Q&A: The benefits of stem cell therapy for autism

Photo Cred: Gustavo Fring/Pexels

By Avery St. Onge

To not consider stem cell therapy as an option for treating autism is the height of close-mindedness, according to Eric Weiss, MD.

Weiss, a plastic surgeon and regenerative medicine doctor in greater Jacksonville, Fla., began researching regenerative medicine after his youngest son, Marston, was diagnosed with autism. Ever since Marston, who’s now 27 years old, was a toddler, Weiss and his wife have been searching for new therapies to help treat his autism. While there’s no cure for the disorder, patients with autism often undergo various therapies including behavioral therapy, nutritional therapy, and pharmacological therapy, yet according to Weiss, none of these treatments address the root cause of autism.

As Weiss was researching various interventions to help Marston, regenerative therapy for autism was gaining traction and treatment piqued Weiss’s interest. Shortly after Weiss learned about the therapy, he enrolled Marston in a clinical trial where scientists tested the efficacy of using umbilical cord blood to treat children with autism spectrum disorder (ASD). According to Weiss, since undergoing stem cell therapy, he’s seen significant improvements in Marston’s symptoms of autism.

Today, Weiss uses umbilical cord stem cells to help heal his patients’ wounds, deformities, and diseases and he also treats patients with autism. We spoke to Weiss about the benefits of stem cell therapy for autism, the evidence supporting it, and what the future of the therapy may look like.

Integrative Practitioner: To start, what is autism?

Eric Weiss: Autism is a diagnosis of abnormal behavior and poor social interaction. There’s a huge spectrum. It usually involves repetitive behaviors, poor social communication, and in many cases, a lack of speech. It’s very unclear what the causes are but we do know that it’s increasing in incidence. In the 70s and 80s, one in 1,000 children in the United States were diagnosed with autism. As of 2021, the The Centers for Disease Control and Prevention (CDC) reported that the incidence of autism was one in 44 children. The same report showed that one in 116 girls, and one in 27 boys are diagnosed with autism, with boys four times as likely to have the disorder. So, the risk of autism is growing tremendously.

Because of this increase in the incidence, in 2005, the National Institutes of Health (NIH) decided to take a closer look into autism and what causes it. There was a landmark paper out of John Hopkins which demonstrated for the first time that kids with autism had inflammation in their brain. Researchers came to this conclusion through a study on autistic kids who had died from unrelated reasons like drowning, car accidents, house fires, etc. They looked at the kids’ fresh brain specimens and found neuroinflammation. They then took another group of living children and did spinal taps on them and looked at their cerebrospinal fluid (CSF fluid) and they all had inflammatory markers. So, now we’re pretty convinced that one of the hallmarks of autism is neuroinflammation. This dovetails very nicely into what we know about most other neurodegenerative disorders. If you look at Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis (ALS), chronic traumatic encephalopathy, stroke, traumatic brain injury, all those have neural inflammation, so it makes sense that autism would, too.

Integrative Practitioner: What is stem cell therapy and how does it relate to autism?

Eric Weiss: First of all, we have these things called stem cells. Stem cells are essentially cells that can divide asymmetrically. These are cells that are designed to help the body heal. Most people are confused, I believe, about the difference between adult stem cells and fetal stem cells. Fetal stem cells are designed to make a baby, and adult stem cells are designed to heal the body. Adult stem cells are made in the bone marrow, and they hang out in various places. They’re designed to repair muscle, skin, bone, cartilage or whatever ails us. The problem is that as we age, we start to lose our stem cells. Just like any other cells, stem cells time out. By the time you’re done with your teenage years, you’ve lost 90 percent of your stem cells and by the time you reach your mid-30s, you’ve lost 95 percent of them. Then, in your 70s and 80s, you’ve lost 99.5 percent of your stem cells, which is why it’s more difficult to recover from trauma, injury, and disease as you age.

We knew that we had these stem cells inside of us, but what we later found out was that one of the greatest repositories of adult stem cells is umbilical cord blood. Umbilical cord blood is just chock full of adult stem cells. This is because after about midway into the second trimester, a fetus’ fetal stem cells are gone, and they only have adult stem cells.

Back in the early 2000s when researchers were studying traumatic brain injury, they found that a secondary injury occurs after brain trauma, caused by neuroinflammation, which may be much worse than the first injury. These traumatic brain injury researchers were looking for something that could turn off neuroinflammation and that was basically when stem cells got discovered. Researchers then narrowed their search to a stem cell in umbilical cord blood called the mesenchymal stem cell (MSC), which has the ability to turn off neuroinflammation and help with neurorestoration. When these scientists figured out they found a cell that could turn off the neuroinflammation related to traumatic brain injury, it didn’t take a leap of faith to apply this research to autism and start turning off neuroinflammation in autistic patients. So, that’s what they did.

Integrative Practitioner: What evidence exists that supports stem cell therapy for autism?

Eric Weiss: There is a significant amount of evidence that supports stem cell therapy for autism. We have to remember that that transfusing somebody or taking an organ out of one person and giving it to another is nothing new. We’ve been doing at least some type of living tissue transplant in humans for over 100 years. If you look at bone marrow transplants, which are full of stem cells, we’ve been doing that for 60 years. There are about 80 different diseases where the primary treatment involves umbilical cord blood stem cells.

When the researchers found that MSC reduced neuroinflammation from traumatic brain injury, they started researching other conditions to try it on. The first thing they tried was cerebral palsy because there’s a neuroinflammatory aspect to it. They found that kids that got umbilical cord blood stem cells got better. The first United States study on stem cell therapy for autism was at Duke University in 2019. They took 25 kids with autism and gave them their own umbilical cord blood. Sixty percent of participants got better, tremendously better. It wasn’t subtle by any stretch of the imagination.

We knew from previous studies that that kids with autism have less than the normal amount of synapses or neuronal connections in their brain, especially in the areas of speech and language. Before and after a single dose of umbilical cord blood in the stem cells, that number increased tremendously, and the kids started talking. They also knew that their EEG were abnormal, and brainwaves showed less than the normal power. After a single dose of stem cell therapy, their power increased, and their EEG started to become normal.

In another experiment, researchers figured out a way where they could objectively measure how much time a kid with autism would spend looking at the face of somebody, trying to interact with them. Before and after a single dose, 60 percent of the participants got significantly better. That study came out in 2019 and jump-started several more studies that all showed 50 percent to about 70 percent of autistic kids benefited from the therapy.

Some of these studies were from Chinese literature and there were some in South American literature, but now there’s some starting to be in United States literature. However, in the United States typically research starts with a phase one study that determines if a therapy is safe. Then it moves to phase two which determines if the therapy works. At phase three researchers figure out dosage and administration. Finally, at phase four researchers decide if the therapy is better than other therapies available.

When Duke did a phase one study, just to see if stem cell therapy for autism was safe, they found all these benefits and they immediately went to a phase two study. In phase two, they incorporated 160 kids in the study and the final data from the study showed that there was no benefit of stem cell therapy for autism. So that kind of flew in the face of the science of the experimental models, and everything else. This was considered a setback, until researchers took a closer look at the raw data. When they looked at the raw data, researchers realized they had studied a group of children with severe autism. They looked at the IQ of the participants and found many of the kids had a very low IQ. They not only had autism, but also significant intellectual disability.

When researchers looked at the group of patients that had IQs of 69 and below and compared them with those with an IQ of 70 and above, the kids with an IQ of 70 and above clearly benefited from stem cell therapy and the kids that had IQs of 69 and less did not show any improvement. So, the question would be, what is it about a low IQ that doesn’t show benefit? These are all single dose experiments so the answer may be that they need higher doses, or it might be that kids with intellectual disabilities aren’t candidates for autism. Just because someone is autistic doesn’t mean they have a low IQ, but there are kids with autism with low IQs. Now more studies are being done that are taking the IQ range of participants into account.

Integrative Practitioner: For the people that do benefit from stem cell therapy, what do those benefits look like and to what extent is the therapy treating autism?

Eric Weiss: Right now, the only studies out there are [for] a single dose, and what they’ve shown is that kids who are given umbilical cord blood continue to improve on their autism rating scales. They come off the curve, and more than double are getting close to normal. They continue to benefit from the therapy up to about six months and then they plateau, but they never go back down. Kids start talking better. They start more social interaction, and they have less aberrant behavior. Those are the big three things.

Now, there are actually experimental animal models of autism, where scientists create neuroinflammation in animals and the animals begin to act like autistic children. There’s a type of mouse called the BTBR mouse that when induced with neuroinflammation, begins showing repetitive behavior, antisocial behavior. When given umbilical cord blood, they get better. Not every one of them, but most. It’s still brand-new science, but it’s looking promising that it will be beneficial to a large proportion of children with autism because it’s getting at the underlying issue of the disorder.

Integrative Practitioner: To be clear, this therapy is being done outside of clinical trials?

Eric Weiss: The long and short of it is yes. The [U.S. Food and Drug Administration] FDA said it could be given on a compassionate need basis because it’s the only known treatment for autism. The umbilical cord blood for autism is considered an IND, which stands for investigational new drug, and it must be bought from a company that is approved by the FDA.

Integrative Practitioner: Are there any dangers involved in this therapy?

Eric Weiss: I would say there are dangers in any potential therapy, but umbilical cord blood and stem cells are the primary treatment of many different diseases. It has been given millions of times all over the world and it has a very high safety threshold. Usually, the only side effects we see is a transient, low-grade fever in the first 24 hours, or a rash. Researchers usually pretreat kids with a little bit of steroid and a little bit of Benadryl and that takes care of most side effects.

One of the dangers is that this therapy is not covered by insurance, so it’s a financial burden to most, and there is a 50 percent chance that it may not benefit the child. However, most of these parents are spending tens of thousands of dollars a year on speech therapy, behavioral therapy, drugs, and really, this one-time therapy has the ability to help more than anything else we can do.

Integrative Practitioner: What does the future of stem cell therapy for autism look like?

Eric Weiss: Obviously, the therapy needs to be thoroughly researched by multiple people in multiple studies. You’re talking about one in every 44 babies born in the United States having autism. Somebody’s got to figure out what the cause is. Somebody has to figure out how to cure it. This is the first big break for treating autism, but I think that umbilical cord blood stem cells will be the biggest thing in medicine since antibiotics. Potentially, we will be able to treat stroke, heart attacks, arthritis, and autoimmune diseases because we’re starting to unlock the cells that are responsible to regrow the tissue that people are missing. Really, it’s teaching the body how to heal itself.

Editor’s note: This interview has been edited and condensed.