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Drug repurposing is something we spend a lot of time thinking about at Findacure, and something that we will be spending more and more time talking about in the coming months. But what exactly is it, and why should rare disease patients care?

In essence, drug repurposing is recycling for pharmaceuticals. When repurposing drugs we are trying to find a new potential use for an existing medicine, and make more use of it. This is possible because most drugs have effects on multiple parts of the body, or multiple biochemical pathways, and most of these may go wrong in multiple different diseases. Generally though, a drug is only approved for one illness, and so is only used for one of the ways in which one of its target pathways has gone wrong. If we can identify the other illnesses for which it has an effect, we can increase the usefulness of the drug, and hopefully help more patients.

Drug repurposing is also a great strategy because doctors and scientists are working with a known medicine. The drug being repurposed will already have passed through a series of clinical trials, and will have been widely used in patients. Consequently we understand its side effects, doses, the way in which it is absorbed into the body, and we may even understand something about the drug’s effect on other diseases. All of this knowledge can be used to help us identify new illnesses that a drug could treat, but will also help to accelerate the clinical trial process. This means repurposed drugs have the potential to reach patients much more quickly, making them a particularly exciting option for rare disease patients.

Unfortunately, repurposing research is hard to fund when it focuses on generic drugs (drugs which are no longer under patent), as companies will struggle to secure market exclusivity for the drug, and thus struggle to make money by finding new uses for it. This means different approaches are needed to fund such research.

A recently launched UK trial to investigate if Aspirin can prevent cancers returning is a fantastic example of the potential of drug repurposing, which has partly been funded by the National Institute for Health Research and Cancer Research UK. Charities can clearly take a big role in repurposing drugs, and this is something Findacure are hoping to do for rare disease patients with our Social Impact Bond.

It is important to remember that big pharma companies are also very interested in recycling drugs, but generally focus on those chemicals that previously failed clinical trials for one illness, which they can target to new patient groups. We call this drug repositioning. Repositioning works well for big pharma, as it reuses shelved products, making the most of previous expensive drug discovery work, and ultimately leading to a new drug that can be sold.

If you’d like to learn more about drug repurposing as a route to find new treatments for rare diseases, then you should attend Findacure’s Scientific Conference on Rare Disease Day 2016. This event will focus on drug repurposing and repositioning, and we’ll be hearing from patients, academics, doctors and charities. You can register for the day on our Eventbrite website.[/two-third][one-third]Recycling-RepurposePharmaceuticSciConfPic_BasicRick pres[/one-third][/row]


by Rick Thompson time to read: 2 min