Are patient-derived models of amyotrophic lateral sclerosis a game changer for novel drug discovery? - PubMed
4 hours ago
- #Amyotrophic lateral sclerosis
- #Drug discovery
- #Patient-derived models
- Patient-derived ALS models are emerging as human-relevant complements to traditional systems.
- A framework evaluates models on disease-relevant phenotypes, patient heterogeneity, and impact on therapy.
- Current models include iPSC-derived neurons, organoids, co-cultures, and organ-chip systems.
- These models are not yet standalone decision tools but serve as filters for target discovery and translation.
- Standardization and clinical links are crucial for their value in drug development.