Research
SEVERAL THERAPIES IN DEVELOPMENT
FOR “OFF” TIMES
TESTING NEW
THERAPIES REQUIRES
THE RIGHT TOOLS
When medication, namely levodopa, is not
working optimally and Parkinson’s symptoms
return, “off” time occurs. These periods
may come on gradually before the next
medication dose is due (i.e., “wearing off”), or
suddenly and unpredictably.
by MAGGIE McGUIRE KUHL
There is currently only one medication
available to quickly relieve sudden “off”
periods, but two MJFF-backed treatments
may soon change that. Sunovion’s
dissolvable, under-the-tongue apomorphine
strip (an updated version of the current
treatment, which is administered via
injection), is showing positive results in Phase
III trials. And Inbrija, an inhaled levodopa
formulation from Acorda, is expected to go to
the FDA for review in the next few months.
There are many challenges to advancing
new treatments, including having the
right tools to assess therapeutic impact.
Parkinson’s symptoms vary from person
to person and even hour to hour, making
it difficult to measure the effects of new
medicines. Supporting development of
scales to evaluate disease and treatment
benefit is one way The Michael J. Fox
Foundation (MJFF) brings new therapies to
patients faster.
To reduce total daily “off” times and
dyskinesia, Foundation-funded NeuroDerm is
advancing Phase III trials of liquid levodopa/
carbidopa. This medication is infused
continuously under the skin through a pump,
similar to the insulin pump for diabetes.
MJFF funded validation of the Unified
Dyskinesia Rating Scale with a $1 million
grant in 2009. This tool measures dyskinesia
(uncontrollable, jerky movements that
are a side effect of levodopa use), which
lets researchers test whether dyskinesia
drugs are working. The scale’s availability
encouraged more companies to work on
these types of therapies.
ADVANCES IN DEEP BRAIN STIMULATION
In 2016, the FDA expanded the indication
for deep brain stimulation (DBS) — a surgical
therapy in which thin wires are placed in the
brain to deliver electrical pulses and decrease
PD motor symptoms. The procedure now is
approved for people earlier in their course of
disease (four years versus five as previously
required) and who have “off” times and/or
dyskinesia.
“There was nothing on the market and
no drugs in the pipeline for dyskinesia.
And when I spoke with company
representatives, I got the feedback that
there wasn’t a good measure, so why
should they go into that area?” says
Christopher Goetz, MD, of Chicago’s Rush
University, who led creation and testing of
the Unified Dyskinesia Rating Scale.
Researchers now are looking to deliver
stimulation on demand, when symptoms
occur, rather than continuously as the
current systems do; use DBS in earlier
stages of disease to perhaps prevent motor
complications; and stimulate different
areas of the brain to treat a broader array of
symptoms.
Company Adamas used the scale to assess
its drug Gocovri, recently approved for
levodopa-induced dyskinesia.
Hear more about this dyskinesia scale and
the critical role that research tools play
in drug development from Dr. Goetz in
a recent podcast. Visit michaeljfox.org/
podcasts or search “The Michael J. Fox
Foundation” in your podcast app.
Stay tuned to learn more about Parkinson’s drug
development progress at michaeljfox.org/
therapiesindevelopment.
9
Fall/Winter 2017