New Study Finds Hope for Autism Treatment in Stem Cell Therapy

This study is just the start of uncovering a possible cure for autism, and more research needs to be done.

It is estimated that one in every 68 children in the United States has some form of autism, according to the Centers for Disease Control and Prevention. Sadly, thirty percent never learn to speak, and many who have early behavioral interventions still struggle on a daily basis. Without an FDA-approved medication to improve symptoms of autism, parents and doctors are always looking for a solution.
A new study may give these families some newfound hope. Twenty-five autistic children age two to six-years-old participated in a cutting edge study at Duke University to see whether a transfusion of their own umbilical cord blood containing rare stem cells could help treat their autism. Each child received 1 billion to 2 billion stem cells through an IV in their arm or leg.
Before the children began the cord blood infusion, they were evaluated based on several behavioral and functional tests focused on expressive vocabulary, eye-tracking, and attention to social stimuli. Additionally, they underwent MRIs and EEGs to track their brain activity. Parents also completed written surveys about their child’s behavior. The children were evaluated again six and 12 months following the infusion.
The results were recently published, highlighting some positive findings. More than two-thirds of the children showed significant improvements from their autistic symptoms. Some children who were not speaking very much had major improvement in their vocabulary and speech. Many children were able to easily play and hold more meaningful conversations. Finally, some children had less repetitive behaviors than they did before the stem cell therapy.
A recent CNN article featured a family who has witnessed the spectacular results of the study in their autistic daughter. Seven-year-old Gracie used to throw tantrums and even kick her own sister. But since she underwent the stem cell therapy, she has been a completely different child. Although she was diagnosed as being on the mild to moderate autism scale, her parents said that the disorder consumed about 75 percent of their daily routine. After the study, that time dropped to only ten percent. She now attends a regular school and is thriving there. The family is thrilled with the changes they have seen in her behavior, and her quality of life has been dramatically improved.
This study is just the start of uncovering a possible cure for autism, and more research needs to be done. This initial trial did have its limitations; it was an uncontrolled safety study and open so that the doctors and families knew that the therapy was being administered.
A more traditional, larger second trial with oversight by the FDA is currently underway. It has a placebo and involves 165 autistic children ranging in age from two to eight-years-old.
During the phase II study, the children on their first visit receive a cord blood infusion (either their own or from a donor) or they get a placebo. They also undergo a behavioral assessment and brain monitoring. On their second visit six months later, they will have another infusion with the preparation that they did not receive the first time. Researchers will monitor them for over a year to evaluate any behavioral improvements.
To follow the results of the study, visit the Duke Center for Autism and Brain Development.