Drug Repurposing Conference, Part 1, London - 29th Feb 2016

On 29th Feb I had the pleasure of going to the Findacure Drug Repurposing for Rare Diseases conference which was held at the Royal Institution in London.  My notes on this conference will take up a few posts. This is part 1 of 3. Part 2 is here. Part 3 is here.

The conference was opened by Dr Rick Thompson, one of the scientific officers at Findacure. He outlined some basic drug re-purposing and rare disease information:

• Rare diseases affect some 350 million people worldwide, around 5% of the population.
• Drug re-purposing is taking an existing drug and demonstrating that if effective for a different group of people

When you have an existing drug it has a known safety profile and known side effects. There is a history of human usage and the pathways of action are known. Once an idea for repurposing is identified these factors can reduce the requirements for clinical trials for the new group/population.

When drugs are repurposed there may be changes to the delivery of the drug, the dose of the drug or the formulation of the drug - although new safety data may be required.

Dr David Cavalla of Numedicus and Healx discussed what the opportunities for drug repurposing are.

In summary this process is quicker, cheaper and less risky. He outlined that with conventional drug development for every approved drug that is out there, there is another 23 drugs which have entered pre-clinical trials but not made it that far. The conventional drug development route takes some 4-9 years and costs 1.8 billion, but drug repurposing takes some 1-4 years and costs 0.3 billion.

Some 90% of drugs on the market already have secondary uses, which may be exploiting the same or different mechanisms, and this is an under-exploited area. Computer models can be used to explore if existing drugs have potential other uses.

David gave some well known examples of drug repurposing:

• Aspirin - 1899: Pain - 1970: Myocardial Infarction and Stroke - 2010: Cancer,
• Viagra - Originally: Erectile dysfunction - Now: Pulmonary arterial hypertension

The website http://www.drugrepurposing.info/ contains a database of compounds and lists some 94 examples of drug repurposing. The issue is that there is little commercial impetus to explore these avenues.

David described that Healx is working on drug repurposing with patient charities and big data. They are using big data to compare drug profiles with disease profiles to examine for potential matches. They know that some drugs can have some protective effects, and if these protective effects happen to be useful in a different disease there's a potential match.

Matt Hammond presented from a patient perspective. His daughter has congenital hyperinsulinism (CHI), and he told everyone about the emotional rollercoaster he and his family have been on since her birth. He described in detail the unknowns and the uncertainty around the whole journey, which you can read here: http://www.diseasespotlight.com/rare-disease-hyperinsulinism/. One of the conversations he had with a specialist led to the discussion of trialling a drug or removing her pancreas. This is an example of drug repurposing.

Matt gave a very useful list of the hidden costs of rare diseases:

• Psychological
• Time (travelling, seeing professionals etc.)
• Finance (travelling)
• Missing out (on other activities and events)
• Form filling (it seems there a lot of forms in some cases)
• Loneliness (being the only one in your situation)
• The need to plan everything (because of the effects of the disease) 

This was one of the most useful lists I've seen.