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Review uncovers therapeutic trade-offs of PIKfyve inhibition across disease models, suggesting that tunable targeting is required to avoid severe lysosomal toxicity.
PIKfyve inhibitors have evolved from niche probes into powerful tools for studying endolysosomal biology. Although PIKfyve inhibition has revealed important roles in membrane trafficking, autophagy, viral entry, and cancer‐cell vulnerability, clinical translation remains limited by a narrow therapeutic window because disruption of PI(3,5)P 2 homeostasis causes profound lysosomal dysfunction. Future progress will depend on tuneable or partial inhibitors, improved selectivity, and targeted delivery. PIKfyve therefore provides a useful model for balancing potency, precision, and safety in lipid kinase drug discovery.
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Selvam et al. (2026) studied this question.
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