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Anavex生命科学公司宣布了自闭症谱系障碍和阿尔茨海默病之间共同生物学的新发现

2026-04-14 19:34

  • Multiple peer-reviewed publications point to biological link between autism spectrum disorder (ASD) and Alzheimer's disease (AD), including shared disruptions in autophagy.
  • Epidemiological data show that autistic adults may be diagnosed with Alzheimer's and related dementias at rates up to 8 times higher than the general population, with onset occurring years or decades earlier than typical.
  • Converging human genetic evidence links numerous high-confidence ASD risk genes — including TSC1/TSC2, PTEN, SHANK3, and FMRP — to impaired cellular autophagy, establishing autophagy dysfunction as a shared molecular substrate across genetically diverse forms of ASD.
  • Synaptic dysfunction in ASD is now understood to arise, in substantial part, from a failure of autophagy-dependent synaptic pruning — causing an excess of poorly regulated synaptic connections and disrupted excitatory–inhibitory balance in neural circuits.
  • The brain's extracellular matrix (ECM) is pathologically altered in ASD and is bidirectionally coupled to autophagy.
  • Restoration of autophagy impairment, now emerging as a central shared pathway in both ASD and AD, is precisely the biological system targeted by blarcamesine through its activation of SIGMAR1.
  • Blarcamesine has demonstrated restoration of autophagy through SIGMAR1 activation in preclinical models and has shown clinical effects in Phase IIb/III trials in early Alzheimer's disease, Phase II/III in Rett syndrome (a neurodevelopmental disorder caused by MECP2 mutation), and Phase II in Parkinson's disease dementia.
  • Collectively, these data provide a scientific basis for advancing blarcamesine into pivotal clinical studies, subject to further evaluation and regulatory considerations.

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