What is PPMI?
PPMI is a landmark observational clinical study that aims to identify biomarkers of Parkinson’s disease (PD) progression by assessing multiple cohorts to develop the largest collection of clinical, imaging and biologic specimens ever created in the Parkinson’s community. PPMI data and specimens are made accessible through this Web site to academic and industry researchers to perform verification studies of PD biomarkers. The PPMI infrastructure is becoming a new model for biomarker research and will continue to add new cohorts. Click here to see a list of existing PPMI cohorts.
PPMI will be conducted in the United States, Western Europe and Australia. The study is sponsored by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and is made possible by restricted donations to the Foundation from a consortium of Parkinson’s drug development stakeholders. PPMI is led by Principal Investigator Ken Marek, MD, President and Senior Scientist of the Institute for Neurodegenerative Disease in New Haven, Connecticut.
What are biomarkers?
A biomarker can be any objectively measurable physical characteristic associated with the presence of disease (diagnostic marker), or any characteristic that changes over time in a way that can be tied to the progression of disease (progression marker). Examples from other health conditions include: Blood pressure, which is objectively and easily measured and, when high, acts as a diagnostic marker of potential heart disease; and, white blood cell count is a progression marker of leukemia. A count that rises to abnormal levels indicates that the condition is getting worse, while a count that returns to normal indicates improvement.
PPMI Data and Specimens
What tests and assessments will be performed on subjects? What data and specimens will be made available through PPMI?
Study participants will be required to complete motor assessments, DaTSCAN™ and MRI imaging, serum, plasma and urine collection, CSF collection, olfaction testing, DNA testing and neuropsychiatric/cognitive testing. All test data and specimens collected as part of this protocol will be submitted to the central study repositories and will be made available through this site.
Who has access to PPMI data and specimens?
Investigators from academic institutions and industry organizations will be allowed to access the repository. Investigators are required to submit basic information about themselves for basic administrative review to ensure legitimacy. Upon approval, investigators will be given immediate access.
What kinds of research can be conducted on the data and specimens?
PPMI data will be available to any researcher who is approved for access to the database. Because PPMI specimens are in limited supply, they will be reserved for biomarker verification studies – or studies to further validate lead marker candidates. PPMI samples cannot be used for discovery research, although other banks of samples exist for this purpose.
Eligibility and Enrollment
Who can take part in PPMI?
Having completed enrollment of the original newly diagnosed and healthy control cohorts in April 2013, PPMI now seeks particpants for the prodromal cohort. PPMI now requires participation of individuals who do not have PD who have REM sleep behavior disorder, a smell deficit or carry a LRRK2 genetic mutation. Participants must be at least 60 years of age.
What is the visit schedule for individuals who enroll in the study?
Participants will be required to make four study visits in their first year, with visits twice a year thereafter. Each visit will include different tests, assessments and biologics sampling, including motor, neuropsychiatric and cognitive assessments; DatSCAN and MRI imaging; and, blood, cerebrospinal fluid (CSF), urine and DNA sampling.
Do participants have to go to a PPMI site to participate in the study?
Yes. Because the study has been designed with highly standardized protocols that must be strictly adhered to across study sites, enrolled subjects must visit an official PPMI clinical site to conduct all study visits.
PPMI Sponsorship and Funding
Is the pharmaceutical industry involved in this study?
Although MJFF is the sole sponsor of PPMI, the Foundation is developing this project as a consortium effort. To this end, MJFF is actively recruiting funders for the study, including industry funding partners. In fact, the Foundation aims to secure half of the needed funds for PPMI from pharmaceutical companies and individuals who share our commitment to developing better tools for drug development. In seeking these partnerships, MJFF is clear that becoming a funding partner in PPMI will not benefit any single entity directly. Rather, the discovery of a PD biomarker is pre-competitive, providing equal benefit to all drug developers working in PD by enabling faster progress toward breakthrough treatments.
Why hasn’t anyone done a study like PPMI before?
In short, everyone acknowledges that the discovery of a biomarker would be transformative to the work being done in the field. However, biomarker development demands significant financial and intellectual resources and offers little short-term return on investment (compared to, say, a patentable new therapy). For individual academic or industry research groups, such a major undertaking does not offer the short-term rewards and long-term competitive advantage often used to assess business decisions to commit resources.
In light of the urgent need for a Parkinson’s biomarker and the ambitious goal to meet that need through PPMI, it is fitting that multiple partners mobilize under the umbrella of a single streamlined and coordinated effort, sharing the expense, risk and, ultimately, the benefits of the research. MJFF is committed to spearheading efforts to accelerate the development of improved therapies for those living with Parkinson’s today. Given this aim, MJFF is uniquely poised and positioned to lead the push and marshal the necessary resources to make PPMI a reality.
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