The Michael J. Fox Foundation Annual Report 2016 – Frameworks for Progress | Page 17

“Cures” — that is, treatments that could slow, stop,
reverse or even prevent Parkinson’s progression,
something no current treatment has been proven to do
— remain patients’ most urgent unmet medical need.
Researchers refer to this kind of treatment as “disease-
modifying,” and three-quarters of The Michael J. Fox
Foundation (MJFF) funding is devoted to its pursuit.
As in many diseases since the completion
of the Human Genome Project in
2003, Parkinson’s research has been
transformed by genetic discoveries. But
these findings are meaningful to patients
only when methodically translated into
practical treatments. MJFF is working to
ensure that translation moves as quickly
and efficiently as possible.
— Alpha-synuclein continues to earn its
status as arguably the highest priority
target in Parkinson’s disease (PD)
research with five patient clinical trials
moving through or completing Phase
I safety trials. In 2016, an experimental
“vaccine” approach, developed by
MJFF-awardee AFFiRiS (an Austrian
biotech) reached a new milestone
by showing the treatment can
successfully harness antibodies against
this protein that forms abnormal,
toxic clumps in the brains and bodies
of people with Parkinson’s.
— Researchers are currently working on
therapies to address GCase dysfunction
in people with GBA (glucosidase, beta,
acid gene) mutations. In late 2016, an
MJFF-funded team reported that GBA
mutations put Parkinson’s patients
at higher risk of cognitive decline,
particularly in the pr esence of certain
“severe” types of GBA mutation. These
findings and other current initiatives
may inform how we develop therapies.
In January 2017, the first trial of a
drug developed by Sanofi Genzyme,
began in people with GBA mutation.
Notable for its potential to treat PD,
the study also represents a major step
forward for personalized medicine in
Parkinson’s disease.
— Industry leaders continue to take notice
of MJFF’s efforts to keep LRRK2 a
priority target. MJFF has applied its
comprehensive roadmap strategy to
LRRK2 through a consortium-based
approach to investigate biology, fund
therapeutic development, create
research tools and recruit individuals
with LRRK2 mutations to participate
in clinical studies. Dozens of academic
laboratories and several pharmaceutical
companies partner with MJFF on active
LRRK2 programs, and clinical testing is
expected by 2018. In 2016, a new biotech
company Denali launched with LRRK2-
based treatments for Parkinson’s core to
its strategic business plan.
In addition to the tremendous promise
of genetics, MJFF is pursuing multiple
therapeutic strategies that work on
non-genetic targets, primarily through
repurposing treatments already approved
for use in other diseases.
— MJFF has helped drive investigations
and trials on several drugs including
isradipine (a drug used to treat high-
blood pressure that may prevent the
death of dopamine-producing cells);
inosine (a dietary supplement that the
body converts into the antioxidant
urate); exenatide (a Type 2 diabetes
drug that could possibly improve
physical and cognitive functions); and
nilotinib (a leukemia drug that could
possibly reduce Parkinson’s-related
toxicity). As evidence and efficacy for
the drugs to treat symptoms of PD
safely are known, the opportunity and
timeline to accelerate for PD patient
use is shortened because it is already
available in the market.
2016 Annual Report
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