An example of this type of targeted approach involves enhancing cell function, and significant preclinical lab-based research has shown that boosting the function of the small ‘power packs’ inside cells called mitochondria can help cells survive. Cure Parkinson’s has several clinical trial programmes dedicated to mitochondria-targeted approaches. An example of this is the UDCA in Parkinson’s UP study. UDCA is a treatment that is used for dissolving gall stones, but recently researchers have found that it also has neuroprotective properties in models of Parkinson’s. Cure Parkinson’s is co-funding the UP study to determine if UDCA could be a useful neuroprotective treatment for Parkinson’s.
A future potential disease halting treatment is a novel drug called Anle138b, which is being developed by the biotech company MODAG. Anle138b is a small molecule that can inhibit the clustering of the Parkinson’s-associated alpha synuclein.
Cure Parkinson’s has funded preclinical research on Anle138b, and this molecule was prioritised by the International Linked Clinical Trials committee. Cure Parkinson’s is now working closely with MODAG to get Anle138b into phase 2 clinical trial for Parkinson’s.
The genetics of Parkinson’s has provided us with insights into the underlying biology of the condition. We now understand more about the biological processes associated with genetic risk factors, and experimental treatments have been developed to target these.
These novel treatments are being clinically tested to see if they will have beneficial effects not just for individuals carrying certain genetic risk factors, but also for the wider Parkinson’s community.