The Chemistry-Driven Drug Design and
Discovery conference (San Francisco;
23–24 June 2003) (http://www.
srinstitute.com/cs258) was the first in
its series and was uniquely organized to
integrate the applied chemistry and
biology topics that currently drive drug
discovery. Topics ranged from new
screening techniques to structural
biology to structure-guided medicinal
chemistry applications. The main
emphasis of the conference was on
structure-based applications in drug
discovery, focusing on hot areas, such
as kinases and GPCRs.
Fragment-based screening and
assembly
There is currently a need for more-
frequent discovery of higher quality
lead compounds to improve the
productivity of R&D. Fragment-based
strategies are, potentially, a powerful
way to achieve this goal. Christopher
Lepre (Vertex Pharmaceuticals;
http://www.vpharm.com) described the
‘SHAPES’ strategy [1], which involves
NMR screening of a small drug-like
fragment-based library to identify initial
weak hits (K
D
values between low μM
and 5 mM) that can be followed-up by
a variety of complementary methods to
produce high-quality leads.
Lepre outlined two successful
applications of SHAPES screening for
lead generation to the lipid binding
protein, aP2, and the MAP kinase, JNK3,
as pre-HTS and post-HTS examples,
respectively. Screening only 100
compounds against aP2 by NMR
methods resulted in 13 hits in the
0.3–800 μM range. X-ray structures of
SHAPES hits bound to aP2, together
with NMR follow-up screens, helped to
generate a pharmacophore model and
the development of several sub-μM
leads for chemical optimization. In the
JNK3 program, SHAPES NMR screening
followed by a virtual screening strategy
identified three scaffold classes with K
i
values between 0.7 and 13 μM . These
three classes of compounds showed
distinct binding modes by x-ray
crystallography; the resulting
information providing an insight that
could be useful across a wide variety of
kinase targets.
Daniel Wyss (Schering-Plough
Research Institute; http://www.schering-
plough.com) described the application
of ‘structure-based NMR screening
approaches’ (SbN) [2] to a variety of
drug discovery targets, for which HTS
has failed to identify suitable leads.
NMR methods were used to identify
lead-like small-molecule hits from
customized fragment libraries. These
often initially weak hits (K
d
values of
μM–mM) were, after some
optimization, turned into viable leads
through focused chemical optimization,
guided by SAR and 3D structural
information. Wyss discussed novel tools
that use the data generated in the NMR
screen to rapidly provide an accurate
structural representation of
protein–ligand complexes under
conditions that are not favorable in
traditional structural work. The
application of SbN to the discovery of
sub-μM competitive inhibitors against
the NS3 protease of the hepatitis C
virus (HCV) was discussed, as was
application to the discovery of lead
inhibitors of the HCV helicase in the low
μM range and leads in the 20–50 nM
range against the tumor biology target,
human MDM2. Wyss concluded by
summarizing the application of SbN as
a primary screening assay to seven drug
discovery programmes, together with
the chemistry approaches that were
used to turn the NMR-detected weak
hits into viable leads.
Johan Oslob (Sunesis Pharmaceuticals;
http://www.sunesis.com) described the
fragment assembly technique,
‘tethering’, which involves cysteine-
captured ligands identified by MS from
a small library of disulfide-containing
molecules. Tethering enabled improved
design of potent small-molecule
antagonists of the IL-2/IL-2R interaction
[3], the most potent analog having an
IC
50
value of 60 nM, weak cell-based
assay activity (EC
50
= 3 μM) and
favorable PK profiles. Oslob
demonstrated that a portion of the
receptor-binding surface of IL-2 is
highly adaptive but that tethering was
still able to effectively probe ligand
preferences and tolerances at this highly
dynamic subsite.
Dean Artis (Plexxikon; http://www.
plexxikon.com) described the
company’s ‘Scaffold-Based Drug
Discovery™’ platform for lead
generation using a highly integrated
structure-guided approach that involves
the screening of >20,000 selected
protein family-targeted scaffold-like
compounds by biochemical assays.
These assays are tuned for the detection
update conference DDT Vol. 8, No. 20 October 2003
1359-6446/03/$ – see front matter ©2003 Elsevier Science Ltd. All rights reserved. PII: S1359-6446(03)02853-8 924 www.drugdiscoverytoday.com
Structure-guided applications in drug
discovery
Daniel F. Wyss , Schering-Plough Research Institute, 2015 Galloping Hill Road, Kenilworth, NJ 07033, USA; tel: +1 908 740 3299,
fax: +1 908 740 7305, e-mail: daniel.wyss@spcorp.com