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The Investigation of Using Cord Blood Stem Cells for Autism Treatment

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Autism spectrum disorder (ASD) and autism describe a complex group of neurodevelopmental disorders that affect social development, language skills, cognitive function and behavioural skills. Children with ASD often struggle with verbal and non-verbal communication, social interactions and play activities.

Individuals with ASD may also have also intellectual disabilities, difficulties in motor coordination and physical health issues including problems sleeping and gastrointestinal problems (Autism Speaks, 2015).

Some key facts about ASD:

  • There is no known cure for autism
  • ASD now affects 1 in 68 children and 1 in 42 boys in the United States
  • Autism is the fastest-growing developmental disorder in the United States
  • People with ASD may communicate, behave, interact and learn differently from other people
  • ASD symptoms can often be recognised before a child turns 3
  • People with autism are more likely to have certain medical conditions including allergies, asthma, epilepsy, persistent viral infections, sensory integration dysfunction, sleeping disorders, and more
  • Autism occurs in all racial, ethnic and socioeconomic groups.
  • A genetic cause can be identified in 20–25% of children on the autism spectrum, however researchers still don’t know what causes autism in many children

Scientists are gradually getting closer to understanding the causes of autism and have identified a number of gene mutations which can cause the disease. They believe that some of the gene mutations only cause autism when they occur in conjunction with specific environmental factors affecting brain development. Those environmental factors including having older parents (mother or father), maternal illness during pregnancy or having difficulties during labour that restrict oxygen flow to the baby (Autism Speaks, 2015). Researchers suspect that the immune system may also play a role in causing autism.

Using Cord Blood Stem Cells for Autism Treatment

The Sutter Neuroscience Institute in the United States is currently conducting the first FDA-regulated clinical trial about the investigation of using cord blood stem cells for autism treatment (Cordblood.com, 2015).

There is some evidence that certain children with autism have dysfunctional immune systems that may be damaging their nervous systems and causing autism. Researchers are interested in determining if fixing the immune system can help reduce or eliminate autism symptoms. Scientists are using umbilical cord blood stem cells for the trial because they have a proven ability to regulate immune system function.

This project is a placebo-controlled study that involves 30 children between the ages of two and seven. The children will receive an autologous stem cell transplant (stem cells from their own umbilical cord blood) to determine if the stem cells can modulate or repair their immune system. If the treatment is a success, researchers believe the children will see an improvement in their language skills and behaviour.

There is another FDA-approved phase I clinical trial at Duke University which involves a test group of Twenty children (age of 24-72 months) who are diagnosed with Autistic Spectrum Disorder (Viacord.com, 2015). The study will attempt to determine the safety of a single intravenous infusion of autologous umbilical cord blood stem cells. If the study can show that using cord blood stem cells for autism treatment is safe, it will give parents more confidence about using cord blood as a treatment option for autism.

The study will also determine how researchers should measure the outcomes of using cord blood stem cells for autism treatment. Details of the trial can be found on the clinical trials.gov website (Clinicaltrials.gov, 2015).

Duke University is also involved in a larger scale USD $41 million 5-year study to assess the effectiveness of stem cells for treating autism, stroke, cerebral palsy and related brain disorders. The project is being managed by Joanne Kurtzberg, MD, chief scientific and medical officer of Duke’s Robertson Cell and Translational Therapy Program, and Geraldine Dawson, PhD, director of the Duke Center for Autism and Brain Development (Psychiatry.duke.edu, 2015).

This Duke University project involves a number of clinical trials which will use umbilical cord blood stem cells to treat 390 children and adults wit autism, 90 adults with stroke and 100 children with cerebral palsy. Researchers believe the umbilical cord stem cells may help repair dysfunctional or damaged areas in the brain.

Researchers are also using stem cells to better understand the interaction between brain chemicals in people with autism (LiveScience.com, 2015). By using cells taken from people with autism, scientists can generate Induced Pluripotent Stem Cells (iPSCs), then prompt those stem cells into becoming brain cells.  Scientists can then study how brain cells from people with autism work in close detail, in a petri dish. The technique can help scientists test new drugs without using human test subjects and closely analyse the interaction of brain cells from people with autism.

Scientists remain positive about using cord blood as a treatment option for autism. If the results from the ongoing investigation of using cord blood stem cells to treat autism are positive, an FDA-approved treatment for autism may be available within the next 5-10 years.

Sources

Autism Speaks II,. (2015). Facts about Autism. Retrieved 21 October 2015, from https://www.autismspeaks.org/what-autism/facts-about-autism 

Autism Speaks,. (2015). What Is Autism?. Retrieved 21 October 2015, from https://www.autismspeaks.org/what-autism 

CDC - Facts about Autism Spectrum Disorders - NCBDDD,. (2015). Facts About ASDs. Retrieved 21 October 2015, from http://www.cdc.gov/ncbddd/autism/facts.html 

Clinicaltrials.gov,. (2015). Autologous Umbilical Cord Blood Infusion for Children With Autism Spectrum Disorder (ASD) - Full Text View - ClinicalTrials.gov. Retrieved 21 October 2015, from https://clinicaltrials.gov/ct2/show/NCT02176317 

Cordblood.com,. (2015). Autism Clinical Trial | CBR®. Retrieved 21 October 2015, from http://www.cordblood.com/stem-cell-research/cord-blood-research/autism 

LiveScience.com,. (2015). Human Stem Cells Shape a New Approach to Autism Research (Op-Ed). Retrieved 21 October 2015, from http://www.livescience.com/50361-autism-reseach-in-human-cells-is-advancing.html 

Miles, J. (2011). Autism spectrum disorders—A genetics review. Genetics In Medicine, 13(4), 278-294. http://dx.doi.org/10.1097/gim.0b013e3181ff67ba 

Nationalautismassociation.org,. (2015). Autism Fact Sheet. Retrieved 21 October 2015, from http://nationalautismassociation.org/resources/autism-fact-sheet/ 

Psychiatry.duke.edu,. (2015). $15 Million Award to Go Toward Exploring New Treatments for Autism, Other Brain Disorders | Duke Psychiatry and Behavioral Sciences. Retrieved 21 October 2015, from http://psychiatry.duke.edu/news/news-archive/marcus-foundation-grant 

Viacord.com,. (2015). Cord Blood and Autism | ViaCord. Retrieved 21 October 2015, from http://www.viacord.com/treatments-and-research/emerging-research/autism-spectrum-disorder/ 

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