Stem Cells, Autism and New Research

By Gita Gupta

Autism affects 1 in 68 U.S. children (1.) FDA-approved treatments for autism address issues like irritability, but there are very few treatments for the core symptoms like impairments in communication and behavior. That’s probably because there isn’t enough research focus on understanding and addressing the abnormal changes seen in the body’s functioning in autism.

During Autism Action Month, we find it refreshing to see a study that targets abnormal changes in the body that happen in autism, with the goal of addressing impairments in behavior and communication.

This new study (2) from Duke University uses a child’s own cord blood, containing various types of stem cells, as a treatment for autism. The results were impressive – more than two-thirds of the children showed significant improvements in adaptive behavior, socialization and communication! At TACA, this gives us hope.

QUICK FACTS ON THE STUDY

The idea behind the study

Earlier research has shown that there are abnormalities in the functioning of the brain in autism, including neuroinflammation and aberrant neural connectivity pathways.

• Neuroinflammation is inflammation of tissue in the brain. Inflammation happens when the immune system fights against something that may turn out to be harmful, such as injury, toxins or infection.
• Aberrant neural connectivity pathways refers to abnormalities in connections between neurons. Neurons are specialized cells that are the basic working unit of the brain and connect with each other to transmit information.

Since these are known abnormalities in the brain in autism, the researchers believe that modulating the immune system and regulation of neural connectivity are logical targets for treatments for autism.

They chose the child’s own umbilical cord blood as the treatment cord blood contains cells that can alter brain connectivity in positive ways, and also suppress inflammation. We also know from earlier research that infusions of a child’s own cord blood back into his body is safe in children with brain injuries like cerebral palsy. Cord blood can be collected from the placenta, or afterbirth, shortly after delivery of the baby without any risk to the baby or mother.

What the study did

The children in the study were aged about 2 ¼ to 6 years, and all had cord blood stored in a blood bank. After thawing and processing, the child’s own cord blood was infused into a vein, while the child was under sedation. The primary goal of this study was to establish the safety of the treatment in autism but the researchers also looked at changes in behavior and communication, at 6 months and 12 months after the infusion.

What the study found

• Cord blood infusions are safe for children with autism – The researchers found that the most common negative effects seen were agitation, skin changes and common childhood infections.
• Cord blood infusions are effective in treating core symptoms of autism – They found significant improvements in socialization, communication, adaptive behavior and eye gaze tracking. Most of the behavioral changes happened within the first 6 months after the infusion, but were sustained between 6 and 12 months after the infusion. They also found that higher nonverbal IQ was associated with greater improvements in behavior

What’s next?

Everyone knew the children were receiving stem cells, because the purpose of this study was to make sure the treatment was safe. There is a second, larger study underway, and this will be a controlled, double-blind study that is intended to establish that the treatment is not only safe, but also effective. The second study is invitation-only, and will enroll 165 children, ages 2 to 7. It’s exciting that this study will not only include children who have cord blood stored in a bank, but also treat children with cord blood from donors.

Open Questions

While this treatment is promising and exciting, the Duke researchers caution that more work needs to be done to prove effectiveness.

The understanding of exact mechanisms is still emerging. Stem cell researchers at other institutions have raised questions around how cells injected intravenously reach the brain, how they target the right places in the brain and the mechanism by which they improve brain function.

In addition, there is no standardization around protocols for processing, dose, route of administration, type of cells etc.

At TACA, we have written about stem cells and autism research in the past (3,4) We know this is a promising treatment and look forward to additional research on this topic that addresses the open questions. Meantime, we at TACA recommend first and foremost that families continue to work with their doctor regarding their child’s treatment plan.

References:

1. http://www.tacanow.org/about-autism/latest-autism-statistics-2/
2. http://onlinelibrary.wiley.com/doi/10.1002/sctm.16-0474/full
3. https://tacanowblog.com/2014/07/14/update-stem-cells-and-autism/
4. https://tacanowblog.com/2012/09/05/stem-cells-and-autism/