Sclerostin: how human mutations have helped reveal a new target for the treatment of osteoporosis.
Publication Title
Drug discovery today
Document Type
Article
Publication Date
7-1-2013
Keywords
washington; isb; Adaptor Proteins, Signal Transducing; Animals; Antibodies; Bone Density Conservation Agents; Bone Morphogenetic Proteins; Drug Discovery; Genetic Markers; Genetic Predisposition to Disease; Humans; Hyperostosis; Molecular Targeted Therapy; Mutation; Osteoporosis; Phenotype; Syndactyly; Treatment Outcome
Abstract
In the 1990s there was a tremendous mood of optimism among pharmaceutical scientists that identification of disease-associated variations in the human genome would result in a surge of new drug targets (the 'gene-to-drug' mantra). To date the expected deluge of new drugs has not arrived. However, a small number of drugs arising directly from the study of rare human disorders showing Mendelian inheritance are now entering late stage clinical trials. Here we describe the advantages of this approach and discuss the background and early clinical trial findings with antibodies directed at a target identified in this way.
Specialty/Research Institute
Institute for Systems Biology
Specialty/Research Institute
Pharmacy
DOI
10.1016/j.drudis.2013.04.001
