Small Molecule InteractionsSmall molecules are a useful tool in research because they affect how proteins work and what biological processes they regulate. Their effects are very similar to the effects caused by random mutations in genes. Therefore, these interactions can be beneficial in developing treatments for genetic mutations [1].
Small Molecules and SMPD1 Through a search on the NCBI database, no small molecules were identified that interact with SMPD1. Interactions with the main protein domains were not found either. The next step used was to check for interactions of small molecules with acid spingomyelinase. This is the enzyme produced from SMPD1, so these small molecules could play a role in its regulation. The search found an inhibitor of acid sphingomyelinase. The picture on the right is the chemical structure of the inhibitor GW4869 [2]. |
Analysis and Discussion
These findings could be useful in developing a treatment for Niemann-Pick type B. GW4869 inhibits acid sphingomyelinase. This inhibition is similar to the effects caused from mutations in SMPD1. Therefore by studying this inhibitor, more can be identifed about how acid sphingomyelinase is inhibited in Niemann-Pick patients. Development of an activator of acid sphingomyelinase could reverse the effects of the mutations. This would be something to look into as a potential treatment method for Niemann-Pick disease.