A life-changing human condition, ASD or more commonly known as Autism Spectrum Disorder leaves one with an incapacity that is devastating. Clinical studies on multiple family histories and twins have shown that some cases of ASD are genetic. However, the majority do not fall under this category and appears suddenly in young children intermittently or idiopathically.

At the Hussman Institute of Autism (HIA) in USA, a team has been studying upcoming methodologies that are related to using of induced pluripotent stem cells, or iPSCs. A group of mature human stem cells from the skin or the blood on being experimentally induced, possess the potential to differentiate into most cell types in the body. Since 2006, the process of induction and differentiation has had period upgradations. “One of the exciting aspects of working with iPSCs is that we can study autism in human neurons that have the exact genetic background of a given individual with autism,” said John P. Hussman, executive director of HIA.

“Mini-brains” or organoids, a resultant from the iPS cells of ASD patients have been produced by another research group from Yale. It came to light that ASD mini-brains are comprised of inhibitory neuron which is a type of nerve cell that multiplies and blocks the production of a protein called FOXG1 and then returns these nerve cells back to their normal population count.

A CIRM funded study led by Rusty Gage on a secondary stem cell model for ASD proposing fresh evidences into the initial neurodevelopmental deficiencies seen in ASD patients were published in the Nature Journal Molecular Psychiatry by the Salk Institute (USA) in partnership with scientists at UC San Diego.

Under clinical conditions, Gage along with team of researchers attempted to create iPS cells particularly from those select ASD patients who had undergone a brain growth of up to 23% faster during their toddler stage. Under constant investigations, the researchers studied the ways by which iPS cells from these select ASD individuals developed into brain stem cells. The next stage of growth turned these brain stem cells into nerve cells. The entire growth progression was mapped and matched to that of healthy iPS cells from normal individuals.

Under close observation of the procedure, the team quickly found an issue with the multiplication of the brain stem cells in order to generate new nerve cells in the brain. The process has been termed as neurogenesis. A surplus amount of nerve cells were being produced as the brain stem cells extracted from ASD iPS cells exhibited additional neurogenesis compared to normal brain stem cells. The nerve cells were incapable of sending signals and establishing a working system of transmission. Due to a lack of the synaptic connections with each other by these additional nerve cells their performance remained anomalous, portraying an inability to be less effective compared to healthy neurons.

IGF-1, a drug that is currently undergoing clinical testing towards probable treatment for autism was used in this instance to treat the nerve cells. The team noticed partial correction in the abnormal activity observed in the ASD neurons. According to a Salk news release, “the group plans to use the patient cells to investigate the molecular mechanisms behind IGF-1’s effects, in particular probing for changes in gene expression with treatment.”

Gage’s opinion is that: “This technology allows us to generate views of neuron development that have historically been intractable. We’re excited by the possibility of using stem cell methods to unravel the biology of autism and to possibly screen for new drug treatments for this debilitating disorder.”

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Research increasingly points to a correlation between cord blood cells and their application in treating neurologic diseases. Can cord blood cells treat autism?

Children on the Autism spectrum often exhibit varying kinds of hypo- and hyperactivity. While some are withdrawn and uncommunicative, others are unable to read verbal cues in communication and respond appropriately. Still others exhibit a ‘static intelligence’ that helps them excel at studies but not at simple tasks or games requiring hand-eye coordination.

Parents and caregivers do all they can to help Autistic children, but it is a hard challenge, such as that faced by a Mumbai-based senior citizen who not only provided medical care and therapy but also set up a school for her Autistic grandson[1]. There is no known cure yet for Autism and allied disorders.

A neural deficit in the brain forms the basis for all of these ‘disconnections’ in behaviour and speech; those with Autism disorders exhibit lower processing abilities and an inability to process multiple points of information simultaneously.[2]At present, there is no known cure for Autism Spectrum Disorders (ASD), while therapy and lessons aimed at improving cognitive and communicative functions can help to improve matters.

However, there are great strides being taken in the field of stem cell research and therapy in bridging the gap between neural deficits, as also cardiovascular, endocrine and orthopaedic ones. Regenerative medicine therapy holds great promise for individuals on the Autism spectrum since embryonic stem cells and non-embryonic stem cells sourced from the umbilical cord blood, adipose tissue and bone marrow may further be used in a breakthrough treatment for ASD. The pluripotent stem cells found from these three sources are present in multiple amounts, of which cord blood cells are the most capable of generating epithelial, hematopoietic and neural tissues.[3]

Doctors and researchers feel that cord blood stem cells may fire the brain’s neural activity and also go a step ahead to generate new neural connections. This is a breakthrough finding that can help Autistic children improve their motor and cognitive skills, among others. Several clinical trials and researchers point to the applications of stem cells derived from cord blood to help in eliminating immune problems in Autistic children.[4]

If stem cells derived from one’s own cord blood may help in regenerative medicine and therapy at a later stage of life, it naturally follows that parents must opt to bank their babies’ cord blood for future use.

It is World Autism Awareness Day today, April 2. As a parent, you may be able to provide the best of material comforts to your child. But consider this: if your child exhibits symptoms of Autistic disorders at a later stage, would you be able to help him with physical therapies alone? As doctors around the world look for a cure for Autism, it is time for parents to give their children a shot at a new life with the disorder – it is time to bank their cord blood for use in the future.

[1] ‘Gritty granny gives her all to help autistic grandson’, by Vinod Kumar Menon, Mid Day, April 2,2015 http://www.mid-day.com/articles/world-autism-day-gritty-granny-gives-her-all-to-help-autistic-grandson/16107195

[2] ‘He talks of aircrafts but doesn’t understand relationships’, by Kamini Lakhani, Sai Connections, January 21, 2015,

[3] ‘The potential of cord blood stem cells for use in regenerative medicine’, by David Harris, Michael Badowski, Nafees Ahmad and Mohamed Gabella, Expert Opinion on Biological Therapy, 2007

[4] Marcus, Mary Brophy, ‘Could stem cells treat autism? Newly approved study may tell’, HealthDay News, October 12, 2012

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For long now, doctors and scientists studying autism and autism spectrum disorders have debated the merits of using stem cells to cure the brain disorder. There have also been attempts in the West to study the effects of one’s own stem cells banked at birth in the generation of viable neurons in the brain.

For instance, an FDA-approved study was conducted in California, USA in 2012, where a group of autistic children (whose disorder was not hereditary or the result of blunt trauma) were infused with their own cord blood cells. The end goal was to map the specific development of these children in correcting deficits of speech, behaviour and language. According to the US Centre for Disease Control and Prevention, 1 in 88 children in the US are autistic. However, the results of the study were said to be inconclusive.

Stem cell therapy for autism is, on paper, a feasible treatment approach for a disorder that has no cure till date. Doctors opine that stem cells may not just help fire neural activity in the brain – thus helping the child improve its condition – but also help generate working neural connection. Stem cells are also being used to help find the underlying causes of the disorder. Thus far, science has been able to conclude that genetics and immunological factors may play a role in the development of autism spectrum disorders in children. The rationale for using stem cell therapy for autism is that stem cells derived from cord blood may help in eliminating immune problems in autistic children.

An extremely promising development in understanding what causes certain autism spectrum disorders is the development of induced pluripotent stem cells (iPSCs) derived from autistic persons. It transpires that iPSCs are capable of transforming from ordinary cells into neurons. The major breakthrough in this finding is that these iPSCs can be cultured in a laboratory. These iPSCs may hold the key to specific alterations that take place in the brain of a foetus, which later develop into autism spectrum disorders after age 2.

Researchers at University of California, San Diego are now examining a new frontier in stem cell therapy for autism: whether human neural connections can be built to mimic some of the interactions taking place in the human brain; the rationale driving this research stems from advances in human tissue engineering.

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