How stem cell therapies might tackle childhood autism

Scientists are researching whether stem cells might be useful in treating childhood Autism Spectrum Disorder, or ASD. Brain scans show differences in the shape and structure of certain brain cells in children living with ASD, compared to children who are not living with ASD. Researchers are investigating whether abnormal or atypical brain cells could be replaced with normal cells, by using stem-cell treatment.

The way stem cells work is that they have the potential to generate new, healthy cells that can replace diseased or abnormal cells. Just like tiny plant stems that are genetically packed with the potential to grow into trees, vegetables, thorns or fruits, human stem cells are packed with the potential to grow into healthy body parts. Using experimental surgeries and transplant methods, stem cells taken from patients’ own skin, blood or bone marrow have effectively replaced failing cells in cases of kidney disease, orthopedic injury, arthritis, ulcers, skin burns and more. Stem cell therapies have restored vision in blind people by replacing failing retina cells with new retina cells.

Newborns’ umbilical cords contain a large number of stem cells, and umbilical cords can be preserved and then used in treatment for the child if disease develops at a later time. Akin to saving blood in a blood bank, an umbilical cord can be saved and frozen in a cord-bank, instead of being discarded. Umbilical cords’ abundance of stem cells means a person can tap into their very own saved cord to access their own stem cells, to be transplanted later in life.

Some experimental studies have shown that cord-blood cells can be safely transplanted to repair damaged brain cells. The topic under the microscope now is whether similar therapies can be achieved for ASD specifically. Research is advancing with regard to other brain disorders, so the question is, can stem cell treatments safely replace abnormal ASD cells too?

Research at Duke University Center for Autism and Brain Development included children living with ASD being treated with cord-blood injections from their own previously-banked umbilical cords, over a one-year period of time. Behavioral improvement was noted on parents’ reports of communication skills and symptoms. Improvement was also noted by clinicians rating symptom severity, degree of improvement, vocabulary, and eye‐tracking measures of attention.

Called “promising” by the researchers, these findings form the foundation for future studies that will be designed to learn more about how stem cell treatments work and how they can be more precisely controlled. Children living with ASD can have excess neurons in the prefrontal cortex area of the brain, and may also have groups of immature, undeveloped cells. The researchers’ major goal is to “harness the power of cellular therapies derived from cord blood and birthing tissues for children with autism spectrum disorder.”

Could the stem cells from cord blood generate normal tissue to replace abnormal tissue? And what kinds of innovative experimental techniques are needed to find out? What biological processes need to be demystified? Challenges abound. Most critically, what has to happen to make the process practical and affordable?

There is no one single cause of ASD. Medical science has not identified predictable origin factors. Numerous theories have spurred studies of biochemical processes, inherited genetics, exposure to chemicals and environmental factors. No one gene has been identified as causing ASD, but researchers are searching for irregular genes that children may have inherited. If there is a single genetic trigger, or a cluster of unstable genes that interfere with brain development that fuels ASD to develop, it has not so far been identified.

ASD’s possible genesis propels a current global conversation due to anti-vaccination advocates’ belief that childhood vaccination causes it, and also due to the resulting spread of contagious diseases. Unvaccinated children in significant numbers have recently contracted sometimes fatal illnesses that are preventable by vaccination.

The American Academy of Pediatrics maintains a list of 50 studies showing how and why vaccines do not cause ASD.