New medicines that remove the harmful androgen receptor in Kennedy's disease (SBMA)
Discovery of Novel Selective Androgen Receptor Degraders (SARDs) for the Treatment of Spinobulbar Muscular Atrophy (SBMA) or Kennedy’s Disease
This work aims to develop small molecules that break down the faulty androgen receptor protein that causes Kennedy's disease to protect nerves and muscles.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Tennessee Health Sci Ctr NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project ID | NIH-11232318 on NIH RePORTER |
What this research studies
Researchers will design and refine selective androgen receptor degraders (SARDs) that target the mutant AR protein found in SBMA. About 60 candidate small molecules will be synthesized and optimized using structure-activity studies. The compounds will be tested in cells, patient-derived stem-cell neurons, and animal models to see if they reduce toxic AR aggregates and improve nerve and muscle function. The team will also study how these compounds cause AR degradation to guide selection of the most promising leads for future clinical testing.
Who could benefit from this research
Good fit: People diagnosed with Kennedy's disease (spinobulbar muscular atrophy) — especially those with early or progressive symptoms — would be the eventual candidates for clinical trials based on this work.
Not a fit: People without the AR mutation, patients with other motor neuron disorders, or those with very advanced irreversible muscle loss are unlikely to benefit from these compounds.
Why it matters
Potential benefit: If successful, these drugs could slow or stop nerve and muscle damage in people with Kennedy's disease.
How similar studies have performed: Lowering testosterone has shown protective effects in animal and cell models, but selective AR degraders are a newer, largely untested therapeutic approach for SBMA.
Where this research is happening
Memphis, United States
- University of Tennessee Health Sci Ctr — Memphis, United States (Active)
Researchers
- Principal investigator: Narayanan, Ramesh — University of Tennessee Health Sci Ctr
- Study coordinator: Narayanan, Ramesh
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.