Stem Cell Research Focusing on Autism's Genetic Mysteries Earns $2.125 Million Grant at Robert Wood Johnson Medical …

Newswise PISCATAWAY, NJ -- The social symbol for autism awareness, a ribbon of brightly-colored puzzle pieces, reflects the complexity of Autism Spectrum Disorder (ASD). A new five-year $2.125 million grant from the New Jersey Governors Council for Medical Research and Treatment of Autism will fund research of induced pluripotent stem cells that may be used to piece together the genetic pathways of autism and lead to new treatments for individuals affected by ASD. The research, led by James H. Millonig, PhD, associate professor of neuroscience and cell biology at the University of Medicine and Dentistry of New Jersey-Robert Wood Johnson Medical School, is being conducted as part of the Robert Wood Johnson Medical School New Jersey Autism Center of Excellence (NJ ACE).

Autism is defined by a spectrum of behavioral and neurological abnormalities, with distinct characteristics for each individual denoting that there are multiple underlying genetic causes, said Dr. Millonig, who also is assistant dean of medical science training at Robert Wood Johnson Medical School and a member of the Center for Advanced Biotechnology and Medicine, a joint institute of the medical school and Rutgers, The State University of New Jersey. Working with my colleagues in neuroscience and at the Child Health Institute of New Jersey and Rutgers, we hope to identify the neurobiological, molecular and genetic basis the biological signature of autism.

Autism is a disorder of the brain, which is mostly comprised of neurons. Induced pluripotent stem cells (iPSCs) are scientifically-derived stem cells from individuals that mimic the traits of embryonic stem cells and can be used to create other cell types. Therefore, the research team will use iPSCs to create human neurons in order to understand how they develop abnormally in individuals with autism. The scientists will then test FDA-approved drugs on the neurons to identify therapeutic treatments that may improve or reverse the disorder.

We know of a lot of genes associated with autism, but dont know when, where and how they act in development, explains Dr. Millonig. We need to look at neurons and determine how they mature differently in order to develop better drug therapies that are tailored to the needs of individuals with autism.

The multi-institutional NJ ACE team includes Emanuel DiCicco-Bloom, MD, professor of neuroscience and cell biology and pediatrics at the medical school; Linda M. Brzustowicz, MD, professor of genetics, Rutgers University; Chi-wei Lu, PhD, assistant professor of obstetrics, gynecology, and reproductive sciences and Zhiping Pang, PhD, assistant professor of neuroscience and cell biology both at the Child Health Institute of New Jersey; and Yong Lin, PhD, at The Cancer Institute of New Jersey. The Child Health Institute of New Jersey and The Cancer Institute of New Jersey are Centers of Excellence at Robert Wood Johnson Medical School. Consultants on the grant include the Rutgers University Cell and DNA Repository; Ronald Hart, PhD, professor of cell biology and neuroscience, Rutgers University; Bonnie Firestein, PhD, professor of cell biology and neuroscience, Rutgers University; Jennifer Moore, PhD, associate director, National Institute of Mental Health Stem Cell Center, and research assistant director, Stem Cell Research Center, Rutgers University.

About UMDNJ-ROBERT WOOD JOHNSON MEDICAL SCHOOL As one of the nations leading comprehensive medical schools, UMDNJ-Robert Wood Johnson Medical School is dedicated to the pursuit of excellence in education, research, health care delivery, and the promotion of community health. In cooperation with Robert Wood Johnson University Hospital, the medical schools principal affiliate, they comprise one of the nation's premier academic medical centers. In addition, Robert Wood Johnson Medical School has 34 other hospital affiliates and ambulatory care sites throughout the region.

As one of the eight schools of the University of Medicine and Dentistry of New Jersey, with 2,800 full-time and volunteer faculty, Robert Wood Johnson Medical School encompasses 22 basic science and clinical departments, and hosts centers and institutes including The Cancer Institute of New Jersey, the Child Health Institute of New Jersey, the Center for Advanced Biotechnology and Medicine, the Environmental and Occupational Health Sciences Institute, and the Stem Cell Institute of New Jersey. The medical school maintains educational programs at the undergraduate, graduate and postgraduate levels for more than 1,500 students on its campuses in New Brunswick, Piscataway, and Camden, and provides continuing education courses for health care professionals and community education programs. To learn more about UMDNJ-Robert Wood Johnson Medical School, log on to rwjms.umdnj.edu. Find us online at http://www.Facebook.com/RWJMS and http://www.twitter.com/UMDNJ_RWJMS. ###

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Stem Cell Research Focusing on Autism's Genetic Mysteries Earns $2.125 Million Grant at Robert Wood Johnson Medical ...

StemCells rises on spinal cord therapy data

NEW YORK (AP) -- Shares of StemCells Inc. jumped Tuesday after the company said it finished treating the first group of patients in a clinical trial of its stem cell therapy.

THE SPARK: StemCells said it has completed treatment for three patients and is now observing their long-term health. All three had suffered a "complete" spinal cord injury at chest level, which means they had no nerve function or feeling below the area that was injured. The company said two of the patients had greater sensory function a year after treatment, and one had recovered to the point that the injury is now considered "incomplete."

StemCells said that as far as it knows, this is the first time a patient has experienced that kind of improvement after treatment with neural stem cells.

The study measured changes in the patients' sensitivity to touch, heat and electrical stimuli. StemCells said one of the patients didn't experience any improvement.

THE BIG PICTURE: The Newark, Calif., company is studying therapies based on purified adult neural stem cells. It had previously reported that two of the three patients had improved six months after they were treated. StemCells is also testing its treatment on patients with complete injuries, and said it hopes those patients will have similar or larger improvements because they still have some nerve function.

Stem cell therapies are still an experimental field, and StemCells is a small company without any approved products.

SHARE ACTION: StemCells shares climbed 38 cents, or 22.8 percent, to $2.02 in afternoon trading. The stock has doubled in value since-mid July, when the company reported positive preclinical data for an experimental Alzheimer's disease therapy.

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StemCells rises on spinal cord therapy data

International Stem Cell Corporation Demonstrates Positive Animal Efficacy Results in Metabolic Liver Disease Program

CARLSBAD, CA--(Marketwire - Feb 12, 2013) - International Stem Cell Corporation ( OTCQB : ISCO ) (www.internationalstemcell.com) ("ISCO" or "the Company") a California-based biotechnology company, today announced the conclusions from its study demonstrating the efficacy and safety of the hepatocyte-like cells (HLC) derived from human parthenogenetic stem cells (hpSC) in a well-established animal model of a congenital liver disorder associated with bilirubin metabolism. The data from this pre-clinical study indicates that implanting HLC in rodents produced both a significant initial decrease and the long-term stabilization of bilirubin levels in blood serum.

Criggler-Najjar syndrome type 1 (CN1) is a rare inherited metabolic disorder in which the sufferer's liver lacks a specific enzyme -- UGT1A1, which is essential for the clearance of the toxin bilirubin. The syndrome results in unconjugated hyperbilirubinemia, a disorder characterized by severe neurological complications which, if left untreated, can lead to irreversible acute encephalopathy. Allogenic hepatocyte transplantation (HT) has been used as an alternative therapeutic option for patients with liver-based metabolic diseases including CN1. However, one of the major factors limiting the clinical advancement of human HT is a shortage of mature, functioning human hepatocytes as well as the limited repopulation capacity of grafted adult cells.

The use of HLCs to treat CN1 has several potential advantages over transplantation of primary hepatocytes. Firstly, HLC would circumvent the shortage of primary cells, as they can be produced and expanded in vitro. Secondly, there is evidence to suggest that grafting HLC may yield better long-term repopulation and persistent metabolic activity than using immature fetal hepatocytes. HLCs can also be given before the onset of bilirubin encephalopathy occurs, and can thus provide sufficient amounts of UGT1A1 to allow the liver to metabolize this toxin.

ISCO has previously reported how these HLC engraft in the liver of Gunn rats, a well-validated model of CN1 where the animals lack UGT1A1 and therefore accumulate toxic levels of unconjugated bilirubin. In addition to this result, no adverse safety signals were detected 16 weeks after the implantation of a therapeutic dose of HLC, and serum levels of bilirubin continued to decline, compared with the control group, up to the conclusion of the observation period at week 19. Moreover, the overall structure and morphology of the liver in all rodents in the treatment group appeared to be undamaged, with no apparent inflammation, tumorigenicity or cell rejection observed.

Dr. Ruslan Semechkin, Vice President - head of R&D for ISCO comments: "This study provides important evidence for the use of our HLC product as a viable source of transplantable cells for the treatment of CN1. Having completed this study, we can now discuss with the FDA the requirements for our Investigational New Drug (IND) application and phase 1 clinical trial."

About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at http://www.internationalstemcell.com.

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Northwestern Medicine researchers investigate stem cell therapy for stroke

Public release date: 11-Feb-2013 [ | E-mail | Share ]

Contact: Megan McCann memccann@nmh.org 312-926-5900 Northwestern Memorial Hospital

Each year, nearly 800,000 Americans suffer a stroke which can result in permanent brain damage, long term disability or death. As a leading cause of adult disability, stroke has an annual burden of more than $62 billion on the United States economy. With the exception of rehabilitation therapy, very few treatments are available to improve the chronic neurologic deficits caused by a stroke. In hopes of expanding therapeutic options, Northwestern Medicine researchers are investigating a novel stem cell therapy, known as SB623, that may hold the key to improving motor function following a disabling stroke.

Northwestern is currently one of only three sites in the nation enrolling participants in a landmark study to test the safety and efficacy of adult stem cell therapy for patients with stable ischemic stroke. Accounting for 87 percent of strokes, ischemic stroke occurs when a blocked artery interrupts the flow of oxygen and blood to the brain. This causes cell death and brain damage which can leave a person with impaired body functions, including paralysis, weakness on one side, difficulty with speech and language, vision issues, and cognitive challenges.

"Two million brain cells die each minute during a stroke making it critical to get treatment fast at the earliest sign of symptoms; once brain damage occurs, there's very little that can be done medically to reverse it," said principal investigator Joshua Rosenow, MD, director of Functional Neurosurgery at Northwestern Memorial Hospital and associate professor of neurosurgery, neurology, and physical medicine and rehabilitation at Northwestern University Feinberg School of Medicine. "While this study is only a preliminary step towards understanding the healing potential of these cells, we are excited about what a successful trial could do for a patient population that currently has very limited therapeutic options."

While the study's primary purpose is to examine the safety of SB623 stem cells, researchers will also seek to determine if the cells are effective in improving stroke symptoms. SB623 is derived from genetically engineered adult bone marrow cells from a healthy adult donor.

"Although not proven in humans, these stem cells have been shown to promote healing and improve function when administered in animal models of stable stroke," said co-investigator Richard Bernstein, MD, director of Northwestern Memorial's Stroke Center and associate professor of neurology at the Feinberg School. "The cells did not replace the neurons destroyed by stroke, but instead they appeared to encourage the brain to heal itself and promote the body's natural regenerative process. Eventually, the implanted stem cells disappeared."

"In this study, the cells are transplanted into the brain using brain mapping technology and scans, allowing us to precisely deposit the cells in the brain adjacent to the area damaged by stroke," explained Rosenow.

Early participants have received 2.5 million cells, but as the study progresses the dose will escalate to 5 million and eventually 10 million cells. Since SB623 cells are allogeneic, a single donor's cells can be used to treat many other individuals. Participants in the study will be followed for up to two years with periodic evaluations for safety and effectiveness in the improvement in motor function.

"Stroke can be a very disabling and life changing event," said Bernstein. "Even just a slight improvement in function could make a huge difference for a person impacted by stroke. To potentially give our patients the opportunity to permanently regain movement or speech is a very exciting prospect. In the animal models, the improvements appeared to remain even after the implanted stem cells disappeared."

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Stem cell discovery gives insight into motor neurone disease

Public release date: 11-Feb-2013 [ | E-mail | Share ]

Contact: Tara Womersley tara.womersley@ed.ac.uk 44-131-650-9836 University of Edinburgh

A discovery using stem cells from a patient with motor neurone disease could help research into treatments for the condition. The study used a patient's skin cells to create motor neurons - nerve cells that control muscle activity - and the cells that support them called astrocytes.

Researchers studied these two types of cells in the laboratory. They found that a protein expressed by abnormalities in a gene linked to motor neurone disease, which is called TDP-43, caused the astrocytes to die.

The study, led by the University of Edinburgh and funded by the Motor Neurone Disease Association, provides fresh insight into the mechanisms involved in the disease.

Although TDP-43 mutations are a rare cause of motor neurone disease (MND), scientists are especially interested in the gene because in the vast majority of MND patients, TDP-43 protein (made by the TDP-43 gene) forms pathological clumps inside motor neurons.

Motor neurons die in MND leading to paralysis and early death.

This study shows for the first time that abnormal TDP-43 protein causes death of astrocytes. The researchers, however, found that the damaged astrocytes were not directly toxic to motor neurons.

Better understanding the role of astrocytes could help to inform research into treatments for MND.

Professor Siddharthan Chandran, of the University of Edinburgh, said: "Motor neurone disease is a devastating and ultimately fatal condition, for which there is no cure or effective treatment. It is not just a question of looking solely at motor neurons, but also the cells that surround them, to understand why motor neurons die. Our aim is to find ways to slow down progression of this devastating disease and ultimately develop a cure."

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Stem cell breakthrough could lead to new bone repair therapies on nanoscale surfaces

Feb. 11, 2013 Scientists at the University of Southampton have created a new method to generate bone cells which could lead to revolutionary bone repair therapies for people with bone fractures or those who need hip replacement surgery due to osteoporosis and osteoarthritis.

The research, carried out by Dr Emmajayne Kingham at the University of Southampton in collaboration with the University of Glasgow and published in the journal Small, cultured human embryonic stem cells on to the surface of plastic materials and assessed their ability to change.

Scientists were able to use the nanotopographical patterns on the biomedical plastic to manipulate human embryonic stem cells towards bone cells. This was done without any chemical enhancement.

The materials, including the biomedical implantable material polycarbonate plastic, which is a versatile plastic used in things from bullet proof windows to CDs, offer an accessible and cheaper way of culturing human embryonic stem cells and presents new opportunities for future medical research in this area.

Professor Richard Oreffo, who led the University of Southampton team, explains: "To generate bone cells for regenerative medicine and further medical research remains a significant challenge. However we have found that by harnessing surface technologies that allow the generation and ultimately scale up of human embryonic stem cells to skeletal cells, we can aid the tissue engineering process. This is very exciting.

"Our research may offer a whole new approach to skeletal regenerative medicine. The use of nanotopographical patterns could enable new cell culture designs, new device designs, and could herald the development of new bone repair therapies as well as further human stem cell research," Professor Oreffo adds.

This latest discovery expands on the close collaborative work previously undertaken by the University of Southampton and the University of Glasgow. In 2011 the team successfully used plastic with embossed nanopatterns to grow and spread adult stem cells while keeping their stem cell characteristics; a process which is cheaper and easier to manufacture than previous ways of working.

Dr Nikolaj Gadegaard, Institute of Molecular, Cell and Systems Biology at the University of Glasgow, says: "Our previous collaborative research showed exciting new ways to control mesenchymal stem cell -- stem cells from the bone marrow of adults -- growth and differentiation on nanoscale patterns.

"This new Southampton-led discovery shows a totally different stem cell source, embryonic, also respond in a similar manner and this really starts to open this new field of discovery up. With more research impetus, it gives us the hope that we can go on to target a wider variety of degenerative conditions than we originally aspired to. This result is of fundamental significance."

The study was funded by the Biotechnology and Biological Sciences Research Council (BBSRC).

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Stem cell breakthrough could lead to new bone repair therapies on nanoscale surfaces

Stem cell discovery gives insight into motor neuron disease

Feb. 11, 2013 A discovery using stem cells from a patient with motor neuron disease could help research into treatments for the condition. The study used a patient's skin cells to create motor neurons -- nerve cells that control muscle activity -- and the cells that support them called astrocytes.

Researchers studied these two types of cells in the laboratory. They found that a protein expressed by abnormalities in a gene linked to motor neuron disease, which is called TDP-43, caused the astrocytes to die.

The study, led by the University of Edinburgh and funded by the Motor Neurone Disease Association, provides fresh insight into the mechanisms involved in the disease.

Although TDP-43 mutations are a rare cause of motor neuron disease (MND), scientists are especially interested in the gene because in the vast majority of MND patients, TDP-43 protein (made by the TDP-43 gene) forms pathological clumps inside motor neurons.

Motor neurons die in MND leading to paralysis and early death.

This study shows for the first time that abnormal TDP-43 protein causes death of astrocytes. The researchers, however, found that the damaged astrocytes were not directly toxic to motor neurons.

Better understanding the role of astrocytes could help to inform research into treatments for MND.

Professor Siddharthan Chandran, of the University of Edinburgh, said: "Motor neuron disease is a devastating and ultimately fatal condition, for which there is no cure or effective treatment. It is not just a question of looking solely at motor neurons, but also the cells that surround them, to understand why motor neurons die. Our aim is to find ways to slow down progression of this devastating disease and ultimately develop a cure."

These findings, published in the journal Proceedings of the National Academy of Sciences., are significant as they show that different mechanisms are at work in different types of MND.

The research, led by the University of Edinburgh's Euan MacDonald Centre for Motor Neurone Research, was carried out in collaboration with King's College, London, Columbia University in New York, the University of California and the Gladstone Institutes in San Francisco.

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Stem cell discovery gives insight into motor neuron disease

Jack Osbourne's stem cell jabs: Ozzy's son flies to German for banned MS treatment

9 Feb 2013 23:11

TV star, who went public about his health crisis last summer, said he is currently feeling well and getting plenty of rest

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STAR Jack Osbourne is to undergo cutting-edge stem cell replacement therapy to help him beat Multiple Sclerosis.

Jack, 27 son of rocker Ozzy and TV star Sharon will go to Germany for the treatment as it is banned in America, where he is based.

He will fly out after covering the Grammy Awards in Los Angeles today for US music channel Fuse TV.

Jack said: There are odd restrictions here in America because everybody still thinks its, like, what it isnt. So Im going to Germany. They clone stem cells from your own blood then inject them back into you.

Hopefully it will repair any damaged cells and nerves and things like that.

Jack, who went public about his health crisis last summer, said he is currently feeling well and getting plenty of rest despite being a new dad to nine-month-old daughter Pearl.

He said: Im good. Thats the thing about MS one minute youre good, one minute youre not. Im hanging on in there, healthy and doing what I got to do. Jack said he and wife Lisa are getting plenty of help with babysitting from his parents, who are relishing their role as grandparents.

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Jack Osbourne's stem cell jabs: Ozzy's son flies to German for banned MS treatment

Estrada, Enrile, Imelda Marcos–how they’re defying age and time with stem-cell therapy

A growing number of wealthy Filipinos have availed themselves of the four-day, three-night treatment in Germany. Last year alone, the clinic attended to 350 Filipinos

DR.GEOFFREY Huertgen, Villa Medicas chief medical director. ALANAH TORRALBA

Jumpstarting the regeneration process to achieve longevity, glowing skin and youthful energy are just three of the claimed benefits derived by patients at Villa Medica.

The Germany-based holistic clinic specializes in fresh-cell therapy, a form of stem-cell treatment sourced from organs and tissues of unborn lamb.

Such claims may sound too good to be true, until you see the likes of former President Joseph Estrada, former first lady Imelda Marcos and Senate President Juan Ponce Enrile strutting about and plotting their next political moves like theyre in their prime.

Except for Enrile, Marcos and Estrada have nearly endorsed the clinic in Edenkoben, an hours drive south of Frankfurt, by agreeing to have their pictures and brief testimonials used in a Villa Medica brochure.

Marcos reportedly started having fresh-cell therapy injections in 1969, less than a decade after Villa Medica opened its doors for business in 1961. But the acknowledged father of fresh-cell therapy, Dr. Paul Niehans, broke ground and started his research in 1931.

Dr. Geoffrey Huertgen, Villa Medicas chief medical director, visited Manila two weeks ago to see former and potential patients. What the clinic promises, he said, is overall good health by enhancing the regeneration process. Youthful attributes such as beautiful skin and increased energy are just byproducts of the clinics holistic philosophy.

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Although fresh-cell therapy claims to help improve the condition of diabetics and kids afflicted with autism and Downs syndrome, to name a few, it doesnt promise a miracle cure for life-threatening diseases such as cancer. And since the clinics approach is organ-specific, it doesnt offer patients a one-size-fits-all therapy.

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Estrada, Enrile, Imelda Marcos–how they’re defying age and time with stem-cell therapy

Bonita Springs stem-cell doctor asks judge to dismiss state's case against him

BONITA SPRINGS The defense for Bonita Springs doctor Zannos Grekos says a state complaint that he committed medical malpractice should be dismissed because prosecutors failed to show how his stem-cell therapy was below a standard of care or responsible for a patient's death, according to new court filings.

Prosecutors with the Florida Department of Health also ignored a state law that patients have the right to seek out alternative medicine, said Richard Ozelie, the Boca Raton attorney for Grekos.

That's what occurred with 69-year-old Domenica Fitzgerald, he said.

"The Florida Legislature authorized its citizenry to seek out, as patients, either complementary or alternative modalities of treatment," he said Friday.

Late Thursday, Ozelie filed a proposed order for Administrative Law Judge J. Lawrence Johnston to consider. The state submitted its proposed order this past Tuesday.

The judge has 30 days to issue a recommendation to the Board of Medicine on potential discipline against Grekos, following a four-day hearing in October in Naples.

The state wants Grekos' license revoked, a $40,000 fine imposed and $200,000 assessed for the state's costs.

In 2010, Fitzgerald sought out stem-cell therapy with Grekos for numbness in her feet that was a side-effect several years earlier from chemotherapy for breast cancer.

In Grekos' Bonita Springs practice in March 2010, he extracted bone marrow aspirate from her and injected it back into her circulatory system without removing bone fragments and fat. She suffered a stroke and was taken off life support April 4, 2010.

The state said Fitzgerald hadn't been fully informed about the procedure and its risks, but Ozelie said that's not true.

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Bonita Springs stem-cell doctor asks judge to dismiss state's case against him