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Modeling Human Cerebellar Development In Vitro in 2D Structure
Published on: September 16, 2022
Advances in cerebellar disorders: pre-clinical models, therapeutic targets, and challenges
Mario Manto1, Jan Cendelin2, Michael Strupp3
1Service des Neurosciences, University of Mons, Mons, Belgium.
Introduction:
Cerebellar ataxias (CAs) represent neurological disorders with multiple etiologies and a high phenotypic variability. Despite progress in the understanding of pathogenesis, few therapies are available so far. Closing the loop between preclinical studies and therapeutic trials is important, given the impact of CAs upon patients' health and the roles of the cerebellum in multiple domains. Because of a rapid advance in research on CAs, it is necessary to summarize the main findings and discuss future directions.
Areas Covered:
We focus our discussion on preclinical models, cerebellar reserve, the therapeutic management of CAs, and suitable surrogate markers. We searched Web of Science and PubMed using keywords relevant to cerebellar diseases, therapy, and preclinical models.
Expert Opinion:
There are many symptomatic and/or disease-modifying therapeutic approaches under investigation. For therapy development, preclinical studies, standardization of disease evaluation, safety assessment, and demonstration of clinical improvements are essential. Stage of the disease and the level of the cerebellar reserve determine the goals of the therapy. Deficits in multiple categories and heterogeneity of CAs may require disease-, stage-, and symptom-specific therapies. More research is needed to clarify how therapies targeting the cerebellum influence both basal ganglia and the cerebral cortex, poorly explored domains in CAs.
Insights
Cerebellar ataxias (CAs) are diverse neurological disorders with limited treatments. Research highlights the need for better preclinical models and targeted therapies to improve patient outcomes.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Cerebellar ataxias (CAs) encompass a group of neurological disorders characterized by diverse causes and significant variability in symptoms.
- Despite advancements in understanding the underlying mechanisms of CAs, effective therapeutic options remain scarce.
- The cerebellum's role extends beyond motor control, impacting various cognitive and emotional functions, making CAs a complex challenge.
Purpose of the Study:
- To review and synthesize current findings in cerebellar ataxia (CA) research, focusing on preclinical models and therapeutic strategies.
- To discuss the importance of bridging the gap between preclinical research and clinical trials for CA.
- To identify future research directions and therapeutic targets for CAs.
Main Methods:
- Systematic literature search of Web of Science and PubMed using keywords related to cerebellar diseases, therapy, and preclinical models.
- Focused discussion on preclinical models, cerebellar reserve, therapeutic management, and surrogate markers for CAs.
- Analysis of existing and emerging therapeutic approaches for CAs.
Main Results:
- Numerous symptomatic and disease-modifying therapeutic strategies for CAs are under investigation.
- Therapy development necessitates robust preclinical studies, standardized evaluations, safety assessments, and demonstrated clinical efficacy.
- The stage of the disease and the patient's cerebellar reserve are critical factors in determining therapeutic goals and approaches.
Conclusions:
- The heterogeneity of CAs and the involvement of multiple neurological domains necessitate tailored, disease-, stage-, and symptom-specific therapies.
- Further research is required to understand the broader impact of cerebellar-targeted therapies on connected brain regions like the basal ganglia and cerebral cortex.
- Closing the gap between preclinical findings and clinical application is crucial for advancing CA treatment.
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