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Hope for the treatment of autism with umbilical cord blood



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Results from a study have revealed encouraging results for the improvement of symptoms of autism in young children following treatment with umbilical cord blood.

Autism spectrum disorder (ASD) impairs the way a person communicates and the way they perceive and interact with the world around them. The causes of autism are not yet fully understood but research suggests that genetic and environmental factors may account for changes in brain development. 

At the Duke Centre for Autism and Brain Development Dr Joanne Kurtzberg and other researchers are bringing new hope to the treatment of autism. 

Cord blood has the ability to cross the blood–brain barrier and differentiate into neurons and other brain cells. There are a few schools of thought on how cord blood can repair brain trauma or neurodegenerative disorders:

  • The transplanted stem cells directly replace dead or dying cells.
  • The transplanted stem cells secret growth factors that indirectly rescue the injured tissue.
  • The transplanted stem cells build a “biobridge” that connects the healthy section of the brain and the damaged section of the brain to facilitate the transport of new neural stem cells to the area in need of repair.

Duke University Medical Centre have found that 70 per cent of the children in their study who received cord blood had improvement in one or more of the core symptoms of autism.

180 children aged 2 – 7 years of age with a confirmed diagnosis of ASD were tested to see whether cord blood infusions would improve symptoms of autism.

The children tested did not have a known genetic cause of ASD, had no other illnesses, were English speaking, had tested negative for Fragile-X, a genetic disorder characterized by mild-to-moderate intellectual disability, and had a negative chromosomal microarray. They were divided into two sections – treatment and placebo. 

Children received autologous cord blood (their own cord blood) if they had a qualifying unit that contained a minimum of 25 million cells per kg of the child’s weight, based on the pre-cryopreservation count. Children lacking an autologous cord blood unit received a >4/6 HLA matching unrelated donor cord blood unit. Both the autologous and donor cord blood units had to contain a minimum cell dose of 25 m.

120 children received cord blood (60 autologous and 60 allogeneic) and 60 children received placebo for their first infusion. Children were analyzed for response at 6 months after their first infusion.  Children crossed over at 6 months to a second infusion. Children receiving cord blood for their first infusion received placebo for their second infusion. Children receiving placebo for their first infusion received cord blood for their second infusion. The type of infusion given at baseline and at 6 months was blinded so that no one interacting with or testing the children knew which infusion was given at each time-point.

The study showed significant improvements in the cord blood group for the subset of children on the study who were 4-7 years of age and were without intellectual disability (NVIQ >70). Improvements were seen in communication (VABS-3 Communication Scale), attention (eye tracking), and increased alpha and beta EEG power. 

In most of the measures, there were no advantages of allogeneic or autologous cord blood identified. But on the Clinical Global Impression-Improvement scale, only children receiving allogeneic cord blood showed improvement, compared to placebo.

It is very important to note that children receiving allogeneic cord blood were given a higher total dose of cells compared to the children receiving autologous cord blood. Thus, it was not possible to know whether this ‘advantage’ of allogeneic cord blood was due to increased dose or the donor cell source.

The study has concluded that there were encouraging results for the sub-group of children ages 4-7 with NVIQ >70. Significant improvements were seen in communication, eye tracking and EEG Bran scans.

References

https://www.jpeds.com/article/S0022-3476(20)30334-6/fulltext
https://parentsguidecordblood.org/en/news/results-duke-act-study-cord-blood-autism-inside-scoop-dr-kurtzberg