Parkinson’s is a progressive condition, meaning that over time the symptoms are going to get worse. Therefore, the first and most critical step in any cure for Parkinson’s is finding a treatment that will slow or stop the progression of the condition.

The progressive loss of dopamine neurons in the brain is one of the classical hallmarks of Parkinson’s. Exactly why these particular cells are vulnerable to this condition is unknown, but awareness of their loss has provided a focal point for research efforts.

Numerous preclinical studies in models of Parkinson’s have pointed towards certain biological pathways that can be targeted. And these projects have led to the initiation of clinical trials by Cure Parkinson’s with the goal of stopping the disease progressing.

A good example of a potential disease-halting treatment is the Cure Parkinson’s supported Adept-PD study. Nortriptyline is a commonly used anti-depressant treatment, but Parkinson’s researchers have reported that this drug also displays the ability to slow disease progression in models of Parkinson’s. The ADepT-PD study is evaluating two anti-depressants (including nortriptyline) in people with Parkinson’s, with Cure Parkinson’s funding a sub-study exploring disease progression.

Nortriptyline and Parkinson’s

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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.

The ambroxol trial programme

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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 MODAG Anle138b clinical trial

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Another neuroprotective approach involves enhancing cellular function, and significant preclinical research has shown that boosting the function of mitochondria can help cells survive. Mitochondria are the power stations of cells – providing all of the energy requirements. Cure Parkinson’s has several clinical trial programs 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.

UDCA and Parkinson’s

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