancy following onset of visual symptoms is about 20 years. The worldwide prevalence of HD is 5-10 cases per 100,000 persons. More than a quarter of a million Americans have HD or are "at risk" of inheriting the disease from an affected parent. Key to the disease process is the formation of specific protein aggregates (essentially abnormal clumps) inside some neurons.

All humans have two copies of the Huntingtin gene (HTT), which codes for the protein Huntingtin (Htt). Part of this gene is a repeated section called a trinucleotide repeat, which varies in length between individuals and may change between generations. When the length of this repeated section reaches a certain threshold, it produces an altered form of the protein, called mutant Huntingtin protein (mHtt). Scientists in the Ellerby lab corrected the mutation by replacing the expanded trinucleotide repeat with a normal repeat using homologous recombination. Homologous recombination is a type of genetic recombination where two molecules of DNA are exchanged. In this case the diseased DNA sequence is exchanged for the normal DNA sequence.

Contributors to the work: Mahru An and Ningzhe Zhang are shared first authors of this study. Other Buck Institute researchers involved in the study include Gary Scott, Daniel Montoro, Tobias Wittkop, and faculty members Sean Mooney and Simon Melov. The work was funded by the Buck Institute and the National Institutes of Health.

About the Buck Institute for Research on Aging The Buck Institute is the U.S.s first and foremost independent research organization devoted to Geroscience focused on the connection between normal aging and chronic disease. Based in Novato, CA, The Buck is dedicated to extending Healthspan, the healthy years of human life and does so utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and those focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimers and Parkinsons, cancer, cardiovascular disease, macular degeneration, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics and bioinformatics. For more information: http://www.thebuck.org.

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Scientists Correct Huntington's Mutation in Induced Pluripotent Stem Cells

Main Category: Huntingtons Disease Also Included In: Stem Cell Research Article Date: 28 Jun 2012 - 9:00 PDT

Current ratings for: Huntington's Research Tool Developed Using Stem Cells

Cedars-Sinai scientists have joined with expert colleagues around the globe in using stem cells to develop a laboratory model for Huntington's disease, allowing researchers for the first time to test directly on human cells potential treatments for this fatal, inherited disorder.

As explained in a paper published June 28 on the Cell Stem Cell website and scheduled for print in the journal's Aug. 3 issue, scientists at Cedars-Sinai's Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntington's disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntington's.

"This Huntington's 'disease in a dish' will enable us for the first time to test therapies on human Huntington's disease neurons," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. "In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. It's a new way of doing trailblazing science."

The Huntington's Disease iPSC Consortium united some of the world's top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntington's patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntington's researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntington's, known to the public, for example, as the cause of folksinger Woody Guthrie's death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntington's results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntington's, nor therapies to slow its progression.

The consortium showed Huntington's cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or "stressing" them, and found that Huntington's neurons died even faster.

"It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques," said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study. "It was very reassuring and significantly strengthens the value of this study."

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Huntington's Research Tool Developed Using Stem Cells

Public release date: 28-Jun-2012 [ | E-mail | Share ]

Contact: Tom Vasich tmvasich@uci.edu 949-824-6455 University of California - Irvine

Irvine, Calif., June 28, 2012 An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients.

The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

UCI scientists were part of a consortium that in 1993 identified the autosomal dominant gene mutation responsible for HD, but there is still no cure, and no treatments are available to even slow its onset or progression. The research, published online today in the journal Cell Stem Cell, is the work of the Huntington's Disease iPSC Consortium. Participants examined several other cell lines and control cell lines to ensure that their results were consistent and reproducible in different labs.

"Our discovery will enable us for the first time to test therapies on human Huntington's disease neurons," said Leslie Thompson, UCI professor of psychiatry & human behavior and neurobiology & behavior, one of the world's leading HD experts and a senior author of the study. "This has been a remarkable time in HD research, with the advent of stem cell technologies that have allowed these scientific advancements. Also, having a team of scientists working together as a consortium has benefited the research tremendously and accelerated its pace."

Leslie Lock, a UCI assistant professor of developmental & cell biology and biological chemistry whose lab helped develop the induced pluripotent stem cells (iPSC), added: "It's exciting to be carrying out work that provides hope for HD patients and their families."

Thompson said that UCI scientists will use the new model to study the specific gene expression changes in human brain cells that trigger the onset of HD, helping them understand how these changes happen and how to correct them.

Huntington's disease afflicts about 30,000 people in the U.S. typically striking in midlife and another 75,000 carry the gene that will eventually lead to it. Caused by a mutation in the gene for a protein called huntingtin, the disease damages brain cells so that individuals with HD progressively lose their ability to walk, talk and reason. It invariably culminates in death. While rare, HD is the most common inherited neurodegenerative disease.

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Human model of Huntington's disease created from skin's stem cells

Newswise LOS ANGELES (EMBARGOED UNTIL NOON EDT ON JUNE 28, 2012) Cedars-Sinai scientists have joined with expert colleagues around the globe in using stem cells to develop a laboratory model for Huntingtons disease, allowing researchers for the first time to test directly on human cells potential treatments for this fatal, inherited disorder.

As explained in a paper published June 28 on the Cell Stem Cell website and scheduled for print in the journals Aug. 3 issue, scientists at Cedars-Sinais Regenerative Medicine Institute and the University of Wisconsin took skin cells from patients with Huntingtons disease and reprogrammed them into powerful stem cells; these were then made into the nervous system cells affected by the disease. Seven laboratories around the world collaborated to demonstrate the cells had hallmarks of Huntingtons.

This Huntingtons disease in a dish will enable us for the first time to test therapies on human Huntingtons disease neurons, said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a senior author of the study. In addition to increasing our understanding of this disorder and offering a new pathway to identifying treatments, this study is remarkable because of the extensive interactions between a large group of scientists focused on developing this model. Its a new way of doing trailblazing science.

The Huntingtons Disease iPSC Consortium united some of the worlds top scientists working on this disease. Cedars-Sinai researchers took skin cells from a several Huntingtons patients, including a six-year-old with a severe juvenile form of the disease. They genetically reprogrammed these tissues into induced pluripotent stem cells, which can be made into any type of cell in the body. The cells lines were banked by scientists at Cedars-Sinai and scrutinized by all consortium members for differences that may have led to the disease. These cell lines are now an important resource for Huntingtons researchers and have been made available via a National Institutes of Health-funded repository at Coriell Institute for Medical Research in New Jersey.

Huntingtons, known to the public, for example, as the cause of folksinger Woody Guthries death, typically strikes patients in midlife. It causes jerky, twitching motions, loss of muscle control, psychiatric disorders and dementia; the disease ultimately is fatal. In rare, severe cases, the disorder appears in childhood.

Researchers believe that Huntingtons results from a mutation in the huntintin gene, leading to production of an abnormal protein and ultimately cell death in specific areas of the brain that control movement and cognition. There is no cure for Huntingtons, nor therapies to slow its progression.

The consortium showed Huntingtons cell deficits or how they differ from normal cells, including that they were less likely to survive cultivation in the petri dish. Scientists tried depriving them of a growth factor present around normal cells, or stressing them, and found that Huntingtons neurons died even faster.

It was great that these characteristics were seen not only in our laboratory, but by all of the consortium members using different techniques, said Virginia Mattis, a post-doctoral scientist at the Cedars-Sinai Regenerative Medicine Institute and one of the lead authors of the study. It was very reassuring and significantly strengthens the value of this study.

This new model will provide the foundation for a new round of experiments by the consortium funded by a new grant from the NIH and the California Institute for Regenerative Medicine.

The Cedars-Sinais Regenerative Medicine Institute has made a major commitment to projects like this Huntingtons study in which stem cell research helps to advance understanding of human disease and open new and innovative methods to identify treatments and cures. The institute has developed an induced pluripotent stem cell core facility and recruited faculty to work in this emerging area of regenerative medicine research.

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Cedars-Sinai Researchers, with Stem Cells and Global Colleagues, Develop Huntington's Research Tool

Public release date: 28-Jun-2012 [ | E-mail | Share ]

Contact: Stephanie Desmon sdesmon1@jhmi.edu 410-955-8665 Johns Hopkins Medical Institutions

Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntington's disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.

By creating "HD in a dish," the researchers say they have taken a major step forward in efforts to better understand what disables and kills the cells in people with HD, and to test the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

Although the autosomal dominant gene mutation responsible for HD was identified in 1993, there is no cure. No treatments are available even to slow its progression.

The research, published in the journal Cell Stem Cell, is the work of a Huntington's Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups. The consortium studied several other HD cell lines and control cell lines in order to make sure results were consistent and reproducible in different labs.

The general midlife onset and progressive brain damage of HD are especially cruel, slowly causing jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and eventually death. In some cases (as in the patient who donated the material for the cells made at Johns Hopkins), the disease can strike earlier, even in childhood.

"Having these cells will allow us to screen for therapeutics in a way we haven't been able to before in Huntington's disease," says Christopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the study's lead researchers. "For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic."

Ross and his team, as well as other collaborators at Johns Hopkins and Emory University, are already testing small molecules for the ability to block HD iPSC degeneration. These small molecules have the potential to be developed into novel drugs for HD.

The ability to generate from stem cells the same neurons found in Huntington's disease may also have implications for similar research in other neurodegenerative diseases such as Alzheimer's and Parkinson's.

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Turning skin cells into brain cells

ScienceDaily (June 28, 2012) Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntington's disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.

By creating "HD in a dish," the researchers say they have taken a major step forward in efforts to better understand what disables and kills the cells in people with HD, and to test the effects of potential drug therapies on cells that are otherwise locked deep in the brain.

Although the autosomal dominant gene mutation responsible for HD was identified in 1993, there is no cure. No treatments are available even to slow its progression.

The research, published in the journal Cell Stem Cell, is the work of a Huntington's Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups. The consortium studied several other HD cell lines and control cell lines in order to make sure results were consistent and reproducible in different labs.

The general midlife onset and progressive brain damage of HD are especially cruel, slowly causing jerky, twitch-like movements, lack of muscle control, psychiatric disorders and dementia, and -- eventually -- death. In some cases (as in the patient who donated the material for the cells made at Johns Hopkins), the disease can strike earlier, even in childhood.

"Having these cells will allow us to screen for therapeutics in a way we haven't been able to before in Huntington's disease," saysChristopher A. Ross, M.D., Ph.D., a professor of psychiatry and behavioral sciences, neurology, pharmacology and neuroscience at the Johns Hopkins University School of Medicine and one of the study's lead researchers. "For the first time, we will be able to study how drugs work on human HD neurons and hopefully take those findings directly to the clinic."

Ross and his team, as well as other collaborators at Johns Hopkins and Emory University, are already testing small molecules for the ability to block HD iPSC degeneration.These small molecules have the potential to be developed into novel drugs for HD.

The ability to generate from stem cells the same neurons found in Huntington's disease may also have implications for similar research in other neurodegenerative diseases such as Alzheimer's and Parkinson's.

To conduct their experiment, Ross took a skin biopsy from a patient with very early onset HD.When seen by Ross at the HD Center at Hopkins, the patient was just seven years old. She had a very severe form of the disease, which rarely appears in childhood, and of the mutation that causes it. Using cells from a patient with a more rapidly progressing form of the disease gave Ross' team the best tools with which to replicate HD in a way that is applicable to patients with all forms of HD.

Her skin cells were grown in culture and then reprogrammed by the lab of Hongjun Song, Ph.D., a professor at Johns Hopkins' Institute for Cell Engineering, into induced pluripotent stem cells. A second cell line was generated in an identical fashion in Dr. Ross's lab from someone without HD. Simultaneously, other HD and control iPS cell lines were generated as part of the NINDS funded HD iPS cell consortium.

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Turning skin cells into brain cells: Huntington's disease in a dish

Disease fight: turning skin cells to neurons June 28th, 2012, 4:04 pm posted by Pat Brennan, science, environment editor

UC Irvine professor Leslie Thompson, with human brain image behind her. Photo by Daniel A. Anderson, UC Irvine.

Using stem cells derived from skin cells, scientists including a UC Irvine team say they have created human neurons that exhibit the effects of Huntingtons disease promising the possibility of testing treatments for the deadly disorder in a petri dish.

Their discovery not only sidesteps ethical issues surrounding the use of human embryonic stem cells, but offers the chance to produce far more diseased neurons, at various stages of disease progression, than ever have been available to researchers before.

This is a relatively new technique where you can reprogram an adult cell, in this case a skin cell, back to this early stem-cell stage, and then guide those into making neurons, said Leslie Thompson, a UC Irvine professor and a senior author of a study announcing the discovery that was published online Thursday.

Huntingtons disease is an inherited, neurodegenerative disorder that is always fatal. It typically strikes in middle age, gradually robbing its victims of the ability to walk and interfering with other basic brain functions.

Huntington's disease cells on their way to becoming neurons. Image courtesy Leslie Thompson, UC Irvine.

Its like Parkinsons in that its a movement disorder in this case, involuntary movements, and rigidity, Thompson said. You know what is going on, but parts of memory are being impaired; you have an impaired ability to walk, think, talk.

Victims typically die of the diseases effects falling, or choking during pneumonia and some especially severe mutations can strike young children. The disease affects about 30,000 people in the United States, and no treatments exist even to slow the onset of symptoms.

The scientists, including UCIs Leslie Lock and Peter Donovan, director of the Sue and Bill Gross Stem Cell Research Center, as well as others from universities around the country and in Italy and Great Britain, used a variety of cell lines to reveal the genetic underpinnings of Huntingtons.

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Disease fight: turning skin cells to neurons

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Stem Cell Therapy - Healthier Looking Skin ,Promote Younger, Reduce Wrinkles - Video

SAN DIEGO CA--(Marketwire -06/29/12)- Medistem Inc. (MEDS) announced today notice of allowance from the United States Patent and Trademark Office (USPTO) for a patent covering the use of fat stem cells, and cells associated with fat stem cells for treatment of diseases related to a dysfunctional immune system. Such diseases include multiple sclerosis, Type 1 diabetes, rheumatoid arthritis and lupus. The allowed patent, entitled "Stem Cell Mediated Treg Activation/Expansion for Therapeutic Immune Modulation" has the earliest priority date of December 2006.

"We have previously published that giving multiple sclerosis patients cells extracted from their own fat tissue, which contains stem cells, appears to confer clinical benefit in a pilot study," said Thomas Ichim, CEO of Medistem. "The current patent that has been allowed, in the broadest interpretation of the claims, gives us exclusive rights to the use of specific types of fat stem cell therapy for autoimmune diseases such as multiple sclerosis."

Subsequent to the filing of the patent application, Medistem together with collaborators at the Lawson Health Sciences Research Institute, Canada, reported data that fat tissue contains high numbers of T regulatory cells, a type of immune cell that is capable of controlling autoimmunity.

This finding was independently confirmed by Dr. Diane Mathis' laboratory at Harvard University, who published a paper in the prestigious journal, Nature Medicine, in which detailed experimental evidence was provided supporting the initial finding that adipose tissue contains high numbers of T regulatory cells. A video describing the paper can be accessed at http://www.youtube.com/watch?v=rEJfGu29Rg8.

The current patent discloses the use of T regulatory cells from fat, combinations with stem cells, and use of fat-derived mononuclear cells. Given that there are currently several groups utilizing this technology in the USA in treating patients, Medistem believes revenue can be generated through enforcement of patent rights.

"Our corporate philosophy has been to remain highly focused on our ongoing clinical stage programs using Medistem's universal donor stem cell, the Endometrial Regenerative Cell (ERC), in the treatment of critical limb ischemia and congestive heart failure," said Dr. Vladimir Bogin, Chairman and President of Medistem. "However, due to the ease of implementation of our fat stem cell technology, combined with the major burden that autoimmune diseases have on our health care system, we are highly incentivized to explore partnering, co-development and licensing opportunities."

Autoimmune conditions occur as a result of the body's immune system "turning on itself" and attacking its own organs or cells. Current treatments for autoimmune conditions are based on "globally" suppressing the immune system by administration of immunosuppressive drugs. This is associated with an increased predisposition to infections and significant side effects. The utilization of stem cells and T regulatory cells offers the potential to selectively suppress pathological immunity while preserving the ability of the body to fight bacteria and viruses. According to the NIH there are approximately 23 million victims of autoimmune conditions.

Links to Documents:

Link to peer-reviewed publication: http://www.translational-medicine.com/content/pdf/1479-5876-7-29.pdf

Link: http://www.marketwire.com/press-release/medistem-files-patent-application-on-therapeutic-cell-population-found-in-fat-tissue-frankfurt-s2u-812298.htm

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Medistem Receives Notice of Patent Allowance Covering Fat Stem Cell Therapy of Autoimmune Diseases

ScienceDaily (June 27, 2012) Stem cells from patients with a rare form of muscular dystrophy have been successfully transplanted into mice affected by the same form of dystrophy, according to a new study published June 27 in Science Translational Medicine.

For the first time, scientists have turned muscular dystrophy patients' fibroblast cells (common cells found in connective tissue) into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice.

The new technique, which was initially developed at the San Raffaele Scientific Institute of Milan and completed at UCL, could be used in the future for treating patients with limb-girdle muscular dystrophy (a rare form in which the shoulders and hips are primarily affected) and, possibly, other forms of muscular dystrophies.

Muscular dystrophies are genetic disorders primarily affecting skeletal muscle that result in greatly impaired mobility and, in severe cases, respiratory and cardiac dysfunction. There is no effective treatment, although several new approaches are entering clinical testing including cell therapy.

In this study, scientists focused on genetically modifying a type of cell called a mesoangioblast, which is derived from blood vessels and has been shown in previous studies to have potential in treating muscular dystrophy. However, the authors found that they could not get a sufficient number of mesoangioblasts from patients with limb-girdle muscular dystrophy because the muscles of the patients were depleted of these cells.

Instead, scientists in this study "reprogrammed" adult cells from patients with limb-girdle muscular dystrophy into stem cells and were able to induce them to differentiate into mesoangioblast-like cells. After these 'progenitor' cells were genetically corrected using a viral vector, they were injected into mice with muscular dystrophy, where they homed-in on damaged muscle fibres.

The researchers also showed that when the same muscle progenitor cells were derived from mice the transplanted cells strengthened damaged muscle and enabled the dystrophic mice to run for longer on a treadmill than dystrophic mice that did not receive the cells.

Dr Francesco Saverio Tedesco, UCL Cell & Developmental Biology, who led the study, said: "This is a major proof of concept study. We have shown that we can bypass the limited amount of patients' muscle stem cells using induced pluripotent stem cells and then produce unlimited numbers of genetically corrected progenitor cells.

"This technique may be useful in the future for treating limb-girdle muscular dystrophy and perhaps other forms of muscular dystrophy."

Professor Giulio Cossu, another UCL author, said: "This procedure is very promising, but it will need to be strenuously validated before it can be translated into a clinical setting, also considering that clinical safety for these "reprogrammed" stem cells has not yet been demonstrated for any disease."

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Successful transplant of patient-derived stem cells into mice with muscular dystrophy

WWL-TVs Sally-Ann Roberts talks about her sisters health battle. Watch Richard Ford on The Colbert Report. TV tweet of the day so far.

TV Linkzilla Daily for 6/27/12 starts now.

When Good Morning America anchor Robin Roberts announced June 11 that shed been diagnosed with a rare form of bone-marrow cancer MDS, or myelodysplastic syndrome -- and would undergo chemotherapy and a bone-marrow transplant, her sister, WWL-TV anchor Sally-Ann Roberts, was cast in a key recovery role.

Sally-Ann Roberts, it turned out, was a perfect match to be a bone-marrow-cell donor for her sister. The New Orleans anchors medical contribution wont come for weeks or perhaps months, but shes already begun efforts to raise awareness of the need for donors.

Her station has launched a Perfect Match Supporting Sally-Ann & Robin page on its website, which now holds several stories on the topic, including a Wednesday (June 27) piece in which WWL staffers sign up to join a bone-marrow registry.

An informational and registration phone-bank, staffed by volunteers from organ- and bone-marrow-donation organizations, will operate from 6-9 p.m. Thursday (June 28), in coordination with the stations morning news block.

In a recent interview, Sally-Ann Roberts said her match is a real blessing, because only 25 percent of people who need a bone-marrow transplant actually find a match among their siblings.

She continued:

The majority of people who need a bone marrow donor have to go outside of their family in order to find one. Sometimes it's like a needle in a haystack, and that's why Robin wants to use this challenge that she's facing right now to try to bring attention to the national narrow donor registry. Millions of people are part of it. If a person -- man woman or child -- is in search of a bone marrow donor they can go to this registry and have a chance to find one. There are many, many people who have used the registry successfully.

The only problem is that minorities are underrepresented in the donor registry. Unlike organ donations, where it really doesn't matter what the ethnic background of an individual is for an organ donation, bone marrow for stem cells has to be aligned with the person's genetic makeup. And that's why if you're African American you will find a match with another African American. Native Americans, the same thing. So that's why every racial group needs to be represented in the marrow-donor registry. That's what were trying to do. Were trying to direct people to BeTheMatch.org. If they do, they'll get a packet in the mail and will be able to do a swab, just the inside of their cheek, and will get a self-addressed stamped envelope. They mail it back with the required information. They may get a phone call, they may never get a phone call. But people who have done so -- I've gone online to listen to some of their stories -- they feel so grateful that they were able to reach out and help another individual in need.

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WWL-TV and Sally-Ann Roberts rally support for bone-marrow donor registry

A mother with three daughters who have Fanconi anemia sued the federal government for the right to compensate bone marrow donors. The U.S. Attorney General will not pursue the case with the Supreme Court, thus making a lower court's ruling law. That means bone marrow donors may now receive vouchers worth up to $3,000. NBC's Dr. Nancy Snyderman reports.

By JoNel Aleccia

Certain bone marrow donors could soon be compensated for their life-saving stem cells after federal officials declined to take the matter to the U.S. Supreme Court, allowing a lower court order to become law.

At least one agency, MoreMarrowDonors.org, hopes to begin a pilot program offering up to $3,000 in scholarships, housing vouchers or charity donations -- but not cash -- in exchange for matching donations of marrow cells derived from blood.

This decision is a total game-changer, said Jeff Rowes, a senior attorney with the Institute for Justice, which filed the lawsuit three years ago on behalf of cancer victims and others seeking bone marrow matches. Any donor, any doctor, any patient across the country can use compensation in order to get bone marrow donors.

That may be the effect of the decision by U.S. Attorney General Eric Holder to forgo a high court review of a 9th U.S. Circuit Court of Appeals ruling that certain kinds of bone marrow donations are exempt from federal rules banning compensation.

Under the ruling, donors who provide marrow cells through a process similar to blood donation, called peripheral blood stem cell apheresis, can be compensated because those cells are no longer regarded as organs or organ parts as defined in the National Organ Transplant Act.

The ruling does not apply, however, to bone marrow obtained through traditional techniques that use a needle to aspirate the cells from the hip.

Although it applies only to nine states covered by the 9th Circuit Court, Rowes expects the effects to be felt nationwide.

The move met with praise from Doreen Flynn, 36, of Lewiston, Maine, the lawsuits namesake and the single mother of three daughters with an incurable blood disorder called Fanconi anemia.

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Bone marrow donors may be compensated after ruling stands

A mother with three daughters who have Fanconi anemia sued the federal government for the right to compensate bone marrow donors. The U.S. Attorney General will not pursue the case with the Supreme Court, thus making a lower court's ruling law. That means bone marrow donors may now receive vouchers worth up to $3,000. NBC's Dr. Nancy Snyderman reports.

By JoNel Aleccia

Certain bone marrow donors could soon be compensated for their life-saving stem cells after federal officials declined to take the matter to the U.S. Supreme Court, allowing a lower court order to become law.

At least one agency, MoreMarrowDonors.org, hopes to begin a pilot program offering up to $3,000 in scholarships, housing vouchers or charity donations -- but not cash -- in exchange for matching donations of marrow cells derived from blood.

This decision is a total game-changer, said Jeff Rowes, a senior attorney with the Institute for Justice, which filed the lawsuit three years ago on behalf of cancer victims and others seeking bone marrow matches. Any donor, any doctor, any patient across the country can use compensation in order to get bone marrow donors.

That may be the effect of the decision by U.S. Attorney General Eric Holder to forgo a high court review of a 9th U.S. Circuit Court of Appeals ruling that certain kinds of bone marrow donations are exempt from federal rules banning compensation.

Under the ruling, donors who provide marrow cells through a process similar to blood donation, called peripheral blood stem cell apheresis, can be compensated because those cells are no longer regarded as organs or organ parts as defined in the National Organ Transplant Act.

The ruling does not apply, however, to bone marrow obtained through traditional techniques that use a needle to aspirate the cells from the hip.

Although it applies only to nine states covered by the 9th Circuit Court, Rowes expects the effects to be felt nationwide.

The move met with praise from Doreen Flynn, 36, of Lewiston, Maine, the lawsuits namesake and the single mother of three daughters with an incurable blood disorder called Fanconi anemia.

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Bone marrow donors soon may be compensated

VATICAN CITY, Italy, June 27, 2012 (GLOBE NEWSWIRE) -- Today, as part of an ongoing mission to advance scientific research on adult stem cell therapies and explore their cultural and ethical implications, Monsignor Tomasz Trafny of the Vatican's Pontifical Council for Culture, joined Dr. Robin Smith, CEO of NeoStem (NYSE MKT:NBS) and Chairman and President of the Stem for Life Foundation, and Dr. Max Gomez, trustee of the Stem for Life Foundation, to present the first copy of their forthcoming book, Our Stem Cells: The Mystery of Life and Secrets of Healing, to The Holy Father, Pope Benedict XVI.

The book is the result of a unique collaboration between the Vatican's Pontifical Council for Culture (via its charitable foundation STOQ International) and the Stem for Life Foundation, and will be available later this year. It includes a special address by His Holiness Benedict XVI, urging increased support and awareness for advancements in adult stem cell research in order to alleviate human suffering.

The book focuses on concepts discussed at the First International Vatican Adult Stem Cell Conference (2011) and presents the reader with an engaging, comprehensive overview of adult stem cells and their vital role in a future of regenerative medicine. In powerful, accessible language the book showcases a wide array of emerging adult stem cell breakthroughs, including their ability to repair damaged hearts and organs, restore sight, kill cancer, cure diabetes, heal burns and stop the march of degenerative diseases, such as Alzheimer's, multiple sclerosis and Lou Gehrig's disease.

"In addition to making the science easy to understand, we filled the book with here-and-now case studies on how adult stem cell therapies are already helping real people suffering needlessly from deadly and debilitating diseases and medical conditions," said Dr. Smith. "Not only does the book speak to the success of our historic partnership with the Vatican, but it sets the stage for our next events."

"This book promotes a powerful dialogue between scientific and religious communities," said Monsignor Tomasz Trafny. "This dialogue needs to find its expression within the important framework of searching for truth and being guided by the highest ethical values. We hope this book will help educate people throughout the world regarding the importance of ethical scientific research and help them understand they do not need to choose between their faith and science; but in fact, the two can work together to profoundly improve humanity."

To preorder the book, go to: http://www.stemforlife.org/ourstemcells

About the Stem for Life Foundation

Stem for Life Foundation (SFLF) is dedicated to improving the quality of life of millions of people suffering from dozens of painful and sometimes debilitating medical conditions by providing information and updates about adult stem cell research, therapy development and possible healthcare applications. SFLF focuses on educating the public, convening the best minds in adult stem cell medicine and research, supporting clinical research, and subsidizing adult stem cell collection and storage for those who need it most.

Understanding that adult stem cell research could lead to better treatments and possibly cures for chronic disease, as well as reduce health care costs and improve quality of life for those with chronic disease and disability, SFLF was established in 2007. SFLF's Board of Trustees and staff are deeply committed to expediting development of stem cell therapies that offer real hope to individuals suffering from a wide-range of life-threatening medical conditions.

About The Pontifical Council for Culture

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The Pontifical Council for Culture and the Stem for Life Foundation Present Groundbreaking Book on Adult Stem Cell ...