Synopsis:

In today’s Grand Round Dr Tom Forbes will discuss primary hyperoxaluria (PH), an ultra-rare and often devastating kidney-stone forming disease which can lead to recurrent kidney stones and end-stage kidney disease. Curative treatment for PH in its most severe form has traditionally required combined kidney and liver transplantation, however a novel class of therapeutics called “RNA interference” look set to improve outcomes substantially. Tom will present an accessible introduction to RNA interference and the range of diseases currently treated with these agents, which are well positioned to be applied to a range of genetic diseases in future.

Speaker:

Dr Tom Forbes is a Paediatric Nephrologist at The Royal Children’s Hospital and a Clinician Scientist Fellow at Murdoch Children’s Research Institute. Tom trained in paediatric nephrology at Birmingham Children’s Hospital and Nottingham University Hospital Queen’s Medical Centre in the UK before completing his fellowship at the Royal Children’s Hospital in 2015. In 2019, Tom finished a PhD with Melissa Little’s Kidney Regeneration laboratory looking at stem-cell derived kidney organoids as functional genomic disease models. He is a co-lead for the MCRI Kidney Flagship and sits on the Steering Committees for the KidGen Renal Genetics Consortium and MRFF funded National Genomic Health Program. Tom’s research interests include kidney genomics, rare genetic kidney disease, antenatal counselling, and neonatal nephrology. Tom is also site principal investigator for two industry sponsored clinical trials examining a new class of therapeutics “RNA interference” to be discussed today.