Stem Cell Therapy Brings Hope to Children with Autism

Autism is a condition which affects an estimated 1 in 45 people in the UK. Around 30% of autistic children will never learn to speak, and many children even with early behavioural interventions still struggle to adapt. Although early intervention and behaviour management strategies help, there are no medically approved treatments that improve the core symptoms of autism.

A recent study by Duke University in North Carolina has shown some promising results that point to the possibility of being able to treat autism using stem cells found in a child’s own cord blood.

First-of-its-Kind

The first-of-its-kind study was lead by Dr. Joanne Kurtzberg, one of the lead researchers at the Carolinas Cord Blood Bank, and Dr. Geraldine Dawson director of the Duke Center for Autism and Brain Development. After seeing successful trials using cord blood to treat children with inherited metabolic disorders and cerebral palsy, they saw a great need for further medical advances in the treatment of autism.

The study involved 25 children with autism whose parents had previously banked blood from their umbilical cord at birth.

In the first treatment each child was given an IV infusion of their own cord blood containing 1-2 billions cells. Three times over the course of a year, an evaluation of the child’s brain activity was carried out, and behavioural observations made.

Positive results

After one year, more than two thirds of children showed significant and continued improvements in behaviour as evaluated by their parents and researchers. This included throwing less tantrums, showing less volatile behaviour, and generally being calmer in every day life.

“Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech,” Kurtzberg says. “Many children were able to attend to play and have meaningful communication in a way that they weren’t before. Some children had less repetitive behaviors than they did when they came onto the study.”

Parents of one of the children, Gracie Gregory, were even able to let her go to a mainstream school, something they previously thought impossible.

Positive but not conclusive

Whilst the research is promising, any results need to be treated cautiously.

As a safety study, not a controlled, double-blind study, it cannot yield definitive proof of positive results. The study was open-label, meaning everyone – the doctors and the families – knew that the therapy was being administered. This means that positive results could be attributed to a number of other factors including a natural improvement of behaviour with age, and the parents subconsciously wanting to see and therefore magnifying any improvements.

A larger second double-blind, placebo-controlled trial is now underway which will involved 165 autistic children between 2 to 8 years old. The added placebo control element, and higher number of children involved, will allow the researchers to better assess the effectiveness of the treatment.

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