UC San Diego researchers receive new CIRM funding

Public release date: 25-May-2012 [ | E-mail | Share ]

Contact: Scott LaFee slafee@ucsd.edu 619-543-6163 University of California - San Diego

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

With a $1.8 million award, Lawrence Goldstein, PhD, professor in the Department of Cellular and Molecular Medicine, Howard Hughes Medical Institute Investigator and director of the UC San Diego Stem Cell Program, and colleagues will continue their work developing new methods to find and test drug candidates for Alzheimer's disease (AD). Currently, there is no effective treatment for AD. The researchers screen novel candidates using purified human brain cells made from human reprogrammed stem cells. Already, they have discovered that these human brain cells exhibit a unique biochemical behavior that indicates early development of AD in a dish.

Mark H. Tuszynski, MD, PhD, professor of neurosciences and director of the Center for Neural Repair at UC San Diego, and colleagues seek to develop more potent stem cell-based treatments for spinal cord injuries. By combining grafts of neural stem cells with scaffolds placed at injury sites, the researchers have reported substantial progress in restoring functional improvement in impaired animal models. The new $4.6 million grant will fund work to identify the optimal human neural stem cells for preclinical development and, in an unprecedented step, test this treatment in appropriate preclinical models of spinal cord injury, providing the strongest validation for human translation.

Amyotrophic lateral sclerosis or ALS (Lou Gehrig's disease) is a progressive neurological condition that is currently incurable. Gene Yeo, PhD, assistant professor in the Department of Cellular and Molecular Medicine, and colleagues will use a $1.6 million grant to exploit recent discoveries that specific mutations in RNA-binding proteins cause neuronal dysfunction and death. They will use neurons generated from patient cells containing the mutations to identify the unique RNA "signature" of these doomed neurons and screen for drug-like compounds that bypass the mutations to correct the RNA signature to obtain healthy neurons.

Eric David Adler, MD, an associate clinical professor of medicine and cardiologist, studies heart failure, including the use of stem cells to treat it. His $1.7 million award will fund research into Danon disease, a type of inherited heart failure that frequently kills patients by their 20s. Adler and colleagues will turn stem cells created from skin cells of patients with Danon disease into heart cells, then screen hundreds of thousands of drug candidates for beneficial effects. The most promising drugs will subsequently be tested on mice with a genetic defect similar to Danon disease, with the ultimate goal of identifying a suitable candidate for human clinical trials. The research may have broader applications for other conditions with similar pathogenesis, such as cancer and Parkinson's disease.

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UC San Diego researchers receive new CIRM funding

State awards stem cell grants to medical researchers

SACRAMENTO California's stem cell agency today approved two grants to UC Davis Health System researchers for their innovative work in regenerative medicine.

Kyriacos A. Athanasiou, distinguished professor of orthopaedic surgery and professor and chair of biomedical engineering, and the Child Family Professor of Engineering at UC Davis, is investigating the use of skin-derived stem cells to heal cartilage injuries and debilitating conditions of the knee such as osteoarthritis.

W. Douglas Boyd, professor of surgery, plans to further refine a novel approach to treating cardiovascular injuries suffered during a heart attack by using stem cells and a tissue-like scaffold to repair cardiac damage.

The pair received individual grants totaling approximately $6.6 million from the California Institute for Regenerative Medicine's (CIRM) governing board.

Athanasiou's and Boyd's multi-year grants were among the proposals submitted to CIRM for its third round of Early Translational Awards, which are intended to enable clinical therapies to be developed more rapidly.

"Both of these scientists are conducting exciting research that could have far-reaching implications in health care," said Jan Nolta, director of the UC Davis Institute for Regenerative Cures and the university's stem cell program director. "Dr. Athanasiou is bioengineering new cartilage that could have the same physiological integrity as the cartilage a person is born with. Dr. Boyd is developing a treatment that uses a paper-thin patch embedded with stem cells to harness their regenerative powers to repair damaged heart muscle."

Boyd, who's a pioneering cardiothoracic surgeon, pointed out in his CIRM proposal that heart disease is the nation's number-one cause of death and disability. An estimated 16.3 million Americans over the age of 20 suffer from coronary heart disease, which in 2007 accounted for an estimated 1 in 6 deaths in the U.S. Boyd plans to use bone-marrow derived stem cells -- known as mesenchymal stem cells -- in combination with a bioengineered framework known as an extracellular matrix, to regenerate damaged heart tissue, block heart disease and restore cardiac function, something currently not possible except in cases of a complete and very invasive heart transplant.

An expert in biomedical engineering, Athanasiou is focusing on developing a cellular therapy using stem cells created from an individual's own skin -- known as autologous skin-derived stem cells -- which have shown great promise in animal models. He plans to use the new funding to conduct extensive toxicology and durability tests to determine the technique's long-term safety and efficacy. Such tests are among the many steps needed to advance toward human clinical trials.

Cartilage is the slippery tissue that covers the ends of bones in joints, allowing bones to glide over each other and absorbing the shock of movement. Cartilage defects from injuries and lifelong wear and tear can eventually degenerate into osteoarthritis. According to the National Institute of Arthritis and Musculoskeletal and Skin Diseases, osteoarthritis is the most common form of arthritis and affects an estimated 27 million Americans over the age of 25.

"For anyone suffering from osteoarthritis or other debilitating cartilage conditions, Dr. Athanasiou's goal of using stem cells to regenerate new tissue could have enormous quality-of-life and economic benefits," said Nolta, who is the recipient of a prior translational grant from CIRM to develop potential therapies for Huntington's disease . "Dr. Boyd's work is equally promising because he's using a bioengineered structure to encourage cardiac tissue repair, which could have important benefits in the treatment of heart disease."

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State awards stem cell grants to medical researchers

5 scientists receive stem-cell research grants

Five scientists from the University of California, San Diego and its School of Medicine have been awarded almost $12 million in new grants from the California Institute for Regenerative Medicine (CIRM) to conduct stem cell-based research into regenerating spinal cord injuries, repairing gene mutations that cause amyotrophic lateral sclerosis and finding new drugs to treat heart failure and Alzheimer's disease.

The awards mark the third round of funding in CIRM's Early Translational Awards program, which supports projects that are in the initial stages of identifying drugs or cell types that could become disease therapies. More than $69 million in awards were announced yesterday, including funding for first-ever collaboratively funded research projects with China and the federal government of Australia.

"With these new awards, the agency now has 52 projects in 33 diseases at varying stages of working toward clinical trials," said Jonathan Thomas, JD, PhD and CIRM governing board chair. "Californians should take pride in being at the center of this worldwide research leading toward new cures. These projects represent the best of California stem cell science and the best international experts who, together, will bring new therapies for patients."

The five new UC San Diego awards are:

CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research at California universities and research institutions and called for the establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities.

The May 24 grants bring UC San Diego's total to more than $112 million in CIRM funding since the first awards in 2006.

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5 scientists receive stem-cell research grants

From stem cell to brain cell: New technique mimics the brain

ScienceDaily (May 24, 2012) 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 brain's 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 brain's 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 brain's 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.

"We have used the new protocol to make dopamine neurons, the type of neuron that is affected by Parkinson's disease, and for the first time, we are seriously talking about these cells as being good enough to move forward for transplantation in patients. The next step is to test the process on a larger scale and to carry out more pre-clinical safety tests," explains Malin Parmar, research team leader.

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From stem cell to brain cell: New technique mimics the brain

City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells

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

City of Hope was granted a $5,217,004 early translational research award by the California Institute for Regenerative Medicine (CIRM) to support the development of a T cell-based immunotherapy that re-directs a patients own immune response against glioma stem cells. City of Hope has been awarded more than $49.7 million in grant support from CIRM since awards were first announced in 2006.

City of Hope is a pioneer in T cell immunotherapy research, helping to develop genetically modified T cells as a treatment for cancer. This strategy, termed adoptive T cell therapy, focuses on redirecting a patients immune system to specifically target tumor cells, and has the potential to become a promising new approach for treatment of cancer.

In this research, we are genetically engineering a central memory T cell that targets proteins expressed by glioma stem cells, said Stephen J. Forman, M.D., Francis and Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation and director of the T Cell Immunotherapy Research Laboratory. Central memory T cells have the potential to establish a persistent, lifelong immunity to help prevent brain tumors from recurring.

The American Cancer Society estimates that more than 22,000 people in the U.S. will be diagnosed with a brain tumor this year, and 13,700 will die from the disease. Glioma is a type of brain tumor that is often difficult to treat and is prone to recurrence. Currently, less than 20 percent of patients with malignant gliomas are living five years after their diagnosis. This poor prognosis is largely due to the persistence of tumor-initiating cancer stem cells, a population of malignant cells similar to normal stem cells in that they are able to reproduce themselves indefinitely. These glioma stem cells are highly resistant to chemotherapy and radiation treatments, making them capable of re-establishing new tumors.

Researchers at City of Hope previously have identified several proteins as potential prime targets for the development of cancer immunotherapies, such as interleukin 13 receptor alpha 2, a receptor found on the surface of glioma cells, and CD19, a protein that is active in lymphoma and leukemia cells. Both investigational therapies are currently in phase I clinical trials. Forman is the principal investigator for the newly granted study which will develop a T cell that targets different proteins expressed by glioma stem cells. Christine Brown, Ph.D., associate research professor, serves as co-principal investigator, and Michael Barish, Ph.D., chair of the Department of Neurosciences, and Behnam Badie, M.D., director of the Brain Tumor Program, serve as co-investigators on the project.

Because cancer stem cells are heterogeneous, our proposed therapy will target multiple antigens to cast as wide a net as possible over this malignant stem cell population, said Brown.

While in this effort, we are targeting a neurological cancer, our approach will lead to future studies targeting other cancers, including those that metastasize to the brain, added Barish.

The CIRM grant will help us to build a targeted T cell therapy against glioma that can offer lasting protection, determine the best way to deliver the treatment, establish an efficient process to manufacture these T cells for treatment, and get approval for a human clinical trial, said Badie.

City of Hope is also a collaborative partner providing process development, stem cell-derived cell products and regulatory affairs support in two other CIRM-funded projects that received early translational research grants. Larry Couture, Ph.D., senior vice president of City of Hopes Sylvia R. & Isador A. Deutch Center for Applied Technology Development and director of the Center for Biomedicine & Genetics, is working with Stanford University and Childrens Hospital of Orange County Research Institute on their respective projects.

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City of Hope Receives $5 Million Grant to Develop T Cell Treatment Targeting Brain Tumor Stem Cells

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

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

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

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

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

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

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

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

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

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

Professor Gepstein said:

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

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

UCI stem cell researcher to receive $4.8 million in state funding

CIRM grant will advance work on multiple sclerosis treatment

Irvine, Calif., May 24, 2012

A UC Irvine immunologist will receive $4.8 million to create a new line of neural stem cells that can be used to treat multiple sclerosis.

The California Institute for Regenerative Medicine awarded the grant Thursday, May 24, to Thomas Lane of the Sue & Bill Gross Stem Cell Research Center at UCI to support early-stage translational research.

CIRMs governing board gave 21 such grants worth $69 million to 11 institutions statewide. The funded projects are considered critical to the institutes mission of translating basic stem cell discoveries into clinical cures. They are expected to either result in candidate drugs or cell therapies or make significant strides toward such treatments, which can then be developed for submission to the Food & Drug Administration for clinical trial.

Lanes grant brings total CIRM funding for UCI to $76.65 million.

I am delighted that CIRM has chosen to support our efforts to advance a novel stem cell-based therapy for multiple sclerosis, said Peter Donovan, director of the Sue & Bill Gross Stem Cell Research Center.

MS is a disease of the central nervous system caused by inflammation and loss of myelin, a fatty tissue that insulates and protects nerve cells. Current treatments are often unable to stop the progression of neurologic disability most likely due to irreversible nerve destruction resulting from myelin deficiencies. The limited ability of the body to repair damaged nerve tissue highlights a critically important and unmet need for MS patients.

In addressing this issue, Lane who also directs UCIs Multiple Sclerosis Research Center will target a stem cell treatment that will not only halt ongoing myelin loss but also encourage the growth of new myelin that can mend damaged nerves.

Our preliminary data are very promising and suggest that this goal is possible, said Lane, a Chancellors Fellow and professor of molecular biology & biochemistry. Research efforts will concentrate on refining techniques for production and rigorous quality control of transplantable cells generated from high-quality human pluripotent stem cell lines, leading to the development of the most therapeutically beneficial cell type for eventual use in patients with MS.

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UCI stem cell researcher to receive $4.8 million in state funding

Stem cells take root in drug development

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

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

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

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

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

About Biostem U.S., Corporation

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

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

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