Fox Focus I Spring / Summer 2021 11
Research Briefs
Dopamine-producing neurons derived from stem cells .
( PHOTO COURTESY OF : NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES )
Clinical Trials Testing Replacement Dopamine Neurons
In Parkinson ’ s disease , brain cells making dopamine — a chemical that coordinates movement — stop working or die . Biotech BlueRock Therapeutics in Cambridge , Massachusetts , is developing cell therapies to replace the dopamine that is lost in Parkinson ’ s to help alleviate symptoms such as tremor , slowness , stiffness , and walking and balance issues . It has begun a trial testing the safety of implanting dopamine neurons made from engineered stem cells in the brains of 10 people with advanced Parkinson ’ s . In previous laboratory models , the stem cell-derived neurons produced and released dopamine as they matured in the brain and eased motor problems . The Foundation funded early work to develop replacement dopamine neurons from stem cells , which has made trials such as this possible .
Drug to Stop Parkinson ’ s Shows Promising Results
A drug to lower the activity of the LRRK2 protein — which is elevated in some people with Parkinson ’ s — has shown positive results in human trials and will progress to the next stage of testing later this year . In early May , biotech Denali Therapeutics in South San Francisco , California , announced final data from its Phase I trials of BIIB122 / DNL151 . The drug was safe in volunteers with and without Parkinson ’ s . Scientists also were able to measure altered LRRK2 activity , showing that BIIB122 / DNL151 is changing biology as intended . Next , larger studies will continue to assess safety as well as evaluate the effect of the drug on Parkinson ’ s symptoms and progression . MJFF has accelerated progress in LRRK2 drug development by bringing together research groups to share resources and troubleshoot common problems , which has been critical to advances such as this .
A molecular model of the LRRK2 protein , which is overactive in people with Parkinson ’ s . Models help scientists design drugs that can modify protein activity . ( PHOTO COURTESY OF : ANDRES LESCHZINER , UNIVERSITY OF CALIFORNIA , SAN DIEGO )