Small-molecule medicines targeting myotonic dystrophy
Design, Synthesis and Efficacy of New Small Molecule Therapeutics to Impede Myotonic Dystrophy
New small-molecule medicines aim to lower the toxic RNAs that cause symptoms in people with myotonic dystrophy.
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 ID | NIH-11307593 on NIH RePORTER |
What this research studies
Researchers are designing and synthesizing modified polycyclic compounds (MPCs) that target the expanded RNAs responsible for myotonic dystrophy. They will vary the molecular core, a benzimidazole side group, and end-group chemistry to create a panel of candidate drugs. Promising compounds are screened in lab-grown cells derived from people with DM1 and DM2 to see if they restore normal RNA processing at very low concentrations. Leads will be tested further for safety and effectiveness before any clinical testing in people.
Who could benefit from this research
Good fit: People diagnosed with myotonic dystrophy type 1 or type 2 who are willing to provide samples or consider joining future clinical testing would be the ideal candidates.
Not a fit: People without myotonic dystrophy or whose symptoms arise from unrelated conditions would not be expected to benefit from this approach.
Why it matters
Potential benefit: If successful, these medicines could reduce the toxic RNAs that drive disease and improve muscle and neurological symptoms for people with myotonic dystrophy.
How similar studies have performed: Earlier small molecules like actinomycin D, microtubule inhibitors, and diamidines showed reversal of toxic RNA effects but were limited by toxicity, while preliminary data for two new MPCs show strong correction of splicing in DM1 and DM2 cell lines at nanomolar concentrations with limited toxicity.
Where this research is happening
Albany, United States
- 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 below.