On March 11, Dr. Mark Frasier, Senior Vice President at the Michael J. Fox Foundation, shared an update on the PPMI study. Listen to a recording of the webinar here. Dr. Frasier summarized some of the key accomplishments of PPMI to date.
Better understanding of UPDRS (Unified Parkinson’s Disease Rating Scale)
This was a relatively new scale when PPMI launched. There wasn’t much data about how this would change as disease progresses. PPMI information about how it evolves over time has already informed clinical trials running now, helping determine what sample size is needed to demonstrate change over time.
Dopamine imaging data
PPMI uncovered that yearly change is on average about 10% in newly diagnosed patients. This was previously unknown. We can see window over several years where dopamine imaging changes. This could potentially be used as a marker.
Cerebral spinal fluid
PPMI data is showing that there are changes in certain proteins in spinal fluid related to PD. At baseline, on average PD subjects have lower alpha synuclein compared to controls. Looking at 300 subjects at initial visit, 6 months and 12 month, it doesn’t look like it changes over time – so it might not be a marker of progression. But since there is work being done on drugs that target alpha synuclein, it could be helpful to see if that drug is changing the levels. We will be analyzing over 2,500 spinal fluid samples this year.
Non motor- studies looking at cognition and memory
PPMI data is showing and reinforcing understanding that ~20% PD individuals have minor cognitive changes in newly diagnosed PD. However that doesn’t seem to change over the initial years. So it is not necessarily early progression marker for the disease. We will be continuing to watch this throughout the study.
DNA – genetic analysis of samples
A research group at the NIH recently developed a genetic risk score. Looking at certain changes in DNA combined with certain clinical observations, they can predict who might have PD based on short list of features. As scoring system is refined, that may help enroll people at risk for the disease into trials focused on prevention.