ScienceDaily (May 22, 2012) Researchers at the Hebrew University of Jerusalem have achieved, for the first time, the generation of neuronal cells from stem cells of Fragile X patients. The discovery paves the way for research that will examine restoration of normal gene expression in Fragile X patients.

Absence of expression of the FMR1 gene is caused by a mutation in the regulatory elements that govern its expression. The abnormal addition of chemical methyl groups to the regulatory elements causes gene silencing in patients, culminating in severe mental retardation.

A potential way to help patients is to find compounds that will clear the abnormal methyl groups from the regulatory elements and reactivate normal gene expression. In their work, the Hebrew University researchers have identified a chemical compound that restored normal gene expression specifically in neuronal cells, the cell type most affected in patients.

The research was conducted in the laboratory of Nissim Benvenisty, the Herbert Cohn Professor of Cancer Research at the Hebrew University, by PhD student Ori Bar-Nur and undergraduate student Inbal Caspi. They demonstrated, for the first time, the generation of brain neuronal cells from patients of Fragile X syndrome in a dish culture. In doing so, they were able to find a substance that restored normal gene expression in patients' cells.

In a previous study conducted in the Benvenisty laboratory, a novel technology was used to induce pluripotent stem cells from skin cells of Fragile X patients. Pluripotent stem cells have the amazing ability to differentiate into any human cell type in a dish culture.

In their latest study (published in the Journal of Molecular Cell Biology), the researchers harnessed this ability to turn the stem cells into neuronal brain cells. After generating the cells, they screened several chemical substances with the aim of finding one that would restore FMR1 normal gene expression. They showed that the substance 5-azaC was able to clear the methyl groups from the regulatory elements of the gene, allowing for the efficient restoration of FMR1 expression in both stem and neuronal brain cells.

The substance 5-azaC has been known for many years to clear methyl groups from regulatory elements of genes, and is also an already established drug for other diseases. However, this is the first time that it has been shown to successfully clear the methylation in neurons or stem cells of Fragile X patients.

In addition, the researchers were able to show that gene expression is maintained even after 5-azaC withdrawal, so there is no need to administer it continuously. This raises hopes for the use of the compound as a potential drug for the benefit of Fragile X patients.

According to Bar-Nur, "There is still a substantial gap between the restoration of gene expression in cultured patients' cells and restoring it in patients; however, the finding that it is possible to restore gene expression in neuronal cells paves the way for further study of its restoration in patients." He concludes: "New technologies developed in recent years in the stem cell field allow us to conduct research that was not possible until recently."

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Stem cell research paves way for progress on dealing with Fragile X

24.05.2012 - (idw) Schwedischer Forschungsrat - The Swedish Research Council

A new technique that converts stem cells into brain cells has been developed by researchers at Lund University. The method is simpler, quicker and safer than previous research has shown and opens the doors to a shorter route to clinical cell transplants. By adding two different molecules, the researchers have discovered a surprisingly simple way of starting the stem cells journey to become finished brain cells. The process mimics the brains natural development by releasing signals that are part of the normal development process. Experiments in animal models have shown that the cells quickly adapt in the brain and behave like normal brain cells.

This technique allows us to fine-tune our steering of stem cells to different types of brain cells. Previous studies have not always used the signals that are activated during the brains normal development. This has caused the transplanted cells to develop tumours or function poorly in the brain, says Agnete Kirkeby, one of the authors of the study.

Since the method effectively imitates the brains own processes, it reduces the risk of tumour formation, one of the most common obstacles in stem cell research. The quick, simple technique makes the cells mature faster, which both makes the transplant safer and helps the cells integrate better into the brain. The results of the study bring stem cell research closer to transplant trials in the human brain.

The research is presented in the report Generation of regionally specified neural progenitors and functional neurons from human embryonic stem cells under defined conditions in the journal Cell Reports.

The study has been conducted as part of the EU 7th Framework Programme project NeuroStemcell.

For more information, please contact: Malin Parmar +46 709 823901, Malin.Parmar@med.lu.se Agnete Kirkeby +45 5168 5353, Agnete.Kirkeby@med.lu.se jQuery(document).ready(function($) { $("fb_share").attr("share_url") = encodeURIComponent(window.location); });

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From stem cell to brain cell new technique mimics the brain

ASTANA, Kazakhstan, May 24, 2012 /PRNewswire/ --Sir Richard Roberts, the eminent British biologist and Nobel Prize laureate, said today European opposition to genetically modified organisms is political rather than scientific in nature.

He also said "personal medicine" based on human genome research holds large-scale promise to improve the health of the world's people on an individualized basis.

Roberts, who won the Nobel in 1993 for his shared discovery of split genes, made his remarks at the Astana Economic Forum, a global conference of scientists, academics, multinational executives and government leaders.

"On a political level, governments must embrace genetically modified organisms (GMOs) and not give way to European prophets of doom, who oppose the use of GMOs for purely political reasons," said Roberts. "It is important to note there is a complete absence of evidence that GMOs can cause any harm. Indeed to any well-informed scientist, traditionally bred plants seem much more likely to be harmful than GMOs."

Roberts predicted growing knowledge of the human genome will yield better medical treatments and diagnostics. "It is just as important that we learn more about the bacteria that colonize our bodies since they are an essential part of what it means to be human," he said.

He also predicated synthetic biology will enable scientists to build novel microorganisms from "scratch."

"Most exciting is the promise of stem cells where the challenge is to understand how they drive their differentiation into all of the other cell types in our bodies," Roberts said. "While I do not advocate prolonging life indefinitely, I am very much in favor of ensuring that as we age, the quality of our life does not diminish."

The annual Astana Economic Forum this year has drawn thousands of participants from more than 80 nations to this rapidly growing Central Asian nation. There has been much focus at the current sessions on the Greek financial crisis and turbulence in the Euro currency, in addition to the broader economic, scientific and international trade issues that are a traditional mainstay at Astana.

Deal making is a big part of both the official and the unofficial agenda at Astana. Multinationals represented include Chevron, Toyota, Nestle, Microsoft, BASF, Total, General Electric.

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Nobelist Speaks Out on Genetic Modification, Synthetic Biology, Stem Cell Research

Editor's Choice Main Category: Bones / Orthopedics Also Included In: Stem Cell Research Article Date: 22 May 2012 - 12:00 PDT

Current ratings for: 'Prochymal - First Stem Cell Drug Approved'

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Prochymal (remestemcel-L) is also the first drug to be approved for the treatment of acute graft-vs-host disease (GvHD) in children, a devastating complication of bone marrow transplantation that kills almost 80% of all affected children, many of which just weeks after they have been diagnosed.

GvHD is the leading cause of transplant-related mortality, caused by an immunologic attack. Severe GvHD can cause blistering of the skin, intestinal hemorrhage and liver failure and is extremely painful with a death rate of up to 80%. At present, the first-line standard therapies for GvHD are steroids. Given that the success rate of steroids is only 30 to 50%, the only other therapy if steroids fail is limited to immunosuppressive agents that are used off-label with little benefit and significant toxicities. Until the approval of Prochymal, there has not been any other therapy for GvHD.

Osiris Therapeutics Inc. was awarded authorization for Prochymal under Health Canada's Notice of Compliance with conditions (NOC/c). A NOC/c is an authorization to market a drug with the condition that the manufacturer undertakes additional studies to verify the clinical benefit. This pathway provides access to treatments for unmet medical conditions and has demonstrated its benefits outweigh its risks in clinical trials.

Andrew Daly, M.D., Clinical Associate Professor from the Department of Medicine and Oncology at the University of Calgary, Canada and leading researcher of Prochymal's phase 3 clinical program declared:

C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris announced:

Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors aged between 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture, producing up to 10,000 doses of Prochymal from a single donor. The drug is a true off-the-shelf stem cell product, which is stored frozen until it is needed. Prochymal is infused through a simple intravenous line without the need to type or immunosuppress the recipient. The drug is currently undergoing Phase 3 trials for refractory Crohn's disease, as well as undergoing clinical trials for the treatment of heart attacks and type-1 diabetes.

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Prochymal - First Stem Cell Drug Approved

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

Contact: Kate McAlpine kmca@umich.edu 734-763-4386 University of Michigan

ANN ARBOR, Mich.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 embryo's 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 U-M School of Dentistry, said, "We turn back the clock, in a way. We're 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 don't 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 don't really know what's 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 patient's immune system.

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Stem-cell-growing surface enables bone repair

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.

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Bone Repair Via Stem-cell-growing Surface

Stem cells have assumed near-mythical status in the popular imagination as a possible cure for every disease under the sun. But while public attention has focused on their potential in regenerative medicine, stem cells have quietly gained a foothold in drug development a move that may hail a huge but unheralded shake-up of the biological sciences.

I think there are tremendous parallels to the early days of recombinant DNA in this field, says James Thomson, director of regenerative biology at the Morgridge Institute for Research in Madison, Wisconsin, and one of the founders of Cellular Dynamics International, also in Madison. I dont think people appreciated what a broad-ranging tool recombinant DNA was in the middle '70s." At the same time, he says, they underestimated the difficulty of using it in treatments.

Now stem cells are in a similar situation, he says, and although therapeutic use is likely to come to fruition eventually, people underappreciate how broadly enabling a research tool it is, he says.

Laboratory-grown stem cells hold much promise for regenerative medicine, but are being increasingly used in drug testing.

MASSIMO BREGA, THE LIGHTHOUSE/SCIENCE PHOTO LIBRARY

Drug companies began dipping a tentative toe into the stem-cell waters about two years ago (see 'Testing time for stem cells'). Now, the pharmaceutical industry is increasingly adopting stem cells for testing the toxicity of drugs and identifying potential new therapies, say those in the field.

Cellular Dynamics sells human heart cells called cardiomyocytes, which are derived from induced pluripotent stem (iPS) cells. Thomson says that essentially all the major pharma companies have bought some. The company also produces brain cells and cells that line blood vessels, and is about to release a line of human liver cells.

Yet Cellular Dynamics is just one of the companies in the field. Three years ago, stem-cell biologist Stephen Minger left his job in UK academia to head GE Healthcares push into stem cells (see 'Top scientist's industry move heralds stem-cell shift'). The medical-technology company, headquartered in Chalfont St. Giles, UK, has been selling human heart cells made from embryonic stem (ES) cells for well over a year, and is due to start selling liver cells soon.

Minger and his team at GE Healthcare assessed the heart cells in a blind trial against a set of unnamed drug compounds to see if the cells would reveal which compounds were toxic. When the compounds were unmasked, Minger says, they found that the cells had been affected by the known toxic compounds. But, crucially, in a number of cases, the cells identified a problem that had only been discovered after the drugs had reached the market and after they had been approved by agencies such as the US Food and Drug Administration (FDA).

These are compounds which went all the way through animal testing, then went through phase I, II, III and then were licensed in many cases by the FDA, says Minger.

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Stem cells take root in drug development