Drug repurposing for ALD: can gene families share therapies?
Funded at Hugo W. Moser Res Inst Kennedy Krieger · NIH-11349416 · Data updated 2026-11-12
A study looking at existing drugs that might help people with X-linked adrenoleukodystrophy (ALD) by using gene-family connections to repurpose therapies.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Hugo W. Moser Res Inst Kennedy Krieger NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | June 16, 2026 |
| Project end | May 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $445,500 |
| NIH project number | 1R21NS148703-01 |
| Project ID | NIH-11349416 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with X-linked ALD or related ABC transporter disorders who may be eligible for future clinical testing or biomarker studies.
✕Less likely to help
Patients without ALD or related transporter disorders may not benefit from this specific drug-repurposing effort.
What this research studies
This project focuses on ALD, a serious neurological condition caused by ABCD1 mutations, and explores whether medications already approved for other conditions could benefit ALD patients. Researchers will search for drugs that can influence shared parts of the ABC transporter family, aiming to find safe, ready-to-use options. The study will leverage insights from related diseases like cystic fibrosis to identify potential therapies that could stabilize cellular function and reduce damage. If a promising drug is found, it could move quickly toward clinical testing in ALD patients. The work is designed to translate laboratory findings into potential treatments for people affected by ALD.
Why it matters
Potential benefit: If successful, the project could identify existing drugs that help ALD patients manage or slow disease progression.
How similar studies have gone: Similar repurposing approaches have shown promise in rapidly advancing therapies for other transporter-related diseases, though ALD-specific results are still early.
Where this research is happening
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Hugo W. Moser Res Inst Kennedy Krieger
Baltimore, United States Active
Researchers
- Principal investigator: Nemeth Mertz, Christina Linnea — Hugo W. Moser Res Inst Kennedy Krieger
- Study coordinator: Nemeth Mertz, Christina Linnea
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.