8 Fox Focus | Research
rather than the disease label— precision medicine has the potential to increase benefit, slow progression and / or ease symptoms.
Research into the complex biology of Parkinson’ s has been the goal of our landmark natural history study, the Parkinson’ s Progression Markers Initiative( PPMI), since it launched in 2010. At the study’ s annual meeting in May, the Foundation announced a new designation for the well-known abbreviation— the Parkinson’ s Precision Medicine Initiative. Read more at right about how scientific progress has led us to this moment in renaming the study.
“ We’ re moving closer to therapies that help keep people with Parkinson’ s stable longer and push off worsening or new symptoms,” said Andrew Siderowf, MD, chief of the Movement Disorders Division at the University of Pennsylvania and PPMI co-principal investigator.“ A PD diagnosis brings a question of‘ What’ s going to happen to me?’ With more disease markers and precision medicine, we can help answer that and bring hope.”
Borrowed Blueprint for Better Disease Outcomes
There’ s precedent for this approach: targeted treatments based on molecular characteristics have led to advances beyond a one-size-fits-all approach for conditions like breast and lung cancer. And in Alzheimer’ s disease, blood tests and brain scans have brought more targeted therapies.
Today, most approved Parkinson’ s treatments( drugs like levodopa / carbidopa or surgical approaches such as deep brain stimulation) help manage symptoms like stiffness and slowness. There are no approved disease-modifying therapies— treatments that target the underlying drivers of disease to slow or stop progression— but more than 75 such treatments are in clinical testing. Some of these studies are recruiting based on genetic factors, with MJFF providing funding, recruitment and / or strategy support:
+ A LRRK2 protein inhibitor, BIIB122, is being tested in multiple studies. The BEACON trial is enrolling people with PD and a LRRK2 genetic variant. And Dr. Siderowf is planning another LRRK2 trial testing multiple therapies at once.
+ The NEULARK trial is screening recently diagnosed people with Parkinson’ s for some factors linked to LRRK2 to test another LRRK2 inhibitor, NEU-411.
+ In Germany, Kathrin Brockmann, MD, is evaluating whether prasinezumab, a drug currently in clinical testing, can slow or prevent cognitive decline in people with PD and a GBA1 gene variant, which is linked to more thinking and memory problems.( The PASADENA trial is testing this drug for impact on movement progression in a general PD population.)
Prevention as the Future of Precision Medicine
In 2023, MJFF announced validation of the alpha-synuclein seed amplification assay( SAA). With this tool, researchers can detect abnormal alpha-synuclein protein activity, which scientists believe causes cell death and symptoms in PD. Data from PPMI showed positive SAA in spinal fluid not only from people diagnosed but( Cont. on page 10)