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New muscular dystrophy treatment approach developed using human stem cells

By Dr. Matthew Watson

ScienceDaily (May 4, 2012) Researchers from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process that -- for the first time -- makes the production of human muscle cells from stem cells efficient and effective.

The research, published May 4 in Cell Stem Cell, outlines the strategy for the development of a rapidly dividing population of skeletal myogenic progenitor cells (muscle-forming cells) derived from induced pluripotent (iPS) cells. iPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

According to U of M researchers -- who were also the first to use ES cells from mice to treat muscular dystrophy -- there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells. This lag has dramatically limited the development of cell therapies or clinical trials for human patients.

The latest research from the U of M provides the proof-of-principle for treating muscular dystrophy with human iPS cells, setting the stage for future human clinical trials.

"One of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response," said principal investigator Rita Perlingeiro, Ph.D., associate professor of medicine in the Medical School's Division of Cardiology. "Up until now, deriving engraftable skeletal muscle stem cells from human pluripotent stem cells hasn't been possible. Our results demonstrate that it is indeed possible and sets the stage for the development of a clinically meaningful treatment approach."

Upon transplantation into mice suffering from muscular dystrophy, human skeletal myogenic progenitor cells provided both extensive and long-term muscle regeneration which resulted in improved muscle function.

To achieve their results, U of M researchers genetically modified two well-characterized human iPS cell lines and an existing human ES cell line with the PAX7 gene. This allowed them to regulate levels of the Pax7 protein, which is essential for the regeneration of skeletal muscle tissue after damage. The researchers found this regulation could prompt nave ES and iPS cells to differentiate into muscle-forming cells.

Up until this point, researchers had struggled to make muscle efficiently from ES and iPS cells. PAX7 -- induced at exactly the right time -- helped determine the fate of human ES and iPS cells, pushing them into becoming human muscle progenitor cells.

Once Dr. Perlingeiro's team was able to pinpoint the optimal timing of differentiation, the cells were well suited to the regrowth needed to treat conditions such as muscular dystrophy. In fact, Pax7-induced muscle progenitors were far more effective than human myoblasts at improving muscle function. Myoblasts, which are cell cultures derived from adult muscle biopsies, had previously been tested in clinical trials for muscular dystrophy, however the myoblasts did not persist after transplantation.

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UCLA scientists measure communication between stem cell-derived motor neurons and muscle cells

By raymumme

Public release date: 4-May-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-206-2805 University of California - Los Angeles Health Sciences

In an effort to identify the underlying causes of neurological disorders that impair motor functions such as walking and breathing, UCLA researchers have developed a novel system to measure the communication between stem cell-derived motor neurons and muscle cells in a Petri dish.

The study provides an important proof of principle that functional motor circuits can be created outside of the body using stem cell-derived neurons and muscle cells, and that the level of communication, or synaptic activity, between the cells could be accurately measured by stimulating motor neurons with an electrode and then measuring the transfer of electrical activity into the muscle cells to which the motor neurons are connected.

When motor neurons are stimulated, they release neurotransmitters that depolarize the membranes of muscle cells, allowing the entry of calcium and other ions that cause them to contract. By measuring the strength of this activity, one can get a good estimation of the overall health of motor neurons. That estimation could shed light on a variety of neurodegenerative diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis, or Lou Gehrig's disease, in which the communication between motor neurons and muscle cells is thought to unravel, said study senior author Bennett G. Novitch, an assistant professor of neurobiology and a scientist with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The findings of the study appear May 4, 2012 in PLoS ONE, a peer-reviewed journal of the Public Library of Science.

"Now that we have this method to measure the strength of the communications between motor neurons and muscle cells, we may be able to begin exploring what happens in the earliest stages of motor neuron disease, before neuronal death becomes prevalent," Novitch said. "This can help us to pinpoint where things begin to go wrong and provide us with new clues into therapeutic interventions that could improve synaptic communication and promote neuronal survival."

Novitch said the synaptic communication activity his team was able to create and measure using mouse embryonic stem cell-derived motor neurons and muscle cells looks very similar what is seen in a mouse, validating that their model is a realistic representation of what is happening in a living organism.

"That gives us a good starting point to try to model what happens in cells that harbor genetic mutations that are associated with neurodegenerative diseases,. To do that, we had to first define an activity profile of normal synaptic communication," he said. "Some research suggests that a breakdown in this communication can be an early indication of disease progression or possibly an initiating event. Neurons that cannot effectively transmit information to muscle cells will eventually withdraw their contacts, causing both the neurons and muscle cells to degenerate over time. Hopefully, we can now create disease models that will allow us to study what is happening."

In this study, Novitch and his team, led by Joy Umbach, an associate professor of molecular and medical pharmacology, used mouse embryonic stem cells to create the motor neurons and previously established lines of muscle precursors to produce muscle fibers. They put both cells together in a Petri dish, and the cells were cultured in such a way to encourage communication. Novitch said the team wanted to see if they would naturally form synaptic contacts and whether or not there was neural transmission between them.

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Stem cell therapy for WCMS student has remarkable results

By JoanneRUSSELL25

When Tyler was born, the umbilical cord was wrapped around his neck, causing a lack of oxygen to his brain that led to Tyler suffering a stroke during delivery. The stroke caused damage to the back of Tylers brain. Tyler was diagnosed with cerebral palsy and his mother, Lisa Biermann, was told to expect the worst: a child who would never walk, talk, or have any chance at a normal life.

Lisa refused to give up hope. She tried everything she could to help Tyler. Tyler could not walk because his feet would not sit flat on the floor. She tried botox injections every three months, braces, casts and even ankle cord surgery. Nothing worked.

Lisa said Tyler could not communicate with her at all. She never knew when he was in pain because he was unable to tell her.

Tyler was considered to be blind, with a prescription that was over nine units nearsighted, and his eyes jumped around. Even with glasses, he could not focus his vision, and doctors did not believe he could see, or ever would see.

Until he was 8 years old, Lisa would carry Tyler from his classes at Woodland Park Elementary.

When Tyler was 8, he had a seizure. Dr. David Steenblock, who is based in California, heard about Tyler and offered to help him with umbilical cord stem cell therapy. Lisa said she thought hard about it, and because she had tried everything else and nothing had worked, she decided to try the stem cell therapy, which Dr. Steenblock told her had no side effects.

In December 2007, Lisa, Dr. Steenblock and his team took Tyler for the treatment, which had to be done in Tijuana, Mexico, because stem cells injection is currently not legal in the United States. Three months later, they went for a second injection.

The stem cells were given to Tyler intravenously for a period of approximately 45 minutes.

Lisa said within weeks, she saw monumental changes in Tyler. All the milestones he never reached as a baby, he began reaching.

Within three months Tyler could put his feet flat on the floor and could walk independently. At six months post-treatment, he no longer needed the painful braces that gave him bunions.

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Stem cell therapy to battle HIV?

By daniellenierenberg

(SACRAMENTO, Calif.) -- UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

"We actually saw an expansion of resistant cells after the viral challenge, because other cells which were not resistant were being killed off, and only the resistant cells remained, which took over the immune system and maintained normal CD4 levels," added Anderson.

The data provided from the study confirm the safety and efficacy of this combination anti-HIV lentiviral vector in a hematopoietic stem cell gene therapy setting for HIV and validated its potential application in future human clinical trials. The team has submitted a grant application for human clinical trials and is currently seeking regulatory approval, which is necessary to move on to clinical trials.

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South Korea Steps Up Stem-Cell Work

By NEVAGiles23

Nature | Health

Regenerative medicine gets a cash boost from the nation's health ministry, but stricter regulations are needed to ensure safety

May 1, 2012

By Soo Bin Park of Nature magazine

Seoul, South Korea

The South Korean health ministry announced last month that research into stem cells and regenerative medicine will receive a funding boost of 33 billion won (US$29 million) in 2012, four times that given in 2011. Overall, six different ministries will invest 100 billion won in stem-cell research this year.

Until last year, public investment in stem cells in South Korea was relatively low and targeted mainly at basic research. But the country's Ministry of Health and Welfare is now expanding its support for clinical research on stem cells, with the money being used to link basic research to intermediate or clinical studies. The aim is to commercialize the research at an early stage.

"From the current research atmosphere and infrastructure, the government has judged that stem-cell studies are now maturing," says Hyung Min Chung, president of Seoul-based biotechnology firm Cha Bio and Diostech and an adviser on the budget plan. He adds that his company is particularly pleased that government investment decisions on developing stem-cell therapies will be made more quickly.

Target market

The government money will be allocated to two areas: rare or incurable diseases for which there is little incentive for private investment, such as spinal cord damage; and common chronic conditions, such as arthritis, for which the aim is to help South Korean companies to capture part of the large potential market for treatments.

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Jets support one fan's quest for a life-saving bone-marrow transplant

By Sykes24Tracey

Earlier this spring, Jets defensive coordinator Mike Pettine spent more than an hour on the phone with Villanova coach Andy Talley. But they weren't talking about football.

Instead, Pettine needed advice on a cause that Talley has championed in Philadelphia: bone marrow donation.

The topic hit close to home for Pettine last month, when he learned through a close friend about Michael Manganiello, a long-time Jets fan from Wayside who was diagnosed with acute myelofibrosis Easter weekend.

The rare condition -- which causes the bone marrow to progressively scar and become unable to produce blood cells -- is aggressively advancing in Manganiello's body, and his survival depends on quickly finding a matching bone marrow donor.

"It was a no-brainer to step in and help," Pettine said last night. "We dont get a lot of opportunities to do something like this, when you have a chance to have such a great impact on a life."

Pettine served as a co-host for the "Match for Michael" event held last night in Eatontown, which raised funds for the Manganiello family and the National Marrow Donor Program, but more importantly added more than 400 new people to the national bone marrow registry. Registering is as easy as taking a cheek swab, and bone marrow donation is now a less invasive procedure, the majority of the time being done via a non-surgical peripheral blood stem cell donation. Donors can usually resume normal activities within two to seven days.

Talley, who founded a non-profit organization two years ago to add donors to the national registry, gave Pettine advice on running such an event. Jets coach Rex Ryan and players Mark Sanchez, Sione Pouha and Aaron Maybin lent their support by attending the event last night, signing autographs and posing for pictures with the newly registered donors.

Manganiello, whose wife described him as the No. 1 Jets fan, was at the Robert Wood Johnson Hospital, where he is currently undergoing chemotherapy, but the Jets coaches and players were trying to reach him in his hospital room by phone.

"He's very selfless -- he would be embarrassed to know we are all here for him, he'd probably feel funny," Margo Manganiello, Michael's wife, said. "I just know were going to get through this together as a family, and I just feel like this must have happened for a reason, for us to give back to other families that are in similar situations, to help them find a cure. Because I'm very optimistic that were going to get there."

In a matter of weeks, the Manganiello family's life has been turned upside down. The 44-year-old father of three had been experiencing fatigue while running and an irregular heartbeat earlier this spring, so his wife forced him to go to the emergency room the day before Easter. It was there that blood tests confirmed the scary diagnosis.

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Local woman donates stem cells through national registry

By Sykes24Tracey

PLATTSBURGH Lois Wenger cites her faith in God as the underlying factor in her ability to help others in need, and she has never faltered in lending a helping hand.

Or in this case some much-needed bone marrow.

Wenger, who works as a support specialist in CVPH Medical Center's Information Services and Support Department, has been donating blood for years. Her blood type is O-negative, which is the universal blood type and can be used by most people in need of a transfusion.

"My older sister is a medical technologist, so it's always been a regular practice (to give blood)," Wenger said.

That practice expanded a few years ago when Wenger heard that the CVPH Blood Donor Center was working with the Rhode Island Blood Donor Center on a plan to increase the national database for bone-marrow donations.

"Only about 5 million people (nationally) were in the database at that time," said Nancy Roberts, a registered nurse at the CVPH Blood Donor Center. "We thought it would be a good idea to send out the word (for needed donors) in our region."

During the past few years, the Donor Center has hosted a bone-marrow registration at the annual Relay for Life fundraising program for the American Cancer Society.

Those successful drives, along with registrations made through the Blood Donor Center, have resulted in about 700 people from the North Country now being listed on the bone-marrow donation registry.

Those potential donors remain anonymous while their specific tissue type (collected by a simple cheek swab when registering) is recorded via bar code.

There is nothing else for the potential donor to do unless they are notified of a potential match anywhere in the country and even across the globe.

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Improved adult-derived human stem cells have fewer genetic changes than expected

By JoanneRUSSELL25

Public release date: 30-Apr-2012 [ | E-mail | Share ]

Contact: Vanessa McMains vmcmain1@jhmi.edu 410-502-9410 Johns Hopkins Medical Institutions

A team of researchers from Johns Hopkins University and the National Human Genome Research Institute has evaluated the whole genomic sequence of stem cells derived from human bone marrow cellsso-called induced pluripotent stem (iPS) cellsand found that relatively few genetic changes occur during stem cell conversion by an improved method. The findings, reported in the March issue of Cell Stem Cell, the official journal of the International Society for Stem Cell Research (ISSCR), will be presented at the annual ISSCR meeting in June.

"Our results show that human iPS cells accrue genetic changes at about the same rate as any replicating cells, which we don't feel is a cause for concern," says Linzhao Cheng, Ph.D., a professor of medicine and oncology, and a member of the Johns Hopkins Institute for Cell Engineering.

Each time a cell divides, it has the chance to make errors and incorporate new genetic changes in its DNA, Cheng explains. Some genetic changes can be harmless, but others can lead to changes in cell behavior that may lead to disease and, in the worst case, to cancer.

In the new study, the researchers showed that iPS cells derived from adult bone marrow cells contain random genetic changes that do not specifically predispose the cells to form cancer.

"Little research was done previously to determine the number of DNA changes in stem cells, but because whole genome sequencing is getting faster and cheaper, we can now more easily assess the genetic stability of these cells derived by various methods and from different tissues," Cheng says. Last year, a study published in Nature suggested higher than expected cancer gene mutation rates in iPS cells created from skin samples, which, according to Cheng, raised great concerns to many in the field pertaining to usefulness and safety of the cells. This study analyzed both viral and the improved, nonviral methods to turn on stem cell genes making the iPS cells

To more thoroughly evaluate the number of genetic changes in iPS cells created by the improved, non-viral method, Cheng's team first converted human blood-forming cells or their support cells, so-called marrow stromal cells (MSCs) in adult bone marrow into iPS cells by turning on specific genes and giving them special nutrients. The researchers isolated DNA from--and sequenced--the genome of each type of iPS cells, in comparison with the original cells from which the iPS cells were derived.

Cheng says they then counted the number of small DNA differences in each cell line compared to the original bone marrow cells. A range of 1,000 to 1,800 changes in the nucleic acid "letters" A, C, T and G occurred across each genome, but only a few changes were found in actual genes--DNA sequences that act as blueprints for our body's proteins. Such genes make up two percent of the genome.

The blood-derived iPS cells contained six and the MSC-derived iPS cells contained 12 DNA letter changes in genes, which led the researchers to conclude that DNA changes in iPS cells are far more likely to occur in the spaces between genes, not in the genes themselves.

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ACT Announces Massachusetts Eye and Ear as Additional Site for Clinical Trial for Dry Age-Related Macular Degeneration …

By JoanneRUSSELL25

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that Massachusetts Eye and Ear (Mass. Eye and Ear) has received institutional review board (IRB) approval to be a site for the companys Phase I/II clinical trial for dry age-related macular degeneration (dry AMD), using human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells.

We are delighted to announce that Mass. Eye and Ear will participate as a site for our clinical trial for dry AMD, said Gary Rabin, ACTs chairman and CEO. Dr. Dean Eliott and his team are deeply committed to finding new treatments for preventing blindness, and we very much look forward to tapping into his expertise and insight into the progression of macular degenerative disorders. The primary teaching hospital for ophthalmology at Harvard Medical School, Mass. Eye and Ear is ranked as among the top ophthalmology hospitals in the country by U.S. News & World Report and has a reputation that is unrivaled.

The Phase I/II trial is a prospective, open-label study designed to determine the safety and tolerability of the hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD. The trial will ultimately enroll 12 patients, with cohorts of three patients each in an ascending dosage format.

Dry AMD represents one of the largest unmet medical needs in ophthalmology, commented Dr. Dean Eliott, M.D. a full time retina surgeon, scientist and Associate Director of the Retina Service at Mass. Eye and Ear. We appreciate the opportunity to get some first-hand experience with the protocol and be involved with the international team that has been assembled around the U.S. and European trials.

Founded in 1824, the Massachusetts Eye and Ear Infirmary is an independent specialty hospital affiliated with Harvard Medical School.

Further information about patient eligibility for the dry AMD study is available at http://www.clinicaltrials.gov; ClinicalTrials.gov Identifier: NCT01344993.

About dry AMD Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. Age-related macular degeneration (AMD) is the leading cause of blindness in people over age 60 in the United States, and the vast majority of cases of AMD are of the dry form, which is currently untreatable.

About hESC-derived RPE Cells The retinal pigment epithelium (RPE) is a highly specialized tissue located between the choroid and the neural retina. RPE cells support, protect and provide nutrition for the light-sensitive photoreceptors. Human embryonic stem cells differentiate into any cell type, including RPE cells, and have a similar expression of RPE-specific genes compared to human RPE cells and demonstrate the full transition from the hESC state.

About Advanced Cell Technology, Inc. Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

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Genetically Modified T Cell Therapy Shown to be Safe, Lasting in Decade-Long Penn Medicine Study of HIV Patients

By daniellenierenberg

PHILADELPHIA HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

The multi-year blood samples also show that the gene-modified T cell population persists in the patients' blood for more than a decade. In fact, models suggest that more than half of the T cells or their progeny are still alive 16 years after infusion, which means one treatment might be able to kill off HIV-infected cells for decades. The prolonged safety data means that it might be possible to test T cell-based gene therapy for the treatment of non-life threatening diseases, like arthritis.

"Until now, we've focused on cancer and HIV-infection, but these data provide a rationale for starting to focus on other disease types," June says. "What we have demonstrated in this study and recent studies is that gene transfer to T cells can endow these cells with enhanced and novel functions. We view this as a personalized medicine platform to target disease using a patient's own cells."

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First fellowships awarded in new Canadian stem cell and regenerative medicine research program

By JoanneRUSSELL25

"See The Potential" program sponsored by Canada's Stem Cell Network and Pfizer

MONTRAL, May 2, 2012 /CNW/ - The first two postdoctoral research fellowships of a new program to promote stem cell research in Canada were announced today by the program's sponsors, Canada's Stem Cell Network and Pfizer.

"See The Potential" is a program established to encourage the work of promising young scientists in the field of stem cell and regenerative medicine research. Under the program, six postdoctoral fellowships will be funded from competitions over the next three years. Fellows will receive a grant of $50,000 per year for up to three years and will conduct two years of stem cell and regenerative medicine research at a recognized research laboratory in Canada as well as another year of research at the Pfizer Neusentis laboratories in the United Kingdom.

The 2011 fellowship recipients that have just been announced, following an internationally publicized competition, are Dr. Corinne Hoesli from Laval University in Qubec City and Dr. Reaz Vawda from University Health Network in Toronto. Dr. Hoesli proposes to conduct research related to engineering artificial blood vessels and is speaking today at the Till and McCulloch Meetings in Montral about the program and her research strategies. The research specialty of Dr. Vawda is comparative investigations on the therapeutic repair function of mesenchymal stem cells in the treatment of spinal cord injury.

"We are very pleased to name these first recipients of the See The Potential postdoctoral fellowships in partnership with Pfizer Inc," said Dr. Verna Skanes, Chair of the Board of the Stem Cell Network. "This program is an exciting way to provide young researchers with the opportunity to develop their research efforts and their careers while building important collaborations for the future with other researchers connected to the Stem Cell Network and, internationally, through Pfizer network. This is exactly the type of collaboration with industry that is the hallmark of translational research and one that can provide benefits to all involved."

Half the program is funded by the Stem Cell Network and other half shared by Pfizer.

"This is an excellent initiative aligned with the Pfizer Neusentis' mission to develop innovative cell therapies to benefit patients through research and development, clinical and business innovation," said academic liaison, Dr. Tim Allsopp, Head of External Research for the Regenerative Medicine activities at Pfizer Neusentis Ltd. "We congratulate our winners and look forward to witnessing the results of their important research."

The second See The Potential fellowship competition is now open with an application deadline set for June 26, 2012. For more information on the competition please visit http://www.seethepotential.ca

Canada's Stem Cell Network The Stem Cell Network, established in 2001, brings together more than 100 leading scientists, clinicians, engineers, and ethicists from universities and hospitals across Canada. The Network supports cutting-edge projects that translate research discoveries into new and better treatments for millions of patients in Canada and around the world. Hosted by the University of Ottawa, the Stem Cell Network is one of Canada's Networks of Centres of Excellence funded through Industry Canada and its three granting councils. http://www.stemcellnetwork.ca

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Juvenile Parkinson’s – Stem cell therapy – Video

By raymumme

01-05-2012 12:12 This video, is a testimonial of a patient from Uruguay that went to Progencell, to get treatment Juvenile Parkinson's . Talks about his experience, the procedure, the outcome and some suggestions. Language spanish with English subtitles, 7:10 min duration aprox.

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Genetically modified T cell therapy appears to be safe, lasting in decade-long study of HIV patients

By Sykes24Tracey

ScienceDaily (May 2, 2012) HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

The multi-year blood samples also show that the gene-modified T cell population persists in the patients' blood for more than a decade. In fact, models suggest that more than half of the T cells or their progeny are still alive 16 years after infusion, which means one treatment might be able to kill off HIV-infected cells for decades. The prolonged safety data means that it might be possible to test T cell-based gene therapy for the treatment of non-life threatening diseases, like arthritis.

"Until now, we've focused on cancer and HIV-infection, but these data provide a rationale for starting to focus on other disease types," June says. "What we have demonstrated in this study and recent studies is that gene transfer to T cells can endow these cells with enhanced and novel functions. We view this as a personalized medicine platform to target disease using a patient's own cells."

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Genetically modified T cell therapy shown to be safe, lasting in decade-long study of HIV patients

By NEVAGiles23

Public release date: 2-May-2012 [ | E-mail | Share ]

Contact: Holly Auer holly.auer@uphs.upenn.edu 215-200-2313 University of Pennsylvania School of Medicine

PHILADELPHIA -- HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

"We have 43 patients and they are all healthy," says senior author Carl June, MD, a professor of Pathology and Laboratory Medicine at Penn Medicine. "And out of those, 41 patients show long term persistence of the modified T cells in their bodies."

Early gene therapy studies raised concern that gene transfer to cells via retroviruses might lead to leukemia in a substantial proportion of patients, due to mutations that may arise in genes when new DNA is inserted. The new long-term data, however, allay that concern in T cells, further buoying the hope generated by work June's team published in 2011 showing the eradication of tumors in patients with chronic lymphocytic leukemia using a similar strategy.

"If you have a safe way to modify cells in patients with HIV, you can potentially develop curative approaches," June says. "Patients now have to take medicine for their whole lives to keep their virus under control, but there are a number of gene therapy approaches that might be curative." A lifetime of anti-HIV drug therapy, by contrast, is expensive and can be accompanied by significant side effects.

They also note that the approach the Penn Medicine team studied may allow patients with cancers and other diseases to avoid the complications and mortality risks associated with more conventional treatments, since patients treated with the modified T cells did not require drugs to weaken their own immune systems in order for the modified cells to proliferate in their bodies after infusion, as is customary for cancer patients who receive stem cell transplants.

To demonstrate the long-term safety of genetically modified T cells, June and colleagues have followed HIV-positive patients who enrolled in three trials between 1998 and 2002. Each patient received one or more infusions of their own T cells that had been genetically modified in the laboratory using a retroviral vector. The vector encoded a chimeric antigen receptor that recognizes the HIV envelope protein and directs the modified T cell to kill any HIV-infected cells it encounters.

As is standard for any trial, the researchers carefully monitored patients for any serious adverse events immediately after infusion -- none of which were seen. Additionally, because of the earlier concerns about long-term side effects, the U.S. Food and Drug Administration also asked the team to follow the patients for up to 15 years to ensure that the modified T cells were not causing blood cancers or other late effects. Therefore, each patient underwent an exam and provided blood samples during each of the subsequent years.

Now, with more than 500 years of combined patient safety data, June and colleagues are confident that the retroviral vector system is safe for modifying T cells. By contrast, June notes, the earlier, worrying side effects were seen when viral vectors were used to modify blood stem cells. The new results show that the target cell for gene modification plays an important role in long-term safety for patients treated. "T cells appear to be a safe haven for gene modification," June says.

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Genetically modified T cell therapy shown to be safe, lasting in decade-long study of HIV patients

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Study using stem cell therapy shows promise in fight against HIV

By NEVAGiles23

Public release date: 1-May-2012 [ | E-mail | Share ]

Contact: Charles Casey charles.casey@ucdmc.ucdavis.edu 916-734-9048 University of California - Davis Health System

UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

a human/rhesus macaque TRIM5 isoform, which disrupts HIV from uncoating in the cytoplasm a CCR5 short hairpin RNA (shRNA), which prevents certain strains of HIV from attaching to target cells a TAR decoy, which stops HIV genes from being expressed inside of the cell by soaking up a critical protein needed for HIV gene expression These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

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Study using stem cell therapy shows promise in fight against HIV

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Stem cell therapy shows promise in fight against HIV

By Sykes24Tracey

ScienceDaily (May 2, 2012) UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

"We actually saw an expansion of resistant cells after the viral challenge, because other cells which were not resistant were being killed off, and only the resistant cells remained, which took over the immune system and maintained normal CD4 levels," added Anderson.

The data provided from the study confirm the safety and efficacy of this combination anti-HIV lentiviral vector in a hematopoietic stem cell gene therapy setting for HIV and validated its potential application in future human clinical trials. The team has submitted a grant application for human clinical trials and is currently seeking regulatory approval, which is necessary to move on to clinical trials.

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Stem cell therapy shows promise in fight against HIV

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Two Drugs Safe for Rare Forms of Kidney Cancer

By Dr. Matthew Watson

(HealthDay News) --
Using a combination of the drugs temsirolimus (Torisel) and Bryostatin appears
to be safe in patients with metastatic kidney cancer, according to early data
from 25 patients in a phase 1 trial.

The researchers said a pathway known as mTOR signaling promotes tumor cell
proliferation and tumor blood vessel development. The temsirolimus-bryostatin
combination blocks two portions of the mTOR signaling pathway, and the early
data suggests the drugs may be active in patients with rare forms of renal cell
cancer that are less likely to respond to other therapies.

"We have certainly seen sustained responses with this combination, which
are encouraging," Dr. Elizabeth Plimack, a medical oncologist and attending
physician at Fox Chase Cancer Center in Philadelphia, said in a news release
from the center.

"Patients with non-clear cell renal cell cancer, including papillary renal
cancer, don't respond as well to tyrosine kinase inhibitors, such as sunitinib [Sutent]
and sorafenib [Nexavar], as patients with clear cell renal cell. So there is an
unmet need for therapy for these patients. We've seen that this combination may
be active to some degree for them," Plimack said.

The findings were to be presented Sunday at the American Society of Clinical
Oncology annual meeting, in Orlando, Fla. Read more…

Source:
http://feeds.feedburner.com/integratedmedicine

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Fish Glow Green After Genetic Engineering – National Geographic

By Dr. Matthew Watson


National Geographic
Fish Glow Green After Genetic Engineering
National Geographic
A genetically engineered fish that glows green from the inside out is helping illuminate what pollutants do inside the body. Endocrine disruptors are substances found in a wide range of industrial products, including plastics, as well as in many female ...

and more »

Source:
http://news.google.com/news?q=genetic-engineering&output=rss

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US biotechnology giant Amgen to invest $200m in Dún Laoghaire, Co Dublin plant … – FinFacts Ireland

By Dr. Matthew Watson


Belfast Telegraph
US biotechnology giant Amgen to invest $200m in Dún Laoghaire, Co Dublin plant ...
FinFacts Ireland
By Finfacts Team US biotechnology giant Amgen and the Irish Government today announced the start of a $200 million-plus expansion programme in Ireland that is expected to result in the creation of 100 jobs. The Taoiseach, Enda Kenny TD and the Tánaiste ...
Amgen to create up to 100 jobs in US$200m expansionSiliconrepublic.com
100 jobs in pipeline as biotech company expands in DublinInsideireland.ie
Amgen Announce Major Expansion Of Dun Laoghaire FacilityeGov monitor
Irish Times -Belfast Telegraph -thejournal.ie
all 84 news articles »

Source:
http://news.google.com/news?q=biotechnology&output=rss

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Amgen’s First CEO – Wall Street Journal

By Dr. Matthew Watson


Wall Street Journal
Amgen's First CEO
Wall Street Journal
Amgen was founded as Applied Molecular Genetics in 1980 with funding from venture capitalists amid a rush to exploit the power of gene splicing. The company worked on several products in its early years, including cloned chicken growth hormone, ...
George Rathmann, Founding CEO of Amgen and Icos, Dies at 84Xconomy

all 24 news articles »

Source:
http://news.google.com/news?q=molecular-genetics&output=rss

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