Autism Clinical Trial

Introduction and Personal Rationale

The most meaningful and transformative experience in my life and professional career was my encounter with Gage and his mother, Renee—a moment that illuminated both the limitations and the untapped potential of cell therapy for autism spectrum disorder (ASD).

I founder the Perinatal Stem Cell Society in 2013 with the mission of advancing safe and affordable stem cell therapy to the clinic.  Renee contacted me unexpectedly, desperate for help. She had done everything "right": she saved her son Gage’s cord blood at birth, recognized early signs of autism, and secured him a coveted spot in a pioneering clinical trial at Duke University. But when she requested her son’s cord blood for the infusion, the unit was found to be contaminated with E. coli and declared unusable. Gage was disqualified.

As the founder of Auxocell Laboratories in 2008, the world’s first company to focus on the umbilical cord-derived mesenchymal stem cells (MSCs) from the Wharton’s Jelly/Cord Tissue, I was deeply immersed in the processing and application of perinatal tissues. I recognized that even if Gage’s cord blood unit had been viable, the cells used in the Duke trial—hematopoietic stem cells—were primarily intended for immune reconstitution and although there was positive data from studies utilizing cord blood to treat ASD I knew there was a better cellular therapy option. In contrast, mesenchymal stem cells from umbilical cord tissue (UC-MSCs), and particularly multilineage-differentiating stress-enduring (MUSE) cells, offer potent immunomodulatory and neuroregenerative properties that may directly target the neuroinflammatory and neural connectivity deficits underlying ASD. 

This insight led to a novel decision: with Renee’s consent, we arranged an innovative, compassionate-use therapy for Gage, providing umbilical cord-derived MSCs processed under rigorous quality standards. Given his age (three years old, near the upper threshold where cell therapy might have the greatest impact) we administered UC-MSCs to Gage. The result was extraordinary. Prior to treatment, Gage was essentially non-verbal; within weeks he spoke a phrase that every parent cherishes but that Renee feared she might never hear: “I love you so much.”

We continued to support Gage’s progress with 2 more additional treatments, and Renee ultimately became a public advocate for this therapeutic approach, sharing their journey at the Perinatal Stem Cell Society conference and founding the Facebook “Cord Tissue for Autism” community.

This personal history is not anecdote alone—it underscores a critical gap in our approach to ASD treatment. Early clinical trials with umbilical cord MSCs have consistently shown encouraging signals of safety and potential efficacy. Furthermore, the unique reparative potential of MUSE cells—a distinct subpopulation of MSCs—has yet to be rigorously evaluated in this context.

Our proposed clinical trial seeks to address this unmet need with a standardized, GMP-grade cellular therapy that combines 20 million high-quality MSCs with 5 million MUSE cells derived from umbilical cord tissue, under strict protocols ensuring purity and potency. We refer to this combination as our “Hero” vial and if you combine 2 or more of the vials it is a “SuperHero” therapeutic dose. 

By building on the foundational work of prior MSC studies, and informed by real-world compassionate use experiences like Gage’s, this trial is designed to rigorously evaluate safety of our ‘SuperHero” protocol while exploring whether this next-generation cell therapy can provide transformative benefits for children with ASD.

Why Now – Passage of Montana’s Right to Try Law (SB 535)

Prior to the passage of Montana’s new Right to Try law (SB 535) in May 2025, the only pathway to bring stem cell therapy to autism children and families required full FDA approval.  

SB 535marks a transformative moment for the development and delivery of innovative therapies for conditions such as autism spectrum disorder (ASD).  This legislation enables licensed experimental treatment centers to provide and sell investigational treatments—including mesenchymal stem cell (MSC) therapies—following the completion of a Phase I safety trial, even before full FDA approval has been obtained.

This creates a unique opportunity for our proposed clinical trial and commercialization plan. Our proprietary MSC + MUSE cell therapy product, manufactured under stringent GMP standards, is designed to address the immunological, inflammatory, and neural deficits implicated in ASD. Building on promising early data from MSC trials and real-world compassionate-use experience, this therapy may offer superior benefits over conventional MSC products alone.

The clinical trial we propose will first establish the safety and preliminary efficacy of this combined MSC + MUSE product through a Phase I/II design. Upon successful demonstration of safety in Phase I, Montana’s regulatory framework will permit us to commercialize the therapy within the state under the Right to Try law. This means we will be able to offer the treatment directly to families of children with ASD, generating revenue to support the ongoing study and a percentage of the revenue generated from families seeking access to this innovative therapy will be used to fund treatments for underserved children, creating a self-sustaining and ethical framework for innovation and equitable care.

This proposal outlines our scientific rationale, trial design, operational plan, and financial model to responsibly implement this novel approach under the regulatory landscape established by Montana’s forward-looking legislation.  We also plan to work with state and federal agencies and to make the amendments that Montana voted into law a become Federal law as well.

Veteran Focus

The Perinatal Stem Cell Society has partnered with the Sabot Foundation to provide stem cell therapy for Veterans.  We plan to offer the clinical trial participant slots to Veteran families with autistic children that qualify for the clinical trial.  After the successful completion of the trial, we plan to have aggressive pricing for Veteran and First responder families and to fund raise to support additional Veteran families. 

Who We Are - Kyle Cetrulo 

Kyle Cetrulo is a pioneering entrepreneur, advocate, and leader in the fields of regenerative medicine, perinatal stem cell science, and cellular therapy commercialization. Since 1998, he has been at the forefront of translating emerging cellular science beginning with his work in the cord blood banking field.  Today he is bringing novel therapeutic platforms to patients and advising on global policy reforms.

Kyle was the founder and former CEO of Auxocell Laboratories, Inc., est. 2008 and recognized for being the first company in the world to focus on the MSCs from umbilical cord tissue.    Kyle is currently the founder and President of StemCellutions, which has developed a GMP-Clinical Grade UC-MUSE MSC product that is set to revolutionize the regenerative medicine field. 

He also founded and serves as President of the Perinatal Stem Cell Society, a global non-profit dedicated to promoting scientific research, education, and responsible clinical translation of perinatal tissues and cells, including MSCs and multilineage-differentiating stress-enduring (MUSE) cells. Through this organization, he has convened leading scientists and clinicians from around the world and helped shape the evolving conversation around stem cell ethics, standards, and applications.  This organization has partnered with the Sabot Foundation to bring stem cell therapy to the Veterans and First Responder Communities. 

His work is deeply informed by real-world patient stories, including a formative experience assisting a family seeking an experimental therapy for a child with autism. This experience has driven his focus on autism spectrum disorder (ASD) as a target for next-generation cell therapies. In addition to his scientific and business leadership, Kyle has played a key role in legislative advocacy, helping advance Right to Try reform efforts and state sponsored legislation that now position Montana as a jurisdiction where families can access investigational treatments following a Phase I trial and expanded access to stem cell therapies in Florida, Utah, Nevada, and Washington State with many more states following suit.

Kyle is known not only for his scientific rigor but also for his dedication to patient-centered innovation and equitable access to care. His current work focuses on building scalable platforms to responsibly commercialize and deliver advanced cell therapies—including MSC + MUSE products—while ensuring that revenue supports expanded access for underserved populations. 

www.perinatalstemcells.com

Sabot Foundation

Founded by Hammond Meredith and Jon Krashna, Sabot Foundation is a 501(c)(3) Charitable Trust created with one goal in mind: to support Veterans and combat veterans and their transition to civilian life. Sabot works with local organizations, cities, foundations, attorneys, health professionals, and others to create a collaborative and cohesive structure of engagement and support for veterans and their families. The Foundation is dedicated to suicide prevention and suicide awareness as well as supporting veterans through comprehensive health and wellness programs to combat the effects of PTS, MST, and MTBI while educating the public about the impact of these conditions by improving veteran quality of life thus ending veteran suicide (which is the main objective of Sabot Foundation).

To achieve that goal, the foundation supports our Veterans by inspiring life-long connections through participation in a variety of holistic, wellness, and therapeutic programs.  Additionally, we provide opportunities to improve their quality of life through housing and family assistance leading to a sustained balanced lifestyle.

The Foundation is dedicated to supporting veterans through comprehensive health and wellness programs to combat the effects of PTS, MST, and MTBI while educating the public about the impact of these conditions. Additionally, we provide opportunities to improve their quality of life through their transition to civilian life and/or sustaining a balanced lifestyle.

Sabot Foundation supports other foundations with similar goals, as well as educational institutions whose missions falls directly in line with our mission – not only to promote awareness but to make it actionable for veterans. The Foundation also utilizes existing relationships with artists, musicians, public figures and the motor sports community in order to mix the genres together. This exclusive approach makes Sabot Foundation a leader in constructive social change with a unique angle in business, providing resources to veterans that will directly aid in their well-being.

https://sabotfoundation.org

Options for Autism

Options for Autism is a nonprofit organization founded in 2022 with the goal of helping children with autism receive adequate medical treatment and therapies. They are committed to ensuring these children and their families have access to the best treatments available so they can lead lives of independence and joy. Our work is fueled by the belief that no child should be left behind due to a lack of resources

https://www.optionsforautism.org

Emergent Wellness

Partner to help manage the data and inputs from the patients.  Data tracking software and program through Heads Up software will enable to continually improve the outcomes for the Autism patients. 

White Lotus Foundation and Endowment

The White Lotus Foundation and the Perinatal Stem Cell Society have partnered to enable the funding of the clinical trial via tax deductible donations running through the White Lotus’s philanthropic branch of the White Lotus Global Initiative.   The White Lotus Foundation and Endowment serves as a sacred steward of resources, channeling funding, wisdom, and collective will toward humanity’s most vital frontiers.

At the heart of the mission lies a clear and unwavering purpose: to support global transformation through grantmaking that nurtures breakthrough innovation, systemic healing, and planetary renewal.  https://www.whitelotusfoundation.org/

All donations are 100% tax deductible and funds will be dispersed on the proposed schedule.

MSCs and MUSE-MSCs

Scientific Background and Rationale

Autism spectrum disorder (ASD) is a complex neurodevelopmental condition characterized by impairments in social communication, restricted interests, and repetitive behaviors. Current therapies primarily focus on behavioral interventions and symptomatic management but do not address the underlying neurobiological dysfunctions, including neuroinflammation, immune dysregulation, and impaired neuronal connectivity.

Mesenchymal stem cells (MSCs) have emerged as a promising therapeutic candidate due to their immunomodulatory, anti-inflammatory, and trophic properties. Early clinical trials using MSCs alone have demonstrated encouraging signals of safety and potential efficacy, but outcomes have been variable, limited by heterogeneity in cell source, dose, and quality.

Multilineage-differentiating stress-enduring (MUSE) cells represent a distinct subpopulation of MSCs with superior regenerative potential. MUSE cells exhibit pluripotent-like capabilities, robust DNA repair mechanisms, and the ability to home specifically to damaged tissues. Critically, MUSE cells can differentiate into functional neurons and glia and integrate into host neural circuits—a key requirement for addressing the neuronal deficits hypothesized to underlie ASD.

StemCellutions’ proprietary cellular therapy combines 20 million high-quality, GMP-grade MSCs with an enriched fraction of 5 million MUSE cells, harvested and purified from umbilical cord tissue under rigorous SOPs. This formulation is designed to synergize the broad immunomodulatory and trophic effects of MSCs with the neural reparative capacity of MUSE cells, potentially offering a superior therapeutic benefit compared to conventional MSC products.  We call this vial a “Hero” dose and when you combine 2 or more vials as we recommend for this clinical trial we refer to the product as a “SuperHero” dose. 

Why this Trial is Likely to Demonstrate Benefit

✓ Enhanced neurorepair: The MUSE-enriched MSC product can differentiate directly into neuronal cells and integrate into neural circuits, potentially restoring functional connectivity in ASD.

✓ Superior homing to damaged tissues: MSCs and MUSE-MSC cells exhibit natural homing properties to sites of inflammation and injury, increasing therapeutic specificity.

✓ Immunomodulation plus regeneration: The combination of MSCs and MUSE cells addresses both immune dysfunction and neural damage, unlike MSC-only therapies that rely predominantly on paracrine signaling.

Benefits of the Proposed Trial

✓ Generate high-quality safety and efficacy data using a next-generation cell therapy platform.

✓ Establish proof-of-concept that MSC/MUSE combination therapy outperforms conventional MSC therapy.

✓ Advance the field toward a disease-modifying therapy for ASD.

Prior MSCs and Autism Clinical Trials

Across all three studies, MSC infusions were consistently safe with mild transient adverse events. Efficacy signals were observed in approximately 40-50% of treated children based on validated ASD-specific scales.  We anticipate an even great efficacy with the combination “SuperHero” treatment.

Sample Size Justification

Given prior MSC trials in ASD have included cohorts of 10–20 patients for Phase I/II trials, and assuming an effect size that warrants statistical comparison between groups, a proposed sample size:

- 15 patients in MSC-alone arm
- 15 patients in MSC+MUSE arm

For an initial Phase I/II trial (safety, tolerability, exploratory efficacy): approximately 30 children total. This size is consistent with early-stage trials and will provide sufficient data to detect clinically meaningful trends while ensuring the trial is ethical, feasible, and informative for larger confirmatory studies.

Clinical Trial Design: StemCellutions MSC + MUSE Therapy in Autism

1. Objectives

Primary Objective:
- To assess the safety and tolerability of intravenous administration of the “SuperHero” MSC + MUSE product in children with autism spectrum disorder (ASD).

Secondary Objectives:
- To evaluate preliminary efficacy of MSC + MUSE therapy versus MSC-only therapy on core symptoms of ASD, as measured by standardized clinical scales (e.g., CARS, CGI-I, VABS-3).

2. Study Design

This is a prospective, randomized, controlled, single-site, Phase I/II clinical trial.
- Total patients: 30 children with ASD (ages 4–12)
- Randomization: 1:1 (MSC-only vs MSC+MUSE)
- Open-label with blinded outcome assessment
- Duration: 12-month follow-up per patient

3. Intervention

Arm 1 (Control):
- 1 rounds of 40 million GMP-grade MSCs intravenously at Day 0.

Total Treatment - 40 Million MSCs

Arm 2 (Experimental):
- 1 rounds of 40 million MSCs + 10 million MUSE cells intravenously at Day 0.

Total Treatment - 40 Million MSCs and 10 Million MUSE

4. Inclusion/Exclusion Criteria

Inclusion Criteria:
- Children aged 4–12 years diagnosed with ASD based on DSM-5 criteria
- Moderate to severe autism severity (e.g., CARS ≥ 30)
- Stable on conventional therapies for ≥ 2 months

Exclusion Criteria:
- Active infection or significant medical condition
- Prior stem cell therapy
- Immunodeficiency or immunosuppressive therapy

5. Outcome Measures

Primary Outcome:
- Incidence and severity of treatment-emergent adverse events (TEAEs) over 12 months

Secondary Outcomes:
- Change in CARS score at 3, 6 and 12 months
- CGI-I score at 3, 6, and 12 months
- Change in VABS-3 scores at 3, 6 and 12 months
- Change in serum inflammatory biomarkers (MDC, TARC)

MeScreen Mitochondria Test 0, 3 and 6 months.

Brain Imaging Diagnostic Tool SPECT (Single Photon Emission Computed Tomography) with Brain Scans at 0, 3 and 6 months

*Add Urine, Stool, Blood Work Panels

6. Statistical Considerations

Sample Size:
- 30 patients (15 per arm) provides adequate feasibility for assessing safety and detecting preliminary efficacy signals.

Analysis:
- Safety: descriptive statistics of adverse events.
- Efficacy: between-group comparisons using ANCOVA adjusting for baseline scores.

Program Summary:

This program proposes a Phase I/II clinical trial evaluating the safety and preliminary efficacy of a next-generation, GMP-grade cellular therapy combining 40 million mesenchymal stem cells (MSCs) and 10 million multilineage-differentiating stress-enduring (MUSE) cells for children with autism spectrum disorder (ASD). The treatment will be administered in one session - Day 0 delivering a robust total cell dose designed to address both neuroinflammatory and neuroregenerative targets implicated in ASD.

Upon successful completion of the Phase I portion, Montana’s innovative Right to Try legislation (SB 535) will allow early commercialization of the therapy through licensed Experimental Treatment Centers (ETCs). The program is designed not only to generate critical clinical data but also to enable patient access and create a sustainable financial model where revenue supports expanded care for underserved populations.

Suggested Disbursement Schedule