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Could Stem Cell Therapy Help With Autism?
May 2018

Back in April of 2017, 25 autistic children participated in a study at Duke University in North Carolina. The study – the first of its kind – aimed to treat the children’s’ autism by transfusing the blood from their own umbilical cord. This blood contained rare stem cells and, after the transfusions, two-thirds of the participants showed improvements in their symptoms.

At the time, skeptics – and even the researchers who created the study – were hesitant to announce the findings as a potential treatment for the disorder. Regardless, it was certainly a much-needed medical advancement as The Center for Disease Control and Prevention estimates that 1 in 68 children suffers from a disorder on the autistic spectrum.

Earlier this month, The Marcus Center for Cellular Cures was established at Duke, where the research began. The new Marcus Center is focused on clinical trials to develop and evaluate cellular and tissue-based therapies, learning to harness the body’s own mechanisms for cellular repair and manufacturing and delivering cell tissues and biomaterials to patients in need. In particular, they’re focused on cures for MS, strokes and – of course – autism.

Geraldine Dawson, a PhD, professor of Psychiatry and Behavioral Sciences and director of the Duke Center for Autism and Brain Development was named co-associate director of the center. She noted that “There currently are no FDA-approved biomedical treatments for autism. Our goal is to develop effective treatments that can significantly improve outcomes for individuals with autism and other developmental disorders.”

Their goal is admirable and has the potential to help hundreds of thousands of people around the world.

As mentioned, 1 in 68 children in America suffers from a disorder on the autistic spectrum. Unfortunately, according to a study conducted by Spectrumnews.org, there isn’t very much reliable information regarding its prevalence in other countries. Regardless, it’s widely considered an epidemic and its consequences weigh heavily both on the children and their parents.

Those suffering with Autism Spectrum Disorder (ASD) have deficits in social skills, have trouble with speech and non-verbal communication and engage in repetitive behaviours. Often, they’ll suffer with debilitating anxiety and, according to Focusforhealth.org, 30 percent of autistic children never speak a word, 20 percent have epilepsy, and – in the most serious cases – children are so frustrated that they self-harm.

After Duke’s 2017 study, CNN reported that Gracie Gregory, a 7-year-old who participated, dramatically improved and her parents reported that the changes were monumental. Her disorder went from taking up 75 percent of her day to just 10 percent.

Duke isn’t alone in their progressive research and because of their initial study, scientists and researchers all over the world have developed their own studies and the results are promising.

At the University of Texas Health Science Center in San Antonio, three scientists carried out a study in a rodent model of autism based on ‘an urgent need for new therapeutic strategies’.

In their study which published in Nature, they sought to restore interneuron function within the GABAergic neurotransmitter system. They used a dual-reporter embryonic stem cell line to generate enriched populations of PV-positive interneurons. These interneurons were then transplanted into the medial prefrontal cortices’ of rodents.  The transplants effectively alleviated deficits in social interaction, helped in cognitive flexibility and reduced the core symptoms of autism.

Likewise, research done at the Hospital for Sick Children and the University of Toronto determined that brain stem cells – in collaboration with the environment they live in – actually build brain circuits during development.

Dr. Freda Miller, a lead in the research, said “Neural stem cells are like “parent” cells that generate their children, the neurons and glia that build brain circuits, in a precisely controlled fashion in response to signals from their environment. These signals ensure that there are enough stem cells to build the brain, to make the correct amounts of neurons and glial cells at the right time and place in the developing brain, and that some stem cells persist into adulthood where they can participate in brain repair. If we can understand what these signals are, and how stem cells respond under normal circumstances, then that information will not only allow us to understand what happens in neurodevelopmental disorders such as autism spectrum disorder but will also provide us with the information we need to activate stem cells in the mature brain to promote repair”.

Worldwide, scientists are asking big, important questions in order to better understand autism. The continued support of stem cell research has helped give these scientists the freedom to explore uncharted territories and is bringing them closer to finding effective treatments and potentially even a cure. Continue to read our blog for further updates.

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