Fox Focus I PPMI Special Edition 13
A Decade of Discovery and Impact
Since its launch in 2010 , PPMI has changed how research is done and what scientists know about the brain . It is a cornerstone of our understanding of disease and has heavily influenced clinical trials . Here , we share just a few examples of the study ’ s impact for the research field so far .
Measures of disease progression : Objective tools to track disease and assess the impact of therapies on progression are critical to testing new treatments ( see page 12 ). PPMI findings are pointing to potential measurements of disease in the body and various methods to collect this critical information :
Over their first four years in PPMI , volunteers recently diagnosed with Parkinson ’ s showed a 28 percent reduction in dopamine activity on a brain scan . Some trials are using this scan to test if therapies slow that dopamine loss , which would help prove if the therapies can effectively slow disease progression .
In people with Parkinson ’ s , blood levels of neurofilament light chain ( NfL ) — a protein that ’ s released when brain cells are damaged — increased over time and correlated with motor symptoms and disease progression . This may be another tool to assess if a treatment is slowing disease .
Largest disease-specific dataset : In 2019 , PPMI released 108 terabytes of data — equivalent to 47,520,000,000 single-spaced typed pages — from its RNA Sequencing Project . The project profiled each PPMI volunteer ’ s RNA , which functions as a messenger from genes to create proteins , at different time points . The data is being used by scientists to explore genetic changes linked to Parkinson ’ s and the impact of gene expression on age , disease progression and even medication use .
Success in recruiting niche populations : Many study sponsors are looking for specific populations — such as , recently diagnosed individuals and genetic mutation carriers — to enroll for years-long trials that may require intensive tests . PPMI has done just that , which gives confidence to companies running and funding these studies . The success of the PPMI model enables other intensive studies that may lead to new treatments .