Identifying biomarkers for Niemann-Pick Type C disease
Official title: Clinically Relevant Biomarkers for Niemann-Pick Type C
Funded at University of Illinois at Chicago · NIH-11177320 · Data updated 2026-11-12
This study is looking at Niemann-Pick Type C, a genetic condition that affects the brain and has no current treatments, to find helpful markers that show how the disease progresses and how well potential treatments might work, which could lead to better care and support for families.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project start | September 15, 2023 |
| Project end | August 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $16,300 |
| NIH project number | 3U01FD008126-02S1 |
| Project ID | NIH-11177320 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Niemann-Pick Type C or those at risk due to family history.
✕Less likely to help
Patients with other lysosomal storage disorders or those without a diagnosis of Niemann-Pick Type C may not benefit from this research.
What this research studies
This research focuses on Niemann-Pick Type C (NPC), a genetic disorder that leads to progressive neurodegeneration and currently has no approved treatments. The team aims to identify and validate biomarkers that can provide insights into disease progression and therapeutic responses. By analyzing a larger cohort of biospecimens, they will develop methods to evaluate these biomarkers, which could help in stratifying patients for future clinical trials and improving parental counseling. The ultimate goal is to enhance understanding of NPC and facilitate the development of effective therapies.
Why it matters
Potential benefit: If successful, this research could lead to better diagnostic tools and targeted therapies for patients with Niemann-Pick Type C.
How similar studies have gone: Previous research has shown promise in identifying biomarkers for other lysosomal storage disorders, suggesting potential success for this approach in NPC.
Where this research is happening
-
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
Related research
Other NIH-funded projects on the same topics.
-
MSC Secretome Eye Drops to Speed Healing of Corneal Wounds
A new eye drop made from stem cell–secreted factors is being tested to help adults with non-healing corneal wounds recover vision faster.
-
UIC Center for Healthy Work
A national program led by UIC aims to improve the health and well-being of workers through collaborative research and practical solutions.
-
Coordinated Oral Health Promotion for Chicago Kids
A long-running study in Chicago is following children and their caregivers to find ways to reduce tooth decay and improve daily life for families.
-
Targeting p38 MAPK to slow ALS-related axonal damage
Aims to block a specific brain-wide signaling pathway (p38 MAPK) to reduce nerve damage in people with various genetic forms of ALS.
-
Precision nanomedicine to calm inflammation and protect bone in gum disease
A new, targeted nanomedicine approach aims to reduce gum inflammation and prevent bone loss in adults with periodontal disease.
-
Role of DPP in Dental Pulp Stem Cells and their Regenerative Potential
A project explores how a natural tooth protein helps dental pulp stem cells repair and regenerate the tooth’s inner tissue.
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.