THE FOX FOCUS...
ON RESEARCH
EXPANDING THE SEARCH FOR BIOMARKERS
The Parkinson’s Progression Markers Initiative marks five years of
progress toward critical objective measures.
Building a Framework for Success
Now at 33 clinical sites on three
continents, PPMI has united thousands
of people with Parkinson’s, control
volunteers, researchers, site teams and
industry partners toward the common goal
of a yardstick against which to measure
Parkinson’s risk, onset and progression.
In a remarkable timeframe — given the
usually slow pace of recruitment for
research studies — PPMI enrolled core
populations of recently diagnosed PD and
control participants within its first
three years.
PPMI has expanded to include people
who have motor symptoms but no evident
dopamine loss as well as people with
known PD risk factors of smell loss and
REM sleep behavior disorder. In 2014,
the study launched a genetics arm
studying people with or without PD who
carry certain mutations associated with PD.
The impact of PPMI is felt globally.
Thanks to the study’s open-access design,
de-identified participant data and
biospecimens are available in real time to
4 THE FOX FOCUS
qualified researchers everywhere, helping
speed the pace of discovery across the
field. Neuroscience investigators have
enthusiastically taken up these
unprecedented resources. In only five
years, researchers have downloaded PPMI
data more than 428,154 times and
submitted 63 biospecimen requests.
“There are valuable insights to uncover
within these data,” said MJFF CEO Todd
Sherer, PhD. “We are grateful to all who
are helping us build this deep inventory,
and we are invigorated by the initial
findings out of PPMI.”
Uncovering Insights on Parkinson’s Risk,
Onset and Progression
Through the study, researchers are
gaining new knowledge about risk factors
for the disease, molecular changes in brain
and body cells that may correlate to PD
onset and progression, and the clinical
symptoms of Parkinson’s.
In August, geneticists from the National
Institutes of Health (NIH) reported
findings using PPMI data showing a
constellation of five factors — smell
function, genetic risk, family history of
PD, age and gender — that, taken
together, were able to differentiate people
with Parkinson’s from those without PD.
Evaluating people at risk for PD with these
criteria may lead to the field’s first
opportunity to predict who will develop
Parkinson’s disease, something that has
never been possible before.
“This study would not have been
possible without PPMI’s comprehensive
data and open-access design,” said lead
investigator Andrew Singleton, PhD, of the
NIH National Institute on Aging. “The
value of PPMI is two-fold: first that the
data is there, and second that it’s
demonstrated to study planners the importance of releasing your data and making it
publicly available.”
Toward its ultimate goal of validating a
biological marker of Parkinson’s
progression, PPMI has found significant
differences in the spinal fluid of people
Planning for the Next Wave of Discovery
As PPMI marks its fifth anniversary,
study leadership continues to think big
and invest time and resources in
ambitious plans. The genetics arm is
currently enrolling people with PDassociated gene mutations (people with
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Third Thursdays Webinars and
Friday Twitter Chats
Join MJFF and expert panelists for live webinars at 12 p.m. ET on the third Thursday
of every month. Learn about the various aspects of living with Parkinson’s disease
and the Foundation’s work to speed medical breakthroughs. Visit michaeljfox.org/
webinars to learn more, watch previous webinars or to register for an upcoming one.
STILL WANT TO LEARN MORE? The Friday following each Third Thursdays
SAM OGDEN
I
n September 2010, when MJFF
launched its landmark biomarker
study, the Parkinson’s Progression
Markers Initiative (PPMI), principal
investigator Ken Marek, MD, said, “While
there is no guarantee that validated
biomarkers will result from the PPMI
study, their im