Drug repurposing for Parkinson’s disease

Drug repurposing is raising significant interest in pharmaceutical research. Repurposing is the redirection of clinically advanced or marketed products into certain diseases rather than in the initially intended indications. Repurposing is based on drugs’ side effect profiles, indicating interaction with more than one cellular targets. Although sometimes arising from serendipity (e.g. Viagra), repurposing can be used as a targeted approach for discovering drugs against diseases using disease-specific screening assays. Parkure’s mission is to find a cure for Parkinson’s disease (PD) by discovering drugs that halt the neurodegeneration process behind the disease. We aim to do that by focusing – although not limiting ourselves to – repurposing drugs using our PD specific assay.

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Figure 1: Taken from [1]. Repurposing of drugs for rapid development. Existing compounds typically have identified molecular targets and primary indications in which they have either reached approval status or been sidelined somewhere along the pipeline, for efficacy or business reasons. If compounds are made available for further research, then investigation may identify new indications for the same target or new molecular targets with separate indications. A parallel pathway to approval for a repurposed indication can be achieved more robustly by relying on prior safety and tolerability experience with the original indication.

The de novo drug discovery and development path can take 13 years and cost close to 2 billion dollars [2]. By using repurposing we can “piggyback” on this development for other indications and shorten the necessary R&D time. In addition to that the availability of enough safety data in some cases can lead directly to small clinical trials, fast-tracking the translation of scientific research to a benefit for patients.

Although repurposing is not a panacea or a replacement for de novo drug discovery, it is however promising, especially in diseases where traditional target based approaches have failed. This potential is reflected by the adoption of repurposing as an approach by major PD non-profits such as the Michael J. Fox Foundation [3].  As also discussed in [3] this adoption can also lead to an adoption of approaches to help with overcoming some of the obstacles associated with it. Parkure aims to be at the forefront of this interaction between small innovative biotech SMEs, pharmaceutical companies and non-profits in order to find a cure for PD. It is worth mentioning that repurposing for PD has already generated some results (amantadine) as well as a few drugs currently in Phase II and III trials [4]. Parkure’s unique position is having a fully developed PD-specific assay for repurposing. When we put this assay to use we will be able to repurpose drugs by testing them in a behaving organisms with PD rather than being based on serendipity or theoretical pathway based inference.

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  1. Strittmatter. Overcoming Drug Development Bottlenecks With Repurposing: Old drugs learn new tricks. Nat Med (2014)
  2. Paul SM, Mytelka DS, Dunwiddie CT, et al. How to improve R&D productivity: the pharmaceutical industry’s grand challenge. Nat Rev Drug Discov (2010)
  3. Shineman et al. Overcoming obstacles to repurposing for neurodegenerative disease. Annals of Clinical and Translational Neurology (2014)
  4. Bolgár et al. Drug repositioning for treatment of movement disorders: from serendipity to rational discovery strategies. Current topics in medicinal chemistry (2013)

Author: Lysimachos Zografos

CEO of Parkure Ltd.

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