A New Drug on Trial for Gaucher's Disease

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In March 1998 a clinical trial started with a drug named OGT 918. The trial involves centres in the UK, Europe and Israel and will examine the effects of the drug on a number of Gaucher patients over a 12 month dosing period .

OGT 918 is formulated as a capsule to be taken orally three times a day. It is an inhibitor of one of the key enzymes responsible for the formation of glycosphingolipids such as glucocerebroside, the fatty material which accumulates in patients with Gaucher's disease.

OGT 918 therapy aims to deplete the initial amount of glycolipid made, allowing residual amount of enzyme to breakdown existing stores of lipids in affected cells.

In June 1998 OGT 918 was granted orphan drug designation by the US Food & Drugs Administration Office of Orphan Product Development for the treatment of Gaucher's disease and Fabryµs disease (another lysosomal storage disease).

Prof Timothy Cox, who is carrying out part of the initial trial at Addenbrooke's Hospital in Cambridge, says: 'This research is of special importance and the results will be eagerly awaited by patients and doctors alike. The idea for this came from Dr Fran Platt and Dr Terry Butters in Oxford and was developed in collaboration with them over the past five years.

Bringing together Gauchers researchers and these members of Prof Dwek's group, and now a new pharmaceutical company (Oxford GlycoSciences), is a powerful source for development. We are excited about this new approach to treatment.'

Prof Cox will speak about the trial at the Gauchers Association Conference in Manchester, UK in April 1999.

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Recent report on OGT 918 (July 1999).

Earlier article on this drug

Source: Gauchers News January 1999

© Copyright Gauchers Association 1999