What side effects are associated with this type of intervention?

Common treatments for Autism, such as human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC), are still under investigation, and their side effects are not fully understood. However, general side effects of stem cell therapies can include immune reactions, infections, and potential for tumor formation. Other biologic therapies may cause mild to moderate adverse effects like fever, headache, and local site reactions. It is crucial to conduct these treatments within well-designed clinical trials to monitor and manage any potential risks.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically using human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) for Autism Spectrum Disorder (ASD). The FDA has approved medications like risperidone and aripiprazole to address irritability associated with ASD, but these do not have regenerative or anti-inflammatory properties similar to hCT-MSC. Current research is exploring the potential of stem cell therapies, including hCT-MSC, for their regenerative and anti-inflammatory benefits in ASD, but these are still in the experimental stages.

What other types of research exist?

Promising novel alternatives for treating autism in 2024 include induced pluripotent stem cells (iPSCs) and exosome-based therapies. iPSCs can be derived from a patient's own cells and have the potential to differentiate into various cell types, offering personalized treatment options. Exosome-based therapies utilize cell-derived vesicles that can modulate immune responses and promote tissue repair, showing potential in reducing inflammation and improving neurological function.

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Mesenchymal Stromal Cells

hCT-MSC for Autism Spectrum Disorder (IMPACT Trial)

Phase 2

Waitlist Available

Led By Jessica Sun, MD

Research Sponsored by Joanne Kurtzberg, MD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months, 12 months

Summary

This trial is testing if special cells from umbilical cords can help children with autism improve their social communication skills by potentially enhancing brain function. Previous studies have shown that umbilical cord blood infusions are safe and may improve social functioning and communication abilities in children with autism.

Who is the study for?

This trial is for children aged 4 to less than 12 years with Autism Spectrum Disorder (ASD), who have a normal immune cell count, no genetic conditions linked to ASD, and are stable on current psychiatric medications. They must be English-speaking and able to visit Duke University twice. Children with known genetic syndromes, metabolic disorders, significant sensory or motor impairments, or unstable medical conditions cannot participate.

What is being tested?

The study tests the effectiveness of human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) versus a placebo in improving social communication skills in children with ASD. It's a Phase II trial where participants will receive either the hCT-MSC treatment or a placebo infusion.

What are the potential side effects?

While specific side effects for hCT-MSC are not detailed here, similar treatments may cause reactions at the infusion site, fever, headaches, fatigue or allergic responses. Close monitoring during and after treatment helps manage any potential side effects.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months, 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months, 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months, 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change on the Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales

Secondary study objectives

CGI-Intervention score

Change in CGI-Severity score

Change in VABS-3 Communication Standard Score

+2 more

Other study objectives

Evidence of formation of anti-HLA antibodies

Incidence and severity of graft versus host disease

Incidence and severity of infusion reactions

+2 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MSCExperimental Treatment1 Intervention

One dose of 6x10e6 cells/kg administered intravenously.

Group II: Placebo InfusionPlacebo Group1 Intervention

Placebo infusion

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cord Tissue Mesenchymal Stromal Cells

2020

Completed Phase 2

~140

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Human umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) are being studied for their potential to improve social communication abilities in children with Autism Spectrum Disorder (ASD). The primary mechanisms of action for hCT-MSC include their regenerative and anti-inflammatory properties. These cells can modulate the immune response, reduce inflammation, and promote tissue repair and regeneration. For Autism patients, these mechanisms are significant because neuroinflammation and immune dysregulation are thought to play roles in the pathophysiology of ASD. By addressing these underlying issues, hCT-MSC therapy may help improve behavioral and cognitive functions in affected individuals.

The Unique Properties of Placental Mesenchymal Stromal Cells: A Novel Source of Therapy for Congenital and Acquired Spinal Cord Injury.Perinatal Brain Injury As a Consequence of Preterm Birth and Intrauterine Inflammation: Designing Targeted Stem Cell Therapies.Adipose tissue-derived stem cells in stroke treatment: from bench to bedside.