Developing new treatments for Niemann-Pick Type C disease
Development of autophagy modulators for evaluation as a therapeutic strategy for Niemann-Pick Type C
This study is looking for new medications that can help people with Niemann-Pick Type C by improving how their cells manage fats and cholesterol, which could lead to better health and a longer life for those living with this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project ID | NIH-10824441 on NIH RePORTER |
What this research studies
This research focuses on Niemann-Pick Type C (NPC), a neurodegenerative disorder caused by mutations in the NPC1 gene, leading to issues with lipid and cholesterol transport in cells. The researchers aim to develop small-molecule drugs that can modulate autophagy, a process that helps maintain cellular health, to restore lipid balance and potentially improve symptoms in NPC patients. By testing these compounds in laboratory models, they hope to identify effective therapies that could extend the lifespan and quality of life for individuals affected by this condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Niemann-Pick Type C disease, particularly children and adolescents.
Not a fit: Patients with other types of Niemann-Pick disease or those without a diagnosis of Niemann-Pick Type C may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve the health and longevity of patients with Niemann-Pick Type C disease.
How similar studies have performed: Previous research has shown promise in using autophagy modulation as a therapeutic strategy for similar neurodegenerative disorders, indicating potential for success in this approach.
Where this research is happening
Chicago, UNITED STATES
- University of Illinois at Chicago — Chicago, United States (Active)
Researchers
- Principal investigator: Cologna, Stephanie M — University of Illinois at Chicago
- Study coordinator: Cologna, Stephanie M
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.