1 Introduction
Successful therapeutic treatment of solid tumors
has been limited during the past decade, and avail-
able anticancer strategies aimed directly at killing
tumor cells leave much room for improvement.
The killing of tumor cells is effective only when
the antitumor drug can reach tumor cells from the
circulation. Due to a solid tumor’s architecture and
high interstitial pressure, however, these require-
ments are not always met, resulting in limited suc-
cess of chemotherapeutic, immunotoxin strategies,
and radiation due to the lack of radiosensitizing
oxygen. Moreover, tumor cells are genetically
unstable and often develop resistance to chemical
agents targeted against them. Because angiogenesis
is a prerequisite for the development of metastases
and the outgrowth of tumors, the use of anti-angiogenic
therapy has come to the forefront, first exemplified
in the clinic with Avastin
®
[1–4].
Regardless of the therapeutic approach or drug
of choice, monotherapy usually has limited effec-
tiveness in treating cancer. Moreover, standard
chemotherapy and radiation therapy always raise
concerns related to systemic effects and toxicity.
In this regard, use of some targeted therapy in
combination with radiation and chemotherapy is
likely to hold the key to future successes in the
clinic. This chapter focuses on the development
of non-peptidic mimetics to sensitize tumors in
order to reduce toxicity from chemotherapy and
radiation therapy and improve outcome. There are
many potential advantages that would attend such
structurally simplified, smaller therapeutic agents,
the most important being increased in vivo expo-
sure and reduced immunogenicity, as well as ease
and cost of production.
2 Designing a Non-Peptidic
Mimetic
The first step to design a peptidomimetic or non-
peptidic mimetic is usually the analysis of the pro-
tein structure of interest, either that of the target or
of the ligand, or both if one is fortunate enough to
have them. Normally structure analysis of the lig-
and yields more useful information, as one would
like to mimic the activity of the ligand, usually to
design an antagonist that tends to be easier than
to design an agonist. This phase of the design often
leads to structural hypotheses that are tested nor-
mally by producing various amino acid substituted
variants of the native sequence. In the case of a
native protein, site-directed mutagenesis is usually
employed by expressing the variant protein using
an appropriate expression system. In the case of a
peptide, variants may be synthesized chemically,
and amino acid residues can be substituted, most
often by using alanine scanning in which native
residues are substituted with alanines one at a time,
and a series of such peptides is produced that basi-
cally “scan” the native sequence. In either instance,
variants of the native sequence are then assessed
Chapter 18
Non-Peptidic Mimetics
as Cancer-Sensitizing Agents
Ruud P.M. Dings, Mark Klein, and Kevin H. Mayo
From: Sensitization of Cancer Cells for Chemo/Immuno/Radio-therapy, 1st Edition. 305
Edited by: Benjamin Bonavida © Humana Press, Totowa, NJ