CardioCel has been initially well received with surgeons in Australia and overseas. Photo: Geoff Fisher

For 10 years researchers at Admedus worked day and night trying to work out how to improve soft tissue repair in the human body.

And with the vital help of CSIRO they have been to develop CardioCel, a life-saving heart patch for the repair and reconstruction of cardiovascular defects.

According to the Children's Heart Foundation, congenital heart disease occurs in one out of 100 births and in at least half of those cases surgery is required and a patch is needed. They state it is the leading cause of birth defect related deaths.

Research undertaken with CSIRO investigated new, potentially ground-breaking applications for CardioCel. The research focused on using stem cells. It found the heart patch has the potential to deliver stem cells and help tissue heal better than other existing products, when used for cardiac repair.

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Derived from animal tissue, the CardioCel patch is engineered over 14 days.

"The first unique feature of this product is that it doesn't calcify in young patients," Professor Leon Neethling, Admedus technical director and heart researcher says.

The flexible patch works like human tissue to cover holes in the heart thereby eliminating the need for repeat surgery.

"In the cardiac repair field it has long been recognised that strong, flexible, biocompatible and calcification-resistant tissue scaffolds would be ideal tissues for repair of heart defects," Admedus' chief operating officer Dr Julian Chick, says.

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Local innovation repairs holes in the heart

Can PRP and Stem Cell Therapy Help You? | Orlando Orthopaedic Center
How can PRP and stem cell therapy help you heal? Orlando Orthopaedic Center #39;s Dr. Matthew R. Willey explains. For more visit http://www.OrlandoOrtho.com.

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$1 M gift from Mr. J. Sebastian van Berkom launches translational research into neurological disease

This news release is available in French.

A patient's very own skin cells may hold the key to new treatments and even cures for devastating neurological diseases. A generous $1 million donation from Mr. J. Sebastian van Berkom, and critical partnerships with Brain Canada, Laval University, Marigold Foundation and the FRQS-Rseau Parkinson Quebec are driving an innovative, iPSC (induced pluripotent stem cell) research platform that will transform research into Parkinson's and other neurological diseases.

Millions of Canadians are affected by diseases of the brain such as ALS, Parkinson's and brain tumours, for which there are limited treatments and no cures. By 2020, neurological conditions will become the leading cause of death and disability. "Everyone's lives are touched in some way by neurological disease, says Mr. van Berkom, President of Van Berkom and Associates Inc." In creating The van Berkom Parkinson's Disease Open-Access Fund, I hope to change lives and support new research that will lead to new treatments and one day cures. The iPSC platform is a new paradigm for neuroscience research and as one of the world's great neuroscience centres, The Neuro is the place to drive it forward."

"This is the ultimate bench to bedside paradigm, from patient to the bench, back to the patient," says Dr. Guy Rouleau, Director of The Neuro. "With a unique interface between fundamental and clinical research, The Neuro is uniquely positioned to be a central hub in the iPSC platform. Partnering with Mr. Van Berkom, a generous and visionary philanthropist, propels The Neuro toward the goal of significantly deepening insight into disease mechanisms with unprecedented efficiency."

Patients' skin cells will be reprogrammed into induced pluripotent stem cells (iPSCs) at Laval University, under the leadership of Dr Jack Puymirat, and then differentiated at The Neuro into disease relevant cells for research. For example, in the case of Parkinson's this could be dopamine neurons. The cells can also be genome-edited, a state-of-the-art technique that can introduce or correct disease associated mutations - creating the most accurate disease models. These iPSCs will be made widely and openly available to researchers across Quebec for neuroscience research. This open-access approach exponentially increases the likelihood of breakthroughs in neurological disease.

"The unique and exciting aspect of this platform is that we are creating the most specific cells for studying disease using the patient's own tissue, which has distinct advantages over using generic cells or animal models," says Dr. Edward Fon, neurologist and co-Director of the Quebec iPSC platform. "Disease models using human samples are increasingly shown to be far more efficacious in trials, as they much more accurately mimic the disease condition. In the iPSC platform, not only can specific mutations be introduced but, cells are from patients' whose specific clinical history and genetic profile are known, a first step on the road toward neurological personalized medicine. The Neuro has access to a large and well-characterized patient population, who can help create a rich clinically-and genetically-derived registry and biobank. The initial targets in the platform will be ALS and Parkinson's disease (PD), using dopamine neurons for PD and both motor neurons and astrocytes for ALS."

The Quebec iPSC core facility is a provincial core headed by Drs. Fon and Puymirat. Reprogrammed cells at Laval University will be created from different sources such as skin biopsies, blood or urine. The Neuro's component of the platform will consist of two core facilities. The iPSC neuronal differentiation core - which differentiate iPSCs into functional neurons, headed by Dr. Eric Shoubridge, and the iPSC genome-editing core providing unprecedented ability to study the influence of disease mutations, headed by Dr. Peter McPherson.

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The Montreal Neurological Institute and Hospital

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Using patients' own cells to accelerate research into neurological disease

(SACRAMENTO, Calif.) - The state stem cell agency, California Institute for Regenerative Medicine (CIRM),awarded a pair of grants totaling more than $7 million to UC Davis School of Medicine researchers who are working to develop stem cell therapies for spina bifida and chronic diabetic wounds. The funding is part of what the agency considers "the most promising" research leading up to human clinical trials using stem cells to treat disease and injury. Diana Farmer, professor and chair of surgery at UC Davis Medical Center, is developing a placental stem cell therapy for spina bifida, the common and devastating birth defect that causes lifelong paralysis as well as bladder and bowel incontinence. She and her team are working on a unique treatment that can be applied in utero - before a baby is born -- in order to reverse spinal cord damage. Roslyn Rivkah Isseroff, a UC Davis professor of dermatology, and Jan Nolta, professor of internal medicine and director of the university's Stem Cell Program, are developing a wound dressing containing stem cells that could be applied to chronic wounds and be a catalyst for rapid healing. This is Isseroff's second CIRM grant, and it will help move her research closer to having a product approved by the U.S. Food and Drug Administration that specifically targets diabetic foot ulcers, a condition affecting more than 6 million people in the country. The CIRM board, which met in Berkeley today, has high hopes for these types of research that the agency funded in this latest round of stem cell grants. "This investment will let us further test the early promise shown by these projects," said Jonathan Thomas, chair of CIRM's governing board. "Preclinical work is vital in examining the feasibility, potential effectiveness and safety of a therapy before we try it on people. These projects all showed compelling evidence that they could be tremendously beneficial to patients. We want to help them build on that earlier research and move the projects to the next level." The CIRM grants are designed to enable the UC Davis research teams to transition from preclinical research to preclinical development over the next 30 months to be able to meet the FDA's rigorous safety and efficacy standards for Investigative New Drugs. As the former surgeon-in-chief at UCSF Benioff Children's Hospital, Farmer helped pioneer fetal surgery techniques for treating spina bifida before birth. The condition, also known as myelomeningocele, is one of the most common and devastating birth defects worldwide, causing lifelong paralysis as well as bowel and bladder incontinence in newborns. Farmer has been investigating different stem cell types and the best way to deliver stem cell-based treatments in the womb for the past six years. She and her research colleagues recently discovered a placental therapy using stem cells that cures spina bifida in animal models. That discovery requires additional testing and FDA approval before the therapy can be used in humans. With the CIRM funding, Farmer and her team plan to optimize their stem cell product, validate its effectiveness, determine the optimal dose and confirm its preliminary safety in preparation for human clinical trials. Isseroff, who also serves as chief of dermatology and director of wound healing services for the VA Northern California Health Care System, has long been frustrated by the challenges of treating the chronic, non-healing wounds of diabetics. In 2010, she and Nolta received a CIRM grant to begin developing a bioengineered product for treating chronic diabetic wounds. Foot ulcers, in particular, affect about 25 percent of all diabetic patients and are responsible for most lower-limb amputations. Isseroff and her research team created a treatment using stem cells derived from bone marrow (mesenchymal stem cells) along with a FDA-approved scaffold to help regenerate dermal tissue and restart the healing process. Their studies found the technique to be highly effective for healing wounds in animal models. With this latest CIRM grant, Isseroff's team will refine their therapeutic technique by determining the safest dosage for regenerating tissue and testing their product in skin-wound models that closely resemble those in diabetic humans. Nolta also plans to create a Master Cell Bank of pure and effective human mesenchymal stem cells, and establish standard operating procedures for use in diabetic wound repair. The results of their efforts will enable UC Davis to move closer to FDA approval for human clinical trials in the next two and a half years. "These amazing research efforts are giant steps forward in turning stem cells into cures," said Nolta, who also directs the UC Davis Institute for Regenerative Cures in Sacramento. "This preclinical research is the most crucial, and often the toughest, stage before we move scientific discoveries from the laboratory bench to the patient's bedside. We are now poised as never before to make a big difference in the lives of people with spina bifida and non-healing diabetic wounds." For more information, visit UC Davis School of Medicine at http://medschool.ucdavis.edu.

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Stem Cell Grants for Spina Bifida and Diabetic Wound Treatments

Tampa, FL. (PRWEB) March 26, 2015

This month, the Lung Institute has started treating people suffering from chronic lung diseases with stem cells extracted from their bone marrow. This treatment protocol is added to the two other treatment options offered by the Lung Institute: venous (blood-derived) and adipose (fat-derived) stem cell therapy.

The bone marrow and adipose treatments offer the highest concentration of stem cells and allow for the cells to be reintroduced directly into the lungs through a nebulizer. Given this added benefit, most patients in the past opted to receive the adipose treatment over venous. However, many patients have other medical conditions that preclude them from choosing the adipose treatment. Since the number of stem cells harvested from a bone marrow procedure matches that of the adipose procedure, patients that have previously only qualified for the venous procedure are now eligible for a treatment option that produces the highest chance of success.

Patients are often surprised by the simplicity of these minimally invasive procedures, but with cutting-edge technology and the patient-centric clinical team at the Lung Institute, patients can rest assured that they are in good hands. Throughout the entire treatment process, patients have the opportunity to get any questions immediately answered by our knowledgeable medical staff. The Lung Institute clinical team remains in contact with patients after treatment and works together with the patients physician and pulmonologist to create a strong support system for the patient.

About the Lung Institute At the Lung Institute, we are changing the lives of hundreds of people across the nation through the innovative technology of regenerative medicine. We are committed to providing patients a more effective way to address pulmonary conditions and improve their quality of life. Our physicians, through their designated practices, have gained worldwide recognition for the successful application of revolutionary minimally invasive stem cell therapies. With over a century of combined medical experience, our doctors have established a patient experience designed with the highest concern for patient safety and quality of care. For more information, visit our website at LungInstitute.com, like us on Facebook, follow us on Twitter or call us today at (855) 313-1149.

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Lung Institute Announces New Treatment with Bone Marrow

My bone marrow began sending me messages long before I Learned to listen..I was exhausted, pale, drained, and tired all of the time. I eventually landed myself in the emergency room, and was handed a full membership to the cancer club. I was diagnosed with multiple myeloma, an incurable blood cancer.

My bone marrow began sending me messages long before I Learned to listen..I was exhausted, pale, drained, and tired all of the time. I eventually landed myself in the emergency room, and was handed a full membership to the cancer club. I was diagnosed with multiple myeloma, an incurable blood cancer.

That was June 23, 2009.

As it turned out I was very fortunate. I beat the statistical odds and circumstances were in my favour. After four months of chemo and steroids, I was able to use my own stem cells in what is called an autologous stell cell transplant.

My stem cell transplant was a journey to my very core. It's like witnessing a rebirth. It's awe-inspiring and essential. Visualizing those 'yellow' cells stream their way back into my bone marrow opened my eyes to the singular power stem cells bring into our world.

But I was also reminded of Michael Pinto the undertaker in Bombay.

'Grave Problems Resurrected here'

That's so not gonna happen. Not on my resurrection.

My passage through illness taught me that the knowledge of the curative properties of stem cells needs to be shared to offer hope of renewed life. If you knew what medical science can do with stem cells, and if you saw what I did in the labs, through microscropes, you too would feel like using both hands to scoop those secrets out into the world.

Occasionally I gloss over my past cancer club membership--my treatment, my illnessbut then I am remember what a profound reboot my body has gone through, and I remember why. It's true that the deepest crises are moments of great opportunity; an event that shocks you into seeing with your heart. It is a place that combines survival with celebration.

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My message in a bottle, writes Lisa Ray

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SuperCells at Centre for Life
Exhibition at Centre for Life, Newcastle. Exhibition presented by the Stem Cell Network and produced by the Sherbrooke Museum of Nature and Science (Quebec, Canada); in partnership with the...

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Scientists at the University of Cambridge have successfully created 'mini-lungs' using stem cells derived from skin cells of patients with cystic fibrosis, and have shown that these can be used to test potential new drugs for this debilitating lung disease.

The research is one of a number of studies that have used stem cells - the body's master cells - to grow 'organoids', 3D clusters of cells that mimic the behaviour and function of specific organs within the body. Other recent examples have been 'mini-brains' to study Alzheimer's disease and 'mini-livers' to model liver disease. Scientists use the technique to model how diseases occur and to screen for potential drugs; they are an alternative to the use of animals in research.

Cystic fibrosis is a monogenic condition - in other words, it is caused by a single genetic mutation in patients, though in some cases the mutation responsible may differ between patients. One of the main features of cystic fibrosis is the lungs become overwhelmed with thickened mucus causing difficulty breathing and increasing the incidence of respiratory infection. Although patients have a shorter than average lifespan, advances in treatment mean the outlook has improved significantly in recent years.

Researchers at the Wellcome Trust-Medical Research Council Cambridge Stem Cell Institute used skin cells from patients with the most common form of cystic fibrosis caused by a mutation in the CFTR gene referred to as the delta-F508 mutation. Approximately three in four cystic fibrosis patients in the UK have this particular mutation. They then reprogrammed the skin cells to an induced pluripotent state, the state at which the cells can develop into any type of cell within the body.

Using these cells - known as induced pluripotent stem cells, or iPS cells - the researchers were able to recreate embryonic lung development in the lab by activating a process known as gastrulation, in which the cells form distinct layers including the endoderm and then the foregut, from which the lung 'grows', and then pushed these cells further to develop into distal airway tissue. The distal airway is the part of the lung responsible for gas exchange and is often implicated in disease, such as cystic fibrosis, some forms of lung cancer and emphysema.

The results of the study are published in the journal Stem Cells and Development.

"In a sense, what we've created are 'mini-lungs'," explains Dr Nick Hannan, who led the study. "While they only represent the distal part of lung tissue, they are grown from human cells and so can be more reliable than using traditional animal models, such as mice. We can use them to learn more about key aspects of serious diseases - in our case, cystic fibrosis."

The genetic mutation delta-F508 causes the CFTR protein found in distal airway tissue to misfold and malfunction, meaning it is not appropriately expressed on the surface of the cell, where its purpose is to facilitate the movement of chloride in and out of the cells. This in turn reduces the movement of water to the inside of the lung; as a consequence, the mucus becomes particular thick and prone to bacterial infection, which over time leads to scarring - the 'fibrosis' in the disease's name.

Using a fluorescent dye that is sensitive to the presence of chloride, the researchers were able to see whether the 'mini-lungs' were functioning correctly. If they were, they would allow passage of the chloride and hence changes in fluorescence; malfunctioning cells from cystic fibrosis patients would not allow such passage and the fluorescence would not change. This technique allowed the researchers to show that the 'mini-lungs' could be used in principle to test potential new drugs: when a small molecule currently the subject of clinical trials was added to the cystic fibrosis 'mini lungs', the fluorescence changed - a sign that the cells were now functioning when compared to the same cells not treated with the small molecule.

"We're confident this process could be scaled up to enable us to screen tens of thousands of compounds and develop mini-lungs with other diseases such as lung cancer and idiopathic pulmonary fibrosis," adds Dr Hannan. "This is far more practical, should provide more reliable data and is also more ethical than using large numbers of mice for such research."

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Scientists grow 'mini-lungs' to aid the study of cystic fibrosis

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Amniotic Stem Cell Therapy Discussed by R3 (844) GET-STEM
http://r3stemcell.com/stem-cell-treatments/amniotic-derived-stem-cell-injections/ Amniotic derived stem cell therapy has become exceptionally popular due to the benefits that are being seen....

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The Alpha Clinic for Cell Therapy and Innovation | City of Hope
A new grant to City of Hope from the California Institute for Regenerative Medicine (CIRM) will make it possible for novel stem cell based therapies developed here at City of Hope to...

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Boston, MA (PRWEB) March 18, 2015

In the vast flow of new scientific research, discoveries, and information, it is not uncommon for important scientific advances to go unappreciated, or even just unnoticed, for surprisingly long periods of time. The Boston stem cell medicine technology start-up company, Asymmetrex is working to make sure that its growing portfolio of adult tissue stem cell technology patents obtains wide notice, appreciation, and investment.

In late 2014, the company started a digital media campaign to achieve greater visibility for its patented technologies that address the major barriers to greater progress in stem cell medicine. These include technologies for identifying, counting, and mass-producing adult tissue stem cells. The two presentations scheduled for the 5th World Congress on Cell and Stem Cell Research in Chicago continue Asymmetrexs efforts to better inform medical, research, and industrial communities focused on advancing stem cell medicine of the companys vision for implementation of its unique technologies.

Asymmetrex holds patents for the only method described for routine production of natural human tissue stem cells that retain their normal function. The company also holds patents for biomarkers that can be used to count tissue stem cells for the first time. The companys most recently developed technology was invented with computer-simulation leader, AlphaSTAR Corporation. In partnership, the two companies created a first-of-its-kind method for monitoring adult tissue stem cell number and function for any human tissue that can be cultured. This advance is the basis for the two companies AlphaSTEM technology for detecting adult tissue stem cell-toxic drug candidates before conventional preclinical testing in animals or clinical trials. Asymmetrex and AlphaSTAR plan to market the new technology to pharmaceutical companies. The implementation of AlphaSTEM technology would accelerate drug development and reduce adverse drug events for volunteers and patients. At full capacity use, AlphaSTEM could reduce U.S. drug development costs by $4-5 billion each year.

About Asymmetrex (http://asymmetrex.com/)

Asymmetrex, LLC is a Massachusetts life sciences company with a focus on developing technologies to advance stem cell medicine. Asymmetrexs founder and director, James L. Sherley, M.D., Ph.D. is an internationally recognized expert on the unique properties of adult tissue stem cells. The companys patent portfolio contains biotechnologies that solve the two main technical problems production and quantification that have stood in the way of successful commercialization of human adult tissue stem cells for regenerative medicine and drug development. In addition, the portfolio includes novel technologies for isolating cancer stem cells and producing induced pluripotent stem cells for disease research purposes. Currently, Asymmetrexs focus is employing its technological advantages to develop facile methods for monitoring adult stem cell number and function in clinically important human tissues.

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Boston Stem Cell Biotech Start-up Asymmetrex Will Present Essential Technologies for Stem Cell Medical Engineering at ...

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Orthopedic Stem Cell Therapy - Queens, New York
Benjamin Bieber MD of #CrossBayPMR in Howard Beach, New York has had great success with #stemcell therapy using your own fat cells. Avoid invasive #jointreplacement surgery and get back to...

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BOSTON (TheStreet) --A question about Neuralstem (CUR - Get Report) and its stem-cell therapy for ALS kicks off this week's Biotech Stock Mailbag.

Steve writes, "If 47% of the people responded well and their progression of the disease slowed considerably, then I see this as a huge success for a disease with no cure. I don't have ALS or know anyone that does, but if I had it, I would immediately want the treatment knowing that there is a 47% chance that I will respond positively to it and it would DRASTICALLY slow the progression of the disease. Wouldn't you agree, or am I missing the point somewhere?"

Eight of the 15 (53%) ALS patients enrolled in the study saw their ALSFRS scores fall from an average of 40 to 14 over nine months. This is a rapid decline in muscle function and suggests NSI-566 accelerates the progression of ALS.

If you believe 47% of patients in the Neuralstem study benefit from NSI-566, you can't ignore the 53% of patients who fare far worse and may actually be harmed by the stem cell therapy.

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Biotech Stock Mailbag: Neuralstem, Genfit, Intercept, Amarin

GERMANTOWN, MD, March 12, 2015 -- Neuralstem, Inc. (NYSE MKT: CUR) announced top line data from the Phase II trial of NSI-566 spinal cord-derived neural stem cells under development for the treatment of amyotrophic lateral sclerosis (ALS). The study met primary safety endpoints. The maximum tolerated dose of 16 million transplanted cells and the surgery was well tolerated.

Secondary efficacy endpoints at nine months post-surgery indicate a 47% response rate to the stem cell treatment, as measured by either near-zero slope of decline or positive slope of ALSFRS score in seven out of 15 patients and by either a near-zero decline, or positive strengthening, of grip strength in seven out of 15 patients. Grip strength is an indicator of direct muscle strength of the lower arm. ALSFRS is a standard clinical test used to evaluate the functional status of ALS patients. The average ALSFRS score for responders at 9 months after treatment was 37. Non-responders scored an average of 14. These scores represent 93%, versus 35%, of the baseline score retained, respectively, by the responders versus non-responders at 9 months, which is a statistically significant difference. As measured by an average slope of decline of ALSFRS, responders' disease progression was -0.007 point per day, while non-responders' disease progression was -0.1 per day, which was again statistically significant. Lung function as measured by Seated Vital Capacity shows that responder patients remained within 94% of their starting scores, versus 71% for non-responder patients. The trial met its primary safety endpoints. Both the surgery and cells were well-tolerated, with one patient experiencing a surgical serious adverse event.

"In this study, cervical intervention was both safe and well-tolerated with up to 8 million cells in 20 bilateral injections," said Karl Johe, PhD, Neuralstem Chief Scientific Officer. "The study also demonstrated biological activity of the cells and stabilization of disease progression in a subset of patients. As in the first trial, there were both responders and non-responders within the same cohort, from patients whose general pre-surgical presentation is fairly similar. However, we believe that through the individual muscle group measurements, we may now be able to differentiate the responders from the non-responders.

"Our therapy involves transplanting NSI-566 cells directly into specific segments of the cord where the cells integrate into the host motor neurons. The cells surround, protect and nurture the patient's remaining motor neurons in those various cord segments. The approximate strength of those remaining motor neuron pools can be measured indirectly through muscle testing of the appropriate areas, such as in the grip strength tests. We believe these types of endpoints, measuring muscle strength, will allow us to effectively predict patients that will respond to treatment, adding a sensitive measure of the therapeutic effects after treatment. Testing this hypothesis will be one of the primary goals of our next trial." The full data is being compiled into a manuscript for publication.

"We believe the top-line data are encouraging," said Eva Feldman MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System, and an unpaid consultant to Neuralstem. "We were able to dose up to 16 million cells in 40 injections, which we believe to be the maximum tolerated dose. As in the first trial, the top-line data show disease stabilization in a subgroup of patients. Perhaps equally as important, we believe the top-line data may support a method of differentiating responders from non-responders, which we believe will support our efforts as we move into the next, larger controlled trial expected to begin this summer."

"The top-line data look very positive and encouraging. If this proportion of patients doing well after treatment can be corroborated in future therapeutic trials, it will be better than any response seen in any previous ALS trials," said site principal investigator, Jonathan D. Glass, MD, Director of the Emory ALS Center. "Elucidating which factors define a patient who may have a therapeutic response to the stem cell treatment will be the next key challenge. We are hopeful that a set of predictive algorithms can be established to help pre-select the responders in our future trials."

"We were very excited to participate as a site in this clinical trial," said Merit Cudkowicz, MD, Chief of Neurology, Massachusetts General Hospital and Co-Chair of the Northeast ALS Consortium (NEALS). "We are hopeful with respect to the top-line results and we need to move swiftly and safely forward to confirm the responder effect and identify people who might benefit from this treatment approach."

The open-label, dose-escalating trial treated 15 ambulatory patients, divided into 5 dosing cohorts, at three centers, Emory University Hospital in Atlanta, Georgia, the ALS Clinic at the University of Michigan Health System, in Ann Arbor, Michigan, and Massachusetts General Hospital in Boston, Massachusetts, and under the direction of principal investigator (PI), Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. Dosing increased from 1 million to 8 million cells in the cervical region of the spinal cord. The final trial cohort also received an additional 8 million cells in the lumbar region of the spinal cord.

The company anticipates commencing a later-stage, multicenter trial of NSI-566 for treatment of ALS in 2015. Neuralstem has received orphan designation by the FDA for NSI-566 in ALS.

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Neuralstem announces topline results of Phase II ALS trial

WASHINGTON: Researchers, including one of Indian-origin, have created a 'heart-on-a-chip' loaded with human cardiac muscle cells that mimic the real organ to serve as a novel tool to screen medicines. Researchers developed a network of pulsating cardiac muscle cells housed in an inch-long silicone de-vice that effectively models human heart tissue, and they have demonstrated the viability of this system as a drug-screening tool by testing it with cardiovascular medications.

This organ-on-a-chip represents a major step forward in the development of accurate, faster methods of testing for drug toxicity, researchers said. "Ultimately, these chips could replace the use of animals to screen drugs for safety and efficacy," said professor Kevin Healy from the University of California, Berkeley. The authors noted a high failure rate associated with the use of non-human animal models to predict human reactions to new drugs.

"It takes about 5 billion on average to develop a drug, and 60% of that figure comes from upfront costs in the research and development phase. Using a well-designed model of a human organ could significantly cut the cost and time of bringing a new drug to market," said Healy.

The heart cells were derived from human-induced pluripotent stem cells, the adult stem cells that can be coaxed to become many different types of tissue. Researchers designed their heart-on-a-chip so that its 3D structure would be comparable to the geometry and spacing of connective tissue fibre in a human heart.

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Drug testing on heart-on-a-chip gets a step closer

Durham, NC (PRWEB) March 11, 2015

Scientists report in the current issue of STEM CELLS Translational Medicine that they have been able to clone a line of defective stem cells behind a rare, but devastating disease called Fanconi Anemia (FA). Their achievement opens the door to drug screening and the potential for a new, safe treatment for this often fatal disease.

FA is a hereditary blood disorder that leads to bone marrow failure (FA-BMF) and cancer. Patients who suffer from FA have a life expectancy of 33 years. Currently, a bone marrow transplant offers the only possibility for a cure. However, this treatment has many risks associated with it, especially for FA patients due to their extreme sensitivity to radiation and chemotherapy.

Although various consequences in hematopoietic stem cells (the cells that give rise to all the other blood cells) have been attributed to FA-BMF, its cause is still unknown, said Megumu K. Saito, M.D., Ph.D., of Kyoto Universitys Center for iPS Cell and Application, and a lead investigator on the study. His laboratory specializes in studying the kinds of pediatric diseases in which a thorough analysis using mouse models or cultured cell lines is not feasible, so they apply disease-specific induced pluripotent stem cells (iPSCs) instead.

To address the FA issue, he explained, our team (including colleagues from Tokai University School of Medicine) established iPSCs from two FA patients who have the FANCA gene mutation that is typical in FA. We were then able to obtain fetal type immature blood cells from these iPSCs.

When observing the iPSCs, the researchers found that the characteristics of immature blood cells from FA-iPSCs were different from control cells. The FA-iPSCs showed an increased DNA double-strand break rate, as well as a sharp reduction of hematopoietic stem cells compared to the control group of non-FA iPSCs.

These data indicate that the hematopoietic consequences in FA patients originate from the earliest hematopoietic stage and highlight the potential usefulness of iPSC technology for explaining how FA-BMF occurs, said Dr. Saito. Since conducting a comprehensive analysis of patient-derived affected stem cells is not feasible without iPSC technology, the technology provides an unprecedented opportunity to gain further insight into this disease.

This work shows promise for identifying the initial pathological event that causes the disease, which would be a first step in working toward a cure, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

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The full article, Pluripotent cell models of Fanconi anemia identify the early pathological defect in human hemoangiogenic progenitors, can be accessed at http://www.stemcellstm.com.

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Stem Cell Clones Could Yield New Drug Treatment for Deadly Blood Disease

The author has posted comments on this articlePTI | Feb 23, 2015, 12.47AM IST

Ten different donor sources have been used so far and new germ-cell lines have been created from all of them, researchers said.

Page 1 of 4

Scientists at Cambridge University collaborated with Israel's Weizmann Institute of Science and used stem cell lines from embryos as well as from the skin of five different adults. Researchers have previously created live baby mice using engineered eggs and sperm, but until now have struggled to create a human version of these 'primordial germ' or stem cells.

Ten different donor sources have been used so far and new germ-cell lines have been created from all of them, researchers said.

"We have succeeded in the first and most important step of this process, which is to show we can make these very early human stem cells in a dish," said Azim Surani, professor of physiology and reproduction at Cambridge, who heads the project.

"We have also discovered that one of the things that happens in these germ cells is that epigenetic mutations, the cell mistakes that occur with age, are wiped out," said Surani, who was involved in research that led to the birth of Louise Brown, the world's first test-tube baby, in 1978.

Jacob Hanna, the specialist leading the project's Israeli arm, said it may be possible to use the technique to create a baby in just two years.

"It has already caused interest from gay groups because of the possibility of making egg and sperm cells from parents of the same sex," he said. The details of the technique were published in the journal Cell.

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Now, same-sex couples can make babies