The allowed claims cover a method of using trehalose (SLS-005) to treat several neurodegenerative conditions including spinocerebellar ataxia (SCA), spinal and bulbar muscular atrophy (SBMA), dentatomral-pailidoluyssan atrophy (DRPLA), Pick’s disease, corticobasaldegeneration (CBD), progressive supranuclear palsy (PSP), and frontotemporal dementia.
Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells. In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.