Autism Stem-Cell Therapy to Be Tested in Kids in Trials

By Ryan Flinn - 2012-08-21T04:01:00Z

Researchers are recruiting autistic children for a study that will test whether injecting stem cells banked from their umbilical cords can lessen symptoms and provide insights into the nature of the disorder.

While stem cells have been promoted, and sold, as a treatment for autism, few clinical trials have been conducted to see whether theyre effective. The study, which begins enrolling patients today, is the first of its kind approved by the U.S. Food and Drug Administration to assess the use of stem cells as a potential autism therapy, said Michael Chez, director of pediatric neurology at Sutter Medical Center in Sacramento, California, and the principal investigator.

About 1 in 88 children in the U.S. are diagnosed with an autism-related condition. The disorder hurts brain development and is linked to poor social interaction and communication skills, repeated body movements, and unusual attachments to objects.

With this study well be able to answer in a firm way that this is truly an observed effect, or we didnt get an observed effect, Chez said in a phone interview.

Thirty children with autism, ages 2 to 7, will be divided in two groups, with one getting the stem cell injection and the other receiving a placebo shot. After six months, the groups will switch. Patients will be monitored for improvement in language as well as irritability and other autism rating scales.

Ricardo Dolmetsch, a neurobiologist at Stanford University in California whose laboratory is studying autism, said he doesnt think the trial will yield much in usable results, though hes glad the idea of using stem cells is being testing.

I commend them for having the guts to actually do it, given that there are all kinds of people out there trying to sell it, he said. On the other hand I dont think its big enough to provide an answer.

Chez theorizes that autism, which has no known cause or cure, may be spurred on by damaged nerve cells. Stem cells, the building blocks of life that can grow into any type of tissue in the body, could repair the damage or create new cells, he said. Such a mechanism would yield results in six to 12 months, the time it takes to create new cells.

Another possibility may be that autism is related to a signaling issue, where cells in the body arent connecting properly. Stem cells may help repair that problem, he said, and would be evident if results are seen within weeks of the injection. A third and more exploratory possibility is the disorder is related to inflammation, an immune system response.

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Autism Stem-Cell Therapy to Be Tested in Kids in Trials

Stem Cell Trial for Autism Launches in U.S.

Stem cell treatment could lower inflammation levels and demonstrate whether autism is an autoimmune disease

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Families with autistic children must navigate a condition where questions outnumber the answers, and therapies remain sparse and largely ineffective. A clinical trial being conducted by the Sutter Neuroscience Institute in Sacramento, California to address this situation began recruiting participants today for a highly experimental stem cell therapy for autism. The institute plans to find 30 autistic children between ages 2 and 7 with cord blood banked at the privately-run Cord Blood Registry, located about 100 miles west of the institute. Already one other clinical trial, with 37 total participants between ages 3 and 12 years old, has been completed in China. The researchers affiliated with Beike Biotechnology in Shenzhen, the firm that sponsored the study, have not yet published any papers from that the trial, which used stem cells from donated cord blood. Mexican researchers are currently recruiting kids for yet another type of autism stem cell trial that will harvest cells from the participants fat tissue.

But for each of these officially registered trials, many more undocumented stem cell therapy treatments take place for clients who are willing to pay enough. Our research is important because many people are going to foreign countries and spending a lot of money on therapy that may not be valid, says Michael Chez, a pediatric neurologist and lead investigator of the study at Sutter.

A major difference between the Sutter trial and those in China is that his will use the childs own stem cells, rather than those from a donor. Chez hypothesizes that one way autologous stem cell infusion might work is by reducing inflammation within the bodys immune system. This would answer previous research that suggests that autism may be an autoimmune disease. One of our exploratory goals will be to look at inflammatory markers in cells, he says.

The studys primary goal, however, will be assessing changes in patients speaking and understanding of vocabulary.For each individual, researchers will create a baseline benchmark that establishes current skill levels. The group will be evenly divided, with one initially receiving an infusion of their own, unmodified cord blood stem cells and the other a placebo treatment of saline injection. Six months later, all of the children will be tested on their ability to comprehend and form words. The groups will then be switched. In the course of the 13-month-long study, both groups will receive only one stem cell therapy infusion.

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Stem Cell Trial for Autism Launches in U.S.

Stem Cell Treatments: False Hope Warning Signs

Unproven, Risky Treatments Mislead Patients to Seek Cutting-Edge Therapy

There's a dark side to stem cells: bogus treatments that prey on patients' hopes when mainstream medicine has little to offer.

Stephen Byer stepped far outside typical medical care when his son, Ben, had ALS. He took Ben to China for stem cell-like treatments, and later helped hundreds of people do the same, believing it would help them.

The unproven procedure could have killed Ben. It didn't -- but it also didn't work. Ben later died of ALS. So did the ALS patients Byer now regrets helping get the treatment.

Why take the chance? For Byer, it started with misleading promises online.

"The Internet, while increasing communication, has spawned a horde of charlatans and creeps," Byer says. "We were suckered into one of the earlier forms of stem cell chicanery."

But not everyone who seeks unapproved stem cell treatments feels ripped off. Even though the stem cell treatments Dawn Gusty got in Tijuana, Mexico, didn't ease her multiple sclerosis, she doesn't look back with regret.

That moment -- when hope surpasses science, and when someone claims to be able to bridge that gap -- may be one of the riskiest for patients to handle. And it's one of the most alarming for stem cell experts.

"It is a very dangerous situation," says Joshua Hare, MD, director of the Interdisciplinary Stem Cell Institute at the University of Miami.

Make no mistake: Hare is all for scientific stem cell research. His concern, he says, is "hype" that glosses over an inconvenient fact: There are no new approved stem cell therapies.

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Stem Cell Treatments: False Hope Warning Signs

Stem cell tranplant decision due in Venice

The toddler, Celeste Carrer, suffers from muscular atrophy or progressive muscle wastage. In the past 18 months she has had several injections of stem cells taken from the bone marrow of her mother Elisabetta that reportedly enabled to her to move her head, arms and legs after near paralysis.

The treatments were carried out by paediatrician Dr Mario Andolina at Brescia Hospital in Italy's north in conjunction with the Stamina Foundation, a non-profit organisation established in 2009 to promote stem cell research.

However the child's treatment was stopped in May after Turin prosecutor Raffaele Guariniello launched an investigation into medical staff at the non-profit foundation.

Police raided the hospital's laboratories while agents from the Agenzia del Farmaco, the national drug agency, blocked the "collection, transport, manipulation, cultivation, storage and administration of human cells" at the hospital and foundation.

"The block on Brescia will unfortunately cause the death of several patients," said Dr Andolina."The therapy annoys people because the stem cell treatment is practically free apart from a bone marrow extraction from relatives. Traditional cures on the other hand cost thousands of euros."

Elisabetta Carrer and her husband won legal approval in January 2011 to begin their child's transplants and are now taking legal action in a bid to resume Celeste's treatments.

"The laboratory is considered appropriate to manage transplants for children from all over Europe and is regarded as cutting edge," the Carrers' lawyer Dario Bianchini told La Repubblica, the Italian daily.

"There is clinical evidence to show that Celeste, after these treatments, has improved." Under a decree approved by former Health Minister Livia Turco in 2006, stem cell research is permitted in Italy when a patient is in a life-threatening situation or worsening.

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Stem cell tranplant decision due in Venice

Stem cell transplant decision due in Venice

The toddler, Celeste Carrer, suffers from muscular atrophy or progressive muscle wastage. In the past 18 months she has had several injections of stem cells taken from the bone marrow of her mother Elisabetta that reportedly enabled to her to move her head, arms and legs after near paralysis.

The treatments were carried out by paediatrician Dr Mario Andolina at Brescia Hospital in Italy's north in conjunction with the Stamina Foundation, a non-profit organisation established in 2009 to promote stem cell research.

However the child's treatment was stopped in May after Turin prosecutor Raffaele Guariniello launched an investigation into medical staff at the non-profit foundation.

Police raided the hospital's laboratories while agents from the Agenzia del Farmaco, the national drug agency, blocked the "collection, transport, manipulation, cultivation, storage and administration of human cells" at the hospital and foundation.

"The block on Brescia will unfortunately cause the death of several patients," said Dr Andolina."The therapy annoys people because the stem cell treatment is practically free apart from a bone marrow extraction from relatives. Traditional cures on the other hand cost thousands of euros."

Elisabetta Carrer and her husband won legal approval in January 2011 to begin their child's transplants and are now taking legal action in a bid to resume Celeste's treatments.

"The laboratory is considered appropriate to manage transplants for children from all over Europe and is regarded as cutting edge," the Carrers' lawyer Dario Bianchini told La Repubblica, the Italian daily.

"There is clinical evidence to show that Celeste, after these treatments, has improved." Under a decree approved by former Health Minister Livia Turco in 2006, stem cell research is permitted in Italy when a patient is in a life-threatening situation or worsening.

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Stem cell transplant decision due in Venice

Stem Cell Therapy: The Hope and the Hype

By Shane Huntington

Pioneering stem cell researcher Prof Martin Pera discusses where stem cell research has been and where its going, the therapeutic potential of stem cell technologies, and what we should and shouldnt expect from this fast-developing research field. With host Dr Shane Huntington.

SHANE HUNTINGTON Over the last couple of decades stem cells have been touted as the key to treating a wide range of diseases, with advantages that exceed surgical repair or even organ transplantation; but work on stem cells, especially those derived from human embryos, has drawn significant public scrutiny. In many countries embryonic stem cell research is tightly regulated, and researchers have been forced to explore other research options. Such restrictions are not present worldwide and, in some countries, regulation has yet to catch up with the science. This can make it especially challenging for patients trying to navigate through the world of therapeutic stem cell technologies, both in trial phase and on the consumer market. To explore these issues and the technology itself we are joined on Up Close by one of the pioneers of stem cell research: Professor Martin Pera, program leader of Stem Cells Australia, and professor of stem cell sciences at the University of Melbourne. Welcome to Up Close, Martin.

MARTIN PERA Thank you.

SHANE HUNTINGTON Could you start by giving us a bit of an overview of what a stem cell is and why these particular cells have generated so much interest over the last two decades?

MARTIN PERA Well, they're really two basic types of stem cells. One type of stem cell we cal pluripotent stem cells. And these can either be derived from very early stage embryos of they can now be created in the lab from adult cells, from patients, through a process we call reprogramming. And pluripotent stem cells are able to give rise to all the tissues of the body. The second class of stem cell is the tissue stem cell, sometimes called adult stem cells. These are stem cells that are minority populations in many adult tissues. They are, essentially, more limited in their potential; they usually only give rise to a few types. Both types of stem cells have two key properties. The first is that they are primitive cells that can give rise to more mature functional cell types like neurons or red blood cells. The second is that they can also divide to produce more stem cells. So stem cells provide a reservoir for tissue regeneration or repair. I think the excitement around pluripotential stem cells in particular has to do with the fact that they can be grown indefinitely in the laboratory and multiplied many times over to make more stem cells and that they can give rise to all the tissues of the body. So for the first time we've got an indefinitely renewable source of any healthy human tissue for use either in the laboratory or in transplantation medicine.

SHANE HUNTINGTON You've been working at the stem cells, essentially, from the beginning, when you and others first discovered that you could, potentially, coax these cells down particular differentiation pathways. Tell us a bit about what that was like back then because I can imagine there would have been an incredible sense of enthusiasm with regards to the possibilities of such cells.

MARTIN PERA Well, we were very excited. Of course, it was known since the 1980s that stem cells could be developed from the mouse embryo. Despite many attempts in the intervening years it proved difficult to derive these cells from other species. Then, in the mid-'90s, Jamie Thomson, in Wisconsin, showed that you could make pluripotent cells from the rhesus monkey embryo. And our laboratory, and a few others, set out to see if we could do this from human embryos. It was incredibly exciting when we first had some success, so that, indeed, we could make these cells and that they could turn in to human tissue-like nerves.

SHANE HUNTINGTON What does it mean in terms of the progress of, essentially, combating a range of diseases at that point in time? It seems as though you had something that looked like it was an answer but, decades later, we're still sort of a fair way off.

MARTIN PERA Well, at those early stages there was incredible promise and incredible potential. One of the most difficult things for us in the field was to convey that promise, and the excitement was not trying to claim that things were going to happen too quickly. To me, it's remarkable that only a little over a decade after human embryonic stem cells were discovered we're already seeing clinical trials of the first human embryonic stem cell derived products for conditions like macular degeneration, a very common cause of blindness. Now, that's remarkable because, as I say, it's only a little over a decade. Even in the drug industry - which is a well travelled paradigm for development of therapeutics - it's not unusual to have 10 to 15 years between the discovery and clinical trials. So I think this is quite remarkable. However, in many areas, we still have a long way to go for many applications.

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Stem Cell Therapy: The Hope and the Hype

Family ties: Trinity woman with leukemia receives stem cells from brother

CHAPEL HILL Kathy DeClue loves her youngest brother Don Hammed for many reasons. These days, Dons selflessness is at the top of her list.

Don donated the stem cells I received during my transplant, said Kathy, 57, of Trinity, in Randolph County. It was a favor I never thought Id have to ask for. But I would have done the same for him if he needed me to.

Kathy needed the stem cell transplant to treat chronic lymphocytic leukemia (CLL), a cancer of the blood cells. For some CLL patients, the disease progresses slowly and they may never need treatment. For others, like Kathy, the disease was on fast-forward and required aggressive medical attention.

From the start, we knew that the CLL was behaving like a high-risk disease and was resistant to just about all the therapies we have, said James M. Coghill, MD, assistant professor of hematology and oncology at the UNC School of Medicine, a member of UNC Lineberger Comprehensive Cancer Center and the leader of Kathys health care team.

The stem cell transplant on April 25 was the best option for trying to get her disease under control, and Kathy had the luxury of three siblings who were a perfect match to donate stem cells. Shes had a supportive family every step of the way. Im sure they would be willing to donate bone marrow again if she needed it.

Besides Don, 46, of Kernersville, N.C., brother Butch Hammed, 56, and sister Rose Tucker, 60, both of Roanoke, Va., were perfect matches. Having multiple matches is unusual as most patients, at best, have a one-in-four chance of getting a perfect match, Dr. Coghill said.

Don got the nod because he was young, healthy and had never been pregnant, Dr. Coghill said. Generally, we try to go with males as donors because female donors who have been pregnant develop antibodies that can increase the chances of graft vs. host disease or rejection.

Rose, the mother of three children, jumped, cried and screamed when she found out she was a perfect match for her sister and her best friend.

I wanted to be a match and her donor so bad, and I was really disappointed when they went with Don, Rose said. They told me Id have enough to do as her caregiver, and as it turned out, I did.

Rose, who was laid off from her child day care job last year, came to Chapel Hill and tended to Kathys every need during the preparations for the transplant and the 100 days post-transplant that Kathy was required to stay at SECU Family House.

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Family ties: Trinity woman with leukemia receives stem cells from brother

World First: Islet Transplant Into Man Using Sernova's Cell Pouch(TM) for Treatment of Diabetes

LONDON, ONTARIO and EDMONTON, ALBERTA--(Marketwire - Aug. 16, 2012) - Sernova Corp. ("Sernova" or the "Company") (SVA.V) and the University of Alberta today announced the treatment of the first patient with insulin-producing islets transplanted into Sernova's Cell Pouch(TM) in a Phase I/II clinical study to treat Type-1 diabetes led by Dr. James Shapiro, Professor of Surgery and Medicine, University of Alberta and Director, Clinical Islet Transplant Program. Sernova will host a conference call at 9 a.m. EDT, today, Aug. 16, 2012, to discuss the company's clinical and business developments.

"The Sernova Cell Pouch(TM) implantation and transplantation processes are simple, rapid minimally invasive procedures, conducted on an outpatient basis under local anesthesia," said Dr. Shapiro. "This offers substantial potential benefit over the Edmonton protocol and the ease of use provides an opportunity for the Cell Pouch(TM) to become the standard of care for people with diabetes if it proves to be effective in these initial trials."

The objectives of the human clinical trial are to assess the safety and efficacy of the Cell Pouch(TM) with transplanted islets in up to 20 patients with Type-1 diabetes. The study is sponsored by Sernova Corp and the University of Alberta. In the study, patients who have met the enrolment criteria and provided informed consent are implanted with the Cell Pouch(TM) prior to transplantation of donor human islets. To prevent islet graft rejection, patients in this study are treated with the best in class standard of care immunosuppression protocol. Interim analysis of the data from this clinical study is expected during H1, 2013. Further information on the trial may be found at http://www.clinicaltrials.gov (Identifier:NCT01652911).

"The Cell Pouch(TM) is a breakthrough technology which has the potential to significantly improve the lives of people living with chronic diseases such as diabetes," said Philip Toleikis, Ph.D., President and CEO of Sernova Corp. "Supported by Sernova's strong preclinical results, the treatment of patients in this clinical trial of the Cell Pouch(TM) further advances our vision for the future of providing millions of diabetic patients with the Cell Pouch(TM), and an unlimited source of insulin-producing cells protected with a local anti-rejection technology."

Conference Call

The conference call to discuss Sernova's clinical and business developments may be accessed by dialing 866-532-1852 for domestic callers and +443-842-7644 for international callers. Please specify to the operator that you would like to join the "Sernova conference call" or "conference ID: 21155379."

About The Cell Pouch(TM)

The Cell Pouch(TM) is a proprietary medical device that, following subcutaneous implantation, incorporates with tissue and microvessels forming a natural environment for transplantation of therapeutic cells. Multiple preclinical studies demonstrated the Cell Pouch(TM) to provide a safe environment for transplantation, as well as long-term efficacy of therapeutic cells. Sernova's goals for the Cell Pouch(TM) for diabetes include providing a safe and natural site for islets to significantly increase the number of patients currently treated with intraportal delivery of donor islets and to provide a safe environment for virtually unlimited available sources of insulin-secreting cells such as insulin-producing stem cells and xenogeneic cells. This vision combined with local anti-rejection protection of the cells could enable millions of patients with insulin-dependent diabetes to be treated without limitation to availability of cells.

About Sernova

Sernova Corp. is a clinical stage health-sciences company focused on commercializing medical technologies. Sernova is currently developing a platform technology for a number of serious disease indications, starting with a novel treatment for insulin-dependent diabetes, using the novel Cell Pouch System(TM) for transplantation and long-term survival of therapeutic cells and its patented Sertolin(TM) cell technology which can provide an immune-protected local environment for therapeutic cells.

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World First: Islet Transplant Into Man Using Sernova's Cell Pouch(TM) for Treatment of Diabetes