Finding New Medicines for CLN3 and CLN6 Batten Disease
Small Molecule Drug Discovery for CLN3 and CLN6 Disease
['FUNDING_R01'] · UNIVERSITY OF NEBRASKA MEDICAL CENTER · NIH-11144477
This research looks for new small molecule medicines to help children with CLN3 and CLN6 types of Batten disease.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF NEBRASKA MEDICAL CENTER (nih funded) |
| Locations | 1 site (OMAHA, UNITED STATES) |
| Trial ID | NIH-11144477 on ClinicalTrials.gov |
What this research studies
Batten disease, specifically CLN3 and CLN6 types, are severe conditions affecting children, causing vision loss, seizures, and cognitive decline, with no current cure. This project aims to discover new drug candidates that can modify the disease's progression. We are focusing on correcting cellular problems like dysfunctional waste removal (autophagy) and cell death (apoptosis) that are common in these diseases. By targeting these issues, we hope to find a treatment that can slow or stop the devastating effects of Batten disease.
Who could benefit from this research
Good fit: This research is focused on understanding and treating CLN3 and CLN6 Batten disease, which primarily affects children, often between 0-11 years old.
Not a fit: Patients without CLN3 or CLN6 Batten disease would not directly benefit from this specific drug discovery effort.
Why it matters
Potential benefit: If successful, this research could lead to the first effective treatments for CLN3 and CLN6 Batten disease, potentially improving the lives of affected children.
How similar studies have performed: Currently, there is no known cure or effective treatment for CLN3 or CLN6 disease, and no single small molecule has been found to synergistically modulate the key cellular processes targeted here.
Where this research is happening
OMAHA, UNITED STATES
- UNIVERSITY OF NEBRASKA MEDICAL CENTER — OMAHA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: TRIPPIER, PAUL — UNIVERSITY OF NEBRASKA MEDICAL CENTER
- Study coordinator: TRIPPIER, PAUL
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.
Conditions: Batten Disease