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“A cheap and widely available drug can help save the lives of patients seriously ill with coronavirus.”

This was the lead headline on a BBC news story a few weeks back, which proclaimed Dexamethasone as the first proven life-saving drug for coronavirus. Put simply, Dexamethsone is an example of drug repurposing in action: a low dose steroid, used since the early 1960’s to treat conditions as common as asthma and arthritis has been shown to significantly reduce the risk of death in those patient most seriously affected by covid-19. The drug is cheap, widely available to patients globally, and already stockpiled in the UK.

While it is no miracle cure, it is a frontline treatment that will save lives, and give clinicians, and crucially patients, another option in the fight against this disease. And it has been identified and delivered to patients in a matter of months.

This is the value of drug repurposing: rapid research; accessible drugs; low cost treatments; the ability to deliver a step change in treatment for patients with a high unmet need.

Back in the early days of the coronavirus pandemic (April to be precise) Findacure ran a webinar designed to highlight the potential of drug repurposing for both covid-19 and rare diseases. In this webinar we stressed the parallels between an emerging infectious disease, and a rare condition, namely:

  • A small number of researchers or clinicians working on something like it.
  • A small patient population, who may be hard to identify.
  • Limited understanding of disease natural history.
  • Limited knowledge of disease pathways and mechanisms.
  • No existing treatments, and often no clinical care pathway.

The fundamental difference, however, between rare conditions and emerging infections like covid-19 is urgency. The pace of spread and the imminent risk to the health of the world’s population creates a sense of overwhelming urgency to meet the need and deliver the treatment. This has led to unprecedented levels of investment in covid-19 research, huge levels of scientific and international collaboration, and the rapid delivery of potential treatments into clinical trials.

This is already starting to pay off for covid-19, and we can all be thankful for this news. But what about rare diseases?

Well, rare disease research soldiers on, but covid-19 has stalled many research programme and clinical trials. Whether through funding being lost or redirected, or clinical trial staff and researchers being directed into the covid-19 response, much work in rare disease has been derailed in the last few months. There is a lack of urgency for change in rare diseases outside of our community.

However, covid-19, and the success of repurposing for it, can create an opportunity for rare disease patients moving forward. Covid-19 has made public the true potential of repurposing, and it has shown how quickly and effectively treatments can be developed for a condition with the right coordination and effort. We need to find a way to take this message and shout it load, to show the vast unmet need of rare disease patients, and ensure that this need can be met with some more urgent action. If it can, then there is a real potential to develop a new drug development pathway for generic drug repurposing, and deliver some meaningful treatments to patients in the near future.

If you’d like to read more about the progress of treatment development in covid-19, then check out this recent article in science.

Hope for rare repurposing in the future?

by Rick Thompson time to read: 3 min