Identifying New Drugs that Inhibit Acid Sphingomyelinase
Author Information
Author(s): Kornhuber Johannes, Muehlbacher Markus, Trapp Stefan, Pechmann Stefanie, Friedl Astrid, Reichel Martin, Mühle Christiane, Terfloth Lothar, Groemer Teja W., Spitzer Gudrun M., Liedl Klaus R., Gulbins Erich, Tripal Philipp
Primary Institution: Department of Psychiatry and Psychotherapy, University of Erlangen, Erlangen, Germany
Hypothesis
FIASMAs have shared structural and physicochemical properties allowing high lysosomal drug concentrations.
Conclusion
The study identified 27 novel functional inhibitors of acid sphingomyelinase (FIASMAs) that could be used to treat various diseases.
Supporting Evidence
- FIASMAs were found to have distinct physicochemical properties.
- Functional inhibition of ASM was associated with good permeability across the blood-brain barrier.
- FIASMAs were identified among various therapeutic drug classes.
Takeaway
The researchers found new drugs that can help stop a harmful enzyme in the body, which could help treat diseases like Alzheimer's.
Methodology
The study used a combination of experimental testing and machine learning to identify and classify compounds based on their ability to inhibit acid sphingomyelinase.
Potential Biases
Selection bias may have influenced the bimodal distribution of functional inhibition.
Limitations
The model is valid only for small drug-like molecules and does not account for in vivo therapeutic conditions.
Statistical Information
P-Value
p<0.001
Statistical Significance
p<0.001
Digital Object Identifier (DOI)
Want to read the original?
Access the complete publication on the publisher's website