Cross-disease small molecule therapy for CAG expansion spinocerebellar ataxias
Official title: Alternative Splicing and Development of Small Molecule Therapeutics in CAG Expansion Spinocerebellar Ataxias
Funded at State University of New York at Albany · NIH-11617807 · Data updated 2026-11-12
A new NIH grant is exploring whether existing and natural small molecules can lower harmful CAG RNA and proteins across several spinocerebellar ataxias to help people with these conditions.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | State University of New York at Albany NIH-funded |
| Lab location | 1 site (Albany, United States) |
| Project start | December 1, 2023 |
| Project end | November 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $63,755 |
| NIH project number | 3R01NS135254-03S1 |
| Project ID | NIH-11617807 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals with spinocerebellar ataxias (e.g., SCA1, SCA3, SCA7) who have been characterized in prior studies or who may participate in related cell-based or translational research.
✕Less likely to help
Patients with SCAs not driven by CAG expansions or those who cannot participate in cellular or translational studies may not benefit.
What this research studies
People with spinocerebellar ataxias (SCAs) face progressive problems with coordination and movement. This project builds on prior work selecting small molecules that target CAG expansions and showing they can reduce harmful RNA and protein in patient cells and animal models. The plan is to screen FDA-approved drugs and natural products to find candidates that work across multiple CAG-SCA types, using a cell-based reporter system and patient-derived cells. If successful, these molecules could move toward therapies that help several related ataxias with a single approach. The work is translational, aiming to translate molecular findings into potential treatments for patients with SCA1, SCA3, SCA7, and related disorders.
Why it matters
Potential benefit: If successful, the approach could lead to cross-disease treatments that reduce disease-causing RNAs and proteins in several SCAs.
How similar studies have gone: Previous work showed that CAG-targeting molecules reduced harmful transcripts in cell lines and models, supporting progression to broader studies.
Where this research is happening
-
State University of New York at Albany
Albany, United States Active
Researchers
- Principal investigator: Berglund, Andrew — State University of New York at Albany
- Study coordinator: Berglund, Andrew
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.