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Drug Repurposing for Rare Diseases 2017

Findacure’s fourth annual conference was held on Tuesday 28th February – Rare Disease Day – at the Royal College of Nursing in London. This year’s conference was the biggest yet, hosting 120 delegates, with a total of fifteen speakers sharing their perspectives on repurposing. Talks came from researchers, patients, clinicians, charities, the pharmaceutical industry, clinical research organisations and more, all coming together to focus on the best ways to improve the delivery of repurposed drugs to the patients that need them.


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.

Click here to view Tim’s slides.

Bev Hart and Georgia Hart - Family affected by Friedreich’s ataxia

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.

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.

Click here to view Richard’s slides.

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.

Click here to view Christine’s slides.

Dr Daniel O’Connor - Medical Assessor, MHRA and Jonathan Underhill - Medicines Clinical Advisor, NICE

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.

Dr Pan Pantziarka - UK Representative, Anticancer fund

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.

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!

Dr Jabe Wilson - Director, Text and Data Analytics, Elsevier

Polly Moyer - Co-founder, Action for MdDs UK

Juliet Hulse - Director of Research Nursing, Illingworth Research Group

Prof. Wyatt Yue - Associate Professor, University of Oxford

Ruth Slater - Trustee, Mast Cell Action

Dr Farhat Khanim - Senior Research Fellow, University of Birmingham


Drug Repurposing for Rare Diseases 2017

by Libbie Read time to read: 11 min