Neutralizing a toxic lipid that damages nerves in Krabbe disease
A Novel Molecular Neutralization Strategy for Cytotoxic Sphingolipid in a Neurogenetic Disorder
This project tests whether cyclodextrins (ring-shaped sugar molecules) can capture the toxic lipid psychosine to protect the brains and nerves of children with Krabbe disease.
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 ID | NIH-11295378 on NIH RePORTER |
What this research studies
From a parent's perspective, researchers are exploring whether hydroxypropyl-beta-cyclodextrin (HPbCD) can bind and neutralize psychosine, the harmful sphingolipid that builds up in Krabbe disease. The team will use the Twitcher mouse model of Krabbe disease to give HPbCD and track effects on lifespan, nerve fiber preservation, and myelination. They will look at brain and nerve tissue under the microscope and measure biochemical markers of psychosine and nerve health. Positive preclinical results could support moving this approach toward studies that involve affected children.
Who could benefit from this research
Good fit: Ideal candidates for future patient-facing work would be infants or young children with a confirmed diagnosis of Krabbe disease (globoid-cell leukodystrophy).
Not a fit: People with neurological disorders that do not involve psychosine accumulation or unrelated conditions are unlikely to benefit from this targeted approach.
Why it matters
Potential benefit: If successful, this approach could lower psychosine toxicity in the brain and help protect myelin and nerve function in children with Krabbe disease.
How similar studies have performed: Cyclodextrins have shown benefit in other lysosomal lipid disorders (for example, Niemann-Pick type C), but using them to neutralize psychosine in Krabbe disease is a novel finding supported mainly by encouraging mouse data so far.
Where this research is happening
New York, United States
- 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
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 below.