The Global Quest To Unravel Autism Focuses On Our Own Stem Cells

10/08/2016

Recent discoveries in genomics and stem cell research shed light on the origin of autism and open up a world of possibilities for treating the neurodevelopmental disorder, which currently has no known cure.

The father of four-year-old Sadiya Nadaf was pleased to hear his daughter utter words, identify shapes, and even interact and play with other children.

Sadiya received stem cell therapy for her autism and has experienced significant improvements in her symptoms. Her father describes just how much her behaviour has improved and shares his hope of helping other families with the treatment:

“Many doctors had told us that there was no treatment available. Many parents like us might have lost hope but this therapy can at help their children develop skills and lead an almost normal life.”

As autism and stem cell research makes progress in the study of how the condition develops, new treatments are emerging and advancing through clinical trials.

In the first-of-its-kind, the Sutter Neuroscience Institute in Sacramento in the US conducted a placebo-controlled study of stem cell therapy in 30 children with autism. The FDA-approved trial treated the children with an infusion of their own umbilical cord blood which was stored in a stem cell bank after their birth.

The trial’s principal investigator, Dr Michael Chez, reported that over the course of a year, 60 percent of the children who received the autism stem cell treatment saw moderate to significant improvements, particularly in language and social skills.

Described as a neuro-developmental disorder, autism spectrum disorder (ASD) and autism are terms for a complex group of conditions affecting brain development. They come in varying degrees, and are generally characterised by difficulties communicating, forming social relationships, and learning language and abstract concepts. With no known cure, treatment for autism currently includes a mix of therapeutic drugs and consistent rehabilitation.

Statistics of its prevalence show that in the US approximately 1 in every 68 children — or as many as 1 in every 45 children according to some reports — currently live with Autism.

“Raising a child with autism is a constant challenge for parents and especially for mothers as it is exhausting. Being a childhood disorder, autism results in speech problems, hyperactivity, aggressive behaviour and difficulty in social interaction. At present, many children in India are affected and being treated with drugs for symptomatic relief, special education, occupational speech and behavioural therapies. Now, stem cell therapy has given them a new hope.” —Dr. Nandini Gokulchandran

This is why finding a cure is the concern of numerous biomedical and stem cell labs around the world. One prominent initiative is led by the Autism Center of Excellence at UC San Diego and Simons Foundation SPARK. They have partnered up to conduct the largest ever autism research study in U.S. to explore the genetics and biology of autism.

Another collaborative project is funded by the QUEST and OHSU Foundations and conducted by investigators at Children’s Hospital of Orange County and researchers at the Department of Pediatrics and the CDRC at OHSU. They are studying autism using recent breakthroughs in stem cell therapy.

Their goal is to create a cellular model of the autistic brain. The researchers plan to use an individual’s own reprogrammed stem cells to create autistic brain cells, so they can discover how the cells express genes and how they compare to cells derived from individuals without the condition.

Prominent institutions and organisations are backing stem cells in the search for an effective autism treatment. Their work will at the very least raise the awareness of autism among the public and scientific and medical communities, and at the best offer thousands of children around the world the chance to live a more normal life.

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