Activating Pantothenate Kinase to Boost CoA for PKAN in Children
Official title: Advancing Pantothenate Kinase Activation Therapy
Funded at St. Jude Children's Research Hospital · NIH-11398174 · Data updated 2026-11-12
A patient-facing program aims to raise CoA levels in the brains of children with PKAN by using a new activator of pantothenate kinase, potentially improving movement and brain health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | September 1, 2025 |
| Project end | May 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $735,373 |
| NIH project number | 5R01NS144311-02 |
| Project ID | NIH-11398174 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children diagnosed with Pantothenate Kinase-Associated Neurodegeneration (PKAN) or related CoA metabolism disorders may be eligible.
✕Less likely to help
Children without PKAN or related CoA metabolism disorders may not benefit from this specific therapy.
What this research studies
This grant supports testing an allosteric activator called PZ-2891 that increases CoA in brain cells and crosses the blood-brain barrier. The approach builds on understanding how different PANK enzymes control CoA levels in tissues and uses a mouse model to show that boosting CoA can improve movement and survival. If successful, the work could lay the groundwork for a therapy that may help children with PKAN and related neurodegenerative features. The project conducted in a pediatric context seeks to translate promising lab findings toward a treatment that addresses a specific childhood movement disorder.
Why it matters
Potential benefit: If successful, it could lead to a brain-penetrant therapy that improves movement and daily function for children with PKAN.
How similar studies have gone: Prior work showed that PZ-2891 elevates CoA in cells and mouse brains and rescues neurodegenerative features in a PKAN mouse model, supporting a translational path.
Where this research is happening
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St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Lee, Richard E. — St. Jude Children's Research Hospital
- Study coordinator: Lee, Richard E.
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.