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Tumor Suppressor MicroRNAs in Clinical and Preclinical Trials for Neurological Disorders
Austin Lui1, Timothy Do1, Omar Alzayat1
1Department of Neurology, University of California at Davis, Davis, CA 95616, USA.
Abstract:
Cancers and neurological disorders are two major types of diseases in humans. We developed the concept called the "Aberrant Cell Cycle Disease (ACCD)" due to the accumulating evidence that shows that two different diseases share the common mechanism of aberrant cell cycle re-entry. The aberrant cell cycle re-entry is manifested as kinase/oncoprotein activation and tumor suppressor (TS) inactivation, which are associated with both tumor growth in cancers and neuronal death in neurological disorders. Therefore, some cancer therapies (e.g., kinase/oncogene inhibition and TS elevation) can be leveraged for neurological treatments. MicroRNA (miR/miRNA) provides a new style of drug-target binding. For example, a single tumor suppressor miRNA (TS-miR/miRNA) can bind to and decrease tens of target kinases/oncogenes, producing much more robust efficacy to block cell cycle re-entry than inhibiting a single kinase/oncogene. In this review, we summarize the miRNAs that are altered in both cancers and neurological disorders, with an emphasis on miRNA drugs that have entered into clinical trials for neurological treatment.
Insights
Aberrant Cell Cycle Disease (ACCD) links cancers and neurological disorders through shared cell cycle re-entry mechanisms. Targeting tumor suppressor microRNAs (miRNAs) offers a novel therapeutic strategy for both conditions.
Area of Science:
- Molecular Biology
- Oncology
- Neuroscience
- Pharmacology
Background:
- Cancers and neurological disorders are significant human diseases.
- Accumulating evidence links these distinct diseases through a common mechanism: aberrant cell cycle re-entry.
- Aberrant cell cycle re-entry involves kinase/oncoprotein activation and tumor suppressor (TS) inactivation, contributing to tumor growth and neuronal death.
Purpose of the Study:
- To introduce the concept of Aberrant Cell Cycle Disease (ACCD).
- To explore the shared molecular mechanisms between cancers and neurological disorders.
- To review the therapeutic potential of microRNAs (miRNAs) for treating ACCDs, particularly focusing on miRNA drugs in clinical trials for neurological conditions.
Main Methods:
- Conceptual development of Aberrant Cell Cycle Disease (ACCD).
- Review of existing evidence linking cell cycle regulation to cancer and neurological disorders.
- Analysis of microRNA (miRNA) roles in both disease types and their therapeutic applications.
Main Results:
- Aberrant cell cycle re-entry is a unifying mechanism in both cancers and neurological disorders.
- Cancer therapies targeting kinase/oncogene inhibition and TS elevation show potential for neurological treatments.
- Tumor suppressor microRNAs (TS-miRNAs) demonstrate potent efficacy by targeting multiple kinases/oncogenes simultaneously, offering a robust approach to block cell cycle re-entry.
Conclusions:
- Aberrant Cell Cycle Disease (ACCD) provides a novel framework for understanding and treating diverse diseases.
- MicroRNA-based therapeutics, especially TS-miRNAs, represent a promising avenue for neurological treatments.
- Further research into miRNA drugs in clinical trials is warranted for both cancer and neurological disorder therapies.
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