BETTER TREATMENT PIPELINES
THE MICHAEL J. FOX FOUNDATION 2025 YEAR IN REVIEW
Investigative Therapeutics Exchange, or LITE— that bridges advances in LRRK2 basic science, or research that aims to identify and characterize fundamental knowledge of a disease, with industryled drug development. Similar efforts are being explored for GBA1.
In addition to genes, evidence shows that inflammation may contribute to the loss of dopamine neurons that leads to Parkinson’ s symptoms. A growing list of clinical trials is currently testing drugs to reduce inflammation. Several of these approaches target the so-called NLRP3 inflammasome, a group of proteins that react to a range of stimuli( for example, bacteria, viruses or misfolded proteins), which plays a central role in inflammation after cells are damaged.
A Drug to Slow or Stop Parkinson’ s Advances
For decades, a drug to slow or stop Parkinson’ s progression was a distant dream. Decades of drug development led to treatments that mitigate symptoms, but disease-modifying therapies remained just out of reach.
Today, that dream is closer to reality than ever before. In June, Swiss pharmaceutical company Roche announced that prasinezumab, a potential disease-modifying therapy, would move forward into Phase III clinical trials. Scientists believe that the drug can bind to toxic alpha-synuclein proteins that build up in the brain and destroy dopamine neurons, limiting their damaging effects. Breaking this cycle has the potential to stop the damage that causes Parkinson’ s symptoms to progressively worsen over time— or better still, prevent symptoms from ever emerging. Prasinezumab isn’ t the first disease-modifying drug to make it this far, but it is one of the first of this type of therapy to reach critical Phase III testing.
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