The Fox Focus Fall/Winter 2016 | Page 3

Category Research Research With Fox Insight — our Foundation’s online observational study — you can share information about symptoms, family neurological history and quality of life by completing online questionnaires every 90 days. Studying the anonymized responses of thousands of volunteers over time will help researchers get a better sense of how Parkinson’s disease (PD) progresses differently across individuals and develop meaningful new therapies for people with PD. More than 4,800 Fox Insight participants (and counting) are actively contributing to Parkinson’s disease research and have completed over 11,000 study visits. Anyone, including those without Parkinson’s disease, can participate to accelerate research and help speed a cure. Take 10 minutes to enroll today at foxinsight.org. Who’s Using Fox Insight: YES 78% NO 22% WITHOUT PD MOST COMMONLY REPORTED SYMPTOMS (includes those with and without PD) Motor symptom: 46% 52% 72% 25% 2% did not specify 3% did not specify AVERAGE AGE 60 AVERAGE AGE 49 by MAGGIE McGUIRE KUHL New results add to the promise of a “vaccine” against the key Parkinson’s protein alpha-synuclein, which researchers hope may slow or stop disease progression. Alpha-synuclein clumps in the cells of nearly everyone with Parkinson’s disease, and scientists believe those aggregates are toxic and cause cell death that leads to symptoms and progression. A therapy that stops alpha-synuclein toxicity may stop Parkinson’s in its tracks. HAS PD WITH PD Photo by Sam Ogden YOUR DAY IS OUR DATA Harnessing the Body’s Natural Defenses to Fight PD SHAKING OR TREMORS Non-motor symptom: DAYTIME SLEEPINESS some who did not react in the first trial produced antibodies with the boost. “This showed that the body is not desensitized to the vaccine and can produce alpha-synuclein antibodies again,” said Kuldip Dave, PhD, who directs MJFF grantmaking in alpha-synuclein. “And that you don’t have only one opportunity for treatment — that if you don’t produce antibodies with a first round of vaccine, you may with follow-up.” Vienna, Austria-based biotech AFFiRiS is testing a drug (called PD01A) to cause the body’s immune system to create alpha-synuclein antibodies. The treatment works like the flu vaccine, activating the body to generate its own natural disease fighters. The big question is if PD01A will slow or stop Parkinson’s disease. Trials are not designed to test efficacy (there is no placebo, for example), but preliminary observations are promising. Many antibody responders from the first trial (42 percent) did not need to increase dopamine medication over the study period (an average of three years). Now researchers will work to assess efficacy. In a 2011 trial, 24 participants with early-stage Parkinson’s received PD01A. That trial showed the treatment was safe, and 50 percent of participants created alpha-synuclein antibodies. Within a year, however, the “responders” saw antibody levels decline, leading the researchers to test a “boost” dose one year later. In early September the company announced its “boost” study — funded with $1.04 million from The Michael J. Fox Foundation (MJFF) — showed an additional vaccine dose is safe and can elicit antibodies against alpha-synuclein. “Our Parkinson’s Disease Phase I boost study data are providing an encouraging signal of target engagement,” said Vera Bürger, head of the AFFiRiS Clinical Department. “AFFiRiS is proactively engaging with the expert community for planning of next steps.” AFFiRiS reported that all volunteers completed the study with no safety concerns. In addition, all responders from the first trial responded again, and Beyond AFFiRiS, four other companies also have therapies in clinical trials that aim to prevent, break up or clear out clumps of alpha-synuclein. 2 3 The Fox Focus Fall/Winter 2016