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Friday, August 24, 2012

Two Dispatches from the Frontiers of Autism Research

From the Autism Speaks Blog

Donated Tissue Makes New Findings Possible

By Jill James, Ph.D., Director of the Autism Metabolic Genomics Laboratory at Arkansas Children’s Hospital Research Institute

Autism Speaks' Autism Tissue Program and its donors enable the study of brain inflammation, cell damage and antioxidants.

Postmortem brain tissue is one of the most precious donations a family can make. I am forever grateful to families who make this remarkable gift. In fact, the research that I do has been made possible by Autism Speaks Autism Tissue Program (ATP), which makes brain tissue available to qualified scientists studying autism.

To help illustrate the importance of these donations, please see today’s news story on the findings we published in Translational Psychiatry. We also had the opportunity to present our results at the Autism Speaks National Conference for Families and Professionals, earlier this month.

In this study, we found lowered levels of the antioxidant glutathione in the postmortem brain tissues of persons who had autism. These results support our previous findings of oxidative stress and damage in plasma and immune cells from children with autism. Together, this suggests that damaging oxidation may be pervasive in autism.

Further, it may be that markers of oxidative stress outside the brain may allow us to gauge oxidative stress in the brain of living persons with autism.

Taken together, these results suggest that a genetic predisposition to insufficient antioxidant protection in the brain may contribute to autism risk. At the least, this may be true in the presence of certain stresses during brain development.

In recent years, research has made it increasingly clear that autism can result from a combination of genetic predisposition and environmental stress during critical periods of early brain development. Our research supports this line of thinking.

These findings represent a small step forward. But we see great potential to deepen our understanding of the underlying biology of autism in ways that may lead to new methods for preventing and treating it.

I can’t emphasize enough how much this scientific progress depends on the support of families such as yours – whether it’s research dollars raised at an Autism Speaks Walk or a post-mortem donation of precious brain tissue. We could not do what we do without your support. Thank you.

Learn more about participating in Autism Speaks Autism Tissue program here and a wide range of other autism studies here. Explore more of the studies Autism Speaks is funding through the grant search, and find more news and perspective on the Autism Speaks Science page.

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From Spoonful of Medicine - A Blog from NATURE Medicine

First US Stem Cell Trial for Autistic Children Launches Today

By Kathleen Raven
August 21, 2012

"Go slowly and think hard about your decision.”

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 participant’s 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 child’s 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 body’s 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 study’s 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.

Not all stem cell scientists who study neurodevelopmental diseases are ready to invest great hope that the autism stem cell trial will succeed. “I wish I could tell you I’m optimistic about the end results,” says James Carroll, a pediatric neurologist at the Georgia Health Sciences University in Augusta who began a clinical trial two years ago to better understand how stem cell therapy affects patients with cerebral palsy. “But so far we have not seen any kind of miraculous recovery in our cerebral palsy patients. I would be delighted if that changes.”

Members in the stem cell therapy patient community think Chez will have no shortage of volunteers for the trial. Jeremy Lowey, who lives in Sacramento and has struggled with a rare condition known as non-verbal learning disorder, arranged for his own stem cell therapy treatment in India last year, which he called life-changing. He receives numerous Facebook requests from parents of autistic children who are curious to know more. He always begins his conversations by saying, “Go slowly and think hard about your decision.”

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