ALAMEDA, Calif.--(BUSINESS WIRE)--BioTime, Inc. (NYSE MKT: BTX) announced that its subsidiary Asterias Biotherapeutics, Inc. (OTCBB: ASTY) has reached an agreement with Cancer Research UK and Cancer Research Technology (CRT), the charitys development and commercialization arm, to conduct a clinical trial of Asterias novel immunotherapy treatment AST-VAC2 in subjects with non-small cell lung cancer.

AST-VAC2 is a non-patient specific (allogeneic) cancer vaccine designed to stimulate patients immune systems to attack telomerase, a protein that is expressed in over 95 percent of cancers but is rarely expressed in normal adult cells.

The vaccine was developed following successful early phase clinical trials of a similar, patient specific (autologous) Asterias vaccine, called AST-VAC1, which was derived from patients blood cells and tested in prostate cancer and acute myeloid leukemia.

Unlike AST-VAC1, and other autologous (patient specific) vaccines that are developed from a patients own cells, AST-VAC2 is derived from human embryonic stem cells (hESCs), meaning it can be produced on a large scale and stored ready for use, rather than having to produce a specific version of the drug for each patient.

The trial of AST-VAC2 will evaluate the safety and toxicity of the vaccine, feasibility, stimulation of patient immune responses to telomerase and AST-VAC2, and clinical outcome after AST-VAC2 administration in patients with resected early-stage lung cancer and in patients with advanced forms of the disease.

Pedro Lichtinger, Asterias chief executive officer, said: The Asterias collaboration with Cancer Research UKs Drug Development Office and CRT represents a major step in advancing our proprietary dendritic cell platform for the potential benefit of patients.

AST-VAC2 is based on a specific mode of action that is complementary and potentially synergistic to other immune therapies. We are delighted to partner with Cancer Research UK to advance this important platform through Phase 1/2 clinical trials. Cancer Research UKs Drug Development Office has the global recognition of having the quality, capability and track record of successfully advancing development programs. We are excited about the possibility of favorably impacting the lives of patients across multiple cancers and are proud to be working with Cancer Research UK.

Under the agreement, Asterias will complete development of the manufacturing process for AST-VAC2. Cancer Research UK will then produce the vaccine and conduct the phase 1/2 clinical trial in the United Kingdom. On completion of the clinical trial, Asterias will have an exclusive first option to acquire a license to the data from the trial on pre-agreed terms including an upfront payment, milestones and royalties on sales of products. If Asterias declines this option, CRT will then have an option to obtain a license to Asterias intellectual property to continue the development and commercialization of AST-VAC2 and related products in exchange for a revenue share to Asterias of development and partnering proceeds.

Dr. Jane Lebkowski, president of research and development at Asterias, said: The use of human embryonic stem cells to derive allogeneic dendritic cells for cancer immunotherapy has the potential to dramatically improve the scalability, consistency, and feasibility of cellular cancer vaccines. We believe this collaboration will enable the acceleration of clinical studies of AST-VAC2 and the collection of important proof-of-concept data for the entire human embryonic stem cell-derived dendritic cell immunotherapy platform.

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BioTime Subsidiary, Asterias Biotherapeutics, and Cancer Research UK and Cancer Research Technology Partner for ...

PUBLIC RELEASE DATE:

5-Sep-2014

Contact: Lisa Newbern lisa.newbern@emory.edu 404-727-7709 Emory Health Sciences

Creating induced pluripotent stem cells or iPS cells allows researchers to establish "disease in a dish" models of conditions ranging from Alzheimer's disease to diabetes. Scientists at Yerkes National Primate Research Center have now applied the technology to a model of Huntington's disease (HD) in transgenic nonhuman primates, allowing them to conveniently assess the efficacy of potential therapies on neuronal cells in the laboratory.

The results were published in Stem Cell Reports.

"A highlight of our model is that our progenitor cells and neurons developed cellular features of HD such as intranuclear inclusions of mutant Huntingtin protein, which most of the currently available cell models do not present," says senior author Anthony Chan, PhD, DVM, associate professor of human genetics at Emory University School of Medicine and Yerkes National Primate Research Center. "We could use these features as a readout for therapy using drugs or a genetic manipulation."

Chan and his colleagues were the first in the world to establish a transgenic nonhuman primate model of HD. HD is an inherited neurodegenerative disorder that leads to the appearance of uncontrolled movements and cognitive impairments, usually in adulthood. It is caused by a mutation that introduces an expanded region where one amino acid (glutamine) is repeated dozens of times in the huntingtin protein.

The non-human primate model has extra copies of the huntingtin gene that contains the expanded glutamine repeats. In the non-human primate model, motor and cognitive deficits appear more quickly than in most cases of Huntington's disease in humans, becoming noticeable within the first two years of the monkeys' development.

First author Richard Carter, PhD, a graduate of Emory's Genetics and Molecular Biology doctoral program, and his colleagues created iPS cells from the transgenic monkeys by reprogramming cells derived from the skin or dental pulp. This technique uses retroviruses to introduce reprogramming factors into somatic cells and induces a fraction of them to become pluripotent stem cells. Pluripotent stem cells are able to differentiate into any type of cell in the body, under the right conditions.

Carter and colleagues induced the iPS cells to become neural progenitor cells and then differentiated neurons. The iPS-derived neural cells developed intracellular and intranuclear aggregates of the mutant huntingtin protein, a classic sign of Huntington's pathology, as well as an increased sensitivity to oxidative stress.

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Disease in a dish approach could aid Huntington's disease discovery

7 hours ago Stem cells. Credit: Nissim Benvenisty - Wikipedia

Induced pluripotent stem cells (iPSCs)adult cells reprogrammed back to an embryonic stem cell-like statemay hold the potential to cure damaged nerves, regrow limbs and organs, and perfectly model a patient's particular disease. Yet through the reprogramming process, these cells can acquire serious genetic and epigenetic abnormalities that lower the cells' quality and limit their therapeutic usefulness.

When the generation of iPSCs was first reported in 2006, efficiency was paramount because only a fraction of a percentage of reprogrammed cells successfully became cell lines. Accordingly, the stem cell field focused on reprogramming efficiency to boost the pool of cells that could be studied. However, as scientists gained an increased understanding of the reprogramming process, they realized that myriad variables, including the ratio of reprogramming factors and the reprogramming environment, can also greatly affect cell quality.

Now researchers working in the lab of Whitehead Institute Founding Member Rudolf Jaenisch together with scientists from the Hebrew University have determined that the reprogramming factors themselves impact the reprogramming efficiency and the quality of the resulting cells. Their work is described in the current issue of the journal Cell Stem Cell.

"Postdoctoral researcher Yosef Buganim and Research Scientist Styliani Markoulaki show that a different combination of reprogramming factors may be less efficient than the original, but can produce much higher quality iPSCs," says Jaenisch, who is also a professor of biology at MIT. "And quality is a really important issue. At this point, it doesn't matter if we get one colony out of 10,000 or one out of 100,000 cells, as long as it is of high quality."

To make iPSCs, scientists expose adult cells to a cocktail of genes that are active in embryonic stem cells. iPSCs can then be pushed to differentiate into almost any other cell type, such as nerve, liver, or muscle cells. Although the original combination of Oct4, Sox2, Klf4, and Myc (OSKM) efficiently reprograms cells, a relatively high percentage of the resulting cells have serious genomic aberrations, including aneuploidy, and trisomy 8, which make them unsuitable for use in clinical research.

Using bioinformatic analysis of a network of 48 genes key to the reprogramming process, Buganim and Markoulaki designed a new combination of genes, Sall4, Nanog, Esrrb, and Lin28 (SNEL). Roughly 80% of SNEL colonies made from mouse cells were of high quality and passed the most stringent pluripotency test currently available, the tetraploid complementation assay. By comparison, only 20-30% of high quality OSKM passed the same test. Buganim hypothesizes that SNEL reprograms cells better because, unlike OSKM, the cocktail does not rely on a potent oncogene like Myc, which may be causing some of the genetic problems. More importantly, the cocktail does not rely on the potent key master regulators Oct4 and Sox2 that might abnormally activate some regions in the adult cell genome.

To better understand why some reprogrammed cells are of high quality while others fall short, Buganim and Markoulaki analyzed SNEL colonies down to the genetic and epigenetic level. On their DNA, SNEL cells have deposits of the histone protein H2AX in locations very similar to those in ESCs, and the position of H2AX seems to predict the quality of the cell. The researchers believe this characteristic could be used to quickly screen for high quality colonies.

But for all of its promise, the current version of SNEL seems unable to reprogram human cells, which are generally more difficult to manipulate than mouse cells.

"We know that SNEL is not the ideal combination of factors," says Buganim, who is currently a Principal Investigator at Hebrew University in Jerusalem. "This work is only a proof of principle that says we must find this ideal combination. SNEL is an example that shows if you use bioinformatics tools you can get better quality. Now we should be able to find the optimal combination and try it in human cells to see if it works."

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New reprogramming factor cocktail produces therapy-grade induced pluripotent stem cells

23 hours ago The biobank comprises three cryotanks, equipped with cooled protective hoods, and a transfer station from which the sample containers are transported via a rail system. There is enough space for approximately 60,000 samples. Credit: Fraunhofer IBMT

For the development of new drugs it is crucial to work with stem cells, as these allow scientists to study the effects of new active pharmaceutical ingredients. But it has always been difficult to derive enough stem cells of the right quality and in the right timeframe. A central biobank is about to remedy the situation.

Human stem cells allow scientists to assess how patients are likely to respond to new drugs and to examine how illnesses come about. For a few years now, it has been possible to take tissue samples from adults and use reverse programming to artificially produce stem cells, which have the potential to create any kind of cell found in the human body. Before this discovery, pharmaceutical researchers had to use adult stem cells or primary cells, which have a more limited potential. Another option is to use stem cells derived from human embryos, but quite apart from the ethical considerations these cells are available only in limited diversity. The new technique makes it possible for instance to reprogram adult skin or blood cells so that they behave in a similar way to embryonic stem cells and can become any type of cell. "These are known as induced pluripotent stem cells, or iPS cells for short," says Dr. Julia Neubauer from the Fraunhofer Institute for Biomedical Engineering IBMT in St. Ingbert, Germany. Although an increasing number of local biobanks have emerged in recent years, none of them fulfills the requirements of the pharmaceutical industry and research institutions. What is needed is a supply of 'ready-to-use' stem cells, which means large numbers of consistently characterized, systematically catalogued cells of suitable quality.

At the beginning of 2014, the IBMT teamed up with 26 industry and research partners to launch a project aimed at establishing a central biobank the European Bank for induced pluripotent Stem Cells (EBiSC) to generate iPS cells from patients with specific diseases or genetic mutations (http://ebisc.org/). Six months into the project and the first cells are available for use in the development of new drugs. By its three-year mark, it is hoped the project will be in a position to offer over 1000 defined and characterized cell lines comprising a hundred million cells. Such quantities are needed because a single drug screening involves testing several million cells. The main biobank facility is being built in the English city of Cambridge and an identical "mirror site" will be set up at the IBMT's Sulzbach location in Germany.

Gently freezing cells

The IBMT was brought on board for EBiSC by virtue of the comprehensive expertise it gained through participation in the EU's "Hyperlab" and "CRYSTAL" projects. For EBiSC, IBMT scientists are responsible for freezing the cells and for automating cell cultivation and the biobank itself. For an efficient long-term storage of functional stem cells, they have to be cooled down to temperatures of below 130 degrees Celsius in a controlled way. The scientists have to prepare the cells so they can survive the cold shock of nitrogen gas. The IBMT has, for instance, developed technologies that allow cells to be frozen in an extremely gentle way. "Cells don't like being removed from the surface they are grown on, but that's what people used to do in order to freeze them. Our method allows the cells to stay adherent," explains Neubauer.

Just as with foodstuffs, stem cells depend on an unbroken cold chain to preserve their functionality and viability. The scientists store the cells in special containers or cryotanks each measuring one by two meters. To remove a particular sample, the scientists have to open the cryotank. The problem is that this exposes all the other samples to warmer ambient air, causing them to begin to thaw out. "It's just like when you go to your refrigerator at home it's not a good idea to leave the door open too long," says Neubauer. She and her colleagues at the IBMT and industry partner Askion GmbH have together developed a stem cell biobank complete with protective hoods that protect the other samples whenever the cryotank is opened. In addition to maintaining the temperature, the hoods help keep another key shelf-life criterion, humidity, at a constant level.

Flawless freezing is important, but it is just as important to automate the whole process. "That not only guarantees consistency, it's what makes it possible to provide large quantities of cells of the required quality in the first place," says Neubauer. And the scientists' cooling process already boasts a finished technology. In their automated biobank, each cell sample is labelled with barcodes to allow them to be tracked. The samples travel along a conveyor belt to the individual cyrotanks, and a computer monitors the entire freezing and storage process.

Now the scientists are working on automating cell cultivation or the multiplying of the cells. There are essentially two possible approaches. One is to use robots that translate each preparation step into a mechanical one. The other is to use stirred bioreactors that provide free-moving cells with the ideal supply of nutrients and oxygen. Both technologies feature in the IBMT's portfolio. "By the time the project is completed, we'll know which is the better method for what we're trying to do," says Neubauer.

Explore further: Animal-free reprogramming of adult cells improves safety

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Central biobank for drug research

A lot of individuals are regularly making an effort to find the best medications available today because of the presence of a lot of illnesses in the world. New treatments and variations of old ones are hitting the market because of this growing need of people and one of the newest alternatives to medication that experts have come up with is referred to as induced Pluripotent Stem Cell Therapy, also called iPS Cell Therapy or iPSC Therapy. What is Induced Pluripotent Stem Cell therapy?

Regardless if the entire thing is controversial, a ton of experts continue to show interest when it comes to stem cell therapy. Grown inside the laboratory, people are injected with transmuted cells to replace cells that are unhealthy. This is what science fiction is made of, but now almost a reality.

The thing about stem cell therapy is that it garnered and continues to garner a lot of bad publicity in line with moral and ethical concerns. Several years ago, people saw to it that no further research was done on embryonic stem cells but in 2006, studies were conducted by the Japanese but this time, they used mouse cells. More and more people became mindful of and interested in iPSC because of this shift in events.

About 5 years ago, the University of Wisconsin found a way to study iPSC with the help of adult human cells. The thing about iPSC is that people only had problems with the studies when embryonic stem cells were utilized. Because of such an event, efforts have been made to include iPSC processes in Regenerative or Reparative Medicine.

Various illnesses can affect daily living from arthritis to diabetes to burns and iPSC therapies can be a solution to these provided that adequate research is conducted. What you have here can also be utilized for diseases that are genetic in nature like cancer for example. Aside from dealing with spinal cord issues, there is also a chance that iPSC can be used to cure Parkinsons and Alzheimers disease.

There is so much potential in stem cell therapy. Imagine how much good it will do to mankind if healthy cells may scientifically be produced in laboratories and injected into patients. For people with cancer, the cancerous cells can easily be replaced with the ones that are healthy.

What you have here can change the way people look at disease and pain.. Not having to rely on the human body for cell regeneration is something that can lead to thousands of opportunities in health. There is still a need to perfect current research efforts on the matter but this is surely beyond science fiction.

Other than yet merely in experimental stage, the therapies are also very costly. These therapies need more time for experimentation and more years are necessary if you want to lower the costs of the therapies. But scientists remain hopeful.

One of the most popular therapies in line with stem cells these days is bone marrow transplantation. There are various patients that have different cancers related to the bone marrow or blood and this is what this transplantation serves to treat. It is a risky procedure, however, and may have several complications.

In various countries, scientists get support for this type of research. It may take years before people can rely on iPS Cell therapy on a regular basis but even if this is so, all the hard work will surely be well worth it because of the countless benefits that this form of therapy can bring. Pain and disease will be no match for science once this form of therapy is completed.

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IPS Cell Therapy | Stem Cells Research

ALAMEDA, Calif.--(BUSINESS WIRE)--BioTime, Inc. (NYSE MKT: BTX) today reported financial results for the first quarter ended June 30, 2014 and highlighted recent corporate accomplishments.

We are pleased with our success to date in building toward our goal of developing both near-term commercial applications of our technologies and maintaining our focus on the power of pluripotent stem cells to create innovative human therapeutics, said Dr. Michael D. West, BioTimes Chief Executive Officer. Near-term product development underway includes our subsidiary OncoCyte Corporations three cancer diagnostic products undergoing clinical studies, mobile health product development in our subsidiary LifeMap Solutions, Inc., our Renevia pivotal clinical trial in Europe, steps to prepare for the marketing of our recently FDA-cleared wound healing product Premvia, and growing research product sales by our ESI BIO division.

BioTimes longer-term major therapeutic product opportunities are based on the broad range of cell-based regenerative therapies planned for development from its pluripotent stem cell technology platform. This platform is protected by over 600 patents and patent applications worldwide within the BioTime family of companies. Our subsidiary Asterias Biotherapeutics, Inc. has submitted an amended IND to the FDA for a Phase 1/2a clinical trial of AST-OPC1 for the treatment of cervical spinal cord injury and is currently awaiting clearance from the FDA for that trial. Asterias is also currently undertaking process development of AST-VAC2, a cancer immunotherapy targeting the important antigen called telomerase, for a potential clinical trial in lung cancer. This progress, along with the appointment of Pedro Lichtinger as Asterias CEO and the award of a $14 million grant from the California Institute for Regenerative Medicine, should fuel the development of these first-in-class therapeutic products. Recently, Asterias shares began to trade publicly under the symbol ASTYV, the first of our subsidiaries to have its shares trade publicly. Lastly, we expect that BioTimes subsidiary Cell Cure Neurosciences Ltd. will soon file its IND to begin a clinical trial of OpRegen for the treatment of age-related macular degeneration. Additional important cell-based product development is underway in our disease-focused subsidiaries OrthoCyte Corporation and ReCyte Therapeutics.

As we saw in the first quarter of this year, our expenses have risen compared to recent quarters, but our progress during the second quarter in streamlining our workforce through shared core resources among our subsidiaries should reduce our cash burn rate in the third quarter. We would like to thank those who share our goal of better health in the coming era of regenerative medicine. Their continued support and the diligent efforts of our collaborators at leading academic medical institutions is critical in advancing our products from the lab bench to the clinic, where they are desperately needed.

Second Quarter and Recent Highlighted Corporate Accomplishments

Financial Results

Revenue

For the six months ended June 30, 2014, on a consolidated basis, total revenue was $2.2 million, up $0.3 million or 19% from $1.8 million for the same period one year ago. The increase in revenue is primarily attributable to a $0.4 million increase in grant income primarily from a grant awarded to BioTimes subsidiary Cell Cure Neurosciences Ltd. (Cell Cure Neurosciences) from Israels Office of the Chief Scientist, offset in part by the decline in license fees of $0.1M primarily due to full recognition of the unamortized balance of the Summit license fees received in advance during the fourth quarter of 2013 as a result of the termination of our license agreements with Summit in 2013.

Expenses

Operating expenses for the six months ended June 30, 2014 were $26.0 million, compared to expenses of $18.0 million for the same period of 2013. The increase in operating expenses is primarily attributable to an increase in staffing, and the expansion of research and development efforts, including additional expenses in the Renevia clinical safety trial program, the development of OpRegen by BioTimes subsidiary Cell Cure Neurosciences for the treatment of dry age related macular degeneration, and the increased staffing and operations of Asterias in connection with the Geron stem cell asset acquisition and by LifeMap Solutions. In addition, during the first six months in 2014, operating expenses included $1.5 million of amortization expense of intangible assets recorded in connection with the Geron stem cell asset acquisition in October 2013.

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BioTime Announces Second Quarter 2014 Results and Recent Developments

PUBLIC RELEASE DATE:

6-Aug-2014

Contact: Robert Miranda cogcomm@aol.com Cell Transplantation Center of Excellence for Aging and Brain Repair

Putnam Valley, NY. (Aug. 6, 2014) Recent studies have shown that transplanting induced pluripotent stem cell-derived neural stem cells (iPS-NSCs) can promote functional recovery after spinal cord injury in rodents and non-human primates. However, a serious drawback to the transplantation of iPS-NSCs is the potential for tumor growth, or tumorogenesis, post-transplantation.

In an effort to better understand this risk and find ways to prevent it, a team of Japanese researchers has completed a study in which they transplanted a human glioblastoma cell line into the intact spinal columns of laboratory mice that were either immunodeficient or immunocompetent and treated with or without immunosuppresant drugs. Bioluminescent imaging was used to track the transplanted cells as they were manipulated by immunorejection.

The researchers found that the withdrawal of immunosuppressant drugs eliminated tumor growth and, in effect, created a 'safety lock' against tumor formation as an adverse outcome of cell transplantation. They also confirmed that withdrawal of immunosuppression led to rejection of tumors formed by transplantation of induced pluripotent stem cell derived neural stem/progenitor cells (iPS-NP/SCs).

Although the central nervous system has shown difficulty in regenerating after damage, transplanting neural stem/progenitor cells (NS/PCs) has shown promise. Yet the problem of tumorogenesis, and increases in teratomas and gliomas after transplantation has been a serious problem. However, this study provides a provisional link to immune therapy that accompanies cell transplantation and the possibility that inducing immunorejection may work to reduce the likelihood of tumorogenesis occurring.

"Our findings suggest that it is possible to induce immunorejection of any type of foreign-grafted tumor cells by immunomodulation," said study co-author Dr. Masaya Nakamura of the Keio University School of Medicine. "However, the tumorogenic mechanisms of induced pluripotent neural stem/progenitor cells (iPS-NS/PCs) are still to be elucidated, and there may be differences between iPS-NS/PCs derived tumors and glioblastoma arising from genetic mutations, abnormal epigenetic modifications and altered cell metabolisms."

The researchers concluded that their model might be a reliable tool to target human spinal cord tumors in preclinical studies and also useful for studying the therapeutic effect of anticancer drugs against malignant tumors.

"This study provides evidence that the use of, and subsequent removal of, immunosuppression can be used to modulate cell survival and potentially remove tumor formation by transplanted glioma cells and provides preliminary data that the same is true for iPS-NS/PCs." said Dr. Paul Sanberg, distinguished professor at the Center of Excellence for Aging and Brain Repair, University of South Florida. "Further study is required to determine if this technique could be used under all circumstances where transplantation of cells can result in tumor formation and its reliability in other organisms and paradigms."

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Researchers seek 'safety lock' against tumor growth after stem cell transplantation

What is Okyanos Cardiac Stem Cell Therapy? Cardiac stem cell therapy is a promising new treatment option for advanced heart disease patients. This short video explores the procedure and benefits of adult stem cell therapy for severe

By: Okyanos Heart Institute

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David's Stories from Detroit David in Detroit for Netroots Nation 2014 On the Bonus Show: A Russian man beats the bank at it's own game, stem-cell therapy gone awry, Rhode Island's accidental legal prostitution experiment

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Kellie van Meurs, pictured with her husband Mark, died while undergoing stem cell treatment in Russia. Photo: Facebook

Supporters of a Brisbane mother-of-two who died while undergoing a controversial stem cell treatment in Russia say it did not cause her death, nor have others been discouraged from seeking it.

Kellie van Meurs suffered from a rare neurological disorder called stiff person syndrome, which causes progressive rigidity of the body and chronic pain.

She travelled to Moscow in late June to undergo an autologous haematopoietic stem cell transplant (HSCT) under the care of Dr Denis Fedorenko from the National Pirogov Medical Surgical Centre.

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Cell Therapy IPS Cell Therapy IPS Cell Therapy

New York, NY (PRWEB) July 23, 2014

The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) have partnered to develop stem cell resources to investigate and explore new treatments and ultimately find a cure for juvenile Batten disease, a fatal illness affecting children.

NYSCF scientists will create induced pluripotent stem (iPS) cell lines from skin samples of young people affected by juvenile Batten disease as well as unaffected family members. IPS cell lines are produced by artificially turning back the clock on skin cells to a time when they were embryonic-like and capable of becoming any cell in the body. Reprogramming juvenile Batten iPS cells to become brain and heart cells, will provide the infrastructure needed to investigate what is going wrong with the cells adversely affected by the disease. Thus far, efforts to study juvenile Batten disease have been done using rodent models or human skin cells; neither of which accurately mimic the disease in the brain, leaving researchers without proper tools to study the disease or a solid platform for testing drugs that prevent, halt, or reverse its progression. This will be the largest and first genetically diverse collection of human iPS cells for a pediatric brain disease.*

In addition to working with BBDF to actively recruit patients and families to donate skin samples, Batten Disease Support and Research Association (BDSRA) is providing resources and technical support, spreading awareness among academic scientists, and notifying its Pharmaceutical partners. Together, BBDF and BDSRA will ensure that juvenile Batten disease and other researchers are aware of and utilize the 48 stem cell lines resulting from this collaboration to further juvenile Batten disease research worldwide.

We know the genetic mutations associated with juvenile Batten disease. This partnership will result in stem cell models of juvenile Batten, giving researchers an unprecedented look at how the disease develops, speeding research towards a cure, said Susan L. Solomon, NYSCF Chief Executive Officer.

Working with NYSCF to generate functional neuronal subtypes from patients and families is a stellar example of one of our key strategies in the fight against juvenile Batten disease: creating resource technology with the potential to transform juvenile Batten disease research and accelerate our timeline to a cure, said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Juvenile Batten disease begins in early childhood between the ages of five and ten. Initial symptoms typically begin with progressive vision loss, followed by personality changes, behavioral problems, and slowed learning. These symptoms are followed by a progressive loss of motor functions, eventually resulting in wheelchair use and premature death. Seizures and psychiatric symptoms can develop at any point in the disease.

Juvenile Batten disease is one disorder in a group of rare, fatal, inherited disorders known as Batten disease. Over 40 different errors (mutations) in the CLN3 segment of DNA (gene) have been attributed to juvenile Batten disease. The pathological hallmark of juvenile Batten is a buildup of lipopigment in the body's tissues. It is not known why lipopigment accumulates or why brain and eventually, heart cells are selectively damaged. It is, however, clear that we need disease-specific tools that reflect human disease in order to figure this out and to build therapy.

NYSCF is a world leader in stem cell research and production with a mission to find cures for the devastating diseases of our time, including juvenile Batten disease. NYSCF has developed the NYSCF Global Stem Cell ArrayTM, an automated robotic technology that standardizes and scales stem cell production and differentiation, enabling the manufacture and analysis of large numbers of identical cells from skin samples of patients. The Array technology allows for the production of large-scale iPS cells that have the potential to become any cell type in the body.

This collaboration brings together the expertise of these two leading non-profit organizations, the support of BDSRA, and the participation of affected families, to create and make available to researchers, juvenile Batten disease iPS cell lines. Building on the NYSCF Research Institutes leading stem cell expertise and unique automated technology and analytics, while taking advantage of the tremendous resources and expertise of BBDF, BDSRA and affected families, this collaboration will move research

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The New York Stem Cell Foundation Partners With Beyond Batten Disease Foundation to Fight Juvenile Batten Disease

PUBLIC RELEASE DATE:

23-Jul-2014

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

NEW YORK, NY -- The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) have partnered to develop stem cell resources to investigate and explore new treatments and ultimately find a cure for juvenile Batten disease, a fatal illness affecting children.

NYSCF scientists will create induced pluripotent stem (iPS) cell lines from skin samples of young people affected by juvenile Batten disease as well as unaffected family members. IPS cell lines are produced by artificially "turning back the clock" on skin cells to a time when they were embryonic-like and capable of becoming any cell in the body. Reprogramming juvenile Batten iPS cells to become brain and heart cells will provide the infrastructure needed to investigate what is going wrong with the cells adversely affected by the disease. Thus far, efforts to study juvenile Batten disease have been done using rodent models or human skin cells, neither of which accurately mimic the disease in the brain, leaving researchers without proper tools to study the disease or a solid platform for testing drugs that prevent, halt, or reverse its progression. This will be the largest and first genetically diverse collection of human iPS cells for a pediatric brain disease.

In addition to working with BBDF to actively recruit patients and families to donate skin samples, Batten Disease Support and Research Association (BDSRA) is providing resources and technical support, spreading awareness among academic scientists, and notifying its Pharmaceutical partners. Together, BBDF and BDSRA will ensure that juvenile Batten disease and other researchers are aware of and utilize the 48 stem cell lines resulting from this collaboration to further juvenile Batten disease research worldwide.

"We know the genetic mutations associated with juvenile Batten disease. This partnership will result in stem cell models of juvenile Batten, giving researchers an unprecedented look at how the disease develops, speeding research towards a cure," said Susan L. Solomon, NYSCF Chief Executive Officer.

"Working with NYSCF to generate functional neuronal subtypes from patients and families is a stellar example of one of our key strategies in the fight against juvenile Batten disease: creating resource technology with the potential to transform juvenile Batten disease research and accelerate our timeline to a cure," said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Juvenile Batten disease begins in early childhood between the ages of five and ten. Initial symptoms typically begin with progressive vision loss, followed by personality changes, behavioral problems, and slowed learning. These symptoms are followed by a progressive loss of motor functions, eventually resulting in wheelchair use and premature death. Seizures and psychiatric symptoms can develop at any point in the disease.

Juvenile Batten disease is one disorder in a group of rare, fatal, inherited disorders known as Batten disease. Over 40 different errors (mutations) in the CLN3 segment of DNA (gene) have been attributed to juvenile Batten disease. The pathological hallmark of juvenile Batten is a buildup of lipopigment in the body's tissues. It is not known why lipopigment accumulates or why brain and eventually, heart cells are selectively damaged. It is, however, clear that we need disease-specific tools that reflect human disease in order to figure this out and to build therapy.

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NYSCF partners with Beyond Batten Disease Foundation to fight juvenile Batten disease

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Newswise Researchers at the National Institutes of Health have developed a technique that will speed up the production of stem-cell derived tissues. The method simultaneously measures the expression of multiple genes, allowing scientists to quickly characterize cells according to their function and stage of development. The technique will help the researchers in their efforts to use patients skin cells to regenerate retinal pigment epithelium (RPE)a tissue in the back of the eye that is affected in several blinding eye diseases. It will also help the scientists search for drugs for personalized treatments.

Progress in stem cell-based therapies has been limited by our capacity to authenticate cells and tissues, said Kapil Bharti, Ph.D., a Stadtman Investigator in the Unit on Ocular and Stem Cell Translational Research at the National Eye Institute (NEI), a part of NIH. This assay expands that capacity and streamlines the process.

The assay was described in a recent issue of Stem Cells Translational Medicine.

The RPE is a single layer of cells that lies adjacent to the retina, where the light-sensitive photoreceptors commonly called rods and cones are located. The RPE supports photoreceptor function. Several diseases cause the RPE to break down, which in turn leads to the loss of photoreceptors and vision.

The stem cells Dr. Bharti is using to make RPE are induced pluripotent (iPS) stem cells, which are produced by reverting mature cells to an immature state, akin to embryonic stem cells. iPS cells can be derived from a patients skin or blood cells, coaxed into other cell types (such as neurons or muscle), and in theory, re-implanted without causing immune rejection.

To verify the identity of RPE made from iPS cells, scientists use microscopy to ensure the tissue looks like RPE and physiological assays to ensure the tissue behaves like RPE. They also use a technique called quantitative RT-PCR to measure the expression of genes that indicate ongoing cell development and function. For example, expression of the gene SOX2 is much higher in iPS cells than mature RPE.

But quantitative RT-PCR only permits the simultaneous measurement of a few genes per sample. Dr. Bharti teamed up with Marc Ferrer, Ph.D., of NIHs National Center for Advancing Translational Sciences (NCATS) to develop a multiplex assaya method for simultaneously measuring multiple genes per RPE sample in a highly automated fashion. The assay is based on a commercially available platform from the biotech company Affymetrix. In the assay, tiny snippets of DNA tethered to beads are used to capture RNA moleculescreated when genes are expressed by cells in the RPE sample. Once captured, the RNA from distinct genes is labeled with a fluorescent tag.

Starting with cells from a skin biopsy, the researchers generated iPS-derived RPE and then measured the expression of eight genes that are markers of development, function, and disease. They measured RNA levels of each gene one at a time using quantitative RT-PCR and then all genes simultaneously using the multiplex assay. When compared, the results correlated.

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Gene Profiling Technique to Accelerate Stem Cell Therapies for Eye Diseases

Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. The approach, the first of its kind, takes advantage of induced pluripotent stem (iPS) cell technology to transform skin cells into retinal cells, which are then used as a patient-specific model for disease study and preclinical testing.

Using this approach, researchers led by Stephen H. Tsang, MD, PhD, showed that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) disrupts the protein that gives retinal cells their structural integrity. They also showed that the effects of these mutations can be reversed with gene therapy. The approach could potentially be used to create personalized therapies for other forms of RP, as well as other genetic diseases. The paper was published recently in the online edition of Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

"The use of patient-specific cell lines for testing the efficacy of gene therapy to precisely correct a patient's genetic deficiency provides yet another tool for advancing the field of personalized medicine," said Dr. Tsang, the Laszlo Z. Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology.

While RP can begin during infancy, the first symptoms typically emerge in early adulthood, starting with night blindness. As the disease progresses, affected individuals lose peripheral vision. In later stages, RP destroys photoreceptors in the macula, which is responsible for fine central vision. RP is estimated to affect at least 75,000 people in the United States and 1.5 million worldwide.

More than 60 different genes have been linked to RP, making it difficult to develop models to study the disease. Animal models, though useful, have significant limitations because of interspecies differences. Researchers also use human retinal cells from eye banks to study RP. As these cells reflect the end stage of the disease process, however, they reveal little about how the disease develops. There are no human tissue culture models of RP, as it would dangerous to harvest retinal cells from patients. Finally, human embryonic stem cells could be useful in RP research, but they are fraught with ethical, legal, and technical issues.

The use of iPS technology offers a way around these limitations and concerns. Researchers can induce the patient's own skin cells to revert to a more basic, embryonic stem cell-like state. Such cells are "pluripotent," meaning that they can be transformed into specialized cells of various types.

In the current study, the CUMC team used iPS technology to transform skin cells taken from two RP patients -- each with a different MFRP mutation -- into retinal cells, creating patient-specific models for studying the disease and testing potential therapies.

By analyzing these cells, the researchers found that the primary effect of MFRP mutations is to disrupt the regulation of actin, the protein that makes up the cytoskeleton, the scaffolding that gives the cell its structural integrity. "Normally, the cytoskeleton looks like a series of connected hexagons," said Dr. Tsang. "If a cell loses this structure, it loses its ability to function."

The researchers also found that MFRP works in tandem with another gene, CTRP5, and that a balance between the two genes is required for normal actin regulation.

In the next phase of the study, the CUMC team used adeno-associated viruses (AAVs) to introduce normal copies of MFRP into the iPS-derived retinal cells, successfully restoring the cells' function. The researchers also used gene therapy to "rescue" mice with RP due to MFRP mutations. According to Dr. Tsang, the mice showed long-term improvement in visual function and restoration of photoreceptor numbers.

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Patient-specific stem cells and personalized gene therapy

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Newswise A team of researchers from the University of California, San Diego School of Medicine, Oregon Health & Science University (OHSU) and Salk Institute for Biological Studies has shown for the first time that stem cells created using different methods produce differing cells. The findings, published in the July 2, 2014 online issue of Nature, provide new insights into the basic biology of stem cells and could ultimately lead to improved stem cell therapies.

Capable of developing into any cell type, pluripotent stem cells offer great promise as the basis for emerging cell transplantation therapies that address a wide array of diseases and conditions, from diabetes and Alzheimers disease to cancer and spinal cord injuries. In theory, stem cells could be created and programmed to replace ailing or absent cells for every organ in the human body.

The gold standard is human embryonic stem cells (ES cells) cultured from discarded embryos generated by in vitro fertilization, but their use has long been limited by ethical and logistical considerations. Scientists have instead turned to two other methods to create stem cells: Somatic cell nuclear transfer (SCNT), in which genetic material from an adult cell is transferred into an empty egg cell, and induced pluripotent stem cells (iPS cells), in which adult cells are reverted back to a stem cell state by artificially turning on targeted genes.

Until now, no one had directly and closely compared the stem cells acquired using these two methods. The scientists found they produced measurably different results. The nuclear transfer ES cells are much more similar to real ES cells than the iPS cells, said co-senior author Louise Laurent, PhD, assistant professor in the Department of Reproductive Medicine at UC San Diego. They are more completely reprogrammed and have fewer alterations in gene expression and DNA methylation levels that are attributable to the reprogramming process itself.

The development and use of iPS cells has grown exponentially in recent years, in no small part due to the fact that they can be generated from adult cells (often from the skin) by temporarily turning on a combination of four genes to induce the adult cells to return to a pluripotent state.

Laurent noted that iPS cell lines have been created from patients to model many different diseases and the ability to make personalized iPS cells from a patient that could be transplanted back into that patient has generated excitement because it would eliminate the need for immunosuppression.

The nuclear transfer method has been pioneered more recently by a team led by Shoukhrat Mitalipov, PhD, professor and director of the Center for Embryonic Cell and Gene Therapy at OSHU. The technique is similar to the process used in cloning, but the pluripotent cells are collected from early embryos before they develop into mature organisms.

For their comparisons, the researchers at UC San Diego, OSHU and Salk created four nuclear transfer ES cell lines and seven iPS cell lines using the same skin cells as the source of donor genetic material, then compared them to two standard human ES lines. All 13 cell lines were shown to be pluripotent using a battery of standard tests.

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New Reprogramming Method Makes Better Stem Cells

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2-Jul-2014

Contact: Kristina Grifantini press@salk.edu Salk Institute

LA JOLLA-Researchers around the world have turned to stem cells, which have the potential to develop into any cell type in the body, for potential regenerative and disease therapeutics.

Now, for the first time, researchers at the Salk Institute, with collaborators from Oregon Health & Science University and the University of California, San Diego, have shown that stem cells created using two different methods are far from identical. The finding could lead to improved avenues for developing stem cell therapies as well as a better understanding of the basic biology of stem cells.

The researchers discovered that stem cells created by moving genetic material from a skin cell into an empty egg cell-rather than coaxing adult cells back to their embryonic state by artificially turning on a small number of genes-more closely resemble human embryonic stem cells, which are considered the gold standard in the field.

"These cells created using eggs' cytoplasm have fewer reprogramming issues, fewer alterations in gene expression levels and are closer to real embryonic stem cells," says co-senior author Joseph R. Ecker, professor and director of Salk's Genomic Analysis Laboratory and co-director of the Center of Excellence for Stem Cell Genomics. The results of the study were published today in Nature.

Human embryonic stem cells (hESCs) are directly pulled from unused embryos discarded from in-vitro fertilization, but ethical and logistical quandaries have restricted their access. In the United States, federal funds have limited the use of hESCs so researchers have turned to other methods to create stem cells. Most commonly, scientists create induced pluripotent stem (iPS) cells by starting with adult cells (often from the skin) and adding a mixture of genes that, when expressed, regress the cells to a pluripotent stem-cell state. Researchers can then coax the new stem cells to develop into cells that resemble those in the brain or in the heart, giving scientists a valuable model for studying human disease in the lab.

Over the past year, a team at OHSU built upon a technique called somatic cell nuclear transfer (the same that is used for cloning an organism, such as Dolly the sheep) to transplant the DNA-containing nucleus of a skin cell into an empty human egg, which then naturally matures into a group of stem cells.

Ecker, holder of the Salk International Council Chair in Genetics, teamed up with Shoukhrat Mitalipov, developer of the new technique and director of the Center for Embryonic Cell and Gene Therapy at OHSU, and UCSD assistant professor Louise Laurent to carry out the first direct comparison of the two approaches. The scientists created four lines of nuclear transfer stem cells all using eggs from a single donor, along with seven lines of iPS cells and two lines of the gold standard hESCs. All cell lines were shown to be able to develop into multiple cell types and had nearly identical DNA content contained within them.

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Some stem cell methods closer to 'gold standard' than others

Salk Institute scientist Joseph Ecker holds a flow cell slide used in a genome sequencing machine. Ecker and colleagues compared the genomes of two kinds of artificial embryonic stem cells for a study comparing their quality.

In a setback for hopes of therapy with a promising kind of artificial embryonic stem cells, a study published in the journal Nature has found that these "induced pluripotent stem cells" have serious quality issues.

However, scientists who performed the study, including researchers from the Salk Institute and UC San Diego, say it should be possible to improve the quality of these IPS cells. They say lessons can be learned from studying a newer technique of making human embryonic stem cells through nuclear transfer, the same technology used to create Dolly the cloned sheep.

In addition, the study does not prove that the quality problems will affect therapy with the cells, said scientists who examined the study. That remains to be tested.

The IPS cells are made from skin cells treated with "reprogramming" factors that turn back the clock, so they very closely resemble embryonic stem cells. The hope is that these IPS cells could be differentiated into cells that can repair injuries or relieve diseases. Because they can be made from a patient's own cells, the cells are genetically matched, reducing worries of immune rejection.

In San Diego, scientists led by Jeanne Loring at The Scripps Research Institute have created IPS cells from the skin cells of Parkinson's disease patients, and turned the IPS cells into neurons that produce dopamine. They hope to get approval next year to implant these cells into the patients, relieving symptoms for many years. The project is online under the name Summit4StemCell.org.

A major concern is that IPS cells display abnormal patterns of gene activation and repression. This is controlled by a process called methylation. This process adds chemicals called methyl groups to DNA, but these "epigenetic" changes do not change the underlying DNA sequence. Methylation represses gene function; removing the methyl groups, or demethylation, activates them.

The Nature study was led by Shoukhrat Mitalipov of Oregon Health & Scence University. Mitalipov made headlines last year for applying nuclear transfer to derive human embryonic stem cells, the first time this has been achieved in human cells. These cells can be made to be a near-perfect genetic match to the patient, and their quality closely resembles those of true embryonic stem cells.

"We know that the embryonic stem cells are the gold standard, and we've been always trying to make patient-matched cells that would match the gold standard," Mitalipov said. "And at this point it looks like the NT (nuclear transfer) cells produce exactly those cells that would be best."

Nuclear transfer involves placing a nucleus from a skin cell into an egg cell that has had its nucleus removed. The cell is then stimulated, and starts dividing in the same way a fertilized egg cell divides to form an embryo.

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Artificial embryonic stem cells have quality problems: study

On a brilliant day in April, tens of thousands of baseball fans stream past Jonathan Thomas's office towards AT&T Park for the first home game of the San Francisco Giants 2014 season. Thomas's standing desk faces away from the window, but the cheering throngs are never far from his mind.

Thomas chairs the board of the California Institute for Regenerative Medicine (CIRM), the US$3-billion agency hailed by scientists around the world for setting a benchmark for stem-cell research funding. But scientists will not be the ones who decide what becomes of CIRM when the cash runs out in 2017. Instead, it will be the orange-and-black-clad masses walking past Thomas's window. And to win their support, Thomas knows that the agency needs to prove that their collective investment has been worthwhile. We need to drive as many projects to the patient as soon as possible, he says.

Californians voted CIRM into existence in 2004, making it the largest funder of stem-cell work in the world. The money the proceeds of bond sales that must be repaid with $3 billion in interest by taxpayers helped to bring 130 scientists to the state, and created several thousand jobs there. It has funded research that led to the publication of more than 1,700 papers, and it has contributed to five early clinical trials.

The institute has navigated a difficult path, however. CIRM had to revamp its structure and practices in response to complaints about inefficiency and potential conflicts of interest. It has also had to adapt its mission to seismic shifts in stem-cell science.

Now, ten years after taking off, the agency is fighting for its future. It has a new president, businessman Randal Mills, who replaces biologist Alan Trounson. Its backers have begun to chart a course for once again reaching out to voters, this time for $5 billion (with another $5 billion in interest) in 2016. And it is under intense pressure to produce results that truly matter to the public.

Whether or not CIRM succeeds, it will serve as a test bed for innovative approaches to funding. It could be a model for moving technologies to patients when conventional funding sources are not interested.

Much of what is celebrated and lamented about CIRM can be traced back to the Palo Alto real-estate developer who conceived of it: Robert Klein. Although officially retired from CIRM he chaired the board from 2004 to 2011 (see 'State of funding') Klein's office is adorned with mementos of the agency: a commemorative shovel from the groundbreaking of a CIRM-funded stem-cell research centre, and a photo of him with former governor Arnold Schwarzenegger at the ribbon-cutting ceremony.

Liz Hafalia/San Francisco Chronicle/Polaris/eyevine

Patient advocates and parents at a 2012 meeting in which US$100 million in CIRM grants were approved.

It was Klein's idea to ask voters to support stem-cell research in 2004, through a ballot measure called Proposition 71. When he succeeded, CIRM instilled a kind of euphoria in stem-cell scientists, who were at the time still reeling from a 2001 decree by then-President George W. Bush that severely limited federal funding for embryonic-stem-cell research. California's commitment removed this roadblock and revealed that many in the state and the country supported the research.

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Stem cells: Hope on the line

Durham, NC (PRWEB) June 27, 2014

A new study released today in STEM CELLS Translational Medicine suggests a new way to produce endothelial progenitor cells in quantities large enough to be feasible for use in developing new cancer treatments.

Endothelial progenitor cells (EPCs) are rare stem cells that circulate in the blood with the ability to differentiate into the cells that make up the lining of blood vessels. With an intrinsic ability to home to tumors, researchers have focused on them as a way to deliver gene therapy straight to the cancer. However, the challenge has been to collect enough EPCs for this use.

This new study, by researchers at the Institute of Bioengineering and Nanotechnology, National University of Singapore and Zhejiang University led by Shu Wang, Ph.D., explored whether human induced pluripotent stem cells (iPSCs) could provide the answer. iPSCs, generated from adult cells, can propagate indefinitely and give rise to every other cell type in the body, much like human embryonic stem cells, which are considered the gold standard for stem cell therapy.

However, human iPS cells can be generated relatively easily through reprogramming, a procedure that circumvents the bioethical controversies associated with deriving embryonic stem cells from human embryos, Dr. Wang said.

After inducing human iPS cells to differentiate into the EPCs, the research team compared the stability and reliability of the induced EPCs with regular EPCs by injecting them into mice with breast cancer that had metastasized (traveled) to the lungs. The results showed that their induced EPCs retained the intrinsic ability to home to tumors, just as regular EPCs do. They also did not promote tumor growth or metastasis.

We next tested the induced EPCs therapeutic potential by infusing them with an anticancer gene and injecting them into the mice, Dr. Wang said. The results indicated that the tumors were reduced and the animals survival rates increased.

Since this approach may use patient's own cells to prepare cellular therapeutics and is based on non-toxic immunotherapy, it holds potential for translation to clinical application and may be particularly valuable as a new type of anti-metastatic cancer therapy.

With the increasing potential of using EPCs as cancer therapeutics, it is important to have a reliable and stable supply of human EPCs, said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. This study demonstrates the feasibility of generating EPs from early-passage human iPS cells.

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New Stem Cell Production Method Could Clear Way for Anticancer Gene Therapy

TOKYO: Japanese stem cell scientists have succeeded in slowing the deterioration of mice with motor neuron disease, possibly paving the way for eventual human treatment, according to a new paper.

A team of researchers from the Kyoto University and Keio University transplanted specially created cells into mice with amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's, or motor neuron disease.

The progress of the creatures' neurological degeneration was slowed by almost eight per cent, according to the paper, which was published on Thursday in the scholarly journal Stem Cell Reports.

ALS is a disorder of motor neurons -- nerves that control movement -- leading to the loss of the ability to control muscles and their eventual atrophy.

While it frequently has no effect on cognitive function, it progresses to affect most of the muscles in the body, including those used to eat and breathe.

British theoretical physicist Stephen Hawking has been almost completely paralysed by the condition.

In their study, the Japanese team used human "iPS" -- induced pluripotent stem cells, building-block cells akin to those found in embryos, which have the potential to turn into any cell in the body.

From the iPS cells they created special progenitor cells and transplanted them into the lumbar spinal cord of ALS mice.

Animals that had been implanted lived 7.8 per cent longer than the control group without the procedure, the paper said.

"The results demonstrated the efficacy of cell therapy for ALS by the use of human iPSCs (human induced pluripotent stem cells) as cell source," the team said in the paper.

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Cell scientists slow degeneration in motor neuron mice

Friday 27 June 2014 22.31

Japanese stem cell scientists have succeeded in slowing the deterioration of mice with motor neurone disease, possibly paving the way for eventual human treatment.

A team of researchers from the Kyoto University and Keio University transplanted specially created cells into mice with amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's, or motor neurone disease.

The progress of the creatures' neurological degeneration was slowed by almost eight percent, according to the paper, which was published Thursday in the scholarly journal Stem Cell Reports.

ALS is a disorder of motor neurones -- nerves that control movement -- leading to the loss of the ability to control muscles and their eventual atrophy.

While it frequently has no effect on cognitive function, it progresses to affect most of the muscles in the body, including those used to eat and breathe.

British theoretical physicist Stephen Hawking has been almost completely paralysed by the condition.

In their study, the Japanese team used human "iPS" -- induced pluripotent stem cells, building-block cells akin to those found in embryos, which have the potential to turn into any cell in the body.

From the iPS cells they created special progenitor cells and transplanted them into the lumbar spinal cord of ALS mice.

Animals that had been implanted lived 7.8% longer than the control group without the procedure, the paper said.

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Scientists slow degeneration in motor neurone mice

The Board of Directors of Asterias is honored that Pedro elected to join Asterias as CEO and is very pleased that he also chose to make a significant financial commitment to the company, said Alfred D. Kingsley, Chairman of the Asterias Board of Directors. With Pedro at the helm, Asterias is focused on its efforts to develop and commercialize therapies that have the potential to treat patients with serious unmet medical needs. In particular, Pedro will lead Asterias through the process of advancing its lead clinical-stage programs, AST-OPC1 for the treatment of spinal cord injury and the AST-VAC2 allogeneic dendritic cell cancer immunotherapy platform.

I believe in Asterias technology, its therapeutic programs, and its talented employees and am committed to making the company a success, remarked Mr. Lichtinger. My efforts as the companys CEO will focus on developing innovative therapies for critically ill and chronically ill patients, and creating significant value over time for Asterias shareholders.

About Asterias

Asterias Biotherapeutics is a biotechnology company focused on the emerging field of regenerative medicine. Our core technologies center on stem cells capable of becoming all of the cell types in the human body, a property called pluripotency. We plan to develop therapies based on pluripotent stem cells to treat diseases or injuries in a variety of medical fields, with an initial focus on the therapeutic applications of oligodendrocyte progenitor cells (AST-OPC1) and antigen-presenting dendritic cells (AST-VAC1 and AST-VAC2) for the fields of neurology and oncology respectively. AST-OPC1 was tested for treatment of spinal cord injury in the worlds first Phase 1 clinical trial using human embryonic stem cell-derived cells. We plan to seek FDA clearance to reinitiate clinical testing of AST-OPC1 in spinal cord injury this year, and are also evaluating its function in nonclinical models of multiple sclerosis and stroke. AST-VAC1 and AST-VAC2 are dendritic cell-based vaccines designed to immunize cancer patients against telomerase, a protein abnormally expressed in over 95% of human cancer types. AST-VAC2 differs from AST-VAC1 in that the dendritic cells presenting telomerase to the immune system are produced from human embryonic stem cells instead of being derived from human blood.

In October of 2013, Asterias acquired the cell therapy assets of Geron Corporation. These assets included INDs for the clinical stage AST-OPC1 and AST-VAC1 programs, banks of cGMP-manufactured AST-OPC1 drug product, cGMP master and working cell banks of human embryonic stem cells, over 400 patents and patent applications filed worldwide including broad issued claims to fundamental platform technologies for the scalable growth of pluripotent stem cells and compositions of matter for several hESC-derived therapeutic cell types, research cell banks, customized reagents and equipment, and various assets relating to the AST-VAC2 program and preclinical programs in cardiology and orthopedics.

Asterias is a member of the BioTime family of companies.

Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

About BioTime

BioTime is a biotechnology company engaged in research and product development in the field of regenerative medicine. Regenerative medicine refers to therapies based on stem cell technology that are designed to rebuild cell and tissue function lost due to degenerative disease or injury. BioTimes focus is on pluripotent stem cell technology based on human embryonic stem (hES) cells and induced pluripotent stem (iPS) cells. hES and iPS cells provide a means of manufacturing every cell type in the human body and therefore show considerable promise for the development of a number of new therapeutic products. BioTimes therapeutic and research products include a wide array of proprietary PureStem progenitors, HyStem hydrogels, culture media, and differentiation kits. BioTime is developing Renevia (a HyStem product) as a biocompatible, implantable hyaluronan and collagen-based matrix for cell delivery in human clinical applications, and is planning to initiate a pivotal clinical trial around Renevia, in 2014. In addition, BioTime has developed Hextend, a blood plasma volume expander for use in surgery, emergency trauma treatment and other applications. Hextend is manufactured and distributed in the U.S. by Hospira, Inc. and in South Korea by CJ HealthCare Corporation, under exclusive licensing agreements.

BioTime is also developing stem cell and other products for research, therapeutic, and diagnostic use through its subsidiaries:

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BioTime Subsidiary Asterias Biotherapeutics Announces Investment by CEO