Existing drugs may hold the key to treating rare diseases, says Cambridge charity



Findacure, a rare diseases charity based in Cambridge, says repurposing existing drugs to treat rare diseases will improve patient outcomes and save the NHS money.


Diseases that affect less than one in 2,000 people are known as ‘rare diseases’. They are some of the world’s most complicated conditions, yet they are also the most underfunded and under-researched. Of the 7,000 rare diseases that have been identified to date, only 400 have a licensed treatment – that’s less than 6%.

So few treatments have been researched for rare diseases because it is not profitable for pharmaceutical companies to develop new drugs for such small patient populations. Drugs that are developed often have an astronomical price tag attached to them, meaning they might not be approved by the cost-effectiveness watchdog NICE and patients are still denied access to them.

Findacure is holding a conference to share their solution to these challenges: repurposing existing, off-patent drugs to treat specific rare diseases.

A classic example of ‘drug repurposing’ is the drug sildenafil, also known as Viagra. Originally developed to treat heart problems, it is now used in patients with erectile dysfunction. Another example is guanfacine, which was originally used to treat ADHD, but is now used to manage some symptoms of fragile X syndrome – a rare disease.

Since early 2016, Findacure has been developing a drug repurposing programme specifically for rare diseases. They hope their research will slow symptom progression, improve patient quality of life, and extend patients’ lives. But Findacure believe their project will also save the NHS money. As the drugs they will be researching are off-patent and already proved to be safe for use in humans. This means that they are much cheaper than newly discovered drugs, and their improvements on patient health could greatly reduce the cost of managing rare diseases to the NHS.

Findacure will be holding a conference on Rare Disease Day, Tuesday 28 February 2017, to share the ideas behind their drug repurposing project. It will take place at the Royal College of Nursing, near Marylebone in London.

The conference will introduce attendees to the concept of drug repurposing, before going on to explore its uses and potential for treating rare diseases. Six talks will be given by a variety of speakers, including clinicians, researchers, pharma reps, regulators, and patients involved in drug repurposing trials. Breaks between talks will be a perfect opportunity for attendees to network with the already equally diverse audience.

The event will also feature the launch of Findacure’s ‘open call’ for submission of drug repurposing projects. The open call will see Findacure representatives touring the country to invite researchers, clinicians, doctors and patient groups to submit and potentially get funding for their ideas.

Findacure would like to welcome any interested persons to join them at the conference. More information, including a full programme and registration, is available on their website: www.findacure.org.uk/scientific-conference-2017/

They also welcome further questions to be directed to their Head of Research, Dr Rick Thompson, on rick@findacure.org.uk.