I write to a patient audience every day. My press releases, blog posts and newsletter articles all grow out of our core message that weíre moving research forward to develop better treatments for patients, and I relish every chance I get to meet the people who are benefiting from breakthroughs.
Saturday I had the opportunity to talk with people with Parkinsonís when I traveled south to Baltimore for a luncheon for the participants of the Parkinsonís Progression Markers Initiative ó a landmark biomarkers study sponsored by The Michael J. Fox Foundation. Dozens of research volunteers came to hear what was new out of PPMI and to ask questions of the principal investigator at Johns Hopkins University, Zoltan Mari, MD, and of me and my colleague Vanessa. PPMI hosts such an event for the volunteers at every study site each year.
Saturday was a rare treat for me, and everyone I spoke to after said it was well worth their time, but maybe that was the chocolate cake talking.
PPMI patients and their loved ones ó many who are enrolled in the study themselves as controls ó talked before the presentation began, swapping stories about how Parkinsonís disease (PD) had impacted them, their family medical histories and how they learned about PPMI.
Vanessa gave a historical reminder of what PPMI is and why the Foundation is supporting this effort. [The CliffsNotes version is itís a large, multi-site study that collects samples and data from PD patients, people who may be at risk of developing PD and controls in an effort to identify and validate biological markers of disease or PD risk.] I introduced myself and touted our Webinar series and email communications as platforms for more regular updates.
Then Zoltan gave updates from PPMI:
- The study is enrolling people with PD risk factors: smell loss, REM sleep behavior disorder and certain genetic mutations.
- Initial analyses has uncovered interesting results, such as that levels of some proteins are lower in the spinal fluid of PD patients than in the spinal fluid of controls.
- Many researchers (affiliated with PPMI and not) are using the available data and biosamples to test their hypotheses. De-identified data from PPMI participants has been downloaded more than 130,000 times.
I knew most of that news, so I was more jazzed for the question and answer session to see what patients cared and wondered about. One man asked a basic but very important question: Where does PPMI fit into the overall Foundation research strategy?
I took this one, telling the crowd that the biomarkers PPMI is working toward are the foundation for all Parkinsonís research. Biological markers would tell us that weíre testing therapies with the right people and help us measure a drugís effect with objective measures rather than waiting to observe clinical symptoms. A reliable, validated Parkinsonís biomarker would accelerate efforts across other MJFF priority areas, bringing better treatments to market faster.
This week Iíve been back at it in New York, but that time to meet our partners in PPMI and to hear what they thought about the study and the research field was extremely motivating. Thanks for inviting me along, Vanessa and Zoltan!