The Michael J. Fox Foundation Annual Report 2017 – Roadmaps for Progress | Page 6

The Michael J. Fox Foundation
2017 Annual Report
Annual Letter from
the CEO and the
Co-Founder
Todd Sherer, PhD, (right) with
David Standaert, MD, PhD, at the
annual PD Therapeutics Conference
Debi Brooks with Michael J. Fox
at an MJFF salon sharing research
progress
Dear Friend,
Understanding Parkinson’s disease is the starting point of every
effort to accelerate the cure. Years of work spent uncovering
Parkinson’s secrets — defining the highly variable patient
experience, shedding light on genetic origins of disease,
mapping molecular pathways — are now paying off in a tangibly
quickening tempo of progress. Researchers are increasingly
linking pathology to clinical symptoms (and vice versa) to
inform therapeutic target and biomarker identification.
4
This has positioned drug makers to
make rapid inroads toward treatments
with potential to slow, stop and perhaps
even prevent Parkinson’s disease (PD)
symptoms and cell loss. 2017 saw the
launch of the first precision medicine
clinical trials targeting genetically
defined forms of Parkinson’s disease
(LRRK2 and GBA). Numerous therapies
acting on other pathologies, or auguring
improved management of disabling
symptoms, continued through clinical
trials, pushing ever closer to market.
Multiple consortia and countless
investigators worked in tandem to fill in
ever-larger sections of the Parkinson’s
puzzle, characterizing cellular pathways
and linking outward dysfunction
to underlying disease progression.
A cornerstone of these efforts, our
Parkinson’s Progression Markers
Initiative (PPMI), is influencing clinical
trial design and vastly improving
understanding of the natural history
of disease. Today, researchers are
maximizing the value of precious PPMI
samples through cutting-edge “omics”
analyses, techniques that are speeding
2017
Progress
in Drug
Development
the identification of novel biomarker
candidates and the nomination of
improved therapeutic targets.
And, as demonstrated in part by the
willingness of patients and families to
participate in PPMI, The Michael J. Fox
Foundation (MJFF) also has invested
in building more onramps for engaging
the PD community. In October 2017, we
launched Fox Insight (foxinsight.org) —
our online clinical study that is gathering
patient-reported data on experience
of PD from, at time of printing, nearly
20,000 people with PD and their loved
ones. The study aims to enroll hundreds
of thousands of people amplifying the
patient voice in Parkinson's research.
In collaboration with 23andMe, Fox
Insight is also capturing genetic data for
continued biological discovery, matching
genotype to phenotype.
While 2017 was a year full of progress
and promise in Parkinson's research,
much work remains to reveal the
molecular fingerprint of the disease,
draw lines between pathological bad
actors and physical manifestation, and
The Parkinson’s drug development pipeline
is abuzz with activity, and new therapies to
treat motor and non-motor symptoms are
market-bound.
Two new Parkinson’s drugs hit pharmacy
shelves in 2017. Newron’s Safinamide
(Xadago), an add-on therapy for those
dealing with persistent symptoms despite
levodopa, received U.S. Food and Drug
Administration (FDA) approval in March.
And the first drug specifically indicated for
levodopa-induced dyskinesia — Adamas’
Gocovri, an extended-release formulation
of amantadine — arrived in August. MJFF
enabled the drug on its path by providing
strategic leadership and $1 million in funding
for the development of a rating scale
instrumental to testing dyskinesia drugs in
transform rapidly accumulating insights
into reliable measures and curative
therapies. This information is critical
to speed development of therapeutics
already under way as well as those still
to be discovered. On the following
pages, we share the Foundation’s
roadmap approach, designed to enable
and structure this work in order to
accelerate knowledge turns.