Autism Clinical Trial

1 in 68 children have autism spectrum disorder (ASD). 93; 
				ASD is about 4.5 times more common among boys (1 in 42) than among girls (1 in 189). 93ASD occurs in all racial, ethnic, and socioeconomic groups. 93; 
				 Age range when most children are diagnosed with ASD. 93


What is autism?

Autism spectrum disorder (ASD) is a complex disorder with a wide range of signs and symptoms. No two children with ASD are exactly alike, however, they may share commonalities such as difficulties with language and social interactions.

While the cause of ASD remains unknown, some researchers believe that it may stem from differences within the immune system, at least in some cases. Previous studies have documented that some children with ASD have certain markers in their blood work that suggest an elevated immune response. 94, 95

Scientists are hoping to find new treatments that "calm" this overactive immune response to help protect the developing brain in young children with autism.

The potential role of newborn stem cells

Cord blood stem cells may have unique advantages:

  • Anti-inflammatory properties: Early research has shown that cells found in cord blood have the ability to regulate inflammation and migrate to the site of neurologic injury. 96, 97
  • Bystander effect: The working theory is that the infused cells secrete factors that promote the body's own repair mechanism in the surrounding tissue(s) via paracrine signaling, or what is commonly referred to as the bystander effect. 98
  • Safe and accessible: Cord blood can be easily collected at the time of birth and stored for potential future use by the newborn donor or close family member.

Phase II clinical trial

Led by Dr. Michael Chez, Director of Pediatric Neurology at Sutter Medical Center, researchers recently published the results of a phase II clinical trial investigating the safety and efficacy of autologous cord blood stem cells for children with ASD that employed a double-blind, placebo-controlled design. 99 Just a few months prior, researchers at Duke University published a phase I safety trial evaluating autologous cord blood in children with ASD. 100 The interest in cord blood as a potential cellular therapy, combined with the positive trends from these early stage research studies, speaks to the momentum of newborn stem cell science. As part of CBR's commitment to advancing the science of newborn stem cells, CBR has provided all institutional funding for the Sutter clinical trial.

Key Terms

Clinical trial: The process of determining the safety and efficacy of a treatment or procedure
Autologous: When the patient’s own cells are used in a transplant or infusion
Double-blind: Participants are randomly assigned to receive the treatment or a placebo, and neither participant or researcher know which test group they belong to until the study is complete
Placebo-controlled: The effects of the drug or treatment being studied are compared against the effects of an inactive or inert substance, like saline
Phase I & phase II: Early stages of clinical research looking at safety and efficacy
Cellular therapy: The use of a biological product of cells or cell parts to replace or repair damaged tissue

Study highlights 99

  • Participants: The study enrolled 30 children with ASD who were between the ages of two and seven, and had their own newborn stem cells banked with CBR.
  • Study Design: The children were randomly assigned to receive either cord blood or a placebo (saline) IV-infusion and were then followed for 24 weeks and evaluated for any changes in symptoms and behavior. At this point, the children were given the opposite infusion and monitored for another 24 weeks and underwent the same evaluation and assessments. Neither the children, their families, nor the researchers knew if they were receiving the cord blood infusion first or the placebo. Children were also evaluated for symptoms of autism at the first study visit before any infusions were administered.
  • Outcomes: This study provides further evidence that autologous cord blood infusions are safe, feasible, and well tolerated in children with ASD. While some participants demonstrated individual gains, these improvements were not characteristic of all participants. There was an overall trend toward improvement related to socialization, but this did not reach statistical significance.

CBR provided funding and supported Dr. Chez's study as part of our
commitment to advance newborn stem cell science.


What's next for autism research?

Researchers believe that this is a worthwhile line of scientific inquiry, and larger studies are needed to establish evidence regarding efficacy of cord blood in children with ASD and whether some children may be more likely to respond to this type of treatment than others.

  • Duke University has a phase II, double-blind clinical trial underway that will evaluate efficacy for autologous and matched, unrelated cord blood infusions for a larger number of children with ASD. The study is fully recruited, and the trial is currently underway. 101
  • Duke University has a phase I, open-label study underway that will investigate safety for matched, unrelated donor umbilical cord tissue-derived mesenchymal stem cells in children with ASD. The study is recruiting by invitation only, and the trial is currently underway. 102

CBR keeps you connected

CBR continues to keep our clients informed and connected. We encourage our clients to participate in the Family Health Registry, where we identify conditions common among CBR families, such as ASD. This helps us to learn more about the conditions and connect individuals who might be candidates to the researchers studying new potential applications.

  • By preserving cord blood stem cells now, families may have the opportunity to use the cells in the future.
  • In the Sutter phase II clinical trial, the 30 children who participated are all CBR clients whose cord blood had been preserved at the time of birth and who met the specific study criteria.
  • CBR is continuing to connect current clients to novel research, including clinical trials for conditions that have no cure today.
  • CBR invests in the future of families - we were the sole institution that provided funding for the Sutter phase II clinical trial.