There hasn’t been much research done recently looking at Stem Cells for Autism. It’s not an area that’s being heavily researched by companies right now.
We’ve broken down each study in detail, but we know it’s a lot to digest.
At the start of the article, we’ve provided an initial summary of what all the research is telling us.
If you want to look at any study in particular, use the Content Table on the left to go to a particular study.
To read more about Autism treatment processes & cost globally, our article on Stem Cell Therapy for Autism will be useful.
Autism Findings
What the Research Says About Stem Cells for Autism
Looking at the latest data, research into stem cell therapy for autism is still in a very early, experimental stage. Unlike more heavily researched areas like osteoarthritis, there are only a handful of recent studies on this topic. While the studies so far suggest the procedures are generally safe for children, the evidence on whether they actually work is weak and highly conflicting.
The most important finding comes from the largest and highest-quality study to date. This trial found that stem cell therapy was no more effective than a placebo.
Some smaller, less reliable studies that did not use a placebo have reported promising improvements. However, research for autism is still really early, and not many companies are exploring this right now.
If you’re looking at Stem Cell Clinics, the biggest risk your taking is going to a clinic following poor standards. That’s why we take our vetting process so seriously. Read more about our process & why we do what we do below
Current Trials looking at Stem Cells for Autism
We’ve only found 1 current trial looking at Stem Cells for Autism.
Phase I Trial on Bone Marrow MSCs for Autism: Jiuzhitang Maker: China
You can read the full study details on ClinicalTrials.gov
This is a planned clinical trial sponsored by the Chinese company Jiuzhitang Maker Cell Technology in collaboration with Capital Medical University.
It’s designed to find out if stem cell infusions from donated bone marrow are safe for people with Autism Spectrum Disorder and to get an early look at whether the treatment might be effective.
Estimated Start Date: September 30, 2025
Expected Completion Date: August 30, 2028
Recruitment Status: Not yet recruiting
Participants
- Sample Size: 42 participants
- Ages: 6 years and older
- Diagnosis: Confirmed diagnosis of Autism Spectrum Disorder
Delivery Method
A single IV infusion
Cell Dosage
This is a dose-escalation study, which means different groups will receive different doses to find the safest amount.
The three planned single-dose levels are:
- Group 1: 0.5 million cells per kilogram of body weight
- Group 2: 1.0 million cells per kilogram of body weight
- Group 3: 2.0 million cells per kilogram of body weight
Cell Type Used
hBMMSC: This stands for human Bone Marrow-derived Mesenchymal Stem Cells
Follow-up: Participants will be monitored for 48 weeks after their infusion
Primary Outcome: The main goal is to assess safety by carefully tracking all side effects
Secondary Outcomes: Researchers will also look for early signs of effectiveness by measuring changes on multiple behavioral and social scales (ABAS, ABC, CGI, ATEC, SRS), and by checking for changes in inflammatory factors in the blood
Previous Trials looking at Stem Cells for Autism
We break down all the latest research on Stem Cells for Autism completed since 2020.
2024 Phase II Umbilical Cord MSC Stem Cell Research for Autism: Duke University: USA
You can read the full study details on ClinicalTrials.gov
This large-scale clinical trial tested whether an intravenous (IV) infusion of mesenchymal stem cells, derived from umbilical cord tissue, could improve social communication abilities in children with Autism Spectrum Disorder. The goal was to evaluate the effectiveness of the stem cell treatment compared to a placebo.
This was a Phase II, randomized, double-blind, crossover trial. This means children were randomly assigned to get either the stem cells or a placebo & neither the families nor the researchers knew which was given.
The “crossover” design also meant that every child in the study eventually received the real stem cell infusion. The work was led by researchers at Duke University in the United States.
Research Summary
The stem cell treatment did not work. It wasn’t found to be more effective than a placebo for improving the social and communication skills of children with autism.
The treatment was generally safe.
Participants
- 137 children with a confirmed diagnosis of Autism Spectrum Disorder
- Ages 4 to 11 years old
- Recruited from Duke University Medical Center in Durham, North Carolina, USA
- Patients were followed for up to 12 months
Delivery Method
- Each patient received a single intravenous (IV) infusion
Cell Type & Source
- Allogeneic human umbilical cord tissue-derived mesenchymal stromal cells
- The cells were isolated and expanded in a lab before use
Dosage
- A single dose of 6 million cells per kilogram of the child’s body weight
Safety
- No deaths occurred during the study
- Serious adverse events were very rare
- Infusion reactions occurred in 20.6% of the stem cell group vs. 2.9% of placebo group
- No product-related infections or Graft Versus Host Disease reported
Clinical Improvements
- Primary goal (VABS-3 social & communication scores) was not met
- Improvements in both groups were small and not statistically significant
- No secondary measures (quality of life, clinician ratings) showed a significant benefit
This large, high-quality study showed that this treatment of umbilical cord stem cells was no better than a placebo for treating the core symptoms of autism.
While mostly safe, the real treatment caused more minor infusion reactions.
The results of this major trial do not support using this stem cell therapy for children with autism.
2022 Phase I Umbilical Cord MSC Stem Cell Research for Toddlers with Autism: Duke University: USA
You can read the full study details on ClinicalTrials.gov.
This clinical trial tested an IV drip of stem cells from donated umbilical cords in very young children with Autism Spectrum Disorder (ASD). The goal was to primarily evaluate the safety and feasibility of this new procedure in toddlers.
This was a Phase I, open-label study. The work was led by researchers at Duke University in the USA.
Research Summary
The primary goal of the study was to check for safety, and the stem cell infusion was found to be safe and well-tolerated in toddlers, with no serious side effects reported.
The study also found that the toddlers showed promising improvements across multiple measures, including in their behavior, socialization, communication skills & language.
Participants
- 12 toddlers with a confirmed diagnosis of Autism Spectrum Disorder.
- Ages ranged from 18 to 48 months old.
- All participants were recruited from Duke University Medical Center in North Carolina.
- The children were monitored for a total of 12 months after the infusion.
Delivery Method
Each toddler received a single intravenous (IV) infusion, which is a drip into a vein.
Cell Type & Source
hCT-MSC: This stands for mesenchymal stem cells that came from the tissue of donated umbilical cords.
Dosage
A single dose of 2 million cells per kilogram of the toddler’s body weight.
Safety
- Treatment was safe and feasible for toddlers.
- No serious adverse events reported during the one-year study.
- No product-related infections or cases of Graft vs. Host Disease.
- One mild infusion reaction occurred.
Clinical Improvements
(Note: There was no placebo group, so improvements cannot be definitively attributed to the stem cells.)
- Problem Behaviors (PDDBI): Average improvement of -5.00 points (lower is better).
- Socialization & Communication (VABS-3): Average improvement of +3.83 points (higher is better).
- Clinician Rating (CGI): 8 out of 12 toddlers (67%) were “minimally improved” or “much improved” at 6 months.
- Language (CDI-2): Toddlers produced an average of 76 new words in the 6 months following infusion.
What We Don’t Know
Because this was a small, uncontrolled study, we can’t say if the observed improvements were due to stem cells, natural development, placebo effect, or other therapies.
Conclusion
This important Phase I study demonstrated that a single IV infusion of umbilical cord stem cells is safe and well-tolerated in toddlers with autism. While not designed to prove effectiveness, the children showed encouraging signs of improvement in behavior, communication, and language.
2021 Bone Marrow Stem Cell Research for Autism: Vinmec Hospital Vietnam
You can read the full study in the journal Stem Cells Translational Medicine.
This study tested a combined therapy for children with severe Autism Spectrum Disorder, two injections of the child’s own bone marrow cells plus an intensive 8-week educational program.
The main goal was to see if this combined approach was safe and if it could improve the core symptoms of autism.
This was an open-label, uncontrolled clinical trial, which means there was no placebo group & everyone involved knew the children were receiving the real treatment. The research was led by a team at Vinmec International Hospital in Hanoi, Vietnam.
Research Summary
The combined therapy of bone marrow cell injections and educational intervention led to significant improvements in autism severity, social communication, language and daily skills over an 18-month period.
The procedure was found to be safe and well-tolerated, with no serious adverse events reported in any of the 30 children.
Participants
- 30 children with a confirmed diagnosis of severe Autism Spectrum Disorder.
- Ages ranged from 3 to 7 years old.
- All children had already been receiving educational intervention for years without enough improvement.
- The children were monitored for 18 months after their first cell injection.
Delivery Method
Each child received two separate injections, six months apart.
The cells were delivered intrathecally, which is an injection into the fluid around the spinal cord.
Cell Type & Source
Autologous bone marrow mononuclear cells (BMMNCs). A mix of cell types, including stem cells, taken from the child’s own bone marrow.
Cell Prep
The BMMNCs were isolated using Ficoll gradient centrifugation on the same day as treatment.
The cells were not lab-cultured or expanded.
Dosage
High dose: Over 40 million cells per kilogram of body weight, for each of the two injections.
Safety
- Treatment was safe and well-tolerated.
- No serious adverse events reported among the 30 children.
- Minor side effects (mild fever or pain) were temporary and easily managed.
Clinical Improvements
(Note: No placebo group. All children also received intensive educational therapy.)
- Autism Severity (CARS): Median score dropped from 50 → 46.5 (lower = better).
- Daily Skills (VABS-II): Median score improved from 53.5 → 60.5 (higher = better).
- Specific Behaviors: Children with no eye contact dropped from 77% → 7%. Hyperactivity reduced by 50%.
- Brain Scans: PET-CT showed improved brain metabolism, but results were not statistically significant.
What We Don’t Know
Because the study had no control group, it’s unclear what caused the improvements — the cell therapy, the education, or both. A placebo effect is also possible.
Conclusion
The study concluded that this combined therapy is safe and may improve symptoms like communication and daily living. However, more rigorous, controlled trials are needed to determine whether the stem cells themselves provide benefit.
Reviews looking at Stem Cells for Autism
A review is a type of scientific paper where researchers summarize and analyze the results of many past studies on a topic, instead of running their own new experiment.
It’s like reading all the available evidence and then explaining what it shows overall. What’s working, what isn’t, and where the gaps are.
Unlike a clinical trial, which tests a treatment on real patients, a review pulls together findings from multiple trials to give a big-picture view.
2022 Review on Stem Cell Research for Autism: China: Gannan Medical University
You can read the full review in the journal Frontiers in Pediatrics.
A team of researchers from Gannan Medical University in China conducted the first-ever meta-analysis on this topic. They wanted to know:
Does stem cell therapy actually work for treating children with autism?
To answer this, the team analyzed 5 controlled clinical trials involving 325 children to see what the combined evidence says about the effectiveness and safety of this treatment.
Research Summary
The analysis suggests that stem cell therapy for children with autism may be safe, as there was no significant difference in side effects compared to control groups.
The results on effectiveness were mixed and contradictory. One autism-specific rating scale (CARS) showed that the treatment worked, while a more general psychiatric scale (CGI) showed it had no effect at all.
The authors strongly concluded that while the results are promising, the current evidence is insufficient and compromised by major limitations and weaknesses in the available research
The researchers searched through multiple scientific databases to find all the existing controlled clinical trials on this topic. They specifically excluded less reliable studies like case reports or trials without a control group to ensure they were only looking at higher-quality evidence.
They examined:
- Effectiveness: Measured improvement in autism symptoms using the Childhood Autism Rating Scale (CARS) and Clinical Global Impression (CGI) scale.
- Safety: Compared adverse reactions between the stem cell and control groups.
- Cell Therapies: Included studies using bone marrow, cord blood, and umbilical cord tissue stem cells.
This was the first meta-analysis of its kind, and the researchers wanted to answer several key questions:
- Is stem cell therapy effective in improving the core symptoms of autism in children?
- Is the treatment safe and well-tolerated by these children?
- Can the combined data offer evidence-based guidance for doctors and families?
The Treatment Appears to be Safe
When researchers combined safety data, they found no significant difference in side effects between stem cell and control groups.
Effectiveness Was a Mixed Bag
- Childhood Autism Rating Scale: Positive results: children who received stem cells showed significantly better scores than controls.
- Clinical Global Impression: Negative results: no difference between the two groups (62% effectiveness in stem cell group vs. 60% in control).
The authors of the review highlighted major weaknesses in the current research.
- Evidence is Insufficient: Not strong enough to make firm recommendations. Guardians should be cautious about enrolling children.
- Lack of Standardization: Varying cell types, doses, and delivery methods made results hard to compare.
- Other Major Issues: Small sample sizes, short follow-up durations, and use of non-specific rating scales limit reliability.
If you’re looking at Stem Cell Clinics abroad, the biggest risk your taking is going to a clinic following poor standards. That’s why we take our vetting process so seriously. Read more about our process & why we do what we do below
Conclusion: Stem Cell Research For Autism
Honestly, there isn’t a lot of data to make an accurate conclusion right now. Not a lot of companies are looking to treat Autism with Stem Cells which says a lot.
There’s a lot more work being done looking at Stem Cells for orthopedic & other neurological issues like Parkinson’s & Alzheimers. But, we’ve analysed all the data so far.
- Does the Research tell us Stem Cells can cure Autism?
- No. Based on the best evidence we have so far, the therapy has not been proven to cure Autism.
- The largest and highest-quality study (the Duke Phase II trial) was a placebo-controlled trial, the gold standard in medicine. It found that the stem cell infusion was no better than a placebo (a saltwater drip) for improving social and communication skills.
- While two smaller, less reliable studies showed some “promising” improvements, they did not have a placebo group, so it’s impossible to know if the stem cells were the true cause of the changes.
- Is it safe for children?
- Yes, it appears to be relatively safe in the short term.
- Across all the completed studies, there were no serious, life-threatening side effects reported.
- The most common issue was a minor infusion reaction (like a rash or feeling unwell), which happened more often in the children who received the real stem cells.
- Is one type of stem cell or delivery method better than another?
- We don’t know
- The studies used different cells (from bone marrow and umbilical cords) and different methods (an IV drip vs. a spinal injection).
- Because the results are so mixed and the studies are so different, it’s impossible to say if one method is better than another.
- What is the final takeaway?
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No, stem cells can’t “100% cure Autism. Data is really early at this stage & its unclear if Autism can even help patients wit Autism.
Common side effects of getting Stem Cells are fever and local pain/swelling at the injection site. We go into all of the side effects in more detail in our Stem Cell Therapy Side Effects Article
Yes, Stem Cells can help your knee arthritis by reducing inflammation and supporting the body’s natural healing process. There’s a few different types of Stem cell treatments for knee issues, our article on Stem Cells for Knee conditions goes into more detail.
Or if you’re interested in reading about any other condition Stem Cells can treat, check out our Areas of Treatment section. It has individual articles on each specific condition & if Stem Cells can help.
Stem Cell Prices vary from $1,320 up to $50,000 depending on your condition & what country your visiting for treatment.
To compare treatment costs, our guide on Stem Cell Costs Globally will be useful.
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