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New technology for a more efficient treatment of Pompe disease and other metabolic disorders
Date:11/19/2012

ing if left untreated. One example is Pompe disease, which affects the muscles.

Treatments for these illnesses

Lysosomal storage diseases can be treated with the lacking enzymes, provided they end up in the patient's lysosomes; this is known as Enzyme Replacement Therapy (ERT). This can be accomplished by adding a specific modified sugar to the enzymes, i.e. mannose-6-phosphate, but this is not easy to do. Until now such modified enzymes could only be produced more or less successfully in mammalian cells. The VIB researchers, together with Oxyrane, have developed a novel, more efficient method that uses yeast cells instead.

A new technology for a more efficient production

The result is impressive the enzyme for the treatment of Pompe's disease is absorbed 17 times as efficiently by patient cells in vitro than the current therapeutic drug. In a Pompe disease mouse model the enzyme also worked better than the current therapeutic product. Based on these results Oxyrane will start clinical trials for Pompe disease, this means that it will be tested in humans for the first time.


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Contact: Kris Van der Beken
kris.vanderbeken@vib.be
32-473-783-435
VIB (the Flanders Institute for Biotechnology)
Source:Eurekalert

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