THE FOX FOCUS...
ON RESEARCH
POTENTIAL DISEASE-MODIFYING THERAPY
ADVANCES TO PHASE III TRIAL
A new partnership focuses on innovation in drug discovery
and accelerating pipeline progress.
by allyse falce
8 THE FOX FOCUS
as a Parkinson’s treatment since
pharmaceutical companies are not
incentivized to invest in a compound
already on the market. People with
Parkinson’s should also discuss taking
inosine and any other over-the-counter
medications or supplements with their
physicians. Inosine could cause serious side
effects, including gout, kidney stones and
even high blood pressure.
‘‘
The Foundation’s early and
step-wise investment has
not only helped us reach
this advanced stage of
testing for disease
modification. The
knowledge gained with
its support also greatly
enhances the prospects for
success of the Phase III trial
‘‘
I
nosine, a nutritional supplement and
precursor to the antioxidant urate,
has shown potential as a therapeutic
option that could slow the
progression of Parkinson’s disease.
Observational studies have shown people
with higher levels of urate have lower risk
of Parkinson’s disease (PD), and, if diagnosed with PD, slower disease progression.
In 2008, The Michael J. Fox Foundation
awarded Michael Schwarzschild, PhD,
of Massachusetts General Hospital $5.6
million (the largest MJFF grant given to
a single investigator thus far) for a Phase
II inosine trial. This study showed that
inosine is safe and tolerable and raises urate
levels in people with early-stage PD.
Based on this, researchers announced
funding from the National Institutes of
Health in September 2015 to conduct a
Phase III trial on inosine to determine its
effect on Parkinson’s disease. Enrollment
of 270 people with early-stage Parkinson’s
at 60 U.S. clinical sites is expected to begin
in early 2016.
“The Foundation’s early and step-wise
investment has not only helped us reach
this advanced stage of testing for disease
modification. The knowledge gained with
its support also greatly enhances the
prospects for success of the Phase III
trial,” said Dr. Schwarzschild.
While inosine is currently commercially
available as a dietary supplement, it has not
been proven as a therapy for Parkinson’s.
Foundation and government funding is
therefore essential for testing its efficacy
Inosine moving forward in clinical
testing represents researchers getting a
better understanding of the mechanisms
of disease, patients potentially having a
disease-modifying therapy and MJFF’s
continued dedication to the pursuit
of a cure.
The Sherer Report continued from page 3 ›
also highlight a great challenge. Lack of a
biological marker of Parkinson’s disease
prevents efficient and confident clinical
testing of even the most promising
therapies.
MJFF has prioritized the identification
of PD biomarkers since our earliest days,
and 2015 marks the five-year anniversary of
our landmark study toward that end. The
Parkinson’s Progression Markers Initiative continues to follow volunteers and is
enrolling new participants. Initial results
are shedding light on potential biomarkers
and teaching us more about the nuances of
this variable disease. Read more about what
the study has accomplished and how it will
continue on page 4.
This year we attained several vital mile
markers on the road to a cure, but there is a
ways to go. With your support, the Foundation will keep working hard to accelerate
our pace, clear the roadblocks and together,
reach our destination.
Biomarkers continued from page 5 ›
and without PD), and investigators believe
this group has much to teach them about
the many paths to Parkinson’s disease and
the many ways we may measure and stop
progression.
It is hard to believe that some of the
study’s first volunteers are reaching the
end of their original commitment. The
initial PPMI design called for five years of
participation, but organizers recently
announced plans to invite these individuals
to extend their time in the study.
“The participation of our volunteers has
proved so valuable already, and we know
there is much more to discover,” said
Dr. Marek. “Having come this far, we are
only more invigorated about the
possibilities of continuing to leverage
the PPMI infrastructure. We hope many
participants will choose to continue their
study visits past the five-year point.”
As he said in 2010, research is risky.
The stakes are too high, though, not to try.
Online Research continued from page 2›
A portion of the study is recruiting
participants with Parkinson’s who also own
an Android smartphone (e.g., Samsung
Galaxy, Motorola DROID) and giving
them a free smartwatch. An app records
patients’ daily movements and streams it to
Fox Insight. Part of MJFF’s collaboration
with tech giant Intel, this allows patients
to contribute data without any demands
on their time.That objective data on motor
fluctuations pairs with the online questionnaires to create a more robust personal
profile of one’s PD.
More than 250 individuals are already
participating in this sub-study, and MJFF
plans to expand the program to include
iPhone users in the coming months.
For more information on the Fox Insight
Wear program, visit michaeljfox.org/
wearfoxinsight/enroll.
While technology is fundamental to
Fox Insight, its success relies on the
contributions of the entire PD community.
The power of this research model comes
from tens of thousands of people sharing
their experiences over time. We hope you
will join Fox Insight as the big data
movement revolutionizes what we know
and how we approach new therapies and a
cu ɔ