Archive for the ‘Gene Therapy Research’ Category

Washington, Feb 16 (ANI)): Scientists have found that gene mutations that shorten the largest human protein are behind idiopathic dilated cardiomyopathy (DCM), a familial heart disease.

For decades, researchers have sought a genetic explanation for DCM, a weakening and enlargement of the heart that puts million of people at risk of heart failure each year.

Because idiopathic DCM occurs as a familial disorder, researchers have long searched for genetic causes, but for most patients the etiology for their heart disease remained unknown.

Now, new work from the lab of Christine Seidman, a Howard Hughes Investigator and the Thomas W. Smith Professor of Medicine and Genetics at Harvard Medical School and Brigham and Women's Hospital, and Jonathan Seidman, the Henrietta B. and Frederick H. Bugher Foundation Professor of Genetics at Harvard Medical School, has found that mutations in the gene TTN account for 18 percent of sporadic and 25 percent of familial DCM.

"Until the development of modern DNA sequencing platforms, the enourmous size of the TTN gene prevented a comprehensive analyses - but now we know TTN is a major cause of DCM," said Christine.

DCM may cause shortness of breath, chest pain, and limited exercise capacity. DCM increases the risk of developing heart failure, for which no cure is available, and the risk of stroke and sudden cardiac death.

These findings will not only help patients understand the cause of their DCM symptoms, but also help to screen family members who might be at risk of developing the condition. Early identification of those at risk allows early intervention with medications that reduce workload on the heart and help prevent the changes in heart muscle, called remodeling, that lead to heart failure.

As DCM progresses, remodeling of the heart tissue makes the heart more prone to disturbances in the normal heart rhythm that can lead to stroke, heart attack and sudden death.

Using next generation sequencing tools that substantially reduce the cost per base (the TTN sequence contains 100,000 bases) by orders of magnitude over earlier standards, the Seidmans were able to perform comprehensive screening for TTN mutations for the first time. They analyzed TTN in 312 DCM patients, 231 HCM patients, and 249 individuals with no disease. Of the many mutations identified, 72 make titin, the largest human protein, shorter.

Called TTN truncating variants, these specific mutations appeared almost exclusively in patients with DCM.

"Our hypothesis is that any variant that shortens titin is going to cause DCM, which will lead to heart failure by the same mechanism," said Jonathan.

A better understanding of pathological mechanism may lead to better and more direct therapies for treatment and prevention of DCM.

The findings have been reported in the New England Journal of Medicine. (ANI)

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'Broken' heart caused by genetic mutation

ScienceDaily (Feb. 15, 2012) — For decades, researchers have sought a genetic explanation for idiopathic dilated cardiomyopathy (DCM), a weakening and enlargement of the heart that puts an estimated 1.6 million Americans at risk of heart failure each year. Because idiopathic DCM occurs as a familial disorder, researchers have long searched for genetic causes, but for most patients the etiology for their heart disease remained unknown.

Now, new work from the lab of Christine Seidman, a Howard Hughes Investigator and the Thomas W. Smith Professor of Medicine and Genetics at Harvard Medical School and Brigham and Women's Hospital, and Jonathan Seidman, the Henrietta B. and Frederick H. Bugher Foundation Professor of Genetics at Harvard Medical School, has found that mutations in the gene TTN account for 18 percent of sporadic and 25 percent of familial DCM.

"Until the development of modern DNA sequencing platforms, the enourmous size of the TTN gene prevented a comprehensive analyses -- but now we know TTN is a major cause of DCM," said Christine Seidman, who reported the findings February 16 in the New England Journal of Medicine.

Idiopathic DCM is one of three different types of cardiomyopathy (the term "idiopathic" indicates that acquired causes for DCM such as atherosclerosis, excess drinking or viral infections have been excluded). It affects only about 4 in 10,000 Americans, but may be under-diagnosed because symptoms often appear late in the course of disease. DCM may cause shortness of breath, chest pain, and limited exercise capacity. DCM increases the risk of developing heart failure, for which no cure is available, and the risk of stroke and sudden cardiac death.

These findings will not only help patients understand the cause of their DCM symptoms, but also help to screen family members who might be at risk of developing the condition. Early identification of those at risk allows early intervention with medications that reduce workload on the heart and help prevent the changes in heart muscle, called remodeling, that lead to heart failure.

As DCM progresses, remodeling of the heart tissue makes the heart more prone to disturbances in the normal heart rhythm that can lead to stroke, heart attack and sudden death. "One of the added values to knowing that you are at risk for developing DCM is that we can do prophylactic screening so that silent arrhythmias are picked up before they become harmful," said Christine Seidman. "The discovery is immediately translatable into clinical practice to provide patients with gene-based diagnosis." The Partner's Laboratory for Molecular Medicine, an HMS affiliate, has incorporated TTN analyses.

The Seidmans and others had previously linked other gene mutations to about 20 to 30 percent of idiopathic DCM cases -- and, with more success, to a related disease, hypertrophic cardiomyopathy. They had examined almost all of the genes linked to muscle units known as sarcomeres, but saved the biggest for last: TTN, which encodes the protein titin. At approximately 33,000 amino acids, titin is the largest human protein.

"Titin was a missing link," said Christine Seidman. "A very large missing link."

The Seidmans' collaborated with researchers from the Imperial College (London) and the University of Washington. Traditional sequencing methods had previously found only a few TTN variants in patients with DCM because complete, accurate sequencing was too expensive.

Using next generation sequencing tools that substantially reduce the cost per base (the TTN sequence contains 100,000 bases) by orders of magnitude over earlier standards, the Seidmans were able to perform comprehensive screening for TTN mutations for the first time. They analyzed TTN in 312 DCM patients, 231 HCM patients, and 249 individuals with no disease.

Of the many mutations identified, 72 make the titin protein shorter.

Called TTN truncating variants, these specific mutations appeared almost exclusively in patients with DCM. "Our hypothesis is that any variant that shortens titin is going to cause DCM, which will lead to heart failure by the same mechanism," said Jonathan Seidman.

To identify the pathological mechanism, the Seidmans plan to model a handful of TTN truncating mutations in mice.

One concern in the search for disease causing genes is that, while there will be many gene variants discovered, only a few will cause disease. This is particularly true for missense mutations that cause single nucleotide changes -- changes that substitute a single amino acid within the protein.

"We often don't know if a missense mutation significantly impacts a protein's function, until we model it and study its effects," said Jonathan Seidman.

However, in the case of truncating mutations, "it's the converse," he continued. "We don't have to model all of those different mutations that truncate titin, becuase they all foreshorten the protein. We can pick a few representative ones and expect that they will reveal a common mechanism."

A better understanding of the mechanism may lead to better and more direct therapies for treatment and prevention of DCM.

This research was funded by Howard Hughes Medical Institute; National Institutes of Health; Leducq Foundation; American Heart Association and Muscular Dystrophy Association; and the UK National Institute for Health Research Cardiovascular Biomedical Research Unit.

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The above story is reprinted from materials provided by Harvard Medical School. The original article was written by Elizabeth Dougherty.

Note: Materials may be edited for content and length. For further information, please contact the source cited above.

Journal Reference:

Daniel S. Herman, Lien Lam, Matthew R.G. Taylor, Libin Wang, Polakit Teekakirikul, Danos Christodoulou, Lauren Conner, Steven R. DePalma, Barbara McDonough, Elizabeth Sparks, Debbie Lin Teodorescu, Allison L. Cirino, Nicholas R. Banner, Dudley J. Pennell, Sharon Graw, Marco Merlo, Andrea Di Lenarda, Gianfranco Sinagra, J. Martijn Bos, Michael J. Ackerman, Richard N. Mitchell, Charles E. Murry, Neal K. Lakdawala, Carolyn Y. Ho, Paul J.R. Barton, Stuart A. Cook, Luisa Mestroni, J.G. Seidman, Christine E. Seidman. Truncations of Titin Causing Dilated Cardiomyopathy. New England Journal of Medicine, 2012; 366 (7): 619 DOI: 10.1056/NEJMoa1110186

Note: If no author is given, the source is cited instead.

Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of ScienceDaily or its staff.

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Genetic mutation implicated in 'broken' heart

Sydney, Feb 16 (IANS) Gene therapy not only helps injured brain cells live longer and regenerate but also changes the shape of the cells, says a new study.

Alan Harvey, professor of anatomy and physiology at The University of Western Australia, said brain cell morphology (study of form and structure of organisms) was very important in determining how a cell communicated with other cells and formed the circuits that allowed the brain to function.

"Our previous work has shown that when growth-promoting genes are introduced into injured brain cells for long periods of time (up to nine months), the cells' capacity for survival and regeneration is significantly increased," he was quoted as saying by the journal Public Library of Science One.

"We have now shown that these same neurons have also changed shape in response to persistent over-expression of the growth factors," said Harvey, who co-authored the study with Jennifer Rodge, according to a statement of Western Australia.

"Any changes in morphology are therefore likely to alter the way neurons receive and transmit information. These changes may be beneficial but could also interfere with normal brain circuits, reducing the benefits of improved survival and regeneration."

Harvey said the results were significant for those involved in designing gene therapy based protocols to treat brain and spinal cord injury and degeneration.

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Gene therapy helps regenerate injured brain cells

CAMBRIDGE, Mass., Feb. 15, 2012 (GLOBE NEWSWIRE) -- Pathfinder Cell Therapy, Inc. ("Pathfinder," or "the Company") (OTCQB:PFND.PK - News), a biotechnology company focused on the treatment of diseases characterized by organ-specific cell damage, today announced that the European Patent Office (EPO) has communicated its intent to grant Pathfinder's first European Patent, EP1896571, which provides intellectual property protection for the Company's cell-based technology throughout this region. The patent covers the Company's proprietary Pathfinder Cells ("PCs"), which are a newly identified non-stem cell mammalian cell type that has demonstrated the ability to stimulate regeneration of local damaged tissue without being incorporated into the new tissue.

PCs are being developed by Pathfinder for the treatment of a number of important human diseases and have demonstrated encouraging preclinical results to date, including the ability to completely reverse diabetes in a mouse model (results published in Rejuvenation Research, April 20101). European Patent EP1896571 includes composition of matter claims for the Company's proprietary PCs, as well as methods of use relating to cell-based therapies, specifically for the treatment of diabetes.

Richard L. Franklin, M.D., Ph.D., Founder, CEO and President of Pathfinder said, "We are pleased to report the allowance of this critical patent in Europe, which protects our core Pathfinder Cell technology. Cell-based therapy using PCs may represent a unique approach to the treatment of diabetes and other diseases where regenerating damaged local tissue may restore normal organ function. We will continue to grow our international patent estate as we work to bring our innovative therapy to patients in need."

About Pathfinder

Pathfinder is developing a novel cell-based therapy and has generated encouraging preclinical data in models of diabetes, renal disease, myocardial infarction, and critical limb ischemia, a severe form of peripheral vascular disease. Leveraging its internal discovery of Pathfinder Cells ("PCs") Pathfinder is pioneering a new field in regenerative medicine.

PCs are a newly identified mammalian cell type present in very low quantities in a variety of organs, including the kidney, liver, pancreas, lymph nodes, myometrium, bone marrow and blood. Early studies indicate that PCs stimulate regeneration of damaged tissues without the cells themselves being incorporated into the newly generated tissue. Based on testing to date, the cells appear to be "immune privileged," and their effects appear to be independent of the tissue source of PCs. For more information please visit: http://www.pathfindercelltherapy.com.

1Karen Stevenson, Daxin Chen, Alan MacIntyre, Liane M McGlynn, Paul Montague, Rawiya Charif, Murali Subramaniam, W.D. George, Anthony P. Payne, R. Wayne Davies, Anthony Dorling, and Paul G. Shiels. Rejuvenation Research. April 2011, 14(2): 163-171. doi:10.1089/rej.2010.1099

FORWARD LOOKING STATEMENTS

This press release contains forward-looking statements. You should be aware that our actual results could differ materially from those contained in the forward-looking statements, which are based on management's current expectations and are subject to a number of risks and uncertainties, including, but not limited to, our inability to obtain additional required financing; costs and delays in the development and/or FDA approval, or the failure to obtain such approval, of our product candidates; uncertainties or differences in interpretation in clinical trial results, if any; our inability to maintain or enter into, and the risks resulting from our dependence upon, collaboration or contractual arrangements necessary for the development, manufacture, commercialization, marketing, sales and distribution of any products; competitive factors; our inability to protect our patents or proprietary rights and obtain necessary rights to third party patents and intellectual property to operate our business; our inability to operate our business without infringing the patents and proprietary rights of others; general economic conditions; the failure of any products to gain market acceptance; technological changes; and government regulation. We do not intend to update any of these factors or to publicly announce the results of any revisions to these forward-looking statements.

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Pathfinder Announces First European Patent Allowance for Pathfinder Cells

Illustration: Rocco Fazzari

Remember Pandora's box? In the future, when the seaside mansions have sunk and my bit of Redfern grunge is absolute waterfront, our children's children will look back gobsmacked by the evils we so blithely loosed; nuclear war, climate change, gene meddling.

The Murdochs and Bransons will have vanished with their palaces beneath tides they helped create but two of the lowly will be revered forever; an organic farmer from Kojanup, Western Australia, and a Brisbane breast cancer survivor.

What do Steve Marsh and Yvonne D'Arcy have in common? Only that both are fighting barehanded to stuff the gene genies back in their box.

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It is all very Promethean; a story of money and power and the urge to possess life itself. When Jonas Salk invented the polio vaccine an interviewer asked him about patenting it. Salk laughed. “Could you patent the sun?”

Few these days have Salk's wisdom. Both Monsanto and Myriad Genetics believe they own the essential stuff of life – and this is what Marsh and D'Arcy are testing. Their two quite separate cases will help shape not just state or country, but the global future of food and health. Small people. Big stuff.

Pivotal to the legal issue is the distinction between art and nature, the made and the found. The made is owned, and can therefore be patented. The found is given, and cannot.

It's no semantic trifle. It's a battle for the commons, as important – perhaps more important – as the enclosure battles of manorial England. Like those enclosures, it is a war waged by the rich against the poor. But now the poor are starting to fight back, in the courts.

Marsh was an organic farmer growing wheat, oats, rye, canola and fat lambs in Kojanup. I say "was", because when in late 2010 his crop was contaminated by modified canola genes from the next-door farm of his childhood friend Michael Baxter, Marsh lost his organic certification. The GM seeds had blown in. He lost 70 per cent of his livelihood. Regaining certification will take years. He had no choice but to sue his mate.

The court case is imminent but the law remains murky. Australian GM farmers are not obliged to inform their neighbours, and remedies are unclear. Yet organic certification rightly takes a zero-tolerance approach since there's virtually no longitudinal research on the safety of GM food and growing evidence of harm.

A letter from ex-army plant pathologist Emeritus Professor Don Huber to the US Agriculture Secretary, Tom Vilsak, in January last year warned of a “previously unknown” pathogen, apparently a micro fungus, pervading Roundup Ready crops. Huber suspected this bug of causing “epidemic” reproductive failure in swine, cattle and horses, with abortion rates up to 45 per cent and recommended an “immediate moratorium”.

Yet, here, governments and farming federations insist on treating these as simple neighbourhood disputes to be sorted over the back fence.

This is moronic, since the stakes are so much higher. Marsh is supported by various green groups, including the Safe Food Foundation, with legal firm Slater & Gordon working pro bono, but it's still costly. The respondent, Baxter, is supported by the WA Pastoralists and Graziers Association. Monsanto denies contributing (though some dispute this).

Maybe Marsh should be thankful Monsanto isn't suing him for patent infringement, as it sued Saskatchewan farmer Percy Schmeiser. Schmeiser was a seed saver. GM contamination set his breeding program back decades, but Monsanto regarded it as a theft and sued him for $37 a hectare. Schmeiser eventually won, but not without a 10-year trail to the Supreme Court.

Patent law is meant to protect inventions, not discoveries. Monsanto's RR gene is clearly synthetic: the argument is about the market control this allows (patents are not supposed to create monopolies), the behaviours used to protect it (allegedly including threats and intimidation) and the unknown effects of genetically modified organisms in the food chain (you can't patent anything harmful).

D'Arcy's case, opening on Monday in the Federal Court in Sydney, takes a different tack. Here the argument is about Myriad's right to own a gene – the "breast cancer gene" BRCA1 – that has been extracted from the human body but not otherwise modified.

But how can a private company have intellectual property rights over something that, for better or worse, inhabits every cell in your body? Myriad argues, predictably, that just isolating the gene renders it “markedly different”. Professor Peter Cashman of the University of Sydney's law school argues the other way (as does, now, the US government).

If Myriad wins, it can continue to monopolise not the therapy – oh, no – but the test, for which it charges $US3200 ($3000). (The American Civil Liberties Union's challenge to private sequestration of human genes opens in the US Supreme Court tomorrow.)

Myriad is not alone. About 10,000 genes, more than a third of the human genome, are now privately owned, including genes for muscular dystrophy and polycystic kidney disease. So the implications of this test case, which will start to determine the legality of "owning" genes that were found, not made, are immense.

It's more than just money. With last year's creation of the first wholly synthetic life form, Craig Venter's replicating single-cell microbe, the capacity to own new life – the Frankenstein faculty – becomes quarantined by the very same corporates, biotechs and pharmas that have repeatedly proved themselves oblivious to the good of the species.

So where's government? Out to lunch, basically. The Gene Technology Act 2000 should have established a clear framework for farmers' GM liabilities but didn't, leaving them to rely on common law torts of negligence and nuisance. This means cases such as Marsh v Baxter will cost millions and destroy communities.

As to the human genome, Senator Bill Heffernan introduced a Patent Amendment Bill last September that would prohibit all patenting of human genes. But with every sandstone university and the Academy of Science opposing it – uh, because it might harm business! – the bill is bogged in indecision.

And all the while the genies fly. Eat your heart out, Prometheus. This stuff makes Pandora look benign.

Read more:
Health of all on line as Davids fight gene giants

The researchers used gene therapy in rats to stimulate production of somatostatin, a seizure-stopping chemical that naturally occurs in the brain. The study was published in the February issue of the journal Neuroscience Letters.

More than 3 million people in the United States have epilepsy, according to the Centers for Disease Control and Prevention. This lifelong disease is characterized by uncontrollable seizures and can keep people from living independently or holding jobs, particularly if they do not respond to seizure-controlling medication. Finding novel ways to prevent these seizures could help people with epilepsy live more normal, symptom-free lives, said Dr. Paul Carney, chief of the division of neurology in the UF College of Medicine department of pediatrics and senior author of the study.

“For years people have focused only on treating the disease, not preventing the disease,” Carney said. “The mantra is no seizures, no side effects.”

People with epilepsy tend to have lower levels of the hormone somatostatin, as do people with Alzheimer’s disease. Although somatostatin, which belongs to a group of protein-like molecules called neuropeptides, is present in the brains of people with epilepsy, scientists have shown that its levels decrease during seizures, said Rabia Zafar, the lead author of the paper and a former postdoctoral associate in Carney’s lab.

To test whether they could prevent seizures by bolstering levels of this hormone, the researchers administered a dose of the gene that triggers somatostatin expression. A harmless virus transported the gene safely through the body.

“There is some somatostatin in the brain anyway, because it’s a neuropeptide, but there was a dramatic increase after the injection,” Zafar said.

Boosting somatostatin levels led to weaker and shorter seizures, and none of the subjects that received the injection suffered the highest level of seizure. Better yet, the treatment did not result in unwanted side effects. The only side effect was positive: subjects learned better after the treatment.

“Being able to restore somatostatin up to normal levels allows the brain to heal itself and that is the idea here,” Carney said. “We’re putting something back in that is normally there and allowing the brain to pick it up as part of its normal machinery. We’re not putting in a drug.”

In addition to epilepsy, studies have shown that somatostatin may play a role in aging and neurodegenerative disorders such as Alzheimer’s disease, Carney said. Somatostatin is a neuromodulator, which means it can alter how nerve cells behave.

In this study, the researchers focused on temporal lobe epilepsy, the most common form of the disease. Although medication helps control seizures in most people with this type of epilepsy, about 30 percent of patients do not respond to therapy, Carney said.

“We need better, more effective treatments for a large population of children and adults who don’t respond to conventional treatments,” he said. “Gene therapy, as well as other forms of treatment, are emerging, and there is the hope and promise they will offer more effective and novel treatments for people with drug-resistant epilepsy.”

But the researchers caution that this study is just a first step. Additional research is needed before the technique can be attempted in humans. Researchers are particularly focused on ensuring the treatment does not cause inflammation and discovering the best way to administer it, either be injection to the brain or a less invasive intravenous infusion.

“What effect a compound is going to have partly depends on where in the seizure circuit that new compound or gene is being placed. You could put the same chemical in two places and get two different results,” said Dr. Edward Bertram III, a professor of neurology at the University of Virginia, who was not involved in the study. “That is going to be the issue as they try to develop this: Where should we be putting this to have the best effect? On the promising side, they put (the gene) in a restricted area and had an effect. That is a great first step.”

Provided by University of Florida (news : web)

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Gene therapy for epilepsy could stop seizures

Stanley, Kan. — It’s a special Valentine’s Day gift for Jake the dog.  His family gave him a stem cell treatment that they hope will extend his life.

Jake is an 11-year-old yellow lab.  He’s been part of the LeBlanc family since he was a puppy.  Jake’s owner, Elizabeth LeBlanc, calls him her “first baby.”  But then Mia and Aidan were born and at ages eight and five years old, they love to play with Jake.

When the LeBlanc’s noticed Jake was having trouble getting around they wanted to help.  They tried medication, but say it didn’t work for very long.  Then Mia saw a segment about a stem cell treatment for dogs on t.v. and asked if they could get it for Jake.  The LeBlanc’s called their veterinarian and found out the Stanley Veterinary Clinic in Stanley, Kansas is the only place in the metro where they can do the entire procedure in house.

Dr. Les Pelfrey, D.V.M. explained the procedure.

“We’re going to collect about 20 grams of fat surgically and then we’re going to process it in our lab here in house then we’re going to reintroduce those stem cells after we activate them back into the affected joints,” said Dr. Les Pelfrey.

The procedure can cost $3000. The dog’s fatty tissue has to be sent off to a lab for the stem cells to be extracted.  But at the Stanley Veterinary Clinic they can process the stem cells in their own lab, cutting the cost to $1800.00.

Jake’s arthritis is affecting his hips, knees, one elbow and one shoulder.  Dr. Pelfrey made an incision and removed the fatty tissue from Jake.  Then veterinary technician Stephanie Pierce took it to the lab to break it down, cook it and then spin it.  The final product?  Stem cells that were then re-injected into Jake’s joints to help him grow cartilage.  Pierce says Jake will “act like a puppy again as far as moving around.”

The LeBlancs can’t wait to see the results.

“For 12 years he’s given us love and joy so we just want to give him a better quality of life,” LeBlanc said.

Jake will spend the night at the Stanley Veterinary Clinic.  He should be able to head home tomorrow.  Jake and the LeBlancs should notice results in the next few weeks.

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Dog Receives First-Ever Stem Cell Therapy in Kansas City

14 February 2012 Last updated at 19:10 ET By James Gallagher Health and science reporter, BBC News

Bone marrow stem cell therapy offers "moderate improvement" to heart attack patients, according to a large UK review of clinical trials.

The analysis by the Cochrane Collaboration looked at 33 trials involving more than 1,700 patients.

It said longer-term studies were needed to see if the experimental therapy affected life expectancy.

The review comes a day after doctors reported the first case of using heart cells to heal heart attack damage.

If a patient survives a heart attack, dead heart muscle is replaced with scar tissue - leaving the patient weaker and possibly on a lifetime of medicine.

Researchers are beginning to show that taking cells from a heart, growing millions of new heart cells in the laboratory and pumping those back into the heart may reduce scar tissue and lead to new heart muscle.

Continue reading the main story “Start Quote

Stem cell therapy may also reduce the number of patients who later die or suffer from heart failure, but currently there is a lack of statistically significant evidence based on the small number of patients treated so far”

End Quote Dr Enca Martin-Rendon Lead researcher

However, the trials are at a very early stage and in only a handful of patients. Using a similar technique with cells taken from the bone marrow, which is a prime source of stem cells, has a much longer pedigree.

The report by Cochrane pooled the data from all 33 bone marrow trials which had taken place up to 2011.

It concluded that bone marrow therapy "may lead to a moderate long-term improvement" in heart function which "might be clinically very important".

Longer life uncertain

It said there was still no evidence of "any significant effect on mortality" in comparison with standard treatment. However, this may be due to the size of the studies and that patients were followed for a short period of time.

Lead author Dr Enca Martin-Rendon, from NHS Blood and Transplant at the John Radcliffe Hospital in Oxford, said: "This new treatment may lead to moderate improvement in heart function over standard treatments.

"Stem cell therapy may also reduce the number of patients who later die or suffer from heart failure, but currently there is a lack of statistically significant evidence based on the small number of patients treated so far."

Prof Anthony Mathur, from Barts and the London School of Medicine and Dentistry, is leading the largest ever trial of stem cells in heart attack patients.

It starts this year, however, he told the BBC that the results could come quite quickly. Three thousand patients across Europe will take part. They will be injected with stem cells five days after a heart attack and then followed for two years to see if the therapy affects life expectancy.

Prof Peter Weissberg, medical director at the British Heart Foundation, said: "This review reflects the consensus of opinion about these trials - cell therapy has a modestly beneficial effect.

"Despite that, no-one knows why, or even if, cell therapies will translate into better survival or sustained improvement in damaged hearts. It's much too early to judge the likely long-term benefits."

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Bone gives 'some' heart healing

Public release date: 14-Feb-2012
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Contact: Jennifer Beal
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Wiley-Blackwell

Stem cell therapy moderately improves heart function after a heart attack, according to a systematic review published in The Cochrane Library. But the researchers behind the review say larger clinical trials are needed to establish whether this benefit translates to a longer life.

In a heart attack, the blood supply to parts of the heart is cut off by a blocked artery, causing damage to the heart tissue. The cells in the affected area start to die. This is called necrosis and in the days and weeks that follow, the necrotic area may grow, eventually leaving a large part of the heart unable to contract and increasing the risk of further heart problems. Stem cell therapy uses cells from the patient's own bone marrow to try to repair and reduce this damage. Currently, the treatment is only available in facilities with links to scientific research.

The authors of the review drew together all the available evidence to ask whether adult bone marrow stem cells can effectively prevent and repair the damage caused by a heart attack. In 2008, a Cochrane review of 13 stem cell therapy clinical trials addressed the same question, but the new review adds 20 more recent trials, drawing its conclusions from all 33. By incorporating longer follow up, the later trials provide a better indication of the effects of the therapy several years after treatment.

The total number of patients involved in trials was 1,765. All had already undergone angioplasty, a conventional treatment that uses a balloon to open the blocked artery and reintroduce the blood supply. The review's findings suggest that stem cell therapy using bone marrow-derived stem cells (BMSCs) can produce a moderate long-term improvement in heart function, which is sustained for up to five years. However, there was not enough data to reach firm conclusions about improvements in survival rates.

"This new treatment may lead to moderate improvement in heart function over standard treatments," said lead author of the study, Enca Martin-Rendon, of the Stem Cell Research laboratory, NHS Blood and Transplant at the John Radcliffe Hospital in Oxford, UK. "Stem cell therapy may also reduce the number of patients who later die or suffer from heart failure, but currently there is a lack of statistically significant evidence based on the small number of patients treated so far."

It is still too early to formulate guidelines for standard practice, according to the review. The authors say further work is required to establish standard methods, including cell dosage, timing of cell transplantation and methods to measure heart function. "The studies were hard to compare because they used so many different methods," said Martin-Rendon. "Larger trials with standardised treatment procedures would help us to know whether this treatment is really effective.

Recently, the task force of the European Society of Cardiology for Stem Cells and Cardiac Repair received funding from the European Union Seventh Framework Programme for Research and Innovation (EU FP7-BAMI) to start such a trial. Principal Investigator for the BAMI trial, and co-author of this Cochrane review, Anthony Mathur, said, ''The BAMI trial will be the largest stem cell therapy trial in patients who have suffered heart attacks and will test whether this treatment prolongs the life of these patients."

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Stem cell treatments improve heart function after heart attack

Hundreds of women with breast cancer should be tested for an aggressive form of the disease resulting from a genetic fault, according to new research.

Women with mutations in the BRCA1 gene have a much higher chance of developing breast cancer but also a higher risk of ovarian cancer.

These patients have up to a 65% chance of developing breast cancer by the time they are 70 and a 40% chance of ovarian cancer.

Researchers are now arguing that all women under 50 who are diagnosed with triple-negative (TN) breast cancer should also be offered testing for faults in the BRCA1 gene.

This could identify hundreds of extra women every year who may benefit from tailored therapy for BRCA1 breast cancer, such as platinum-based chemotherapy.

Clinical trials on radiotherapy have also suggested a certain type of radiotherapy could benefit women with BRCA1 mutations more than others.

Women would also be able to tell their families if they have the BRCA1 gene, meaning close relatives could be tested for the hereditary gene.

The research, published in the British Journal of Cancer, looked at more than 300 women with TN breast cancer. Almost one in five diagnosed under the age of 50 were found to have the BRCA1 mutation. This fault means breast cancer is more likely to develop at a younger age.

Current guidance from the National Institute for Health and Clinical Excellence (Nice) recommends BRCA1 testing is offered if the likelihood of detecting a mutation is greater than 20%.

However, based on their findings, researchers from the Institute of Cancer Research estimate more than one in three women with TN breast cancer caused by BRCA1 mutations would not have been tested using the current criteria.

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Study backs breast cancer gene test

SAN DIEGO--(BUSINESS WIRE)--

Pathway Genomics, a genetic testing laboratory specializing in genetic testing for nutrition and exercise response, inherited disease, prescription drug response and health condition risks, has received membership in the American Clinical Laboratory Association (ACLA). As the nation’s leading industry group to advocate lab interests with the U.S. Food and Drug Administration (FDA) and Congress, ACLA was created in 1971 and is committed to promoting public awareness about the value of laboratory services in preventing illness, diagnosing disease, and monitoring medical treatment.

“ACLA is proud to welcome Pathway Genomics as its newest member,” said Alan Mertz, president of ACLA. “Pathway Genomics is another example of our nation’s laboratories that are at the forefront of genetic testing and personalized medicine.”

In addition to Pathway’s ACLA membership, the San Diego-based company was recently accredited by the College of American Pathologists (CAP), and the recipient of a Certificate of Accreditation from the U.S. Department of Health and Human Services’ Centers for Medicare and Medicaid, under the Clinical Laboratory Improvement Amendments (CLIA) of 1988.

“ACLA is an essential organization within the laboratory community,” said Jim Plante, Pathway’s founder and CEO. “The approval of our membership is representative of the importance of genetic testing, and the promise and value of personalized health care.”

Using saliva samples collected from patients, Pathway provides genetic tests for drug responses, nutrition and exercise response, inherited genetic conditions, and risk of many diseases. Pathway consists of more than 40 scientific and medical professionals, including medical doctors, molecular geneticists, and genetic counselors, as well as an expert scientific advisory board.

About Pathway Genomics

Pathway Genomics owns and operates an on-site genetic testing laboratory that is accredited by the College of American Pathologists (CAP), accredited in accordance with the U.S. Health and Human Services’ Clinical Laboratory Improvement Amendments (CLIA) of 1988, and licensed by the state of California. The company uses customized and scientifically validated genetic testing technologies to generate personalized genetic reports, which address a variety of genetic issues, including an individual’s carrier status for recessive genetic conditions, food metabolism and exercise response, prescription drug response, and propensity to develop certain diseases, such as heart disease, type 2 diabetes, and cancer. Pathway’s services are available to patients through their physicians and select health management programs.

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American Clinical Laboratory Association Welcomes Pathway Genomics as New Member

Seattle Genetics (SGEN) reported Q4 EPS of (24c), compared with analysts' consensus estimate of (31c). The company's revenue also came in higher than expected. Seattle Genetics added that it expects its revenue from collaboration and licenses in 2012 to be $55M-$65M. Over the next year, the company expects to initiate multiple Phase III trials of its ADCETRIS drug for various indications, including CTCL and Hodgkin lymphoma. However, the company declined to provide guidance for ADCETRIS sales, saying on its conference call that it does not have enough experience with the market. Moreover, in a note to investors, Leerink Swann lowered its 2012-2014 U.S. ADCETRIS sales estimates, as the firm believes that the drug's uptake will be slower than it had previously expected. Seattle Genetics' guidance for an increase in gross-to-net discounts for the drug also was a factor in the firm's decision to reduce its sales estimates. However, Leerink continues to have a positive overall outlook on the company and maintains an Outperform rating on the shares. In early trading, Seattle Genetics fell 59c, or 3.11%, to $18.37.

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Seattle Genetics drops after providing no ADCETRIS sales guidance

February 14, 2012, 8:37 AM EST

By Ryan Flinn

Feb. 14 (Bloomberg) -- Stem cells grown from patients’ own cardiac tissue can heal damage once thought to be permanent after a heart attack, according to a study that suggests the experimental approach may one day help stave off heart failure.

In a trial of 25 heart-attack patients, 17 who got the stem cell treatment showed a 50 percent reduction in cardiac scar tissue compared with no improvement for the eight who received standard care. The results, from the first of three sets of clinical trials generally needed for regulatory approval, were published today in the medical journal Lancet.

“The findings in this paper are encouraging,” Deepak Srivastava, director of the San Francisco-based Gladstone Institute of Cardiovascular Disease, said in an interview. “There’s a dire need for new therapies for people with heart failure, it’s still the No. 1 cause of death in men and women.”

The study, by researchers from Cedars-Sinai Heart Institute in Los Angeles and Johns Hopkins University in Baltimore, tested the approach in patients who recently suffered a heart attack, with the goal that repairing the damage might help stave off failure. While patients getting the stem cells showed no more improvement in heart function than those who didn’t get the experimental therapy, the theory is that new tissue regenerated by the stem cells can strengthen the heart, said Eduardo Marban, the study’s lead author.

“What our trial was designed to do is to reverse the injury once it’s happened,” said Marban, director of Cedars- Sinai Heart Institute. “The quantitative outcome that we had in this paper is to shift patients from a high-risk group to a low- risk group.”

Minimally Invasive

The stem cells were implanted within five weeks after patients suffering heart attacks. Doctors removed heart tissue, about the size of half a raisin, using a minimally invasive procedure that involved a thin needle threaded through the veins. After cultivating the stem cells from the tissue, doctors reinserted them using a second minimally invasive procedure. Patients got 12.5 million cells to 25 million cells.

A year after the procedure, six patients in the stem cell group had serious side effects, including a heart attack, chest pain, a coronary bypass, implantation of a defibrillator, and two other events unrelated to the heart. One of patient’s side effects were possibly linked to the treatment, the study found.

While the main goal of the trial was to examine the safety of the procedure, the decrease in scar tissue in those treated merits a larger study that focuses on broader clinical outcomes, researchers said in the paper.

Heart Regeneration

“If we can regenerate the whole heart, then the patient would be completely normal,” Marban said. “We haven’t fulfilled that yet, but we’ve gotten rid of half of the injury, and that’s a good start.”

While the study resulted in patients having an increase in muscle mass and a shrinkage of scar size, the amount of blood flowing out of the heart, or the ejection fraction, wasn’t different between the control group and stem-cell therapy group. The measurement is important because poor blood flow deprives the body of oxygen and nutrients it needs to function properly, Srivastava said.

“The patients don’t have a functional benefit in this study,” said Srivastava, who wasn’t not involved in the trial.

The technology is being developed by closely held Capricor Inc., which will further test it in 200 patients for the second of three trials typically required for regulatory approval. Marban is a founder of the Los Angeles-based company and chairman of its scientific advisory board. His wife, Lisa Marban, is also a founder and chief executive officer.

--Editors: Angela Zimm, Andrew Pollack

-0- Feb/13/2012 22:32 GMT

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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Scarred Hearts Can Be Mended With Stem Cell Therapy, Study Shows

COLUMBIA, SC (WIS) - Cutting-edge arthritis treatment for our four-legged family members is now available in Columbia.

Banks Animal Hospital is the first in the area to offer in-house Stem Cell therapy. It uses your pets own body to heal itself.

Take 13-year-old Maggie, for example. The energetic pup has a limp that usually keeps her from jumping or going up stairs.

"Today when everybody's out there filming her little limp it's not as pronounced because she wants to please," said Maggie's owner, Beth Phibbs. "She's just a great dog."

But a great attitude wasn't enough to repair a bad case of cervical spine arthritis.

So Monday, Beth brought Maggie to Banks Animal Hospital for the Stem Cell therapy. Like many, Beth had never heard of Stem Cell work in animals. "Until Dr. Banks mentioned it to me I was like, beg your pardon?"

"There's no down side, no side effects because you're using your own cells," said Dr Ken Banks.

Banks and his staff first gather some of Maggie's blood and fat. Both are good places to find the repair cells they're after. Adult stem cells, not the controversial embryonic kind, are then separated and spun down.

"The repair system in Maggie's body has failed," said Jason Richardson of MediVet-America. "It's fallen asleep at the wheel, we're taking these repair cells, activating them so a chronic condition like osteo arthritis to Maggie will now be an acute illness."

This kind of treatment used to take days with material being shipped across the country, but now it can be done in hours.

"The ability to do it same day, convenience, the ability to do it in clinic saves a lot of money to the doctor which he can then pass on to the patient," said Richardson.

The treatment will still run you around $2,000, but Richardson says that's half of what the similar treatment use to cost.

When it's over, Maggie should be able to live out her life pain and drug free -- something Phibbs is looking forward to.

"I'm hoping in a couple of weeks she's gonna have a new lease on life," said Phibbs.

Copyright 2012 WIS. All rights reserved.

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Vet offers stem cell therapy for dogs

Teva Pharmaceutical Industries Ltd. (Nasdaq: TEVA; TASE: TEVA) and Gamida Cell Ltd. have completed enrollment of 100 patients for their Phase III clinical trial of StemEx, a cell therapy for the treatment of blood cancers, such as leukemia and lymphoma. StemEx is intended for adolescents and adults who cannot find a family related, matched bone marrow donor.

Teva and Gamida Cell are conducting the multi-center international clinical trial through their joint venture, which they own in equal shares. StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted together with the remaining, non-manipulated cells from the same unit.

Gamida Cell president and CEO Dr. Yael Margolin said, "The joint venture is planning to announce the safety and efficacy results of the Phase III StemEx trial in 2012 and to launch the product into the market in 2013. It is our hope that StemEx will provide the answer for the thousands of leukemia and lymphoma patients unable to find a matched, related bone marrow donor."

Margolin added, ?StemEx may be the first allogeneic cell therapy to be brought to market. This is a source of pride for Gamida Cell, as it further confirms the company?s leadership as a pioneer in cell therapy. In addition to StemEx, Gamida Cell is developing a diverse pipeline of products for the treatment of cancer, hematological diseases such as sickle cell disease and thalassemia, as well as autoimmune and metabolic diseases and conditions helped by regenerative medicine."

Nochi Dankner's life sciences investment arm Clal Biotechnology Industries Ltd. (TASE: CBI) owns 20% of Gamida Cell and Mordechai Zisser-controlled Elbit Medical Technologies Ltd. (TASE:EMTC) owns 31.6%.

Published by Globes [online], Israel business news - http://www.globes-online.com - on February 14, 2012

? Copyright of Globes Publisher Itonut (1983) Ltd. 2012

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Teva-Gamida Cell complete enrollment for blood cancer trial

WALKER, Mich. (WOOD) - Dogs who received the first in-clinic stem cell therapy in West Michigan returned to the vets who treated them Monday morning.

Boris and Natasha returned to Kelley's Animal Clinic for their 60-day checkup after receiving stem cell treatment in December 2011.

Dr. James Kelley and his staff of vets removed fat tissue from the dogs and activated it with an enzyme before injecting it into their back legs.

This adult animal stem cell technology is different from the controversial embryonic stem cell therapy.

Kelley said both dogs are doing amazingly well and that the procedure has done more than just help their arthritis.

"We're finding that not only the joints are affected, the rest of the animal is affected as well," said Kelley. "The skin is better. The attitude in these dogs is much improved."

Kelley and his staff have done 16 stem cell treatments since the first on Boris and Natasha, and he said all the dogs are showing signs of improvement after a short period of time.

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Dogs who got stem cell therapy are well

A promising stem cell therapy approach could soon provide a way to regenerate heart muscle damaged by heart attacks.

Researchers at Cedars-Sinai Heart Institute and The Johns Hopkins University harvested stem cells from the hearts of 17 heart attack patients and after prepping the cells, infused them back into the patients' hearts. Their study is published in the current issue of The Lancet.

The patients received the stem cell infusions about three months after their heart attacks.

Researchers found that six months after treatment, patients had significantly less scarring of the heart muscle and also showed a considerable increase the amount of healthy heart muscle, compared to eight post-heart attack patients studied who did not receive the stem cell infusions. One year after, scar size was reduced by about 50 percent.

"The damaged tissue of the heart was replaced by what looks like healthy myocardium," said Dr. Peter Johnston, a study co-author and an assistant professor of medicine at The Johns Hopkins University School of Medicine. "It's functioning better than the damaged myocardium in the control subjects, and there's evidence it's starting to contract and generate electrical signals the way healthy heart tissue does."

While this research is an early study designed to demonstrate that this stem cell therapy is safe, cardiologists say it's an approach that could potentially benefit millions of people who have suffered heart attacks. Damage to the heart muscle is permanent and irreparable, and little can be done to compensate for loss of heart function.

"In the U.S., six million patients have heart failure, and the vast majority have it because of a prior heart attack," said Johnston.

The damaged scar tissue that results from a heart attack diminishes heart function, which can ultimately lead to enlargement of the heart.

At best, Johnston said, there are measures doctors can try to reduce or compensate for the damage, but in many cases, heart failure ultimately sets in, often requiring mechanical support or a transplant.

"This type of therapy can save people's lives and reduce the chances of developing heart failure," he said.

Cardiac Regeneration A Promising Field

Other researchers have also had positive early results in experiments with stem cell therapy using different types of cells, including bone marrow cells and a combination of bone marrow and heart cells.

"It's exciting that studies using a number of different cell types are yielding similar results," said Dr. Joshua Hare, professor of cardiology and director of the University of Miami Interdisciplinary Stem Cell Institute.

The next steps, he said, include determining what the optimal cell types are and how much of the cells are needed to regenerate damaged tissue.

"We also need to move to larger clinical trials and measure whether patients are improving clinically and exhibiting a better quality of life after the therapy."

In an accompanying comment, Drs. Chung-Wah Siu amd Hung-Fat Tse of the University of Hong Kong wrote that given the promising results of these studies, health care providers will hopefully recognize the benefits that cardiac regeneration can offer.

And Hare added that someday, this type of regeneration can possibly offer hope to others who suffered other types of organ damage.

"This stategy might work in other organs," he said. "Maybe this can work in the brain, perhaps for people who had strokes."

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Stem Cells May Help Regenerate Heart Muscle

SOUTH SAN FRANCISCO, CA--(Marketwire -02/14/12)- VistaGen Therapeutics, Inc. (OTC.BB: VSTA.OB - News) (OTCQB: VSTA.OB - News), a biotechnology company applying stem cell technology for drug rescue and cell therapy, has identified its initial Top 10 drug rescue candidates and plans to launch two formal drug rescue programs by the end of next quarter.

VistaGen's goal for each of its stem cell technology-based drug rescue programs is to generate and license a new, safer variant of a once-promising large market drug candidate previously discontinued by a pharmaceutical company no earlier than late-preclinical development.

"We are now at an advanced stage in our business model," said Shawn Singh, VistaGen's Chief Executive Officer. "After more than a decade of focused investment in pluripotent stem cell research and development, we are now at the threshold where game-changing science becomes therapeutically relevant to patients and commercially relevant to our shareholders. We have positioned our company and our stem cell technology platform to pursue multiple large market opportunities. We plan to launch two drug rescue programs by the end of the next quarter."

Over the past year, VistaGen, working with its network of strategic partners, identified over 525 once-promising new drug candidates that meet the Company's preliminary screening criteria for heart toxicity-focused drug rescue using CardioSafe 3D™, its human heart cell-based bioassay system. After internally narrowing the field to 35 compounds, VistaGen, working together with its external drug rescue advisors, including former senior pharmaceutical industry executives with drug safety and medicinal chemistry expertise, analyzed and carefully narrowed the group of 35 to the current Top 10.

About VistaGen Therapeutics

VistaGen is a biotechnology company applying human pluripotent stem cell technology for drug rescue and cell therapy. VistaGen's drug rescue activities combine its human pluripotent stem cell technology platform, Human Clinical Trials in a Test Tube™, with modern medicinal chemistry to generate new chemical variants of once-promising small-molecule drug candidates. These are once-promising drug candidates discontinued by pharmaceutical companies during development due to heart toxicity, despite positive efficacy data demonstrating their potential therapeutic and commercial benefits. VistaGen uses its pluripotent stem cell technology to generate early indications, or predictions, of how humans will ultimately respond to new drug candidates before they are ever tested in humans.

Additionally, VistaGen's oral small molecule prodrug candidate, AV-101 (4-Cl-KYN), is in Phase 1b development for treatment of neuropathic pain. Unlike other NMDA receptor antagonists developed previously, AV-101 readily crosses the blood-brain barrier and is then efficiently converted into 7-chlorokynurenic acid (7-Cl-KYNA), one of the most potent and specific glycineB site antagonists currently known, and has been shown to reduce seizures and excitotoxic neuronal death. Neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system, affects approximately 1.8 million people in the U.S. alone. To date, VistaGen has been awarded over $8.5 million from the NIH for development of AV-101. The Company anticipates pursuing Phase 2 development for neuropathic pain and other neurological indications, including depression, epilepsy, and/or Parkinson's disease in the event it receives additional non-dilutive development grant funding from the NIH or private foundations.

Visit VistaGen at http://www.VistaGen.com, follow VistaGen at http://www.twitter.com/VistaGen or view VistaGen's Facebook page at http://www.facebook.com/VistaGen.

Cautionary Statement Regarding Forward Looking Statements

The statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the success of VistaGen's stem cell technology-based drug rescue activities, ongoing AV-101 clinical studies, its ability to enter into drug rescue collaborations and/or licensing arrangements with respect to one or more drug rescue variants, risks and uncertainties relating to the availability of substantial additional capital to support VistaGen's research, drug rescue, development and commercialization activities, and the success of its research and development plans and strategies, including those plans and strategies related to AV-101 and any drug rescue variant identified and developed by VistaGen. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

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VistaGen Updates Pipeline of Stem Cell Technology-Based Drug Rescue Candidates

MONDAY, Feb. 13 (HealthDay News) -- Stem cell therapy's promise for healing damaged tissues may have gotten a bit closer to reality. In a small, early study, heart damage was reversed in heart-attack patients treated with their own cardiac stem cells, researchers report.

The cells, called cardiosphere-derived stem cells, regrew damaged heart muscle and reversed scarring one year later, the authors say.

Up until now, heart specialists' best tool to help minimize damage following a heart attack has been to surgically clear blocked arteries.

"In our treatment, we dissolved scar and replaced it with living heart muscle. Such 'therapeutic regeneration' has long been the holy grail of cell therapy, but had never been accomplished before; we now seem to have done it," said study author Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles.

However, outside experts cautioned that the findings are preliminary and the treatment is far from ready for widespread use among heart-attack survivors.

The study, published online Feb. 14 in The Lancet, involved 25 middle-aged patients (average age 53) who had suffered a heart attack. Seventeen underwent stem cell infusions while eight received standard post-heart attack care, including medication and exercise therapy.

The stem cells were obtained using a minimally invasive procedure, according to the researchers from Cedars-Sinai and the Johns Hopkins Hospital in Baltimore.

Patients received a local anesthetic and then a catheter was threaded through a neck vein down to the heart, where a tiny portion of muscle was taken. The sample provided all the researchers needed to generate a supply of new stem cells -- 12 million to 25 million -- that were then transplanted back into the heart-attack patient during a second minimally invasive procedure.

One year after the procedure, the infusion patients' cardiac scar sizes had shrunk by about half. Scar size was reduced from 24 percent to 12 percent of the heart, the team said. In contrast, the patients receiving standard care experienced no scar shrinkage.

Initial muscle damage and healed tissue were measured using MRI scans.

After six months, four patients in the stem-cell group experienced serious adverse events compared with only one patient in the control group. At one year, two more stem-cell patients had a serious complication. However, only one such event -- a heart attack -- might have been related to the treatment, according to the study.

In a news release, Marban said that "the effects are substantial and surprisingly larger in humans than they were in animal tests."

Other experts were cautiously optimistic. Cardiac expert Dr. Bernard Gersh, a professor of medicine at Mayo Clinic, is not affiliated with the research but is familiar with the findings.

"This study demonstrates that it is safe and feasible to administer these cardiac-derived stem cells and the results are interesting and encouraging," he said.

Another specialist said that while provocative and promising, the findings remain early, phase-one research. "It's a proof-of-concept study," said interventional cardiologist Dr. Thomas Povsic, an assistant professor of medicine at the Duke Clinical Research Institute, in Durham, N.C.

And Dr. Chip Lavie, medical director of Cardiac Rehabilitation and Prevention at the John Ochsner Heart and Vascular Institute, in New Orleans, also discussed the results. He said that while the study showed that the cardiac stem cells reduced scar tissue and increased the area of live heart tissue in heart attack patients with moderately damaged overall heart tissue, it did not demonstrate a reduction in heart size or any improvement in the heart's pumping ability.

"It did not improve the ejection fraction, which is a very important measurement used to define the overall heart's pumping ability," Lavie noted. "Certainly, much larger studies of various types of heart attack patients will be needed before this even comes close to being a viable potential therapy for the large number of heart attack initial survivors."

Povsic concurred that much larger studies are needed. "The next step is showing it really helps patients in some kind of meaningful way, by either preventing death, healing them or making them feel better."

It's unclear what the cost will be, Povsic added. "What society is going to be willing to pay for this is going to be based on how much good it ends up doing. If they truly regenerate a heart and prevent a heart transplant, that would save a lot money."

Marban, who invented the stem cell treatment, said the while it would not replace bypass surgery or angioplasty, "it might be useful in treating 'irreversible' injury that may persist after those procedures."

As a rough estimate, he said that if larger, phase 2 trials were successful, the treatment might be available to the general public by about 2016.

More information

The U.S. National Heart, Lung, and Blood Institute describes current heart attack treatment.

Original post:
Stem Cell Treatment Might Reverse Heart Attack Damage

13-02-2012 18:16 Dr. John Wagner, Manager, IBM Research Collaboratory for Life Science Victoria will be home to one of Australia's fastest supercomputers and the world's greenest supercomputer, the IBM [NYSE: IBM] Blue Gene/Q, which will be housed at the Victorian Life Sciences Computation Initiative (VLSCI) hosted by the University of Melbourne, and is aimed at advancing the study of human disease.

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IBM's Blue Gene/Q Boosts Human Health Research in Victoria - Video

Public release date: 14-Feb-2012
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Contact: Liz Williams
williams@wehi.edu.au
61-405-279-095
Walter and Eliza Hall Institute

The role of a known cancer-causing gene in the development of the most lethal type of ovarian cancer is being investigated by researchers from the Walter and Eliza Hall Institute after they were awarded a Cure Cancer Australia Foundation (CCAF) grant.

Dr Rachael Rutkowski, from the institute's Stem Cells and Cancer division, was awarded $180,000 to study the role of the known cancer-causing gene in the development of high-grade serous ovarian cancers. This gene belongs to the MYC family of cancer-causing genes that are overproduced in more than 50 per cent of human cancers.

Dr Rutkowski said that the MYC gene family had recently gained attention as a potential cause of some high-grade serous ovarian cancers that are associated with poor clinical outcomes.

"The Cure Cancer Australia funding will allow us to develop better disease models that we can use to discover whether the MYC gene family has a significant role in ovarian cancer development," Dr Rutkowski said. "It could also help us identify new therapeutic targets and biomarkers for diagnosis of this devastating disease."

Ovarian cancer is the sixth most common cause of cancer death in Australian women. Each year, more than 1200 Australian women are diagnosed with ovarian cancer, and around 800 will die from the disease.

Epithelial ovarian cancer accounts for 90 per cent of all ovarian cancers; epithelial referring to the tissue from which the cancer develops. Despite efforts to develop better screening tools, 80 per cent of epithelial ovarian cancers have spread beyond the ovary before they are diagnosed and 70 per cent are generally incurable. High-grade serous ovarian cancers are the most lethal type of epithelial ovarian cancer, and account for approximately 40 per cent of all epithelial ovarian cancers.

Associate Professor Clare Scott, who heads the ovarian cancer research program at the Walter and Eliza Hall Institute and is a medical oncologist at The Royal Melbourne Hospital, said new treatments for ovarian cancer were urgently needed.

"High-grade serous ovarian cancers are aggressive, difficult-to-treat cancers that often have a poor prognosis," Associate Professor Scott said. "The cancers are typically not diagnosed until after they have spread, and are often resistant to chemotherapy drugs. It is imperative that we improve our understanding of how ovarian cancers develop so we can identify molecular or cellular targets for new therapeutic agents."

Changes in the levels of MYC-family proteins have been identified as a potential cause of at least 15-20 per cent of high-grade serous ovarian cancers, and are associated with poor clinical outcomes.

Dr Rutkowski said that the research team would develop pre-clinical models of ovarian cancer with high levels of MYC-family proteins, and abnormal p53 signaling, to determine whether the MYC gene family is involved in the development and chemotherapy-resistance of these cancers.

"We are hoping that we will identify molecular targets for the development of new therapeutic agents to treat ovarian cancer," Dr Rutkowski said. "Similar studies have yielded new therapeutic targets for breast cancer; for example, the treatment of HER2-positive breast cancers, which typically had a poor prognosis, has been revolutionised by the development of Her2-targeted therapeutic approaches. We are looking to accelerate the development of similar targeted therapies for ovarian cancer."

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Role of known cancer gene in ovarian cancer investigated

LAUSANNE, Switzerland--(BUSINESS WIRE)--

Gene Signal, a company focused on developing innovative drugs to manage angiogenesis based conditions, today announced the publication of data demonstrating the significant activity of aganirsen (GS-101, eye drops) in two important models of retinal neovascular disease, wet age-related macular degeneration (AMD) and ischemic retinopathy. Gene Signal’s aganirsen is an antisense oligonucleotide that is expected to complete a phase III trial for the treatment of progressive neovascularisation in the cornea in 2012. These data, published online in Investigative Ophthalmology & Visual Science (doi:10.1167/iovs.11-9064), demonstrate the ability of aganirsen to reach and exert activity on the retina.

“Age-related macular degeneration and ischemic retinopathies are major causes of blindness that are associated with neovascularisation. This can lead to sub-retinal and intravitreal hemorrhage, and tragically retinal detachment, which causes blindness,” explained lead author Dr. Sylvain Chemtob, Université de Montréal, Montreal, Canada.

“For the first time in this study, we have demonstrated that IRS-1, an angiogenic protein, is expressed in the retina and that aganirsen is able to effectively attenuate neovascularization by inhibiting IRS-1 expression, without affecting normal vascularisation, ” noted co-lead author Dr. Matthew Lawrence from RxGen Inc., Hamden, CT, USA.

Aganirsen blocks pathological neovascularization by inhibiting IRS-1. Clinical studies to date have shown that aganirsen is able to safely and effectively inhibit the development of progressive corneal neovascularization secondary to infectious keratitis or chemical burns both of which could lead to corneal graft replacement.

“The only effective drugs approved to target neovascularization in AMD and ischemic retinopathies are intraocular injections of anti-VEGF compounds, such as Lucentis. The studies reported in Investigative Ophthalmology & Visual Science, if confirmed in clinical trials, show unprecedented evidence that topical aganirsen is an innovative compound that may offer advantages over currently available drugs due to its topical delivery and different mode of action as well as an excellent efficacy and safety profile. A topical agent for retinal neovascular disease would revolutionize treatment,” noted Eric Viaud, CEO of Gene Signal.

Study Details

Aganirsen (topical emulsion) was applied daily in non-human primates following laser induced choroidal neovascularisation (CNV), a model of wet age-related macular degeneration [AMD]), and in newborn rats following oxygen-induced retinopathy (OIR), a model of ischemic retinopathy. Retinal aganirsen concentrations were assessed in monkeys following topical delivery (21.5, 43 or 86 ?g). Clinical significance was further evaluated by determination of IRS-1 expression in monkey and human retinal biopsies.

Topical application of aganirsen inhibited neovascular lesion development dose-dependently in African green monkeys, with incidence of high-grade CNV lesions (grade IV) decreasing from 20.5% in vehicle-treated animals to 1.7% (p<0.05) at the 86 ?g dose. Topical aganirsen inhibited retinal neovascularization following OIR in rats (p<0.05); furthermore, a single intravitreal injection of aganirsen reduced OIR as effectively as ranibizumab (Lucentis), and the effects of both compounds were additive. Topical applications of aganirsen did not interfere with physiological retinal vessel development in newborn animals.

About Gene Signal

Gene Signal (www.genesignal.com) is developing a robust pipeline of novel antisense oligonucleotides, proteins and monoclonal antibodies to treat a range of conditions based on its innovative angiogenesis modulating technology. The company’s most advanced therapeutic product is aganirsen (GS-101), an antisense oligonucleotide that has nearly completed phase III for the prevention of corneal graft rejection. Aganirsen is also entering phase II clinical trials for additional angiogenesis based diseases, such as wet age-related macular degeneration (AMD), neovascular glaucoma, and dermal indications. Antisense oligonucleotides confer distinctive advantages versus other biologics: they can be readily transported across cell membranes, are associated with low immunogenicity, and can be produced by simple chemical synthesis, unlike larger proteins and monoclonal antibodies that require cell culture and complex purification steps.

Through world leading expertise in discovering genes involved in the regulation of angiogenesis, Gene Signal has built a significant intellectual property portfolio that has relevance in multiple disease areas. Gene Signal plans to seek partnership with the pharmaceutical industry for the next steps of development and marketing. The company was founded in 2000 and has assembled an outstanding leadership team that includes scientific, medical, regulatory and business professionals with successful track records in developing and commercialising state of the art drugs. Gene Signal’s headquarters are in Lausanne (PSE, EPFL), Switzerland, with its research programs based in France (Bioparc Genopole, Evry) and product development in Canada (Montréal).

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Gene Signal and Collaborators Demonstrate Successful Activity of Topical Aganirsen in Models of Retinal Neovascular ...

ScienceDaily (Feb. 13, 2012) — Novel gene abnormalities discovered in a subpopulation of lung and colorectal tumors could potentially identify patients with a good chance of responding to highly specific "targeted" drugs already in use for treating other cancers, scientists report.

The genetic alterations -- pieces of two genes fused together -- showed up in a massive search of the DNA in stored tumor samples of non-small cell lung cancer and colorectal cancer, said researchers from Dana-Farber Cancer Institute and Foundation Medicine, Inc. These specific genetic abnormalities had not been previously linked to the two cancer types.

Their results were published online by the journal Nature Medicine. Other cancers with similar genetic alterations often respond to "targeted" drugs s that block overactive proteins called tyrosine kinase inhibitors. This suggests that the same drugs also may be effective against lung and colorectal tumors driven by the newly found gene fusions. Because these drugs are already approved to treat cancer, it should be possible to move rapidly to clinical trials in colorectal and lung cancer, the authors said.

If the trials are successful, physicians could potentially test patients' tumors for the presence of the gene fusions and prescribe a medication matched to those alterations, said Pasi A. Jänne, MD, PhD, a thoracic oncologist at Dana-Farber and co-senior author of the report along with Philip J. Stephens, PhD, and Maureen Cronin, PhD, of Foundation Medicine. Doron Lipson, PhD, is the paper's first author. "This is a textbook example of personalized medicine for lung cancer -- a genetic alteration found in a subset of patients that we can now look for and use as a means to select particular therapies," Jänne said.

"In the past, although these targeted drugs were available, they were not chosen for a particular subset, but instead given to everybody," he explained. "This will increase the likelihood of those therapies being more successful."

The researchers estimate that less than 1 percent of Caucasians and about 2 percent of Asians with lung cancers carry this alteration -- a fusion gene labeled KIF5B-RET. However, they said the finding opens a significant therapeutic opportunity.

"In a common indication like non-small cell lung cancer, identifying even a small subpopulation of individuals with gene fusions who may be responsive to a targeted therapy has the potential for major therapeutic impact," said Stephens, executive director of cancer genomics at Foundation Medicine. "This joint research with Dana-Farber translates genomic research to the clinic and we expect that it may quickly have a positive impact for patients."

The American Cancer Society projects that 226,160 Americans will be diagnosed with lung cancer in 2012 and 160,340 will die of the disease.

Foundation Medicine scientists identified the novel fusion gene in a DNA tumor sample removed from a 44-year-old man with non-small cell lung cancer (NSCLC) who had never smoked. The hybrid gene is composed of a piece of a cell growth gene, RET, and part of another gene, KIF5B. This abnormal gene combination causes RET to act like a growth switch stuck in the "on" position, spurring uncontrolled cell division.

The company formed a collaboration with Jänne and his Dana-Farber colleagues to follow up the discovery. "We looked for the RET fusion gene in a larger collection of lung tumor samples to determine how common it is, and if it is acting as an oncogene [a gene that drives cancer]" said Jänne. They searched samples from 121 Caucasian patients and 405 Asian patients who had never smoked or had rarely smoked in the past. The RET fusion gene was detected in 1 of the Caucasian samples (0.8 percent) and 9 of the Asian patient samples (2 percent).

Thyroid cancers containing RET gene hybrids are known to respond to certain targeted drugs that inhibit RET. When Dana-Farber investigators tested three such inhibitors -- sorafenib, sunitinib and vandetinib -- on cultured cells containing the newly discovered RET mutation, each of the drugs killed those cells, the scientists reported. Jänne noted that some patients with NSCLC have responded to treatment with these inhibitor drugs. The researchers want to find out whether those patients had RET mutations in their tumors.

The Foundation Medicine scientists also sequenced DNA samples from 40 patients with colorectal cancer. Along with numerous known mutations, the researchers identified a novel gene alteration, C2orf44-ALK, that causes a 90-fold overexpression of the ALK protein leading to cancerous proliferation. Overexpressed ALK is also found in a small percentage of lung cancer cases and can be inhibited by the targeted drug crizotinib. This raises the possibility of using crizotinib to target the C2orf44-ALK fusion gene in colorectal cancer, the researchers said.

In addition to Cronin, Jänne, Lipson and Stephens, the paper's co-authors were from Sharett Institute of Oncology, Jerusalem; TEVA Pharmaceutical Industries, Petach Tikva, Israel; Samsung Medical Center, Seoul, Korea; Nagoya City University Graduate School of Medical Sciences, Japan; Asan Medical Center, Seoul, Korea; and Albany Medical Center, New York.

The research was funded in part by the Dana-Farber/Harvard Cancer Center Lung Cancer SPORE grant from the National Cancer Institute and the Cammarata Family Foundation Research Fund.

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The above story is reprinted from materials provided by Dana-Farber Cancer Institute, via Newswise.

Note: Materials may be edited for content and length. For further information, please contact the source cited above.

Journal Reference:

Doron Lipson, Marzia Capelletti, Roman Yelensky, Geoff Otto, Alex Parker, Mirna Jarosz, John A Curran, Sohail Balasubramanian, Troy Bloom, Kristina W Brennan, Amy Donahue, Sean R Downing, Garrett M Frampton, Lazaro Garcia, Frank Juhn, Kathy C Mitchell, Emily White, Jared White, Zac Zwirko, Tamar Peretz, Hovav Nechushtan, Lior Soussan-Gutman, Jhingook Kim, Hidefumi Sasaki, Hyeong Ryul Kim, Seung-il Park, Dalia Ercan, Christine E Sheehan, Jeffrey S Ross, Maureen T Cronin, Pasi A Jänne, Philip J Stephens. Identification of new ALK and RET gene fusions from colorectal and lung cancer biopsies. Nature Medicine, 2012; DOI: 10.1038/nm.2673

Note: If no author is given, the source is cited instead.

Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of ScienceDaily or its staff.

Originally posted here:
Newly identified fusion genes in lung and colorectal cancer may guide treatment with 'targeted' drugs

(RTTNews.com) - Biotechnology company Seattle Genetics Inc. (SGEN) Monday reported a loss for the fourth quarter that narrowed from a year ago, due mainly to sales of Hodgkin Lymphoma drug Adcetris approved last August. Loss for the quarter was smaller when compared to analyst estimates, while revenues too came in ahead of expectations.

Looking ahead, Seattle Genetics detailed its revenue forecast for 2012. Nonetheless, investors were not too impressed with the results, sending Seattle Genetics shares down 7 percent in after hours trade on the Nasdaq.

The Bothell, Washington-based company reported fourth-quarter net loss of $27 million or $0.24 per share compared to net loss of $34.5 million or $0.34 per share last year.

On average, 14 analysts polled by Thomson Reuters expected a loss of $0.31 per share for the quarter. Analysts' estimates typically exclude special items.

Results for the 2011 quarter include an $8.7 million valuation adjustment related to holdings in auction rate securities.

The company reported revenues of $48.9 million, compared to $8.1 million in the prior year. Street analysts expected revenues of $39.12 million.

Adcetris product sales for the quarter was $33.2 million. Adcetris (brentuximab vedotin) was approved by the FDA last August for the treatment of patients with Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not ASCT candidates.

The drug has been also approved for the treatment of patients with systemic anaplastic large cell lymphoma after failure of at least one prior multi-agent chemotherapy regimen. Adcetris is the first drug approved by the FDA for Hodgkin lymphoma in more than 30 years.

CEO Clay Siegall said, "...We are also executing on a broad clinical development program of Adcetris to evaluate its potential in earlier lines of therapy for Hodgkin lymphoma and mature T-cell lymphomas, as well as in other CD30-positive malignancies."

Total expenses for the quarter rose to $67.6 million from $43 million last year, reflecting higher selling expenses related to the launch of Adcetris, and increased research expenses.

Seattle Genetics expects full year 2012 revenues from collaboration and license agreements between $55 million and $65 million.

Analysts currently expect the company to report revenues of $196.21 million for 2012.

SGEN closed Monday on the Nasdaq at $18.96, up $0.36 or 1.96%, on a volume of 1.6 million shares. In after hours, the stock lost $1.34 or 7.07%.

For comments and feedback: contact editorial@rttnews.com

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View original post here:
Seattle Genetics Loss Narrows; But Stock Down - Update

Helped by revenue growth, Seattle Genetics, Inc. narrowed its loss in the fourth quarter. Seattle Genetics is a biotechnology company focused on the development and commercialization of monoclonal antibody-based therapies for the treatment of cancer and autoimmune disease.

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Seattle Genetics Earnings Cheat Sheet for the Fourth Quarter

Results: Loss narrowed to $27.2 million (loss of 24 cents per diluted share) from $34.5 million (loss of 34 cents per share) in the same quarter a year earlier.

Revenue: Rose more than sixfold to $48.9 million from the year earlier quarter.

Actual vs. Wall St. Expectations: Seattle Genetics, Inc. beat the mean analyst estimate of a loss of 30 cents per share. It beat the average revenue estimate of $39.1 million.

Quoting Management: “We are pleased with the successful launch of ADCETRIS and our execution in bringing this drug to patients in need,” said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. “Our commercialization initiatives continue to focus on expanding awareness of ADCETRIS among oncologists, particularly in the community setting, and ensuring an efficient reimbursement process.”

Key Stats:

The company has now beaten estimates the last two quarters. In the third quarter, it topped expectations with a loss of -35 cents versus a mean estimate of a loss of 46 cents per share.

The company’s revenue has now risen for two straight quarters. In the third quarter, revenue increased 29.2% to $20.7 million from the year earlier quarter.

Looking Forward: Expectations for the company’s next quarter performance are higher than they were ninety days ago. The average estimate for the first quarter of the next fiscal year is now at a loss of 25 cents per share, up from a loss of 31 cents. For the fiscal year, the average estimate has moved from a loss of $1.45 a share to a loss of $1.40 over the last ninety days.

(Company fundamentals provided by Xignite Financials. Earnings estimates provided by Zacks)

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To contact the reporter on this story: Derek Hoffman at staff.writers@wallstcheatsheet.com

To contact the editor responsible for this story: Damien Hoffman at editors@wallstcheatsheet.com

Excerpt from:
Seattle Genetics Inc. Earnings: Beats Analysts’ Estimates as Loss Narrows