High Throughput Screening (HTS) - New Technology Analyzes Treatment Compounds Fast

Research TeamUpdates

High Throughput Screening is used to develop medications. It allows a researcher to quickly conduct millions of chemical, genetic or pharmacological tests. Through this process active compounds, antibodies or genes that affect a particular biomolecular pathway can be rapidly identified. The results of these experiments provide starting points for drug design and for understanding the role of a particular biochemical process in the human body. Tens of thousands of compounds are being tested and will be shared with other centers that are testing an additional 20,000 compounds.

Two drug strategies are currently being studied: lower polyglucosans by lowering glycogen synthase or lower polyglucosans by increasing glycogen branching enzyme. Polyglucosans are an abnormal form of glycogen that cannot be broken down and used for energy in the body. These abnormal glycogen molecules accumulate within cells and cause damage.

1) The enzyme glycogen synthase is involved in converting glucose into glycogen. Finding a small molecule medication that safely slows down the enzyme glycogen synthase will provide a treatment for APBD. By slowing further build up of glycogen it is hoped that the body will then be able to clear polyglucosans that have already formed. Thus, stopping the disease would not only be a treatment, but could ultimately be a cure.

2) Drugs are being tested that will lower polyglucosan bodies by increasing glycogen branching enzyme (GBE) activity. GBE is an enzyme that plays a part in converting glucose to glycogen. Small molecules that bind to and stabilize the mutated GBE will be identified in order to correct the deficiency that results in APBD. A “library” of chemical compounds that are already being used to treat other disorders will be screened to find those that stabilize GBE. This should result in decreased levels of polyglucosans, representing a new treatment approach for APBD and a cure.


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