Using endocannabinoids to treat Niemann-Pick diseases
Endocannabinoid-Based Treatment for the Neurologic Niemann-Pick Diseases
This study is looking at a new treatment for Niemann-Pick Type A/B and Type C diseases, which are serious brain disorders caused by genetic issues, and it aims to see if using special compounds called endocannabinoids can help lower harmful fats in the brain and improve how the brain works.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-10901849 on NIH RePORTER |
What this research studies
This research focuses on two severe neurological disorders, Niemann-Pick Type A/B and Type C diseases, which are caused by genetic defects leading to harmful accumulations of certain lipids in the body. The study explores a novel treatment approach using endocannabinoids, which are compounds that can influence various bodily functions. By inhibiting an enzyme that breaks down these compounds, the researchers aim to reduce harmful lipid levels in the brain and improve neurological function. The research involves both laboratory studies and potential clinical applications to address the urgent need for effective therapies for these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Niemann-Pick Type A/B or Type C diseases.
Not a fit: Patients with other neurological disorders not related to Niemann-Pick diseases may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new treatment option that significantly improves the quality of life and extends the lifespan of patients with Niemann-Pick diseases.
How similar studies have performed: Preliminary studies have shown promising results using similar endocannabinoid-based approaches in animal models, indicating potential for success in human applications.
Where this research is happening
New York, United States
- Icahn School of Medicine at Mount Sinai — New York, United States (Active)
Researchers
- Principal investigator: Schuchman, Edward H. — Icahn School of Medicine at Mount Sinai
- Study coordinator: Schuchman, Edward H.
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.