Dr Fran Platt

Prof. Platt's  research focuses on glycosphingolipids (GSL) and glycosphingolipid (GSL) lysosomal storage diseases. She and her colleagues (in particular, Dr Terry Butters) have pioneered a novel approach to treat these inherited metabolic diseases that has led directly to the development of an effective drug (miglustat) for type 1 Gaucher disease. This drug is now approved worldwide. Clinical trials are in progress in neuronopathic GSL storage diseases based upon efficacy of the drug miglustat in mouse models of these currently untreatable human diseases. The first report of efficacy of miglustat therapy in a neuronopathic storage disease  (Niemann-Pick disease type C) was recently published, following a clinical trial. Her group also studies engineered mice that lack key enzymes in the GSL biosynthetic pathway to study GSL functions in vivo, including in the brain and the immune system.

Prof. Platt’s research is currently focused in five main areas 1) therapeutic strategies for treating lysosomal storage diseases involving the brain, 2) mechanisms of pathogenesis in storage diseases, 3) glycosphingolipid functions and presentation within the immune system, 4) the mechanism through which imino sugar drugs cause male infertility in mice and 5) the characterisation of a new disease resulting from defective ganglioside biosynthesis.

Prof. Platt obtained a BSc (Zoology) at Imperial College University of London and a PhD from the University of Bath (Animal Physiology). She was a post-doctoral fellow at Washington University Medical School in St Louis, USA (Immunology). Since returning to the UK in 1989 (to the Biochemistry Department, University of Oxford) she has focused on how the abnormal accumulation of glycosphingolipids results in pathology in the lysosomal storage diseases.

She was a Lister Institute Senior Research Fellow from 1996-2002 and moved to the Department of Pharmacology in April 2006.