By Kathleen Raven of Nature Medicine

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.”

This article is reproduced with permission from Nature Medicine. The article was first published on August 21, 2012.

9 Comments

1. 1. BigInScience 02:21 PM 8/21/12

   Fascinating article Kathleen. I typically browse through hundreds of articles on science and health a day, and it seems like autoimmunity and inflammation have become much more discussed as contributing to a greater diversity of conditions including autism. I've written several articles on both topics recently at BigInScience.com
   
   Although the methods utilized in the above clinical trials seem rather crude at this point, I'm glad to see U.S. institutions utilizing stem cells to potentially treat conditions like Autism. I hope this will deter some from pursuing fraudulent "stem cell" treatments outside the country (such treatments were recently highlighted in a 60 Minutes episode http://www.cbsnews.com/video/watch/?id=7394380n).
   
   Fascinating development Kathleen!

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2. 2. Aer-O-Head 03:56 PM 8/21/12

   That's simply ridiculous, junk science at its best. They're simply throwing stem cell technology at autism just because it's been successful in other areas, with tissues and cellular materials. Stem cells basically regenerate cellular growth, but nothing indicates that autism is a cellular problem. They're basically playing with grant money (or whatever) to play games with people's minds and lives, without any foreseeable benefit. In other words, they're speculating that this just might work ... maybe -- "let's just mix these two chemicals together to see what happens." This will likely raise people's hopes and expectations (parents are desperate for solutions with their autistic children), without any basis to suppose that they just might have something here. In my opinion, that is just plain all wrong. This is just experimentation with human beings, a la Dr. Josef Mengele.

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3. 3. ejwillingham 08:12 PM 8/21/12

   This is terrifying, given some of the adverse effects that stem cells have had in the pediatric population. What is the scientific rationale or evidence-based argument for trying stem cells for autism, particularly in the context of that catch-all bogeyman, "inflammation"? This sounds like a fishing expedition using, as the above poster commented, junk science. Ugh.

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4. 4. StemCellBlogger 02:06 AM 8/22/12

   Adult stem cells safety and efficacy: Used for over 40 years successfully in bone marrow transplants. Used successfully for years in PRP treatments. Thousands of successful adult stem cell trials and studies and thousands of patients treated successfully around the world. Blood, hearts, tracheae and other organs created with adult stem cells...all of this was done with zero to virtually no adverse affects and no controversy...and 'ejwillingham' there are many, many autistic spectrum patients who have derived significant benefit from stem cell treatments. www.repairstemcell.wordpress.com

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5. 5. pknoepfler 12:31 PM 8/22/12

   "StemCellBlogger" is someone who is paid a commission to get patients to go to clinics so you can see their motivation for their simplistic cheerleader-like comments.
   
   Overall, I agree with Dr. Carroll quoted in the article that the odds are very low this trial will succeed. I wish it were different.
   
   More research needs to be done to figure out what kids get autism. Without knowing the "why" of a disease, it is very difficult to treat it, right? For more thoughts on this check out my blog post on this trial and more general concepts related to using stem cells for autism as well as stem cell treatments more generally: http://tinyurl.com/c9ajjqa
   
   Paul Knoepfler PhD
   Associate Professor
   UC Davis School of Medicine

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6. 6. bucketofsquid 05:27 PM 8/22/12

   Unless you want to volunteer your brain or that of your autistic child, these shots in the dark are about all we really have. Things that seem to work in animal testing frequently don't translate directly to humans. We are decades from having a computerized model of the entirety of chemical processes in the brain and its various cells.
   
   Better a shot in the dark than nothing at all. The concerns about side effects of stem cell treatment are quite valid and should be clearly explained. the initial trail should be on a small group to avoid unneeded risk. I'm wondering if the persons own stem cells will minimize side effects compared to donor stem cells.

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7. 7. pmb1995 12:49 PM 8/27/12

   I have friends who went to Costa Rica for stem cell treatment for autism. Unfortunately, it cost thousands of dollars and was not successful. Hopefully this study will have better results.

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8. 8. katwomanjulie 03:53 PM 1/22/13

   I think it's ignorant to call this junk science. The Catholic Church used to call Galileo "junk science" when he thought the planets revolved around the Sun at the not the Earth. Need I say what the truth really was? The point of science is to be open minded, form hypothesis and experiment with hopefully the outcome helping man kind. Where would be if it wasn't for Madame Curie exposing herself to radiation? She made a break through in science and it cost her, her life. With that being said, I think it's important for all pregnant woman to get the facts about umbilical cord stem cells. A great resource is http://genecord.com/. Most cultures toss out the umbilical cord, we might as well use it for science purposes.

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9. 9. suzan khuluqi 12:15 PM 3/6/13

   thank you for such a nice article, my son is 6 years old ,diagnosed at the age of 2 years to have autisim ,we have been through many treatement modalities using the dan protocol ,our sun already has umbilical CORD BLOOD at the baby cord institution in USA ,both parents are M.D and wondering about the possibilties of involving our son in your study ...we will appreciat it if you contact us soon.

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