Investigating oral blarcamesine for treatment of early Alzheimer's disease.

INTRODUCTION

Alzheimer's disease (AD) is the most common neurodegenerative dementia, affecting millions globally. Therapies primarily consist of managing symptoms and limiting progression. Further advances in treatment include disease-modifying therapies, many of which have unfavorable safety profiles and provide only modest improvement in cognitive function. The need for novel disease-modifying therapies continues to grow as the burden of AD in the population increases.

AREAS COVERED: We outline approved disease-modifying therapies, including autophagy restoration through SIGMAR1 activation and its affect on neuronal homeostasis, and how oral blarcamesine, a SIGMAR1 agonist, demonstrates promise for early‑onset AD management. We then describe the Phase IIa and IIb/III clinical trials supporting the efficacy of blarcamesine in patients with AD and other neurodegenerative diseases. We also discuss recent preclinical evidence supporting a preventive role for blarcamesine in AD. A PubMed search was conducted for relevant literature regarding this topic using keywords including, but not limited to, 'blarcamesine,' 'disease-modifying therapy,' 'precision medicine,' 'sigma-1 receptor,' and 'SIGMAR1 agonist.'

EXPERT OPINION: Blarcamesine is a potential oral disease-modifying therapeutic candidate for early-stage AD that acts upstream of amyloid-beta pathogenesis and could prevent AD progression early in the course of AD, with emerging preclinical evidence also supporting its potential for disease prevention.