Kidney condition ‘CG3’ treatment uncovered by Newcastle University researchers

A treatment for a rare kidney condition called ‘CG3’ has been discovered by researchers at Newcastle University. The treatment, iptacopan, provides hope for patients who have been suffering with the debilitating disease which was previously untreatable. The breakthrough signifies a major step forward in understanding C3G, with more research needed to understand how best to administer the drug. 

C3G (complement 3 glomerulopathy) is a result of the complement system becoming overactive and damaging the kidneys, specifically the glomeruli which are needed to filter the blood to remove toxins and produce urine. The complement system is part of the immune system made up of proteins responsible for killing pathogens, helping the body fight infection. However, in individuals with C3G the complement system mistakes the glomeruli as targets causing irreversible damage. This reduces their ability to filter the blood, resulting in protein loss from kidneys. Most individuals develop end-stage kidney failure and require kidney dialysis. In many patients C3G returns despite receiving kidney transplants, rendering the transplant unsuccessful. 

The treatment called iptacopan, made by Novartis, was used in a phase III trial, called APPEAR-C3G. The trial involved patients from around the world and compared iptacopan to a placebo, an inactive treatment. The treatment was shown to be effective and safe, preventing damage to the kidneys by the complement system. 

Professor of complement therapeutics at Newcastle University, David Kavanagh, was the study’s senior author. He stated: “For the first time we have a treatment that targets the underlying causes of C3G where previously there was no effective treatment”. Dr Edwin Wong, honorary senior clinical lecturer at Newcastle University and UK chief investigator for the trial added: “We are grateful to the patients who took part in this trial, and the teams whose hard work and dedication got us to this point”.

Whilst C3G is a rare condition, affecting 1 in 500,000 people in the UK, the effects on the individual and their family are detrimental. The development of this treatment is significant not only for patients with C3G but also for individuals suffering with other diseases involving dysregulation in the complement system.