The Fox Focus Fall/Winter 2018 | Page 6

Category Research Research News Briefs POSITIVE LRRK2 RESULTS IN HUMAN STUDIES In August, San Francisco biotech Denali Therapeutics announced that its experimental LRRK2 drug DNL201 was safe and met its target goals in a study in healthy volunteers. The company is now planning trials in patients with Parkinson's, including those with and without a LRRK2 mutation. An estimated three percent of people with Parkinson’s have a LRRK2 mutation, which increases activity of the LRRK2 protein. Recent findings from a Foundation-funded study showed that people with idiopathic (i.e., cause unknown) Parkinson’s also have increased LRRK2 activity, meaning drugs like Denali’s may work for a widespread population — not just those with the mutation. GENE THERAPY ADVANCES MJFF PARTNERS WITH IBM ON DATA MINING Two treatments that introduce genes into the brain to help boost what is lost with Parkinson’s disease have hit recent milestones. Voyager Therapeutics started a Phase II trial of its gene therapy to help the brain convert levodopa (Sinemet) to dopamine and control movement symptoms. Our Foundation funded early development of this therapy at the University of California, San Francisco. And biotech Prevail got a $75 million investment to advance its gene therapy to address GBA gene mutations, a leading genetic cause of Parkinson’s. A $1 million Foundation grant to IBM Research will apply sophisticated data analysis technologies to look for characteristics (e.g., genetic mutations, demographics, protein levels) shared in common among different Parkinson’s disease subtypes. IBM is combing data from the Foundation’s Parkinson’s Progression Markers Initiative study to create predictive models of, for example, fast progressors, or people who have more problems with tremor than with gait. These models could help researchers improve study design and select appropriate participants. 6 The Fox Focus