Filling the funding gap and accelerating drug discovery By Dr Arthur Roach, Director of Research at Parkinson’s UK Published: 27 April 2018 With limited treatments and no cure for Parkinson’s, Parkinson’s UK has always been committed to combing the research world for promising studies. In too many cases, however, this effort simply isn’t enough: so often, drug development is stalled in the early stages due to a lack of funding. This is especially true in the area of neurodegenerative conditions, which is often viewed as difficult and plagued by expensive, high-profile failures. For this reason, we developed the Parkinson’s Virtual Biotech project, a ground-breaking initiative to fill the funding gap and accelerate drug discovery at the very high standard needed to attract investment from large pharma companies during late-stage clinical studies. No bricks, mortar or additional staff The Virtual Biotech allows Parkinson’s UK to provide leadership and critical funding in partnership with a range of other organisations that have the facilities and staff to carry out scientific work on a contract basis. Together, we push studies through to a stage at which large pharma companies will take interest and provide the huge sums needed to get treatments out of the lab and into the pharmacy. And, because it’s virtual, there are no bricks, mortar or additional staff to consider. Partnering with biotech companies With one project already under our biotech belt, last month, we embarked upon a second. We partnered with US biotech Neurolixis to accelerate the development of a promising new drug that could prevent one of the most debilitating side effects of Parkinson’s medication – dyskinesia. Caused by taking levodopa – the main drug used to treat the condition at a stage when many dopamine terminals have already been lost – dyskinesias are involuntary muscle movements. They include twitches, jerks, twisting or writhing movements, and restlessness. This side-effect is impacting the lives of thousands of people in the UK and can make everyday tasks such as eating, walking and writing almost impossible. Around half (40-50 per cent) of all people with Parkinson’s will experience dyskinesia after just five years of taking levodopa, and up to 80 per cent of people will experience it at later stages. Repurposing drugs Parkinson’s UK is now providing more than £780,000 for Neurolixis to carry out the final research needed before the drug, NLX-112, can progress to human clinical trials. This includes drug formulation, preparation of clinical trial materials and safety and efficacy testing in a marmoset model of Parkinson’s. The partnership came about because of Parkinson’s UK’s particular interest in the repurposing of drugs. NLX-112 was previously discovered and developed by French pharma company, Pierre Fabre Médicament, as a potential treatment for pain. After reaching phase two clinical trials, it was out-licensed to Neurolixis, which identified an opportunity to re-purpose the drug for the treatment of levodopa-induced dyskinesia. A timescale that presents huge hope Because this drug has already reached phase two clinical trials in the past, we already know a lot about its safety. This means that, should this last stage of pre-clinical trials go well, we do not have to spend time on many of the usual steps and it can progress directly to phase 2 clinical trials. If it succeeds in these trials, we could be seeing a new treatment for people with Parkinson’s within as little as five years. In a landscape where most drug development takes decades, this timescale presents huge hope, showing the benefits of strategic, collaborative thinking to bring about faster, smarter developments in research. Last year marked 200 years since Parkinson’s was defined as a condition, and levodopa was developed more than 50 years ago. People with Parkinson’s have waited too long for better treatments and we must do everything possible to bring about the day when no one fears this condition.