Clinical trials with cord blood stem cells are being conducted for a number of disorders. Already, stem cells are being used to treat more than 80 different diseases. Over the past year, researchers at Duke University in Durham, NC have been studying the use of autologous (using patient’s own cells) umbilical cord blood infusions for children with Autism Spectrum Disorder. A smaller ongoing study exploring the treatment of autism with cord blood stem cells was initiated in 2012 by the Sutter Institute in Sacramento, CA.
What is Autism Spectrum Disorder?
According to the Center for Disease Control (CDC), Autism Spectrum Disorder (also known as ASD) is “a group of developmental disabilities that can cause significant social, communication and behavioral challenges.” ASD occurs in all racial, ethnic, and socioeconomic groups. It is almost five times more common among boys than among girls. CDC estimates that about 1 in 68 children has been identified with ASD. It typically begins before age three and then lasts throughout the person’s life.
What is a Clinical Trial?
Clinical trials provide a thorough investigation of a potential treatment and follow strict guidelines to ensure safety and effectiveness. It is a multi-step process, broken down into phases:
Phase 0 and 1 studies are small and have safety as the main goal, such as determining if there are any severe events, and how the drug is metabolized and excreted.
Phase 2 studies center on determining the drug’s effectiveness for a given condition. Participants are often compared with other test groups receiving a placebo (an inactive substance) or a standard treatment for the condition.
Phase 3 expands the test population and further ascertains safety and effectiveness, often looking at different dosages and test populations. At this stage, the FDA can approve or reject the intervention.
Phase 4 studies, if approved, often take place to determine long-term trends of the intervention.
Cord Blood Stem Cells for Autism
The Duke University clinical trials using cord blood stem cells for ASD treatment are currently in Phase 1. For this stage of the trial, the team recruited children who have an autism diagnosis but who do not have other genetic disorders, such as the related Rett syndrome.
First, researchers infuse children with their own cord blood cells. These are cells that were banked at birth with a private cord blood bank. Over time, the researchers record any changes in behavior and learning abilities, as well as any changes to their autism symptoms. These changes are measured by tests such as the Autism Diagnostic Observation Schedule.
Although it is a smaller study, the similar Sutter Institute clinical trial is in Phase 2 and has been incorporating the use of placebo infusions.
One thing is known: potential autism treatment with cord blood stem cells could have a significant impact on the lives of those identified with Autism Spectrum Disorder as well as their families.