The whole Findacure team are starting to get excited as our annual conference, Drug Repurposing for Rare Diseases, is less than a month away. Having just released our shiny new conference website with loads of information about the upcoming event, we thought that this would be an opportune moment to preview some of the exciting talks that we will be featuring on the day.

As always, we have a jam-packed programme, featuring speakers from patient groups, academia, industry, and even the UK reimbursement body NICE. We will have more announcements to make over the coming weeks, but for now let’s take a closer look at three of our talks on the day.

Repurposing saracatinib to treat Fibrodysplasia Ossificans Progressiva (FOP)

Fibrodysplasia Ossificans Progressiva – FOP for short – is a truly scary condition. Affected individuals’ soft tissue responds very differently to damage or distress. Bone cells invade these regions of damage and cause bone to form. This means that over the course of their lives they develop bony sheets within the body that restrict movement, or lock their body into a fixed position. This process will ultimately kill the patient.

While exceptionally rare, there has been active research into FOP for a number of years. We will be joined on the 24th of February by Dr Alex Bullock from the University of Oxford. Alex has led a project to understand the molecular basis for the condition. Based on this work, he has been involved in identifying an existing drug – saracatinib – that has now been shown to be effective in FOP mouse models. This has resulted in funding from the EU to run an in human trial for this drug. We will hear from Alex about the condition, his work, and the outlook and prospects for this exciting repurposing opportunity.

Dr Alex Bullock – Principal Investigator, Structural Genomics Consortium, Nuffield Department of Clinical Medicine, Oxford University

Daniel Lewi, Co-founder, CATS Foundation

Working together to rapidly advance a clinical trial

Over the last few years of our drug repurposing conference a number of speakers and delegates have raised the concept of a running single trail for a drug across multiple conditions. This is something that has the potential to increase the efficiency of drug development for rare diseases, and potentially pool the power of multiple small patient populations. Our talk from Will Evans, Chair of Niemann Pick UK, and Dan Lewi, Co-founder of CATS Foundation will give us some insight into this type of trial, and the impact and benefit it can have on different patient groups. Both groups are involved in trials for the same repurposed drug for their distinct conditions, and have worked closely with the controlling company to help with trial design and recruitment. This could be a great new template for rare disease drug development in years to come.

Dr William Evans, Chair, NPUK

Kelly Vance, Secretary & Patient Representative, LAM Action

From Despair to Hope:  A repurposed drug transforms the outlook of women with LAM

A talk from a patient or carer has been a staple of our event over the last five years, and is always a highlight for the audience. This year’s patient talk will be given by Kelly Vance, a patient living with the rare lung disease Lymphangioleiomyomatosis (LAM) since 1996. Kelly will recount her experiences with the condition and her involvement in a patient movement that helped to drive research and treatment development. Thanks to a lot of work from the patients, clinicians, and industry representatives. The drug rapamycin was ultimately repurposed for the LAM, and became a widely available and effective treatment for LAM, helping women living with the condition live longer, and with less of an impact from the disease.

As you can see, the conference is set to be an excellent day, with engaging talks that will highlight some real successes and innovations in the rare repurposing space. We’d love to see you there, so secure your ticket today.

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Speaker Spotlight – Drug Repurposing for Rare Diseases 2020

by Rick Thompson time to read: 5 min
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