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Updated: Jul 22, 2025

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
Published on: May 2, 2025
Neural Cells for Neurodegenerative Diseases in Clinical Trials
Yiping Fan1,2,3, Eyleen L K Goh4, Jerry Kok Yen Chan1,2,3
1Department of Reproductive Medicine, KK Women's and Children's Hospital, Singapore, Singapore.
Cellular therapies show promise for neurodegenerative diseases (ND), with numerous clinical trials underway using various cell types. Further randomized studies are needed to confirm efficacy, though cost and rare disease prevalence pose challenges.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Clinical Trials
Background:
- Neurodegenerative diseases (ND) encompass a range of debilitating central and peripheral nervous system disorders with no current cure.
- Current treatments for ND focus on slowing disease progression and managing symptoms.
Approach:
- This review compiles data from 150 clinical trials investigating cellular therapies for seven distinct ND.
- The trials utilize diverse cell types, including neural stem progenitors, differentiated neural cells, and non-neural cells derived from various sources like embryonic and induced pluripotent stem cells.
Key Points:
- 33 trials involved neural stem progenitors, 8 used differentiated neural cells, and 109 employed non-neural cells.
- Early-phase clinical trials have yielded encouraging results, suggesting therapeutic potential.
- Cellular therapies offer a novel approach to treating neurodegenerative conditions.
Conclusions:
- Encouraging outcomes from early trials necessitate further investigation in randomized controlled settings.
- The high cost of trials and the rarity of certain neurodegenerative diseases may impede definitive large-scale studies.
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