A New and Improved Testing Option for Evaluating Potential Drugs for Sanfilippo
In the fight to cure Sanfilippo syndrome, a key basic requirement is to be able to measure critical substances and to do so as accurately, easily, and inexpensively as possible.
The study by Dr. Joshua Atienza and colleagues (University of Toronto-Scarborough), which received grant support from Cure Sanfilippo Foundation and Sanfilippo Children’s Foundation, reported this April a new fluorometric-coupled enzyme assay testing method for NDST1 [ N-deacetylase/N-sulfotransferase], one of the key enzymes in the synthesis of heparan sulfate whose build up and failure to be cleared from the body is the cause of Sanfilippo.
Previous testing methods have required radioactive assays using tritium (H3) or sulfur 35 (S35) which though accurate and valuable are costly and associated with radioactive waste issues.
The authors described in detail a new dynamic fluorometric coupled assay that assesses the sulfotransferase activity of mNDST1 in the Glycobiology article.
Specifically, Dr. Atienza et al. feel, “Our data demonstrate the capability of the mNDST1-coupled enzyme assay to characterize and validate the inhibitory effects of candidate drugs.” The test was created to help measure the use and effectiveness of new drugs for Sanfilippo which would block or inhibit the formation of heparan sulfate thus reducing build up and tissue damage. This method has just been developed and has not yet been used on patient samples.
The article concluded, “We expect our mNDST1-coupled assay to be instrumental in the identification of drugs that modulate the activity of NDST1 as the gatekeeper of heparan sulfate synthesis. Such drugs may represent potential treatments for patients with mucopolysaccharidoses such as Sanfilippo syndrome.”
Dr. Cara O’Neill, Chief Science Officer of Cure Sanfilippo Foundation, noted that, “Basic science research such as this creates the tools needed to develop treatments for the children. Cure Sanfilippo Foundation is honored to support this research and congratulates the team on their novel work.”
Read the complete Glycobiology article about the new enzyme assay testing for Sanfilippo.