Investigating a pathway to improve symptoms of autism spectrum disorder

Targeting the Pigment Epithelium-Derived Factor (PEDF) Signaling Pathway in Neurodevelopmental Disorders

['FUNDING_R21'] · DREXEL UNIVERSITY · NIH-11026376

This study is looking at how a specific protein called PEDF might help create new treatments for autism that improve a variety of symptoms, not just irritability, by promoting brain growth and connections, using both animal models and human cells to see how it affects social behaviors.

Quick facts

Phase['FUNDING_R21']
Study typeNih_funding
SexAll
SponsorDREXEL UNIVERSITY (nih funded)
Locations1 site (PHILADELPHIA, UNITED STATES)
Trial IDNIH-11026376 on ClinicalTrials.gov

What this research studies

This research focuses on the role of the Pigment Epithelium-Derived Factor (PEDF) signaling pathway in autism spectrum disorder (ASD). It aims to develop new medications that can address the full range of ASD symptoms, not just irritability, by enhancing neuronal growth and connectivity. The study utilizes animal models and human-derived stem cells to explore how PEDF influences neurite formation and social interaction behaviors. By understanding these mechanisms, the research seeks to identify potential therapeutic targets for improving outcomes in individuals with ASD.

Who could benefit from this research

Good fit: Ideal candidates for this research include children and adults diagnosed with autism spectrum disorder.

Not a fit: Patients without a diagnosis of autism spectrum disorder or those with unrelated neurodevelopmental disorders may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new treatments that significantly improve the quality of life for individuals with autism spectrum disorder.

How similar studies have performed: Previous research has shown promise in targeting similar pathways for neurodevelopmental disorders, indicating potential for success in this approach.

Where this research is happening

PHILADELPHIA, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Conditions: adult with autism spectrum disorder, agyria syndrome

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.