Back in 2017, an initial study began to investigate whether infusions of cord blood can improve the symptoms of autism spectrum disorder (ASD) in young children. Following Phase I and Phase II trials, the results were revealed in the Journal of Pediatrics in June 2020.
The initial Phase I study was carried out on 25 autistic children, aged 2-6. It concluded that approximately 70% of the children showed improvements in one or more core symptoms of autism. The randomised, placebo-controlled Phase II trial, led by Drs. Joanne Kurtzberg and Geraldine Dawson, was designed to confirm these findings by evaluating the effects of autologous and allogenic cord blood on 180 children.
The children taking part ranged from 2-7 years of age and all had confirmed cases of autism with no known genetic cause. Based on previous results, the children with a non-verbal IQ >70 showed the most improvement after an infusion of autologous cord blood. Therefore, attempts were made to only include children with nonverbal IQ>70.
The Phase II study involved giving 60 patients autologous cord blood, 60 allogeneic cord blood and 60 a placebo. The children who received autologous cord blood had to have 25 million cells per kg of the child’s weight. Those with less received cord blood from an HLA matching unrelated donor.
For the children who received cord blood instead of a placebo, significant improvements were noticed in those aged 4-7 with a non-verbal IQ higher than 70. Particularly, improvements were seen in communication, eye tracking and EEG brain scans.
In most of the measurements, there were no advantages of allogeneic over autologous cord blood identified or vice versa. However, it's important to note that autologous cord blood is an 100% DNA match to each child, if stored in a private stem cell bank at birth, provides a more readily available, perfect match for potential future treatments.
Based on lessons learned from this study, Dr. Joanne Kurtzberg recently opened a Phase II clinical trial investigating cord tissue mesenchymal stromal cells (MSCs) to treat autism. You can find out more about it here.