Drug Repurposing for Rare Diseases Networking Event 2015

When you are trying to find new treatments for rare diseases it is crucial that you have the right friends, with the right knowledge. With so much to learn about rare diseases, their treatment, and the patient experience, no one person can easily have all the knowledge and energy required to do everything themselves – meeting the right people, or building an excellent network, really matters. That is why we at Findacure hosted our first scientific networking event last night, with the simple aim of bringing people with an interest in drug repurposing together to make friends, and share knowledge – and boy, did we share!

Over 60 delegates came through the door, representing the huge range of disciplines that influence the rare disease space – doctors, pharma companies, academics, biotechs, students, patient groups, consultancies, charities, and even NHS England were represented – and the conversation never died. Many people came up to me during the night to let me know that they had arranged meetings at the event, and were hoping to form new collaborations as a consequence, which will hopefully help to move rare disease research forwards.

A big highlight of the event were our six, five minute “lightning talks”. These aimed to give the audience a snap-shot of the work been done in drug repurposing and rare disease research today. First Professor Khalid Hussain told us about his work on congenital hyperinsulinism, a rare disease that effects newborn babies’ ability to regulate blood glucose, starving their brain of fuel. Traditionally, the worst effected patients have their pancreas removed, consigning them to a life of severe diabetes. Prof Hussain has been involved in case studies that have shown that the drug sirolimus can help these patients regulate their blood sugar level, and retain their pancreas. We then heard from Dr John Pearson from the University of Birmingham’s drug repurposing unit, who discussed three different projects they are working on, and the different problems they have faced in securing the large amounts of funding required to bring early stage research to patients. This need for large amounts of funding was also highlighted by Professor Sir Tom Blundell, who discussed his experience in forming a pharmaceutical company, as well as how understanding the structure of mutated proteins can help us identify treatments for rare diseases.

While delivering treatments to patients was a key theme of the evening, Dr Thomas Hiemstra from the new Patient Led Research Hub at Addenbrooke’s Hospital discussed their aims to put patients at the centre of clinical trial design – an exciting initiative that we believe could be hugely beneficial to patients and researchers alike. We also heard from Dr Edmund Jessop, from the specialised commissioning team at NHS England, who told us the success story of repurposing the drug mexiletine for myotonia. Edmund then gave us a brief overview of some of the financial considerations central to NHS commissioning, helping us to understand the ways in which repurposed drugs could save the NHS money.

The final talk of the night was delivered by Dr Dorian Bevec, told us how we can use data mining techniques and computer algorithms to identify drug repurposing ideas based on the connections between scientific papers.

As you can see, we had a really diverse programme, and a really successful night. Thank you to all of our speakers for giving excellent talks (and keeping to time), and to everyone who attended the event to make it such a success. For those who could not attend, watch this space, as we are hoping to release some materials from the event in the coming weeks.