A collaboration between Cure Parkinson’s and the Van Andel Institute has secured funding for a phase 1b clinical trial to evaluate the efficacy of low-dose lithium in slowing the progression of Parkinson’s disease.
LONDON (May 29, 2024) — Cure Parkinson’s and the Van Andel Institute (VAI) have jointly announced funding for a phase 1b clinical trial that will explore the potential of low-dose lithium, a medication traditionally prescribed for mood disorders, to slow the progression of Parkinson’s disease. The trial will be led by Dr. Thomas Guttuso at the University of Buffalo, expanding upon a growing body of research investigating alternative therapeutic uses for established medications.
Background on Lithium and Its Emerging Role in Parkinson’s Treatment
Lithium has been utilized as a psychiatric medication since it gained approval in the United States in 1970 for the treatment of bipolar disorder. Its primary function is to stabilize mood, but recent investigations have pivoted towards its neuroprotective potential, particularly in the context of neurodegenerative diseases such as Parkinson’s.
The interest in lithium as a treatment for Parkinson’s stems from preliminary studies suggesting that low doses may provide protective benefits against the loss of dopamine neurons, which are critically affected in Parkinson’s disease. Dr. Simon Stott, Director of Research at Cure Parkinson’s, emphasized the significant potential of drug repurposing: “Drug repurposing represents a means of rapidly bringing new treatments to patients by testing agents that we already know a lot about. If low-dose lithium can help slow the progression of Parkinson’s, then this would be an important development for the Parkinson’s community.”
Details of the Phase 1b Clinical Trial
The upcoming trial aims to recruit 20 additional participants, complementing 15 individuals already funded by the U.S. National Institutes of Health (NIH). This will bring the total cohort size to 35 participants who will be monitored to assess the effects of lithium on specific biological indicators associated with Parkinson’s disease progression.
Dr. Darren Moore, Chair of VAI’s Department of Neurodegenerative Science, highlighted the complexity of Parkinson’s disease and the necessity for sophisticated treatment strategies: “Parkinson’s is an immensely complex disorder that requires an equally sophisticated approach to seeking treatments that slow or stop progression. The International Linked Clinical Trials initiative enables us to prioritize medications like lithium that are known to treat other diseases and that may have use in Parkinson’s.”
Biomarkers of Interest in the Trial
The trial will focus on two key biomarkers associated with the progression of Parkinson’s disease: levels of the protein Nurr1 in the blood and levels of “free water” in the brain. Nurr1 is critical for the development of new dopamine neurons, and studies have shown that its levels decrease in individuals with Parkinson’s. A 2019 pilot study conducted by Dr. Guttuso indicated that low-dose lithium might enhance Nurr1 levels, potentially aiding in the survival of dopamine neurons.
In addition to monitoring Nurr1 levels, researchers will also evaluate the levels of free water in the brain, which is considered a marker of dopamine neuron loss in Parkinson’s disease. Free water refers to the fluid in the brain that is not contained within cells. The aforementioned pilot study suggested that low-dose lithium could mitigate the increase of free water, a finding that the new phase 1 trial aims to validate with a larger participant group.
Dr. Guttuso remarked on the significance of these biomarkers: “Because brain free water is one of our leading disease progression biomarkers in Parkinson’s disease, if this study is able to replicate our earlier findings associating low-dose lithium therapy with reductions in free water, this would support low-dose lithium’s ability to slow Parkinson’s progression.”
Future Implications of the Trial
The overarching objective of the phase 1b trial is to determine whether low-dose lithium should continue to be investigated as a viable treatment option for Parkinson’s disease. Recruitment for the study is expected to commence in mid-June 2024.
The decision to investigate lithium’s potential in treating Parkinson’s is informed by early preclinical data pointing toward its protective effects on dopamine neurons at low dosages. Following the positive indications from the 2019 pilot study, low-dose lithium was prioritized by the International Linked Clinical Trials (iLCT) program, a collaborative initiative formed between Cure Parkinson’s and VAI aimed at facilitating the clinical evaluation of drugs that could slow, stop, or reverse the progression of Parkinson’s.
If successful, the trial could pave the way for new treatment strategies that are both accessible and cost-effective for those living with Parkinson’s disease. The implications could extend beyond just the clinical benefits, potentially altering the landscape of Parkinson’s research and treatment paradigms.
As research continues, the collaboration between Cure Parkinson’s and VAI exemplifies the potential for innovative approaches to longstanding medical challenges. With funding commitments and a dedicated research team, the trial represents a hopeful step toward enhancing the quality of life for individuals affected by Parkinson’s.
Research reported in this publication is supported in part by the National Center for Advancing Translational Sciences of the National Institutes of Health under award no. UL1TR001412. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health or other funders.
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