Drug Repurposing for Rare Diseases 2016

Findacure’s Scientific Officer, Dr Rick Thompson, opened the day with an introduction to Findacure, an overview of drug repurposing, and our plans for a social impact bond. The microphone was then passed to Dr Annabel Griffiths and Dr Sana Eljamel from Costello Medical Consulting, who have been working with Findacure to develop an understanding of the impact one specific rare disease, congenital hyperinsulinism, on the NHS. Sana and Annabel reviewed some of the challenges of modelling the costs of a rare disease, before presenting some of their preliminary results.

 

The second talk given by Dr David Cavalla, an expert in drug repurposing, and Chief Scientific Officer at Healx. David gave us an overview of drug repurposing as an approach, highlighting many successful cases where new uses have been identified for old drugs in both rare and common diseases. He outlined the 2000 year story of aspirin (first used by Herodotus), which is even now undergoing new clinical trials for new diseases, and discussed some of his own work in repurposing a ß-blocker to prevent weight loss in chronic diseases (cachexia).

 

Next up was Matt Hammond, the father of Scarlett, a three year old girl born with congenital hyperinsulinism. Scarlett was one of the first children treated with a repurposed drug, sirolimus, and Matt shared her story with us. It was great to have a strong patient perspective at the conference. The audience really appreciated Matt’s honest talk, and laughed in all of the right places.

After the tea break, we returned to session two, which formed a case study on Ataxia. We were lucky to have two talks which explored the collaborative projects in drug discovery and repurposing that are being run in this neurodegenerative disease. Firstly Dr Julie Vallortigara from Ataxia UK discussed the charity’s work in drug repurposing, including a project that studied the impact of vitamin B3 on Friedreich’s ataxia.

Julie was followed by a joint project from Dr Michele Lufino, an Ataxia UK research fellow from the University of Oxford, and Dr Nick Clarke, Head of the Rare Disease Unit at Pfizer. Nick and Michele discussed their collaboration, which is working to find new treatments for Friedreich’s ataxia, and both the benefits and challenges of academic-industry partnerships.

After lunch we returned to a talk by Dr Jack Scannell, an academic with experience in both finance and biotechnology. Jack gave a fascinating and challenging talk, which discussed the merits of user-led innovation – research driven by clinical practice and patient experience – over a traditional lab-based model. Jack was followed by a lightning talk session, where six delegates presented on their work in rare disease drug repurposing, covering patient registries, funding opportunities, structural biology, zebrafish, text mining, and RareOmics.

 

The day’s final talk was delivered by Dr Victoria Parker, from Addenbrooke’s Hospital in Cambridge. Victoria gave us a fantastic overview of a group of rare segmental overgrowth disorders, and the challenges she has faced in setting up a clinical trial for a repurposed drug that may well help to reduce the mass of the patients’ over-grown tissue.