Nootropics
Evidence: moderate_human
Benzylpiperizine-aminopyridine compound that stimulates neurogenesis of human hippocampus-derived neural stem cells in vitro and increases hippocampal volume in vivo. Mechanism is independent of serotonin or norepinephrine reuptake inhibition — fundamentally distinct from traditional antidepressants. Activates the TrkB receptor (BDNF receptor) and downstream Akt/PI3K signaling pathways to promote synaptic plasticity, long-term potentiation, and neuronal survival. Enhances BDNF expression in hippocampal subregions critical for memory consolidation and mood regulation. Originally developed as ALTO-100 (Alto Neuroscience) for treatment-resistant depression with cognitive impairment.
Standard: 40 mg once daily (for educational context — investigational compound, not approved for any indication)
Maintenance: 40 mg/day based on Phase 2 data suggesting this dose has the best benefit-risk profile
Administration: oral
Timing: Once daily, time of day not definitively established from clinical data. With or without food.
Duration: Phase 2 trial used 12-week treatment duration. Long-term safety data unavailable.
FOR EDUCATIONAL PURPOSES ONLY — NSI-189/ALTO-100 is an investigational compound with no regulatory approval. The Phase 2 trial (Fava et al. 2019, n=220) failed its primary endpoint (MADRS) but showed statistically significant improvements on secondary measures of cognition and self-rated depression at the 40mg dose. The 80mg dose showed benefit specifically in moderately (not severely) depressed patients. The pro-cognitive effects are the most compelling aspect — improvements on CogScreen measures with effect sizes up to d=1.12, which is unusually large. The hippocampal neurogenesis mechanism is genuinely novel and represents a new pharmacological approach distinct from all existing antidepressants. Alto Neuroscience is using biomarker-guided patient selection (EEG phenotyping) to improve signal detection in ongoing trials. The compound's future depends on whether this precision psychiatry approach can demonstrate efficacy in targeted populations.
Investigational new drug — not commercially available through legitimate pharmaceutical channels. Neuralstem developed the compound, later sold to Alto Neuroscience (now ALTO-100). Available through research chemical vendors, but purity and identity verification are essential — demand mass spectrometry and HPLC analysis from vendors. No pharmaceutical-grade product exists for consumer use.
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