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Approaches to develop therapeutics to treat frontotemporal dementia
Lisa P Elia1, Terry Reisine2, Amela Alijagic1
1Center for Systems and Therapeutics and Taube/Koret Center for Neurodegenerative Disease Research, San Francisco, CA, USA; The J. David Gladstone Institutes, San Francisco, CA, USA.
Neuropharmacology
|January 22, 2020
Summary
Frontotemporal degeneration (FTD) is a neurodegenerative disease with no effective therapies. This review explores strategies to increase progranulin (PGRN) levels for treating GRN-linked FTD and potentially other neurodegenerative disorders.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Frontotemporal degeneration (FTD) is a progressive neurodegenerative disorder characterized by frontal and temporal lobe degeneration, neuroinflammation, and behavioral changes.
- Currently, no effective therapies exist for FTD, which affects cognition, personality, and behavior.
- While most FTD cases are sporadic, 30-40% are heritable, often linked to mutations in specific genes, including GRN.
Purpose of the Study:
- To review therapeutic strategies for GRN-linked frontotemporal degeneration (FTD) by focusing on increasing progranulin (PGRN) levels.
- To identify novel genetic and chemical modulators that can restore neuronal PGRN levels and function.
- To explore the potential broader applications of PGRN-based therapies for other neurodegenerative diseases like Alzheimer's disease (AD).
Main Methods:
- Review of existing literature on GRN mutations, progranulin (PGRN) biology, and FTD pathogenesis.
- Analysis of mechanisms linking PGRN deficiency to neuroinflammation, lysosomal dysfunction, and neurodegeneration.
- Identification and discussion of potential therapeutic targets and approaches for modulating neuronal PGRN levels.
Main Results:
- GRN mutations lead to reduced brain PGRN levels, causing lysosomal dysfunction, TDP43 aggregation, and neuroinflammation.
- PGRN insufficiency is implicated in the pathogenesis of both FTD and Alzheimer's disease (AD).
- Modulating genetic and chemical pathways offers a potential strategy to increase PGRN levels and counteract disease mechanisms.
Conclusions:
- Restoring functional PGRN levels presents a promising therapeutic avenue for GRN-linked FTD.
- Therapies developed for GRN-linked FTD may have significant implications for treating other neurodegenerative conditions.
- Further research into genetic and chemical modulators is crucial for developing effective disease-modifying treatments for FTD and related disorders.
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