Vol 8, No 3
- Drug Discovery and Proof of Concept
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ecent reports have highlighted the imbalance
between rising costs in drug discovery and the produc-
tion of new molecular entities for the market,
1,2
leading
to a long-term loss of efficiency. Remarkably, this decline
in productivity has occurred despite the fact that bio-
medical research benefits from large governmental and
private investments, and despite the comprehensive
improvements in our knowledge of human genes result-
ing from large sequencing projects.
The tremendous efforts that have to be invested for
drug target identification, follow-up validation studies,
and clinical trials, in combination with the high failure
rate as a consequence of individual response to drugs,
has imposed high costs on the development of drugs.
Understanding individual response to a drug, what
determines its efficacy and tolerability in the patients
body, is the major bottleneck in drug development and
S t a t e o f t h e a r t
R
Copyright © 2006 LLS SAS. All rights reserved
www.dialogues-cns.org
Expression profiling of drug response
from genes to pathways
Ralf Herwig, PhD; Hans Lehrach, PhD
Keywords:
drug discovery; functional genomics; microarray; bioinformatics;
data
integration; database; systems biology
Author affiliations:
Max Planck Institute for Molecular Genetics, Department
of Vertebrate Genomics, Berlin, Germany
Address for correspondence:
Dr Ralf Herwig, Max Planck Institute for Molecular
Genetics, Department of Vertebrate Genomics, Ihnestr. 73, D-14195 Berlin,
Germany
(e-mail: herwig@molgen.mpg.de)
Understanding individual response to a drugwhat determines its efficacy and tolerabilityis the major bottleneck in
current drug development and clinical trials. Intracellular response and metabolism, for example through cytochrome P-
450 enzymes, may either enhance or decrease the effect of different drugs, dependent on the genetic variant. Microarrays
offer the potential to screen the genetic composition of the individual patient. However, experiments are noisy and
must be accompanied by solid and robust data analysis. Furthermore, recent research aims at the combination of high-
throughput data with methods of mathematical modeling, enabling problem-oriented assistance in the drug discovery
process. This article will discuss state-of-the-art DNA array technology platforms and the basic elements of data analysis
and bioinformatics research in drug discovery. Enhancing single-gene analysis, we will present a new method for inter-
preting gene expression changes in the context of entire pathways. Furthermore, we will introduce the concept of sys-
tems biology as a new paradigm for drug development and highlight our recent researchthe development of a mod-
eling and simulation platform for biomedical applications. We discuss the potentials of systems biology for modeling the
drug response of the individual patient.
© 2006, LLS SAS
Dialogues Clin Neurosci
. 2006;8:283-293.