was dampened by the development of resis-
tance of the Bcr-Abl kinase to imatinib. The
structural basis for this resistance involves
the emergence of point mutations in the gene
encoding Bcr-Abl, for example leading to the
change of Thr315 to isoleucine (T315I), which
prevents the drug from binding to the kinase.
Studies have shown that a single residue in
the ATP binding pocket of kinases, named
the gatekeeper residue, controls sensitivity
to small-molecule inhibitors. Mutation of
the gatekeeper to a larger amino acid confers
resistance to the drugs. For example, T315I
enzymes are frequently deregulated in human
disease. Several multitargeted tyrosine kinase
inhibitors have shown impressive clinical
activity, and their efficacy has been attributed
to a selectivity profile that includes multiple
targets
5,6
. Examples are the approved multi-
target kinase inhibitors sorafenib, sunitinib
and imatinib (Gleevec), which are currently
in use for renal cancer, gastrointestinal stromal
tumors and chronic myelogenous leukemia
(CML), respectively. However, the early suc-
cess of imatinib (which inhibits Bcr-Abl, c-Kit
and PDGFR tyrosine kinases) in treating CML
Kinases remove a phosphate group from ATP
and covalently attach it to kinase substrates,
thereby altering their function. Protein kinases
mainly phosphorylate serine, threonine and
tyrosine residues, whereas lipid kinases such as
the phosphoinositide 3-kinases (PI3Ks) modify
the inositol ring of phosphoinositides. Over the
last decade, protein kinases have been an attrac-
tive target for therapeutic intervention in many
diseases, including inflammation, diabetes
and cancer. Most kinase inhibitors target the
ATP binding site of protein kinases and show
competitive inhibition by binding to the affin-
ity pocket, a conserved structural motif located
close to the ATP binding pocket. Kinases recog-
nize distinct substrates among others through
subtle differences in their catalytic structures.
These differences permit the development of
relatively selective inhibitors. However, the
diversity and the size of the kinome (comprising
more than 500 kinases
1
) challenge the develop-
ment of truly selective inhibitors
2,3
. Typically
drug discovery efforts have focused on develop-
ing compounds that are specific for a particular
target kinase, but inhibitors with broader speci-
ficity may offer an improved therapeutic benefit.
In this issue, Apsel et al.
4
identify compounds
that inhibit both protein tyrosine kinases and
lipid PI3Ks, both of which are well known as
potent oncoproteins.
Tyrosine kinases (such as c-Kit, Src and
Bcr-Abl) are among the most targeted by phar-
maceutical companies, mainly because these
Killing two kinase families with one stone
Benoit Bilanges, Neil Torbett & Bart Vanhaesebroeck
Multitargeted protein kinase inhibitors have shown great promise in cancer therapy, but the selectivity profiles
of these compounds have largely relied on serendipity or ‘off-target’ activities rather than rational drug design.
Purposefully designed compounds with activity against multiple target kinases bring us a step closer to
personalized medicine.
Benoit Bilanges and Bart Vanhaesebroeck
are in the Centre for Cell Signalling, Institute
of Cancer, Barts and The London School of
Medicine & Dentistry, Charterhouse Square,
London EC1M 6BQ, UK. Neil Torbett is at
Piramed Pharma, 957 Buckingham Avenue,
Slough, Berkshire SL1 4NL, UK.
e-mail: bart.vanh@qmul.ac.uk
Figure 1 Size and shape selectivity of dual PI3K and tyrosine kinase inhibitors. The variable size and
shape of the gatekeeper residue in both lipid and protein kinases create a “filter” to select the binding
of the PP121 compound. A catalytic residue conserved between tyrosine kinases and PI3Ks is targeted
by the PP121 compound. This interaction stabilizes the enzyme in an ‘active’ conformation, without
allowing access to ATP and hence functionally inactivating the kinase. This interaction is responsible
for the potency of the drug. H, hydrogen bond.
Tyr kinase
Ser/Thr kinase
PI3K
Gatekeeper
residue
Active
ATP
Inactive
H
Differentially
oriented
gatekeeper
residue
Active Inactive
H
PP121
inhibitor
Bulky
gatekeeper
residue
Active Active
648 VOLUME 4 NUMBER 11 NOVEMBER 2008 NATURE CHEMICAL BIOLOGY
NEWS AND VIEWS
© 2008 Nature Publishing Group http://www.nature.com/naturechemicalbiology