Drug Repurposing for Rare Diseases 2017
Dr Rick Thompson - Head of Research, Findacure
Findacure’s Head of Research, Dr Rick Thompson, chaired the conference and gave the opening talk. He described Findacure’s work proving that our Rare Disease Drug Repurposing Social Impact Bond can work as a new route to fund generic drug repurposing. Rick also announced the launch of Findacure’s Rare Repurposing Open Call, a project together with Healx and Cures Within Reach which seeks new ideas for drug repurposing projects that are struggling to reach the clinic.
Professor Tim Barrett - Director, NIHR Clinical Research Facility, University of Birmingham
Rick was followed by Professor Tim Barret, a paediatric clinician, who is heavily involved with a number of different rare disease specialist services. Tim discussed his work on Wolfram syndrome, a rare degenerative condition that causes diabetes along with gradual loss of eyesight and hearing. In the nineties Tim was involved in a key study that outlined the natural history of this condition. After attending a meeting organised by a Wolfram patient’s mother in France, he and a number of collaborators were encouraged to use their knowledge to find a new treatment for the condition. Since then Tim has been involved in a project to repurpose a drug intended to slow disease progression in Wolfram.
Bev Hart and Georgia Hart - Family affected by Friedreich’s ataxia
Dr Richard Wyse - Director of Research and Development, The Cure Parkinson’s Trust
After a break for coffee, we returned to learn about the world of drug repurposing for Parkinson’s disease. Dr Richard Wyse is the Director of Research and Development at The Cure Parkinson’s Trust, a charity dedicated to identifying disease modifying treatments for this devastating condition. While Parkinson’s is not normally considered a rare disease, Richard revealed that in their search for treatments, they have identified a wide range of different mechanisms and causes of the condition: Parkinson’s is in fact a collection of many rare conditions. To address this complex collection of conditions, the Trust have set up an expert panel who review a huge number of different repurposing opportunities in Parkinson’s, and have enabled the launch of multiple clinical trials over the past few years.
Dr Christine Charman - External Program Lead, TAK-celerator, Takeda
Richard was followed by Dr Christine Charman from Takeda, a pharmaceutical company who are setting up a new accelerator model to drive the development of treatments into rare diseases. This TAK-celerator has a strong focus on collaboration, aiming to combine the best of the skills in big pharma with those of academic researchers, small biotech companies, and patient groups. The TAK-celerator is a great example of the collaborative approach being developed in the orphan drug industry.
Dr Daniel O’Connor - Medical Assessor, MHRA and Jonathan Underhill - Medicines Clinical Advisor, NICE
Dr Pan Pantziarka - UK Representative, Anticancer fund
Lightning talk session
When registering, all delegates were welcome to submit a 5-minute ‘lightning talk’ about their own experiences in drug repurposing for rare diseases. The Findacure team chose our favourite six suggestions, all of which were delivered excellently in the day’s dedicated session. Thank you to everyone to took on the challenge!