Drug Repurposing for Rare Diseases 2017

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.

 

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.

Click here to view Tim’s slides.

Following Tim was no easy challenge, but it was met by Bev and Georgia Hart, a family affected by Friedreich’s ataxia. They responded to our call for patient speakers in 2016 and shared their personal story with our audience. Georgia was diagnosed with Friedreich’s in her teens. We heard from both Georgia and her mum, Bev, about their lives before ataxia, the impact of the diagnosis, and its impact upon their lives since. Georgia has been involved in a clinical trial in the USA for a repurposed treatment, and we learnt how and why the family got involved with this and their reaction to the discontinuation of the study.

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.

Click here to view Richard’s slides.

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.

Click here to view Christine’s slides.

After lunch Dr Daniel O’Connor from the MHRA highlighted some of the existing rules in the regulatory framework that can help repurposed drugs for rare diseases reach the market. He highlighted the real benefit of early interaction with the regulators in any drug development programme, and a desire within the MHRA to help move products to market. Jonathan Underhill from NICE then went on to discuss the development of NICE advice on drugs, including off-label treatments. He highlighted the importance of communicating and disseminating this advice successfully to ensure that the best treatments reach patients.

Our final talk came from Pan Panziarka of the Anticancer Fund, a foundation dedicated to repurposing drugs, previously unused for cancers, for use in cancers with a high unmet need, in particular for its rare forms. Pan took us through their work on repurposing the drug propranolol for the treatment of angiosarcoma, from the work done to identify this as a repurposing opportunity through to the studies and trials run to test its efficacy. Pan raised a key point – efficacy (the effectiveness of the drug) is always of central importance in repurposing. Patients want to take the best treatment available and should not accept a less effective treatment for financial reasons. When promoting repurposing it is crucial to highlight the efficacy and impact of treatments relative to anything else available to patients.