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Diving into drug repurposing

Findacure, and particularly Rick, has been talking about drug repurposing for a number of years hoping to convince the rare disease world, whether industry, researchers, or patient groups, that it is great way to develop rare disease treatments.

While we have dedicated a lot of time to running our annual conference to showcase excellence in the field, there has been less emphasis on educating the patient community. We thought that it was about time this changed, so on Tuesday 5th November we partnered with the MCDS-Therapy project to host our Diving into Drug Repurposing workshop at White and Case in London. This day-long event aimed to help patient organisations understand:

  •  drug repurposing and its relevance to rare diseases
  • the main ways to deliver repurposed drugs to patients
  • how to identify repurposing opportunities
  • the role patient groups can play in the drug repurposing research process



Rick Thompson - CEO, Findacure

The event began with our very own CEO, Rick Thompson, taking the stage to introduce the audience to drug repurposing (apparently it is just recycling for drugs). He highlighted the decline in pharmaceutical company productivity and the high cost of many orphan medicines as two big reasons why repurposing is an interesting approach for rare diseases. By speeding up the drug development process and lowering the cost through repurposing, we have the chance to deliver more drugs to patients at a lower cost.

After convincing everyone that repurposing is a good thing, Rick focused on the strategic considerations for rare diseases – understanding the best approach to deliver a treatment for your condition from the outset is key to success. Whether the repurposing candidate is currently under-patent, or generic can make a huge difference to your strategy and options. The route to access is also crucial to consider – the ideal option is of course to receive a marketing approval for the rare disease, an update to the approved product labelling. Unfortunately, for many ultrarare conditions this may not be realistic. In these cases, the aim must be to deliver strong evidence to encourage an update of the relevant clinical guidance, as this could be the only route to deliver benefit to patients.

Watch Rick’s talk

Download Rick’s slides

Ian Roberts - Chief Scientific Officer, Healx

Following Rick was Ian Roberts, Chief Scientific Officer at Healx. Ian covered the different approaches to identifying drug repurposing opportunities, from the traditional non-systematic approach of serendipity – or clinician driven innovation – to more modern approaches that rely on artificial intelligence. These approaches have advanced dramatically in the last five years and can now be applied to a wealth of data, whether natural language from the scientific literature, or ‘omic’ data covering all of the DNA, proteins and even metabolic processes within the body. By combining all these data sets, new repurposing candidates and combinations can be identified for testing.

At the end of Ian’s talk his colleague Karine Proulx wrapped things up by introducing Healx’s Rare Treatment Accelerator. This is an exciting opportunity for patient groups to work with Healx, at no financial cost, to identify and trial repurposing opportunities for their disease.

Download Ian’s slides

Evgenia Mengou - Trustee, aPODD Foundation

After these first two talks, we spent time working in small groups discussing the different stages of drug development and the ways that patient groups can contribute to research. The rest of the day focussed on examples of repurposing projects. Evgenia Mengou of the aPODD foundation discussed their work in the field of repurposing for rare childhood cancers. aPODD is a parent founded organisation driving research into treatments for rare childhood cancers- an area shockingly neglected given the public awareness as cancer as health concern. The foundation is involved in two repurposing programmes, both currently testing candidates in a preclinical setting. You can hear Evie’s thought on drug repurposing and aPODD’s work in our short video below.


Mike Briggs - Professor of Skeletal Genetics, Newcastle University

Prof. Mike Briggs, Project Coordinator of our partner organisation MCDS-Therapy, delivered the day’s final talk. MCDS-Therapy is an EU funded academic led clinical trial to develop a repurposed generic drug, carbamazepine, to treat metaphyseal chondrodysplasia type Schmid (MCDS). The project is a fantastic illustration of how basic research scientists can engage with the wider research and clinical community to drive the development of new treatments. It began with research into a specific protein – collagen 10 – but led to the identification of a disease mechanism, a repurposing opportunity, and a trip to the EMA for scientific advice to set up a clinical trial.

The consortium is now recruiting their first patients, having overcome a number of logistical challenges during project development. Mike shared a number of his insights and challenges from the project so far, helping to highlight the potential of academic led clinical trials, but also the benefit of experienced individuals within the clinical trial team.

Download Mike’s slides

We had a fantastic day at the workshop, and the quality of the talks was exceptionally high. If you missed the workshop you will soon be able to dive into our new E-learning Portal guide on this topic. You can also learn more at our Drug Repurposing for Rare Diseases Conference on 24th February 2020. 


Diving into drug repurposing

by Mary Rose Roberts time to read: 6 min