Using AI and patient-derived lab-grown cells to find treatments for myotonic dystrophy
Official title: AI/ML and iPSC-Derived Organoids for Myotonic Dystrophy Drug Discovery
Funded at Greenstone Biosciences, INC. · NIH-11098495 · Data updated 2026-11-12
This project combines artificial intelligence with patient-derived stem-cell models to speed discovery of safer, more effective medicines for adults with myotonic dystrophy type 1.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Greenstone Biosciences, INC. NIH-funded |
| Lab location | 1 site (Palo Alto, United States) |
| Project start | June 1, 2024 |
| Project end | May 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $192,500 |
| NIH project number | 5R21TR004938-02 |
| Project ID | NIH-11098495 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with myotonic dystrophy type 1, especially those willing to donate a small blood or skin sample for patient-derived cell models or join a related biobank, are the ideal participants for contributing to this work.
✕Less likely to help
People without DM1, children, or patients seeking an immediate therapeutic benefit should not expect direct clinical benefit from this lab-based research.
What this research studies
From a patient's perspective, researchers grow cells made from people with myotonic dystrophy in the lab to make small organ-like tissues that mimic the disease. They use AI and machine learning to screen many chemical compounds on those patient-derived models to predict which ones might work best and be safest. The goal is a “clinical trial in a dish” approach that can prioritize the most promising drug candidates and reduce the need for large early human trials. This work is preclinical but aims to make it faster and less risky for new DM1 treatments to reach patients.
Why it matters
Potential benefit: If successful, this approach could speed up discovery of treatments for DM1 and help bring safer drug candidates to human trials sooner.
How similar studies have gone: Similar iPSC-based 'trial-in-a-dish' and AI-driven drug-discovery approaches have shown promising laboratory results in other diseases, but they remain largely preclinical and have not yet produced widely available DM1 treatments.
Where this research is happening
-
Greenstone Biosciences, INC.
Palo Alto, United States Active
Researchers
- Principal investigator: Pang, Paul — Greenstone Biosciences, INC.
- Study coordinator: Pang, Paul
Related research
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Biological factors that drive variability in myotonic dystrophy
This grant explores why myotonic dystrophy can look very different from person to person by studying its genetic changes and how they affect muscle in adults.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.