Therapies targeting brain damage in Niemann-Pick type C
Therapeutic Targets for Niemann-Pick Type C Neurodegeneration
['FUNDING_R01'] · UNIVERSITY OF MICHIGAN AT ANN ARBOR · NIH-11124077
Testing ways to reduce harmful lipid buildup in the brains of people with Niemann-Pick type C to protect nerve cells and slow disease.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF MICHIGAN AT ANN ARBOR (nih funded) |
| Locations | 1 site (ANN ARBOR, UNITED STATES) |
| Trial ID | NIH-11124077 on ClinicalTrials.gov |
What this research studies
Researchers are studying how mutations in the NPC1 gene cause cholesterol and other lipids to build up inside brain cells and lead to neurodegeneration. They will examine how this storage harms both neurons and the oligodendrocytes that support them and will test approaches to reduce storage or correct misfolded NPC1 protein. The team uses laboratory models, cell-based systems (including human-derived cells where possible), and animal experiments to find molecular targets that drugs could act on. The goal is to identify promising targets that could be pursued in future therapies for people with NPC.
Who could benefit from this research
Good fit: People diagnosed with Niemann-Pick type C, especially those with confirmed NPC1 mutations or early neurological symptoms, would be the most relevant group for related future studies.
Not a fit: People without NPC or with unrelated neurodegenerative conditions are unlikely to receive direct benefit from this specific research in the near term.
Why it matters
Potential benefit: If successful, this work could reveal drug targets that lead to treatments slowing or preventing brain degeneration in NPC patients.
How similar studies have performed: Some preclinical and early clinical approaches (for example cholesterol-modulating agents and molecular chaperones) have shown promise, but no disease-modifying therapy for NPC is yet approved.
Where this research is happening
ANN ARBOR, UNITED STATES
- UNIVERSITY OF MICHIGAN AT ANN ARBOR — ANN ARBOR, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LIEBERMAN, ANDREW P — UNIVERSITY OF MICHIGAN AT ANN ARBOR
- Study coordinator: LIEBERMAN, ANDREW P
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: CNS Diseases, CNS disorder