Investigating CD38 as a potential treatment target for spinal and bulbar muscular atrophy
Official title: Defining the role of CD38 and its potential as a therapeutic target in SBMA
Funded at Philadelphia College of Osteopathic Med · NIH-11020175 · Data updated 2026-11-12
This study is looking at a condition called spinal and bulbar muscular atrophy (SBMA) that mainly affects men, and it aims to find out how a specific enzyme called CD38 might help improve energy in muscle cells, which could lead to new treatments for people with this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Philadelphia College of Osteopathic Med NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | January 1, 2025 |
| Project end | December 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $438,365 |
| NIH project number | 1R01NS140084-01 |
| Project ID | NIH-11020175 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adult males diagnosed with spinal and bulbar muscular atrophy.
✕Less likely to help
Patients with other neuromuscular disorders or those not diagnosed with SBMA may not benefit from this research.
What this research studies
This research focuses on spinal and bulbar muscular atrophy (SBMA), a progressive neuromuscular disease primarily affecting men. The study aims to understand the role of CD38, an enzyme that influences energy metabolism in muscle cells, and how it may serve as a therapeutic target. Using advanced stem cell technology and CRISPR gene editing, researchers will analyze metabolic changes in muscle cells derived from SBMA patients. The goal is to identify ways to restore energy levels in affected muscles, which could lead to new treatment options.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that improve muscle function and quality of life for patients with SBMA.
How similar studies have gone: While the approach of targeting CD38 in SBMA is novel, similar strategies targeting metabolic pathways in other neuromuscular diseases have shown promise.
Where this research is happening
-
Philadelphia College of Osteopathic Med
Philadelphia, United States Active
Researchers
- Principal investigator: Montie, Heather L — Philadelphia College of Osteopathic Med
- Study coordinator: Montie, Heather L
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.