Autism researchers have been given the go-ahead by the U.S. Food and Drug Administration to launch a small study in children with autism that evaluates whether a child's own stem cells may be an effective treatment.
Thirty children with the disorder, aged 2 to 7, will receive injections of their own stem cells banked by their parents after their births.
Scientists at Sutter Neuroscience Institute, in Sacramento, Calif., said the placebo-controlled study will evaluate whether the stem cell therapy helps improve language and behavior in the youngsters.
There is anecdotal evidence that stem cell infusions may have a benefit in other conditions such as cerebral palsy, said lead study investigator Dr. Michael Chez, director of pediatric neurology at the institute.
"We're hoping we'll see in the autism population a group of patients that also responds,"
Other autism and stem cell research is going on abroad, but this study is the first to use a child's own cord blood stem cells.
Chez said the study will involve only patients whose autism is not linked to a genetic syndrome or brain injury, and all of the children will eventually receive the stem cells.
Two infusions will take place during the 13-month study. At the start of the research, the children will be split into two groups, half receiving an infusion of stem cells and half receiving a placebo. At six months, the groups will swap therapies. The infusions will be conducted on an outpatient basis with close monitoring, Chez said.
"We're working with Sutter Children's Hospital, who does our oncology infusions with the same-age children,"
"They are very experienced nurses who work with preschool and school-age kids to help them get through medical experiences."
Each child and his or her parents will be given a private room with a television and videos, beverages, and perhaps a visit from the hospital's canine therapy dog, and then a topical anesthetic will be applied to the arm to numb the skin before intravenous needle placement. A hematology expert will be giving the infusions and monitoring for safety, said Chez, who noted that each child will be watched closely for an hour and a half before heading home. They will be seen the next day as well for a safety check.
At six, 12 and 24 weeks, the researchers will measure behavioral and language changes in the children, and other changes noted by parents and the children's doctors will be logged as well.
"Parental observations like socialization and irritability scale as secondary measures are important," Chez said. Brain electrical activity and immune markers such as levels of pro-inflammatory proteins in the blood also will be tracked.
One in 88 children -- about five times as many boys as girls -- are diagnosed with autism, according to the U.S. Centers for Disease Control and Prevention. But its cause is unknown and there is no cure for the disorder, which begins in early childhood and affects social, behavioral and language development.
Genetics and environmental and immunological factors are thought to play a role, and possibly the interaction of these and other variables, Chez said.
Autism is not actually one disorder, but stretches across a spectrum in which some children have more severe symptoms than others.
One theory suggests autism occurs because cells in the brain, called neurons, are not connecting normally. Stem cells may address this issue.
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