Fran 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”. Fran 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.
Fran 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. Fran’s research therefore spans basic science and translation.
We warmly congratulate Fran on this distinguished and well-deserved honour.
Fran commented “It’s a great honour to have been elected and I hope this will help raise awareness about the devastating diseases we work on. I am truly indebted to past and present members of my lab and our collaborators for their outstanding work over the years”.
The Department of Pharmacology now has three Fellows of The Royal Society: Peter Somogyi, Antony Galione, and now Fran.