Rydr Rudgers suffers from cerebral palsy. He couldn’t move or talk before a cord blood stem cell treatment undertaken during a Duke University study.
His doctor, Michael Chez, M.D. is medical director of Pediatric Neurology at Sutter Neuroscience Institute. He is now heading up the first stem cell clinical trial on autism and hopes for similar results.
Dr. Chez says autism shares some of the same symptoms as cerebral palsy and that there is plenty of anecdotal evidence that using cord blood stem cells from the patients' own umbilical cord can regenerate brain cells. The study which will employ placebos will determine scientifically whether such treatments make improvements in young autism patients.
Sutter Neuroscience Institute got FDA approval for the landmark study and will enroll 30 kids with autism age 2 to 7 to receive injections of their own stem cells. Most parents are given the option of saving their childrens' ubilical cords after birth.
Elisa Rudgers is glad she did. Now four years old, Rydr is walking, talking and eating on his own. That wasn't possible without three stem cell therapy injections over several years. After each injection he made a marked improvement in motor skills.
"It's amazing from where he started and we believe it has t do with the cord blood and all therapy he's gone thorugh since birth," said Rudgers.
Dr. Chez says such therapy is much safer than using randomly donated stem cells and has the potential to evolve into an effective autism treatment.
About 1 in 88 children in the United States 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 we’ll be able to answer in a firm way that this is truly an observed effect, or we didn’t get an observed effect,’’ Chez said.
Chez says it's not known exactly how stem cells regenerate brain cell functions and that eventually a smaller portion of the stem material will be injected to fight the symtoms of autism.
"Probably this will be looked back on as a crude first step but if we see progress it will lead to more progress," said Chez.
How the stem cell trial will work
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.
The study, which began enrolling patients Tuesday, is the first of its kind approved by the 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, and the principal investigator.
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.
The study is designed to keep clinicians, researchers, and parents in the dark about what arm of the study they’re in to prevent bias, Chez said.
‘‘Parents want so desperately to see a response, and therapists want to see a response, if you don’t have an appropriately blinded control study, you get an elevation of observation of response,’’ he said. ‘‘That’s true for any disease that has no cure, but more so with something subjective like autism.’’
Theories on the root cause of autism
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 aren’t 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.