Stem cell studies a long way from autism answers

A recent post in the Nature blog Spoonful of Medicine talked about how the Sutter Neuroscience Institute in Sacramento has started recruiting kids for a clinical trial on an experimental stem cell therapy for autism. The author, Kathleen Raven (@sci2mrow), reported that the research team there hopes their study answers questions about a disease with "few, largely ineffective treatments."

The California team will recruit 30 autistic children between ages 2 and 7 who have cord blood banked at a Cord Blood Registry near the institute. That’s because the Sacramento study will use each child’s own stem cells as treatment, and because cord blood is a stem cell source that avoids the debate surrounding embryonic stem cells. Other autism stem cell trials underway include a Chinese study using cells donated from other children’s cord blood, and a Mexican study where stem cells will be harvested from each child’s fatty tissue.

We asked Alan Percy, M.D., professor in the UAB School of Medicine’s Division of Pediatric Neurology, and Fred Biasini, Ph.D., associate professor in the Department of Psychology, for their take on the studies. Their short answer was that the three studies are unlikely to soon answer the question of whether or not stem cells can treat autism. The Chinese study is complete but has not published any papers and the Mexican study is still recruiting.

Percy says the most important, eventual achievement of the California trial may be to prove with rigorous science that such treatments do not work. The problem, he says, is that some parents are so hungry for answers that they will pursue therapies even If the science is not there. In many cases they leave the U.S. to gain access to treatments in places with much less rigorous patient protection laws in place. Percy sees many pursue this course but no evidence of treatment success.

While the techniques used in the Sutter study have a very small chance of working, Percy says, they may provide insights into better ways to use a patient’s own stem cells in future attempts to create treatments.
The idea that stem cells might improve an autistic child’s ability to speak is based on the theory that the disease has some autoimmune roots. Immune cells in the brain mistakenly attack brain cells to cause inflammation. The hope is that injected stem cells would recognize the ‘autoimmune’ cells as foreigand clean them out. The problem, says Percy, is that such cells would have to enter the central nervous system, an obstacle that has stymied many promising treatments.

UAB is one of the 14 study sites within the Autism and Developmental Disabilities Monitoring (ADDM) Network, says Biasini. Funded by the Centers for Disease Control, the network had been working in recent years to determine how common autism is and how that changes over time and by region. So far, they have determined that about 1 in 88 children have a confirmed autism spectrum disorder.

Biasini also mentioned the following as samples of key UAB autism research:

• An effort led by Biasini and Maria Hopkins, Ph.D., an assistant professor of psychology in the UAB College of Arts and Sciences, seeks to understand how the ability to interpret facial expressions, or not, affects social skills in children with autism with the goal of improving social interactions.
• Work by Sarah O’Kelley, Ph.D., assistant professor in UAB Department of Psychiatry and Behavioral Neurobiology, who is studying social interventions such as the organizing of social skills groups, to see which are most effective in improving social skills in children and adolescents with ASD.
• Kristi Menear, Ph.D., associate professor in the Department of Human Studies within the UAB College of Arts and Sciences, is looking at the impact of exercise on cognitive and social development in adolescents with an ASD.  Exercise has been shown to reduce the impact of various mental and physical health conditions, and her study will look at whether the effect is there in autism as well.
• Irena Bukelis, a psychiatry resident at UAB, is examining the types of medications prescribed to children suspected of having an ASD, which typically include antipsychotic, ADHD and antidepressant drugs. The work will help researchers understand the medication-related issues faced by families as they seek to address a child’s cognitive, social and language challenges, often before they receive an official diagnosis of ASD.

For more information on the state of autism research, Biasini recommends a new and thorough review of current work just published by the Interagency Autism Coordinating Committee (IACC), organized under the auspices of the U.S. Department of Health & Human Services.  Another strong source of information, research resources and registries is Autism Speaks.