The Parkinson's Academy - masterclasses for researchers The Cure Parkinson’s Trust (CPT) in collaboration with the Parkinson’s Academy hosted a meeting in December 2018 dedicated to research engagement and involvement. The meeting presented on ways to become involved in Parkinson’s clinical research and was chaired by Dr Camille Carroll, Professor Oliver Bandmann and Dr Peter Fletcher covering a range of topics from research idea to successful study closure and everything in between! For a number of years the Parkinson's Academy has received feedback about including sessions on research in its curriculum. Taking these responses on board and working closely with CPT and the National Institute for Health Research (NIHR), Dr Carroll and Professor Bandmann and the academy’s Dr Peter Fletcher, designed the 'Research Engagement Meeting' for professionals who are becoming or aspire to become involved with Parkinson’s research on many different levels. The result was a stimulating meeting defining the landscape of Parkinson’s research showcasing how to move from a great research idea to delivering it into clinic; how to get started in research and the support available; and embracing the considerations of those living with the condition with pragmatic approaches from those who have just started the journey. In his introduction to the meeting, CPT Trustee and Parkinson’s Academy Founding Faculty member, Dr Peter Fletcher noted that we are at an exciting time for becoming involved in Parkinson’s research and, importantly, in making that research available to people with Parkinson's. Dr Richard Wyse, CPT's Research and Development Director and Professor Bandmann, lead researcher of the CPT part-funded UDCA trial, both presented. Professor Bandmann has been working for the last 10 years to identify potential drugs that might target mitochondrial dysfunction in Parkinson’s. Given that animal models in Parkinson’s are imperfect, the team in Sheffield tested 2,000 drugs in genetically stratified patient tissue – tissue from people living with Parkinson’s (PwP) with the PARKIN gene mutation. They tested for mechanisms leading to cell death and screened drugs that affect mitochondrial dysfunction - a recognised Parkinson's link. They found the drug, ursodeoxycholic acid (UDCA), a safe drug that had been used to treat liver disease for 30 years, crossed the blood brain barrier and then tested this in a second genetic model, LRRK2, because mitochondrial dysfunction is a feature and appears early in LRRK2 mutation carriers. This energetic and productive pre-clinical work did not automatically result in UDCA being able to move forward and Professor Bandmann eloquently explained in his presentation how programmes can stall with a lack of funding. For the next 5 years he applied for funding from a variety of major research funders but as there is no commercial interest in such an old drug, it proved a real challenge. Other issues included bias against phenotypic screening and diseases without biomarkers, and CTU costs, given that Parkinson’s neuroprotective trials need to include many patients and need to run for a long time. Throughout the trial design and planning stages, Professor Bandmann carried out further preclinical work, to evaluate dose and identify ways of proving target engagement. The trial that is just getting underway in Sheffield and London (The UP Study) will use Phosphorous MR Spectroscopy to measure mitochondrial function which is a key readout comparing these with animal models and skin fibroblasts. To support the clinical data, the team will also use a novel sensor system. In 2015 CPT developed a dossier on UDCA which was highly prioritised by the LCT committee and gave support to Professor Bandmann's work. Finally in 2018, Professor Bandmann secured funding from the JP Moulton Foundation and CPT. View Professor Bandmann's presentation here. Dr Richard Wyse opened the meeting by discussing disease modification in Parkinson’s through the Linked Clinical Trials programme (LCT). It is well-known that Parkinson’s symptomatic therapies have many imperfections. Encouragingly there is research into both better symptomatic and disease-modifying treatments. CPT's sole focus is to halt progression of the disease and ultimately reverse it, and in addition to CPT's preclinical and regenerative medicine programmes, the extensive LCT programme prioritises drugs, many repurposed from other disease areas, which are prioritised and evaluated by a committee of Parkinson’s experts of world renown. Since its inception the programme has evaluated more than 2,000 drugs and 100 different biological targets relevant to Parkinson’s developing 173 detailed dossiers of which 120 have been discussed by the LCT committee, which has led on to the prioritisation of 49 drugs and bioactive compounds of which 10 drugs have now moved into clinical trials and a further 7 are due to start in 2019. Trials in the LCT programme include:• PD-Stat a clinical trial of 230 people with mid-stage Parkinson’s using simvastatin is currently running across 23 centres in England. Read more in-depth about the trial here... • Aim-PD is a small study of people with GBA and idiopathic Parkinson’s testing a drug called Ambroxol, usually used to alleviate mucus build-up, which has just completed at the Royal Free Hospital in London • And the now completed 2017 study of Bydureon, a once weekly injectable treatment for type II diabetes which produced interesting results and is now moving forward into further trials. Read more in-depth information here... This dynamic programme of clinical trials of potentially disease-modifying treatments has created the opportunity for more interested and dedicated professionals to join this growing research effort.