The Michael J. Fox Foundation Annual Report 2017 – Roadmaps for Progress | Seite 8

The Michael J. Fox Foundation
2017 Annual Report
Roadmaps
for Progress
Our roadmap approach layers
strategic funding and non-
financial resources around targets,
pathways and symptoms to deepen
understanding of Parkinson’s and
speed translation.
Cohorts
All therapeutic development
begins with the study of the
human disease.
MJFF’s extensive constituency comprises
more than one million patients, families
and supporters — the most active and
engaged community in all of Parkinson’s.
We leverage this network to assemble
and fund cohorts of populations of
interest (e.g., those with key genetic
mutations, or the newly diagnosed). This
work significantly speeds the process of
gathering well-characterized data and
samples to enable greater understanding
of Parkinson’s, new measurement
methods and next-generation therapies.
The Foundation also is assembling broad
cohorts of potential research volunteers
mobilized to participate in Parkinson’s
studies and trials.
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Tools Biomarkers
Freeing researchers from making
their own tools leads to better
and faster results. Objective disease
measures speed
drug development.
The Foundation pioneered a new model
for creating and distributing critical
Parkinson’s research tools through
direct funding to contract research
organizations and field experts. This
innovation has freed scientists and drug
makers from years and millions of dollars
devoted to tool making so they can focus
on driving scientific discovery. MJFF also
characterizes the best methods and uses
for these tools to encourage replicable
research. In the clinic, our recruitment
tools and online Fox Trial Finder
matching tool help study sponsors enroll
volunteers, addressing the systemic
problem of slow recruitment. MJFF leads a landmark public-private
partnership, among other efforts, to
identify and validate candidates for
Parkinson’s biomarkers (objective
indicators of disease such as blood
sugar and diabetes) and develop tests
to measure Parkinson’s pathology
and symptoms. Toward these goals,
researchers are looking at biological
factors such as protein levels in addition
to phenotypic measures including, for
example, eye movement and activity
levels (via wearable devices). Objective
disease tests would speed drug
development by identifying people most
likely to respond to treatment, tracking
disease progression and assessing
therapeutic impact.
Biology
Characterization of
disease is the backbone
of all research progress.
MJFF funds basic and translational
science to identify and characterize
disease biology, vital to measure
Parkinson’s onset/progression or
target the disease with new treatments.
Exploring cellular functions, defining
protein structures, and studying
pathological mechanisms in the presence
of genetic mutations tee up field-wide
advances in therapeutic experimentation
and optimization. The Foundation
pumps tens of millions of dollars a year
into this critical work.
Therapies
Transforming early-stage
ideas into therapies
requires strategy.
The Foundation’s donor-raised capital
behaves differently from that of
commercial or government research
funders in that tangible patient benefit
is the only ROI we seek. This frees,
indeed obligates, us to seek out and “de-
risk” pre-clinical therapeutic studies by
helping assemble the data required to
attract bigger funders who can advance
these projects through more expensive
later stages of testing. In addition,
MJFF funds trials of repurposed drugs
approved for other conditions but
that have shown evidence in treating
Parkinson’s disease.
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