6 Fox Focus | Research
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U.S. Food and Drug Administration (FDA)
to treat the symptoms of Parkinson’s —
these treatments represent a wide array of
approaches. Two new drugs crossed the
finish line in 2019 alone. This summer, the
FDA approved Nourianz (istradefylline), a
new medication for Parkinson’s “off” time,
when symptoms return between medication
doses, and Myobloc (rimabotulinumtoxinB)
for chronic or excessive drooling in PD.
Late last year, Inbrija, another therapy for
addressing “off” episodes in Parkinson’s was
approved. MJFF provided early support for
Inbrija and for another “off” time therapy, an
under-the-tongue strip of apomorphine from
Sunovion, which is currently being reviewed
by the FDA.
At MJFF, our in-house team of scientists
is focused on identifying and accelerating
the development of therapies targeting
critical stages and symptoms of PD.
Therapies addressing motor and non-motor
symptoms that can impact quality of life
such as movement challenges, cognitive
impairment, gait and balance issues, and
mood changes, to name a few, are something
MJFF actively monitors and funds as part of
annual programing.
Toward a Cure
We are also making extraordinary strides in
therapies that target the underlying biology
of Parkinson’s, aiming to slow or stop its
progression so symptoms don’t get worse.
Today, the most dynamic target area is
alpha-synuclein. In people with Parkinson’s,
this protein clumps into Lewy bodies that
can damage our brain cells. A vaccine from
Austrian biotech AFFiRiS that received
MJFF-monitored
Priority Clinical Programs
September 2019
52
Disease-
Modifying
Therapies
46
Symptomatic
Therapies
financial support from MJFF entered clinical
studies in 2012. A treatment from Prothena
(now partnering with Roche) followed in 2014,
with a different antibody from Biogen entering
in 2015. Today, 11 therapies targeting alpha-
synuclein are in human clinical trials and one
more is expected to begin a Phase I trial soon.
MJFF has supported six of these through direct
financial investment or by providing resources
to help the trial succeed.
Another priority target area in the pipeline is
GBA. Mutations in the GBA gene increase a
person’s risk of Parkinson’s. The first GBA
trial, for a repurposed drug called ambroxol
(used to treat respiratory diseases), entered
trials in 2015. There are now up to four GBA-
targeted therapies that have reached the
clinic. Two of these received MJFF support.
We funded 15 other GBA projects this year
through our partnership with The Silverstein
Foundation for Parkinson’s with GBA. These
critical investments will help more GBA-
targeting therapies reach human clinical
trials in the future.