Finding drug targets in neurons to help people with TSC-related epilepsy, autism, and anxiety
Official title: Evaluating dendritic DJ-1 targets as a framework for identifying pharmacotherapies for TSC-related neurological disorders
Funded at North Carolina Agri & Tech St Univ · NIH-11362268 · Data updated 2026-11-12
This project looks for proteins in nerve cell dendrites that could be turned into new medicines for people with tuberous sclerosis complex (TSC) who have seizures, autism, or anxiety.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | North Carolina Agri & Tech St Univ NIH-funded |
| Lab location | 1 site (Greensboro, United States) |
| Project start | May 20, 2023 |
| Project end | April 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $139,380 |
| NIH project number | 5R16NS134542-04 |
| Project ID | NIH-11362268 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with tuberous sclerosis complex, especially those experiencing epilepsy, autism spectrum disorder features, or anxiety, are the likely eventual beneficiaries and target population for therapies arising from this research.
✕Less likely to help
People whose conditions are unrelated to TSC or to mTOR-driven synaptic dysfunction are unlikely to receive direct benefit from this specific work.
What this research studies
From my point of view, researchers are using TSC as a model to see how overactive mTOR disrupts proteins at synapses and contributes to epilepsy, autism, and anxiety. They focus on a protein called DJ-1 in dendrites to find other proteins whose low levels might be fixed by drugs. The team will use laboratory models of TSC (cell and animal models and molecular experiments) to identify candidate drug targets and test how manipulating these proteins affects synaptic function. The goal is to produce a list of promising molecular targets that medicinal chemists or drug developers could pursue next.
Why it matters
Potential benefit: If successful, this work could point to new drug targets that lead to medicines reducing seizures, autism-related behaviors, or anxiety in people with TSC.
How similar studies have gone: mTOR-blocking drugs like everolimus have improved some TSC symptoms in patients, but targeting dendritic DJ-1 to find new pharmacotherapies is a newer approach with limited prior human testing.
Where this research is happening
-
North Carolina Agri & Tech St Univ
Greensboro, United States Active
Researchers
- Principal investigator: Niere, Farr — North Carolina Agri & Tech St Univ
- Study coordinator: Niere, Farr
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.