Can Cord Blood Stem Cells Treat Autism?

While scientists don’t know exactly what causes autism, they have looked to cord blood stem cells to treat the symptoms because of their regenerative properties.

Here, we look at what researchers know about autism, and why there is reason for both optimism and skepticism in the latest trial research. We also offer some valuable tips on how to evaluate the clinical trials to form your own conclusions.

What Is Autism?

Autism Spectrum Disorder (autism) is a complex condition that affects 1 in 44 children in the U.S., across all racial, ethnic, and socioeconomic groups, according to the Centers for Disease Control [*]. In the last 10 years, autism rates have doubled, a fact some attribute to growing awareness of autism and changes to the condition’s diagnostic criteria [*]. Treatments vary widely [*].

Its symptoms are varied and even today, scientists are still trying to identify its nuances, which typically include social awkwardness, anxiety, repetitive behaviors, and resistance to change.

On the high-functioning end of the spectrum, an individual may have a special capability such as photographic memory or math proficiency. On the low end, there are children with intellectual disabilities and those who don’t talk at all.

Almost a third of children with autism have an intellectual disability and a larger number have issues with motor skills. Boys are 4 times more likely to be diagnosed with autism than girls. According to Johns Hopkins School of Public Health, 1 in 34 boys has autism (2.97%) while only 1 in 145 girls do (.69%). These numbers may be deceptive though, because boys and girls often display different symptoms, with girls more likely to have anxiety versus repetitive behaviors common in boys. It’s possible that some girls with autism are being misdiagnosed with anxiety or depression instead of autism.

The word autism was first used to describe the disorder in the 1920s by a Ukranian-born Jewish female psychologist named Grunya Sukhareva, writing 20 years before Hans Asperger and Leo Kanner, who until recently were credited with the discovery of autism [*].

Almost a century later, researchers still don’t know exactly what causes autism and thus have no means of preventing it, reducing its severity or its risk of occurring. This is the problem at the crux of treating autism in a targeted manner.

What Causes Autism? The Latest Research

Scientists recognize genetics has a role in autism, but the research to fully understand this may be years, or even decades, away. What scientists do know is that genetic mutations called copy-number variations, in which large chunks of an essential and specific genome get deleted or duplicated, are present in a substantial number of autistic individuals [*].

In at least 30% of autism cases, these de novo mutations (meaning they weren’t present in either parent) play out differently in boys than girls; It takes a larger hit to the gene to make a girl autistic. This led genetic scientists studying autism to think that the mothers passed a strong autism-causing variant to their offspring, but further research debunked that theory.

The opposite proved true: In families with more than one sibling affected by autism, the siblings shared more of their father’s genome [*].

Immune dysregulation is another promising area of research into the cause of autism. Researchers have determined that the mother’s immune system may be triggered during pregnancy to impair fetal development. For instance, if she suffers from a high fever during pregnancy in the second trimester, the chance of the baby developing autism increases by 40% [*].

It’s also possible that a maternal-fetal conflict could cause autism, where the mother’s immune system reacts to an antigen (protein) from the father’s genome which it then attacks in the fetus [*].

Other causes noted in recent research include synaptic pathology, hormone imbalances, and exposure to neurotoxins [*]. Researchers have also suggested autoimmune disorders among the parents increase the risk of autism in offspring [*].

A healthy pregnancy is the first line of defense against autism, but it is by no means a guarantee.

What Is Cord Blood?

Cord blood is gathered from a newborn’s umbilical cord after birth. It’s a precious resource of stem cells — the building blocks of our blood and immune systems. Once considered medical waste, the umbilical cord contains both hematopoietic stem cells (in the cord blood) and mesenchymal stem cells (in the cord tissue). These stem cells have different properties.

There are more than 80 FDA-approved cord blood stem cell treatments for diseases such as leukemia, sickle cell anemia, and other heritable blood disorders. Stem cells from cord blood offer several advantages over stem cells from bone marrow: they’re more readily available, easier to match, cord blood collection is non-invasive, carry a lower risk of infection, and present less risk of graft-versus-host-disease (GVHD).

The ability of stem cells to develop into various cell types, and the ease with which they can be expanded in culture, has led to a great deal of interest in their use in therapies targeting a wide range of diseases, including autism.

How Can Cord Blood Help Autism?

Cord blood stem cells exert a paracrine effect, meaning that these cells can stimulate the patient’s own cells to repair diseased tissue, reduce inflammation, promote the growth of blood vessels (angiogenesis) and stimulate the proliferation and migration of neural stem cells without contributing to such tissue directly [*].

Researchers don’t know exactly what combination of genetics, epigenetics, and immunological components cause autism, so the clinical trials vary based on what hypothesis the researchers proceeded from — among many other factors discussed in a moment.

During the early phase of brain development, there is a close relationship between the immune and nervous systems, leading researchers to hypothesize that there is a link between neuroinflammation, microglial activation, and immune dysregulation in autism patients [*][*].

Recent reports suggest that stem cell transplantation using MSCs may improve neurological conditions resulting from stroke, amyotrophic lateral sclerosis, Alzheimer’s disease, spinal cord injury, or Parkinson’s disease [*].

Clinical Trials for Cord Blood and Autism

Chinese Clinical Trial - 2013

This study was the first to present a safety and efficacy analysis of using allogeneic human cord blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells (UCMSCs) for autism in addition to conventional behavioral therapy.

This non-randomized, open-label, single center phase I/II trial studied 37 children (age 3-12, almost all male) who were given intravenous infusion as well as multiple injections of the cord blood preparations, which were not derived from the children in the study but rather, donated and expanded by Shenzhen Beike Cell Engineering Research Institute (SBCERI).

The study divided the participants into three groups: the Control group received only conventional behavioral therapies; the CBMNC group received CBMNCs and behavioral therapies; and the Combination group received a combination of CBMNCs and UCMSCs and behavioral therapies.

The researchers deemed the treatments safe and effective, although some participants developed low-grade fever that resolved on its own. Both the CBMNC and Combination groups saw significant behavioral improvements compared with the Control group, using the CARS and ABC assessments. The Combination group saw the most improvement overall in visual, emotional, and intellectual responses, body use, adaption to change, fear or nervousness, nonverbal communication, and activity level, as well as in lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech.

Along with the encouraging results, the authors note the limitations of this proof-of-concept study. First, the subjects were not randomized or stratified based on disease severity or another demographic variable. The small number of subjects, and the fact they were only followed for 24 weeks, may introduce bias as to the safety and efficacy measures. This was an open label trial, meaning that neither evaluators nor subjects were blinded, which may have introduced bias as to the behavioral measurements. Finally, the exact mode of action was not known or clarified in this study. In other words, they don’t know exactly how or why they got the results they did. [*]

Duke Autism Center Phase I Trial - 2017

Much has been made on the various cord blood blogs about the research from Dr. Joanne Kurtzberg and her team at the Duke Autism Center, who have been studying cord blood treatment for autism.

The Center’s open-label, Phase 1 clinical trial found some behavioral improvements in a small number of autistic children treated with a single infusion of their own cord blood, leading the authors to conclude that such treatment is “safe and feasible” [*]. But the authors note this caveat:

“While these results provide some promise for future work with cord blood-derived therapies in autism, it is important to note the limitations of this study. As an uncontrolled open-label study, it is not possible to determine whether the observed behavioral changes were due to the treatment or reflect the natural course of development during the preschool period.”

Duke Autism Center White Matter Study - 2019

In a second study in 2019 from these researchers and this same group of 25 autistic children ages 2-6, the authors sought to understand whether the improvements they observed in social function, communication abilities and reduced clinical symptoms in the first trial were associated with changes to brain structural connectivity: white matter.

White matter tracts are the synapses in the brain that transfer information. The researchers of this study hypothesized that damage to these tracts may be the cause of the abnormal behaviors associated with autism.

The authors in this study believe their results support a finding that the improvements they saw in the earlier study were associated with increased structural connectivity in brain networks supporting social, communication, and language abilities [*].

However, a 2020 study on white matter in both adults and children with autism found that the white matter tracts that are the most impacted in autism may not be (at least directly) responsible for the behavioral deficits in autism [*].

Duke Autism Center Phase II Trial - 2020

In 2020, the Duke Autism Center researchers completed a Phase II study that was double-blind, randomized, and placebo-controlled. It did not show significant benefits to the study participants.

When they removed children with intellectual disabilities from the analysis, they reported some improvements in communication, eye tracking, and EEG measurements [*].

The authors have recruited for another clinical trial.

The Trouble With Evaluating Clinical Trials for Autism

What makes these trials and others so hard to evaluate include:

• The range of therapeutic targets — e.g., immune dysregulation, synaptic pathology, etc. — because no one knows the precise cause of autism.
• The small audience for these trials — limited to mostly white children who have cord blood stored privately or publicly for autologous infusion treatments.
• Patient stratification — as seen in the Duke study that separated the children with intellectual disabilities from the group to demonstrate results for a subset.
• The method of evaluation — there seem to be several autism scales and evaluation criteria in use to evaluate behavioral changes.
• The lack of a baseline showing what might be natural improvement with advancing age in autistic children.
• The administration via IV into the bloodstream — how do these cells get into the brain? Is one infusion enough? Is it the best method? Is it the only method?

To better evaluate these studies (and make for less ambiguous future studies [*]), we need to know:

• What specific dysfunction or therapeutic target is being proposed?
• What evidence is there that this dysfunction is expressed in a particular group of autism patients?
• What is the mode of action by which the cell therapy seeks to rebalance that dysfunction?
• If the dysfunction is reversed, is there evidence it will bring about an improvement in the core symptoms of autism?
• And finally, is there any evidence that once autism manifests it is reversible [*]?

Conclusion

If you’re at a loss for what conclusion to draw here, you’re not alone. There are those who find the research done to date on cord blood for autism very promising [*] and are optimistic a cord blood stem cell treatment might improve the symptoms of autism. There are also those who greet the Duke studies to treat autism with skepticism [*].

And there are those in the scientific community who find the results ambiguous or inconclusive because of the ways the studies were conducted and the limited number of participants [*].

The biggest hurdle to effective medical treatment, not to mention prevention, is our lack of understanding as to what causes autism. Stem cell treatments targeting immune dysregulation or white matter in autism patients have shown promise, but there is no firm evidence that autism can be reversed by cord blood stem cell transplant (or any other therapy for that matter). At least, not yet.

For a disorder that is becoming ever more prevalent for reasons we don’t fully understand, there is hope that each successive trial will bring us closer to a stem cell treatment that will improve the symptoms, if not eventually find a cure.

80 Good Reasons to Save Cord Blood

With more than 80 FDA-approved treatments using cord blood stem cells for a wide range of heritable diseases and 5,200+ clinical trials initiated, there are a lot of compelling reasons to save your baby’s cord blood and cord tissue with an honest and ethical cord blood bank such as MiracleCord.

To learn more about what cord blood can do for your child and your family right now, download MiracleCord’s Info Kit.

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