Preclinical development of scyllo-inositol for the treatment of Alzheimer's disease (AD) has been investigated in both in vitro and in vivo models with positive results. scyllo-Inositol stabilized a small conformer of Aβ42 in vitro, neutralized cell derived Aβ trimers and promoted low molecular weight Aβ species in vivo. These interactions resulted in decreased neuronal toxicity, increased long-term potentiation (LTP) and ablation of cognitive deficits in multiple mouse models of AD. scyllo-Inositol bioavailability, pharmacokinetics, and small animal toxicology studies demonstrated the potential for translation to human patients. The results of Phase I and Phase II clinical trials for AD are presented. Furthermore, the use of this compound for imaging and other amyloid related disorders is discussed.
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