NIH-funded research R01 grant

GDF11’s role in brain development and autism-related disorders

Official title: Investigating the role of Growth differentiation factor 11 (GDF11) in neurodevelopmental disorders

Funded at University of Virginia · NIH-11432464 · Data updated 2026-11-12

A new NIH grant tests whether adjusting a growth factor called GDF11 can improve brain function and behaviors in models related to autism and other neurodevelopmental disorders.

More Neurodevelopmental Disorders research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Virginia
NIH-funded
Lab location1 site (Charlottesville, United States)
Project startSeptember 1, 2026
Project endMay 31, 2031
Funding year2026
Award (this year)$490,593
NIH project number1R01NS150458-01
Project IDNIH-11432464 on NIH RePORTER

Who could benefit from this research

Could help

Children or young people with or at risk for neurodevelopmental disorders related to MECP2 alterations or GDF11-related abnormalities, in future translational contexts.

Less likely to help

Individuals without these specific neurodevelopmental profiles may not benefit from this therapy approach.

What this research studies

This project focuses on a protein called GDF11, which helps regulate brain development and is linked to neurodevelopmental disorders like MECP2-related conditions. Using animal models and other preclinical systems, researchers will see if lowering or normalizing GDF11 levels improves behavioral symptoms after birth. They will also test an FDA-approved drug that inhibits GDF11 to see if it can reduce deficits in these models. The work aims to translate findings toward potential treatments for affected children, with a focus on measurable behavioral improvements.

Why it matters

Potential benefit: If successful, the research could lead to a new treatment approach that improves brain function and behavior for some children with neurodevelopmental disorders.

How similar studies have gone: Preclinical studies have shown that adjusting GDF11 can modify related behavioral symptoms in mouse models, supporting further exploration.

Where this research is happening

  • University of Virginia
    Charlottesville, United States
    Active

Researchers

  • Principal investigator: Bajikar, Sameer — University of Virginia
  • Study coordinator: Bajikar, Sameer

Related research

Other NIH-funded projects on the same topics.

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.
Last reviewed 2026-11-14 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.