Endocannabinoid treatment for neurologic Niemann‑Pick disease (types A/B and C)
Endocannabinoid-Based Treatment for the Neurologic Niemann-Pick Diseases
['FUNDING_R01'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11166609
Testing whether drugs that boost the body's natural endocannabinoids can reduce harmful brain lipid buildup and help people with Niemann‑Pick disease types A/B and C.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11166609 on ClinicalTrials.gov |
What this research studies
This project focuses on two fatal lysosomal storage disorders, Niemann‑Pick types A/B and C, that cause serious brain disease. Researchers are using FAAH inhibitor drugs to raise levels of the body's own endocannabinoids and have seen these drugs lower sphingomyelin buildup and improve brain outcomes in mouse models. They also found that the CB1 cannabinoid receptor becomes trapped in lysosomes in affected neurons, a finding seen in mouse tissues and in brain tissue from a patient with NPA. The team aims to move these findings toward a new endocannabinoid‑based therapy to slow neurological decline.
Who could benefit from this research
Good fit: People diagnosed with Niemann‑Pick disease type A, B, or C, especially those with neurologic symptoms, would be the intended candidates.
Not a fit: People without Niemann‑Pick disease or those with very advanced, irreversible neurological damage may not receive benefit.
Why it matters
Potential benefit: Could reduce toxic lipid buildup in the brain and slow or improve neurologic decline in people with Niemann‑Pick disease.
How similar studies have performed: Preclinical studies in mouse models showed reduced sphingomyelin and extended lifespan with FAAH inhibitors, but human clinical benefit has not yet been established.
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease