Therapeutic antibodies for brain disorders

Sci Transl Med. 2011 May 25;3(84):84ps20. doi: 10.1126/scitranslmed.3002620.

Abstract

The enzyme β-secretase (BACE1) remains an important potential disease-modifying target for developing drugs to treat Alzheimer's disease. However, finding selective BACE1 inhibitors that can penetrate the brain has proved challenging. In this issue of Science Translational Medicine, a pair of studies describes a new approach to inhibiting BACE1 using a human monoclonal antibody that uses receptor-mediated transcytosis to cross the blood brain barrier (Atwal et al. and Yu et al.). The authors engineer a low-affinity bispecific monoclonal antibody targeting both BACE1 and the transferrin receptor and show that this antibody enters the brain more readily and inhibits BACE1 activity more efficiently than does a monospecific antibody against BACE1 alone. These findings should stimulate attempts to use receptor-mediated transcytosis to increase brain uptake of therapeutic antibodies for a variety of brain disorders.

MeSH terms

  • Alzheimer Disease / drug therapy
  • Alzheimer Disease / immunology
  • Amyloid Precursor Protein Secretases / immunology
  • Antibodies / pharmacology
  • Antibodies / therapeutic use*
  • Aspartic Acid Endopeptidases / immunology
  • Brain / drug effects
  • Brain / metabolism
  • Brain / pathology
  • Brain Diseases / drug therapy*
  • Brain Diseases / immunology
  • Humans

Substances

  • Antibodies
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE1 protein, human