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Autism stem cell trial reports improvement in symptoms

10 April 2017
Appeared in BioNews 896

Children with autism spectrum disorder (ASD) who received infusions of their own stem cells from banked umbilical cord blood as part of a clinical trial have no apparent lasting adverse effects after one year.

Parents of some of the 25 children in the study – who were aged between two to five – reported that their kids showed reduced signs of ASD after the treatment. However, improvements in behaviour were correlated with the child's IQ (which was measured before the study) and not with the quantity of stem cells the children received. This may mean that improvements seen were due to the children learning new skills and coping methods and not to the therapy.

Reduction in ASD-related behaviour was measured by questioning the parents and by clinician-administered tests. Around 40 percent of the children in the trial exhibited no improvement.

A similar study reported on in 2016, led by Dr Michael Chez, director of Paediatric Neurology at the Sutter Institute, California and involving 30 children, showed a similar percentage of reported improvement (see BioNews 670).

The team involved in the latest trial, led by Dr Geraldine Dawson and Dr Joanne Kurtzberg from Duke University Medical Center, North Carolina, are now running a second, larger trial on whether cord blood can treat autism. The double-blind, placebo-controlled trial involving 165 autistic children aged two to eight will be overseen by the US Food and Drug Administration.

Some observers have questioned the science underlying the study. 'It's probably premature to run large trials without evidence that they have a therapeutic effect that [we] understand,' said Dr Arnold Kriegstein, director of the Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, San Francisco, who was not involved in the research. 'I think it would be marvellous if this trial worked, but it really seems more like a "Hail Mary pass" than a rational therapy,' he said in 2014.

Cord blood stem cells can be collected from the umbilical cord and placenta and then stored in cord blood banks for future use in cellular therapies or blood stem cell transplantation.

Previous research has shown that cord blood stem cells can help reduce inflammation and signal cells to help repair damaged brain areas. While there is a hypothesis that some signs of autism are due to inflammatory processes in the brain, autism spectrum disorders are complex and may have many possible causes, including differences in the development of neuronal connections or mitochondrial function. Blood stem cells cannot replace neurons, although they may be able to enter the brain after infusion into the blood.

20 May 2019 - by Dr Loredana Guglielmi 
The number of new parents paying to bank their baby's umbilical cord blood for future use in stem cell therapies or transplantation in case of illness has increased by 59 percent in the space of four years in the UK...
4 December 2017 - by Dr Kristien Hens and Dr Leni Van Goidsenhoven 
Popular scientific publications regularly have articles with headlines suggesting that 'autism is hereditary' or that the 'risk for developing autism' is mostly caused by genetics...
10 July 2017 - by Dr Lea Goetz 
International experts are calling for global action on unproven and potentially dangerous stem cell therapies and their misleading marketing to the public...
13 March 2017 - by Jenny Sharpe 
A study that sequenced the whole genomes of over 5000 people has discovered 18 genes associated with autism spectrum disorder (ASD)...
6 March 2017 - by Jamie Rickman 
Researchers have discovered that genetic variants associated with Autism Spectrum Disorder (ASD) also correlate with higher intelligence...
7 November 2016 - by Dr Helen Robertson 
Children with autism spectrum disorder have twice as many harmful mutations in their mitochondrial DNA as their siblings, a study has found...
1 February 2016 - by Dr Julia Hill 
A Chinese laboratory has created genetically modified monkeys that show symptoms of autism...
28 August 2012 - by Nishat Hyder 
Stem cells harvested from patients' own umbilical cord blood are to be trialled in the US as treatment for children with autism...
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