.- Stem cell researchers are launching the first FDA-approved clinical trial to examine whether cord blood stem cells can improve the condition of children with autism.
“This is the start of a new age of research in stem cell therapies for chronic diseases such as autism, and a natural step to determine whether patients receive some benefit from an infusion of their own cord blood stem cells,” Dr. Michael Chez, the study’s principal investigator, said Aug. 21.
Dr. Chez is the director of Pediatric Neurology with the Sutter Neuroscience Institute in Sacramento, Calif. The institute has joined with the major stem cell bank CBR (Cord Blood Registry) to examine the effect of the stem cell therapy on autistic children.
The clinical study will enroll 30 children ages two through seven who are diagnosed with autism and meet other criteria, CBR says. Participants will receive two infusions, one of the child’s own cord blood stem cells and one of a placebo, over 13 months. The trial intends to determine whether the cells help improve patients’ language and behavior.
One in 88 children in the U.S. are diagnosed with autism, a developmental disorder that affects social, language and behavioral skills.
Chez said there is evidence that some autistic children have dysfunctional immune systems that damage or delay their nervous system development.
“Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients which would also improve language and some behavior in children who have no obvious reason to have become autistic,” he said.
Umbilical cord blood contains unique stem cells that have been used for more than 20 years to treat some cancers, blood diseases and immune disorders.
Dr. Maureen L. Condic, associate professor of Neurobiology and Anatomy at the University of Utah, told CNA she is “intrigued” by the study but “cautious.”
“Autism is a very complex and poorly understood condition,” she said Aug. 22.
Umbilical cord stem cells are obtained from a patient at birth and are therefore genetically identical to the patient. This means that they will not be rejected by the patient’s immune system.
Embryonic stem cells, by contrast, are obtained from human embryos destroyed to produce stem cells.
“They are likely to be rejected by the patient's immune system, unless the patient is treated with immune-suppressing drugs,” she added. “Undifferentiated embryonic stem cells produce tumors when injected into patients, and cannot be used for therapies without first manipulating these cells to produce a mature cell type.”
Catholic ethics rejects the use of embryonic stem cells because they are derived from destroying a human embryo.