Early assessment, diagnosis and treatment of Parkinson's Plus Related Syndromes (ExPRESS)

Progressive supranuclear palsy (PSP), Multiple System Atrophy (MSA) and Corticobasal syndrome (CBS) are rare brain disorders, which cause rapidly progressive disability. The last decade has seen rapid progress and recognition that PPS are not only tractable disorders in their own right, but also provide ideal conditions in which to evaluate therapeutic strategies which will potentially have a wide impact in the treatment of more common dementias and movement disorders. Here, we plan to build on scientific and organisational opportunities in the UK, to improve early diagnosis and treatment for PPS. We will do so in partnership with the NHS and a network of collaborating clinicians, and charities supporting families affected by PPS including the Progressive Supranuclear Palsy Association (PSPA) and Multiple System Atrophy Trust (MSAT). We will engage with national research platforms for discovery science and translation, including the Dementia Research Institute (DRI), Dementia Platform UK (DPUK), and National Institute for Health Research - Clinical Research Network and Biomedical Research Centres (NIHR - CRN/BRC). In addition, a number of drug companies have indicated that they would like to collaborate in this initiative. A major challenge for the development of PPS research has been to build the infrastructure required to enable national network activities and nationally-representative patient recruitment. UK charities helped to establish a skeleton network of UK sites (the PSPA and MSAT funded PROSPECT-M study), which has been successful in proof-of-concept for recruitment and coordination across specialist clinics. There is significant interest from both pharmaceutical industry and university researchers in developing new trials of disease modifying therapies. Early diagnosis will require new diagnostic markers (for example specialist blood tests, spinal fluid tests, and pathology-specific brain scans), likely developed with industry. To maximise the impact of forthcoming trials, we need to meet the challenge of recruiting patients with early stage disease, improving diagnostic and prognostic accuracy and developing new trajectory models for individualised approaches. Within our proposed ExPRESS (Early assessment, diagnosis and treatment of Parkinson's Plus Related Syndromes) study we will link together clinicians and therapists who see patients with early stage disease and establish collaborative links with disease charities (PSP Association, MSA Trust). We will link together with professional bodies (Association of British Neurologists Movement Disorders Group, British Geriatric Society Movement Disorders Group), industry partners, and linked UK scientific research consortia such as the UK Dementia Research Institute (DRI), Dementia Platform UK (DPUK), UK Brain Bank Network (BBN) and Health Data Research UK (HDRUK) to maximise the scientific potential of the UK's activity in this area and to improve the potential to develop new treatments. We will recruit patients who have been referred from primary care with suspected Parkinson's disease and develop a clinical algorithm which will enable early detection and recruitment of patients likely to have Parkinson's Plus Syndromes (PPS). This will improve clinical diagnostic pathways and early disease diagnosis. However, we envisage that emerging technologies such as protein based brain scans, advanced MRI scans, specialist spinal fluid and blood tests, and digital tests, for example delivered through smartphone apps, will rapidly accelerate our ability to identify patients with these rare movement disorders. Ultimately, we hope that these early stage patients will be recruited to clinical trials which slow down or stop the progression of these disabling conditions.