The progressive and incurable brain disorder currently affects approximately 100 patients worldwide
LifeArc has announced that it has funded a pivotal clinical trial at the University of Cambridge for £750,000 to re-purpose a UK-licensed medicine to treat the ultra-rare genetic disease neuroferritinopathy.
The recently launched DefINe trial aims to stop the progression of the disease with an existing drug known as Ferriprox (deferiprone), manufactured by Chiesi Global Rare Diseases.
Affecting approximately 100 patients worldwide, neuroferritinopathy is a progressive and incurable brain disorder caused by changes in a gene that produces the ferritin light chain protein.
This change can lead to a build-up of iron in the brain, which can cause severe symptoms such as loss of speech and swallowing.
With currently no effective treatments available to treat the condition, the new randomised, placebo-controlled trial aims to reduce the iron accumulation in the brain using Ferriprox, an affordable oral tablet licensed in the UK to reduce iron levels in blood conditions including thalassaemia.
If successful, Ferriprox could be used to treat other neurodegenerative conditions linked to iron build-up in the brain.
The DefINe trial will evaluate the safety and efficacy of Ferrirox in 40 patients, who will undergo state-of-the-art 7T magnetic resonance imaging scanning to monitor iron levels in the brain, to form the basis of an application for licensing in the UK under exceptional circumstances, often used for rare conditions.
As well as LifeArc’s contribution, Swiss life sciences company Lipomed has offered to provide a cost-effective generic form of Ferriprox, Deferiprone Lipomed and a placebo to the trial, totalling £250,000.
Dr Catriona Crombie, head of LifeArc’s Rare Disease Translational Challenge, said: “Drug repurposing trials like this are an increasingly effective way of taking treatments that have already been approved and applying them to new conditions and diseases.”
Patrick Chinnery, trial lead and professor of neurology, University of Cambridge, commented: “The trial [could] open the possibility of using a similar approach for other neurodegenerative conditions linked to the build-up of iron in the brain, including Parkinson’s disease.”

https://pharma.com/news/lifearc-provides-750000-fund-for-clinical-trial-to-treat-patients-with-neuroferritinopathy/