Drug Repurposing for Fundamental Diseases 2015

To celebrate Rare Disease Day 2015, we ran our second scientific conference on 27th February at BMA House. We were joined by over 50 delegates representing patient groups, research teams, universities, hospitals, biotech companies, and drug repositioning companies.

Programme

Dr Nick Sireau - Findacure

The day was opened by Findacure’s co-founder and Chairman, Dr Nick Sireau. He explained the exciting potential drug repositioning has for fundamental diseases, sharing his personal story with the drug nitisinone. Nitisinone began its life as a weed killer, before being identified as treatment for hereditary tyrosinemia type 1. It is now being trialled for usage in alkaptonuria (AKU) patients, the rare fundamental disease affecting both of Nick’s children. If the trial is successful, this repositioned drug would be the first identified treatment for AKU.

Dr Bruce Bloom - Cures Within Reach

Nick was followed by Dr Bruce Bloom of Cures Within Reach who continued with this theme. He emphasised that with pharma only generating (on average) 20 to 30 new therapies per year, it will take hundreds of years to find treatments for the 7,000 known rare diseases. He stressed that drug repositioning has the potential to accelerate the drug development process for rare fundamental diseases, especially as repositioning has a significantly higher success rate than de novo research.

 

Prof. Michael Briggs - Newcastle University

Our second speaker, Prof. Michael Briggs of Newcastle University, delved into the science behind drug repositioning in heritable connective disorders. He highlighted the importance of identifying disease mechanisms and having disease models in place in order to properly assess the impact of a repositioned drug. A key insight from Prof Briggs was the divide that often exists between what patients want from research and what researchers assume needs to be done. He shared a story from the achondroplasia community, where there are families affected by dwarfism for generations who do not want a ‘cure’ as they do not see anything which needs to be ‘cured’.

Dr Farid Khan - Protein Technologies

Our final speaker was Dr Farid Khan of Protein Technologies who spoke on the benefits of repositioning. He stressed the speed at which small molecules can be screened for fundamental diseases, presenting three case studies (PKU, AKU, and Malaria) in which potentially effective drugs were identified in under a year. Dr Khan stressed that with 30,000 drugs approved over the past 100 years, almost all the pathways in the human body have an existing effective and safe drug. Therefore, drug repositioning could hold the key for hundreds of untreated conditions.