A therapeutic antibody targeting BACE1 inhibits amyloid-β production in vivo

Sci Transl Med. 2011 May 25;3(84):84ra43. doi: 10.1126/scitranslmed.3002254.

Abstract

Reducing production of amyloid-β (Aβ) peptide by direct inhibition of the enzymes that process amyloid precursor protein (APP) is a central therapeutic strategy for treating Alzheimer's disease. However, small-molecule inhibitors of the β-secretase (BACE1) and γ-secretase APP processing enzymes have shown a lack of target selectivity and poor penetrance of the blood-brain barrier (BBB). Here, we have developed a high-affinity, phage-derived human antibody that targets BACE1 (anti-BACE1) and is anti-amyloidogenic. Anti-BACE1 reduces endogenous BACE1 activity and Aβ production in human cell lines expressing APP and in cultured primary neurons. Anti-BACE1 is highly selective and does not inhibit the related enzymes BACE2 or cathepsin D. Competitive binding assays and x-ray crystallography indicate that anti-BACE1 binds noncompetitively to an exosite on BACE1 and not to the catalytic site. Systemic dosing of mice and nonhuman primates with anti-BACE1 resulted in sustained reductions in peripheral Aβ peptide concentrations. Anti-BACE1 also reduces central nervous system Aβ concentrations in mouse and monkey, consistent with a measurable uptake of antibody across the BBB. Thus, BACE1 can be targeted in a highly selective manner through passive immunization with anti-BACE1, providing a potential approach for treating Alzheimer's disease. Nevertheless, therapeutic success with anti-BACE1 will depend on improving antibody uptake into the brain.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Amyloid Precursor Protein Secretases / antagonists & inhibitors*
  • Amyloid Precursor Protein Secretases / chemistry
  • Amyloid Precursor Protein Secretases / deficiency
  • Amyloid Precursor Protein Secretases / metabolism
  • Amyloid beta-Peptides / biosynthesis*
  • Amyloid beta-Peptides / cerebrospinal fluid
  • Animals
  • Antibodies / chemistry
  • Antibodies / metabolism
  • Antibodies / pharmacology*
  • Antibodies / therapeutic use*
  • Aspartic Acid Endopeptidases / antagonists & inhibitors*
  • Aspartic Acid Endopeptidases / chemistry
  • Aspartic Acid Endopeptidases / deficiency
  • Aspartic Acid Endopeptidases / metabolism
  • Biological Assay
  • Brain / drug effects
  • Brain / metabolism
  • Brain / pathology
  • Crystallography, X-Ray
  • Endocytosis / drug effects
  • Humans
  • Macaca fascicularis
  • Mice
  • Models, Molecular
  • Molecular Sequence Data
  • Neurons / drug effects
  • Neurons / metabolism
  • Peptide Library
  • Protein Binding / drug effects
  • Treatment Outcome

Substances

  • Amyloid beta-Peptides
  • Antibodies
  • Peptide Library
  • Amyloid Precursor Protein Secretases
  • Aspartic Acid Endopeptidases
  • BACE1 protein, human
  • Bace1 protein, mouse