Results revealed from Duke University’s autism study

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 1 and Phase 2 trials, the results were revealed in the Journal of Pediatrics in June 2020.

The initial Phase 1 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 2 trial, led by Drs Joanne Kurtzberg and Geraldine Dawson, was designed to confirm these findings by evaluating the effects of autologous and allogeneic 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 2 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.

Key findings
• Throughout this final study, no severe adverse effects were observed
• On the Socialization Scale, no benefits of cord blood over placebo were found however, this analysis was flawed because only 101 of the 180 children enrolled on the study had a non-verbal IQ >70. The minimum needed was 143 children.
• In the cord blood group, significant improvements were noticed in children aged 4-7 with a non-verbal IQ higher than 70
• In most of the measures, there were no advantages of allogeneic or autologous cord blood identified. However, on the Clinical Global Impression-Improvement scale, only children receiving allogeneic cord blood showed improvement compared to the placebo
• In the study, allogeneic cord blood was given at a higher dose than autologous cord blood, meaning that it wasn’t possible to know whether this advantage was due to cell source or increased dose

In conclusion, these results demonstrate encouraging news for children aged 4-7 with a non-verbal IQ >70. Particularly, improvements were seen in communication, eye tracking and EEG brain scans.

Based on lessons learned from this study, Dr Joanne Kurtzberg recently opened a Phase 2 clinical trial investigating cord tissue mesenchymal stromal cells (MSCs) to treat autism. You can find out more about it here.