A new method to neutralize harmful molecules in a genetic brain disorder
Official title: A Novel Molecular Neutralization Strategy for Cytotoxic Sphingolipid in a Neurogenetic Disorder
Funded at Columbia University Health Sciences · NIH-11051851 · Data updated 2026-11-12
This study is exploring a new way to help children with Krabbe disease by using a special substance to help their bodies better absorb treatments that protect their nervous system from harmful toxins.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | March 8, 2024 |
| Project end | February 28, 2029 |
| Funding year | 2025 |
| Award (this year) | $539,304 |
| NIH project number | 5R01NS137638-02 |
| Project ID | NIH-11051851 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with globoid-cell leukodystrophy (Krabbe disease).
✕Less likely to help
Patients with other neurological disorders or those over the age of 11 may not receive benefits from this research.
What this research studies
This research investigates a novel approach using cyclodextrins to neutralize cytotoxic sphingolipids in children with globoid-cell leukodystrophy (GLD), also known as Krabbe disease. The study aims to enhance the absorption of therapeutic agents across the blood-brain barrier, which is crucial for treating this condition. By utilizing hydroxypropyl-beta-cyclodextrin, the researchers observed improved survival and preservation of nerve cells in animal models. This innovative strategy could potentially lead to new treatments that protect the nervous system from damage caused by toxic substances.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option that improves the quality of life and lifespan for children affected by Krabbe disease.
How similar studies have gone: Previous studies have shown promise in using cyclodextrins for drug delivery across the blood-brain barrier, indicating potential success for this novel approach.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Maegawa, Gustavo Henrique Boff — Columbia University Health Sciences
- Study coordinator: Maegawa, Gustavo Henrique Boff
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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.