Oxford biochemists win award for `significant discovery'

Clinical trials are underway on a drug developed after a discovery by two Oxford biochemists, which could help treat patients with the inherited disorder Gaucher disease. People with the disease lack sufficient amounts of an enzyme which helps break down glycolipid molecules within the body. It leads to spleen and liver enlargement, haematological problems, and severe bone disease. Dr Fran Platt, Reader in Glycobiology, and Dr Terry Butters, University Research Lecturer in the Glycobiology Institute of the Department of Biochemistry, have developed a strategy called `substrate deprivation therapy' which uses a drug to block the manufacture of the glycolipid which causes Gaucher and similar diseases. Dr Platt said they made the discovery while investigating the anti-viral properties of drugs which have structures similar to sugars. `At the outset, there was no reason to believe these sugars should work on diseases like Gaucher, but they turn out to have a unique ability to do so. It was really a chance observation, but we realised the implications of what we had discovered and quickly diverted our research in this direction.' The drug N-butyldeoxynojirimycin (OGT 918) developed by Dr Platt and Dr Butters is undergoing clinical trials in Gaucher patients in the UK, Holland, and Israel which should be completed early next year. A second trial has started in the USA in Fabry disease which is in the same class of disorders as Gaucher disease. The clinical trials are being run by university spin-off company Oxford GlycoSciences. Currently, treatments for Gaucher disease are limited and for the majority of types of these disorders, there are currently no therapies at all. Dr Butters said: `The drug-based therapy we have developed should work for all types of storage disorders, and may be used to treat other diseases including Tay-Sachs, Sandhoff disease, and Niemann Pick type C, for which there are also no credible therapies.' In recognition of their `significant discovery', the Gaucher Association awarded Dr Platt and Dr Butters the Alan Gordon Memorial Award, presented in memory of a member of their association who suffered from the disease. The work in Oxford is part-funded by Oxford GlycoSciences, and childhood disease charities including Action Research and CLIMB (Children Living with Inherited Metabolic Diseases). Dr Platt is funded by the Lister Institute of Preventative Medicine. Dr Platt and Dr Butters are pictured, above left, receiving their award.