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Findacure: Drug Repositioning for Rare Diseases

March 9, 2015

The Orphan Druganaut Blog is honored to have Flóra Raffai, the Head of Development from Findacure, as a Guest Blogger. Findacure is a charity which focuses around fundamental diseases – rare, often genetic, diseases from which the basic mechanisms of dysfunction can be extrapolated and applied to our understanding of more common conditions.

At the current pharmaceutical rate of drug development, it would take several hundred years to develop a treatment for each one of the known 7,000 rare diseases. One of the methods being considered to speed up this process is drug repositioning: clinically testing drugs, devices, and nutriceuticals approved for one disease indication to create a ‘new’ treatment in a different disease indication. To explore the potential of drug repositioning for rare diseases, Findacure organised a scientific conference last week. Dr Bruce Bloom of Cures Within Reach, Prof Michael Briggs of Newcastle University, and Dr Farid Khan of Protein Technologies were invited to present on the topic.

A point stressed by all speakers was the huge potential of drug repositioning compared to de novo research. Firstly, drug repositioning has a much higher rate of success, with 10 to 30% of projects succeeding. This is significantly higher than the 1 in 10,000 success rate for projects pursuing new molecules. Dr Khan added to this point by sharing a piece of research he conducted, which found that over the past 100 years over 30,000 drugs have been developed, which collectively cover almost all human pathways. As a result, it is highly likely that there is already an existing effective treatment for most conditions.  A key example cited was that of Nitisinone, a drug which began its life as a weed-killer, before being identified as treatment for hereditary tyrosinemia type 1. It is now being trialled for usage in alkaptonuria patients, an ultra-rare condition. If the trial is successful, this repositioned drug would be the first identified treatment for alkaptonuria.

Despite this potential, there are key barriers raised by the speakers which need to be overcome. Firstly, there is a lack of financial incentive for pharmaceutical companies to engage in drug repositioning, especially for generic drugs where patents have run out. Dr Bloom offered a solution in the form of social financing, specifically social impact bonds. Cures Within Reach and Findacure are currently collaborating to establish a system of social impact bonds in the UK, where the government would pay on success. This should be in place by 2016, allowing small drug repositioning projects to be funded.

A second barrier highlighted by Prof Briggs is the divide between what patients want from research and what researchers assume needs to be done. He shared a story from the achondroplasia community, where families have been affected by dwarfism for generations. These communities do not support research for a ‘cure.’ Because the dwarfism has become part of their culture, they do not identify it as something in need of a ‘cure’. They are more interested in palliative care, easing joint pains and discomfort, than they are in a drug to increase bone growth. The solution suggested was for greater involvement of patient groups in research and drug development. Through innovative meetings, such as medical ‘hacking’ days, patient needs can be effectively communicated to researchers, who can then directly respond with new projects.

By combining social financing with innovative collaborations, drug repositioning can become a viable option for accelerating treatments for neglected rare conditions. It also highlights the fundamental importance of rare conditions, demonstrating the common mechanisms across an array of diseases, which is continually adding to our understanding of human physiology. A key learning from the conference was indeed that rare diseases are truly fundamental diseases.

To listen to the presentations from the conference, please follow the links below:

•   Dr Bruce Bloom, Cures Within Reach – Innovative Partnerships and Financing Models for Rare Disease Drug Repositioning

•   Prof Michael Briggs, Newcastle University – Repositioning of Small Molecules for Inherited Connective Tissue Diseases

•   Dr. Farid Khan, Protein Technologies – Collaborative Drug Repositioning: Case Studies between Universities, SMEs, and Charities.

If you are interested in finding out more about future Findacure events, contact Flóra Raffai via

Picture courtesy of Findacure.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

  1. Thank you for an excellent article. I am the mother of a three year old terminally ill RARE child with SMA Type 1. We are extremely proactive managing Hunter’s SMA. After following a study where Celebrex increased SMN in the SMA mice model, Hunter started taking Celebrex off label. Drug repurposing is important in the SMA community, and the Rare Disease community in general. We support the Open Act, as it affords pharmaceutical companies an incentive via extended patent protection to explore repurposing trials. This is important, as individual Rare Disease communities are oftentimes too small to warrant the cost a repurposing trial. By extending the patent protection an additional six months, the benefit is greatly increased.

    • Thank you for visiting & your personal story with SMA. If you are interested in sharing your story or your organization as a Guest Blogger, there is an open invitation. Kind Regards, Ann

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