The Fox Focus Fall/Winter 2017 - Page 9

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 podcasts or search “The Michael J. Fox Foundation” in your podcast app. Stay tuned to learn more about Parkinson’s drug development progress at therapiesindevelopment. 9 Fall/Winter 2017