September 23, 2013 | by Darcy Blake | The Michael J. Fox Roundtable included a four-speaker discussion of Parkinson’s Disease moderated by Dave Iverson, NPR journalist and contributing editor at MJFF. The title of the discussion was Speeding Breakthroughs for People with Parkinsons’s disease: MJFF’s Model, Role and Impact.
Deborah W. Brooks, MJFF Co-Founder
The evening began with an introduction by Deborah W. Brooks, co-founder of The Michael J. Fox Foundation, who praised the MJFF team of neuroscientists whom she described as those who “devour science.” She explained how MJFF has now paired them with folks focused on deliverables, and she described the exciting research they are providing in spite of the time (15 to 30 years) and money (one billion dollars) it takes to bring a drug to market.
The other roundtable speakers were Melanie M. Brandabur, MD, The Parkinson’s Institute, Philip A. Starr, MD, UCSF Department of Neurological Surgery and Ryan Watts, Phd, Genetech.
Dr. Melanie Brandabur, Motor Disorder Specialist
Dr. Melanie Brandabur, described the work of motor disorder specialists as “those always trying to ride the wave” and “smooth the bump” between medication on and off times. We now actually “under-medicate in general, particularly in California where everyone wants holistic treatment, because we’re so fearful of dyskinesia,” she explained. Dr. Brandabur discussed secondary symptoms such as swallowing, speaking difficulties, cognitive impairment, hallucinations and G.I. problems, and new remedies for them. “Partnership” between patient and doctor is necessary to make it all work, she added.
Ryan Watts, Medical Researcher
Watts concurred with Dr. Brandabur on the subject of dyskinesia and he said that treating it is one of the most robust parts of their program. He discussed that in order to avoid dyskinesia, they are working on an inhaler treatment that would avoid the digestive system for a more sustained benefit. He included the work researchers are doing to slow down the disease by targeting the cause. He mentioned research with the protein alpha synuclein, and the first trial of vaccine which is currently going on in Europe. He also talked about the protein kinase function that makes LRRK2 a target.
Dr. Philip Starr, Neurosurgeon
Dr. Starr discussed how there has been a recent early stem study of 250 patients who have had PD for 5-7 years prior to surgery since the initial early studies of an older group of DBS patients, who had PD for 10-15 years prior to surgery. Based on this study, research seems to indicate that DBS is effective for patients who are younger in disease progression than originally presumed. He discussed the small number of possible complications from having DBS, and why PD patients don’t just start out with DBS before trying medications.
Dave Iverson, Moderator
Dave Iverson compared signing up for trials to “online dating.” You put your name out there, and see who gets back to you, but you don’t have to go out with them,” he said. This is your doorway into helping with research.
There are 72 active trials seeking 2,000 volunteers in California, Brooks said, and promoted foxtrialfinder.org, an online site that connects volunteers in the PD community with clinical trials. “I am personally dedicated to as many meeting as many PD patients as possible. If I could sign them up for a trial, I’d be even happier,” she said.
Iverson wrapped up the evening with a final question from the audience, “what brings you excitement about current PD research?” Ryan said he’s really excited with at least four studies that Genetech is doing which focus on being able to treat PD through biomarkers, including their work with alpha synuclein. Dr. Brandabur predicted that many things will coalesce in the future including the work of Parkinsons’s Institute genetic researchers Dr. Birgitt Schuele (its all in her petri dish) and Dr. Jeremy Nichols in his LRRK2 research. Dr. Starr say that although DBS therapy has changed little in the past 20 years, there is some new research using brain signals to control patient motor symptoms that is now on the horizon. A very informative and positive evening for all of us with PD.
MICHAEL J. FOX FOUNDATION FOX TRIAL FINDER: https://foxtrialfinder.michaeljfox.org