The Sutter Neuroscience Institute in Sacramento plans to launch groundbreaking research today to discover whether infusing umbilical cord stem cells into the bloodstreams of autistic children will help them overcome debilitating characteristics of the condition.
The clinical trial is the first of its kind to be approved by the U.S. Food and Drug Administration, said Dr. Michael Chez, the principal investigator and Sutter's director of pediatric neurology.
One of the world's foremost autism experts at the institute, which is recognized for its practice of pediatric neuroscience, Chez said the research may help identify a valuable new tool in the struggle against autism spectrum disorders, which now affect about one in 88 children nationwide, and one in 54 boys.
"This is an exciting trial, because it's exposing us to the new frontier of stem cells and whether they may have some positive effect on this disease," Chez said. "This is the start of a new age of research in stem cell therapies for chronic diseases such as autism."
Recent research has revealed robust new uses for stem cells. Stem cells, for example, can now be developed from heart muscle in cardiac patients and injected back into the heart for improved functioning. Doctors are also having success using stem cells to treat leukemia and bone marrow diseases.
Autism is the leading cause of delayed development in children, typically surfacing before 3 years of age. The condition is characterized by impaired communication, repetitive thoughts and behavior and difficulty in socialization.
The likelihood of a child's being given a diagnosis of an autism spectrum disorder increased more than 20 percent from 2006 to 2008, according to a report this year by the Centers for Disease Control and Prevention. Locally, more than 3,830 students in the four-county Sacramento region were counted as autistic in December 2011, up 13 percent from the previous year, according to state data.
The Sutter clinical trial follows promising research in using cord blood stem cells to help children with cerebral palsy, a brain disorder that shares some characteristics with autism, to improve motor function.
Chez became the point man for developing protocols for the clinical trial over a year ago, after being contacted by Cord Blood Registry, the world's largest cord blood bank. The Bay Area company has collected and frozen 400,000 samples of umbilical cord blood for individuals and families.
The umbilical cord that links a mother's placenta to her fetus is imbued with blood stem cells. Since the 1990s, parents increasingly have opted to have their babies' umbilical cords frozen in storage as a sort of insurance against future disease. The trend now extends to about 5 percent of parents.
The basic idea behind stem cell therapy is that these original blood cells can be used to repair malfunctioning organs and systems and that using one's own stem cells, rather than a donor's, lessens the risk of autoimmune responses.
Chez has been following closely the case of a young cerebral palsy patient whose cord blood stem cells spurred marked improvement after infusions at Duke University.
"He's not officially diagnosed with autism, but he has some overlap with some of his symptoms," Chez said of the patient. The boy, who had extreme difficulty moving his mouth, was treated three times with stem cells.
With the first infusion, Chez said, the boy improved. With the second, he was able to eat. With the third, he was talking.
"Every time he got an infusion, he made a huge jump," Chez said.
Scientists believe that stem cells work by sending "messengers" to blood cells, perhaps even sending signals to the brain, Chez said.
"One of the theories that could be the reason why we see results in cerebral palsy patients is that malfunctioning cells might see a signal from the stem cells to go back and function correctly," he said.
Chez is hoping to see similar results in the brains of autistic children.
Autism is thought to have multiple risk factors, including genetic, environmental and immunological components.
It is the immunological component that interests Chez most. Much of his research focuses on the relationship between a child's immune system and the central nervous system. Evidence suggests that some children with autism have dysfunctional immune systems that may damage or delay development of the nervous system.
"Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients who have no obvious reason to become autistic," Chez said.
The study launched today will recruit 30 autistic children between the ages of 2 and 7 whose cord blood stem cells are stored at Cord Blood Registry's facilities.
In what's called a double-blind study, in which neither patients nor researchers know which subjects receive treatments and which receive placebos, 15 of the children will get an infusion of their cord blood and 15 will receive saline solutions.
After six months, the two groups will switch.
Over the next year-and-a-half, the children will be closely monitored for improvements or changes in behavior.
Chez's co-investigator is Dr. Michael Carroll, medical director of the Blood and Marrow Transplantation and Hematological Malignancies Program at Sutter Medical Center in Sacramento.
Said Carroll, "I've seen how stem cell therapy has changed my field of medicine and how I care for my blood cancer patients. I am eager to see how our work can open new doors for patients and families dealing with autism."
The study is being conducted in conjunction with the Sutter Institute for Medical Research. Researchers have set up a hotline providing more information at (888) 536-9826.