Frances Platt

Frances Platt is a biochemist and pharmacologist. She studies a group of rare genetic disorders called lysosomal storage diseases with a view to developing new drug therapies. The lysosome, a compartment inside cells, is responsible for breaking down big molecules. When the lysosome goes wrong molecules build up in the lysosome and this accumulation of material is called 'storage'. Frances focuses on a subgroup of these diseases in which fatty molecules called sphingolipids are stored. These rare diseases mainly affect infants and children, but adults can also develop these disorders. They are all progressive in nature and most affect the brain leading to neurodegeneration.

 

Frances found that a drug she was studying as an anti-viral compound could be used to treat lysosomal diseases. Her research with colleagues in Oxford led to the approval of the drug miglustat for treating Gaucher disease and Niemann-Pick disease type C. She has also developed other drugs for treating these rare diseases that are currently in clinical trials. Frances's research therefore spans basic science and translation.