research focus REVIEWS
DDT Vol. 4, No. 9 September 1999 1359-6446/99/$ – see front matter © Elsevier Science Ltd. All rights reserved. PII: S1359-6446(99)01383-5 431
I
on channels make good drug targets because they are
physiologically essential, are pharmacologically access-
ible, are encoded by a variety of genes and usually
operate as multimeric protein assemblies, resulting in a
high degree of functional and anatomical specificity
1,2
.
Through molecular cloning, heterologous expression and
electrophysiological characterization by patch-clamping, it
is clear that the complexity of ion-channel biology also
offers multiple opportunities for small-molecule drugs to
achieve a specific, desired functional effect. For example,
small molecules might influence a variety of biophysical
properties of ion channels, such as voltage-dependence,
permeability, use-dependence, activation and inactivation.
In contrast to simple blockers or openers, the discovery of
modulatory compounds could allow the development of
drugs that specifically act on cells or tissues exhibiting
aberrant levels of ion-channel activity.
To screen compounds against functional ion channels or
assemblies, it is necessary to reconstitute and measure
channel function in a relevant biological context, such as
in a cell. It is also preferable for the assay to be compat-
ible with automated high-throughput screening (HTS) of
large compound libraries. These assays would ideally be
sensitive and fast, allow probing of various functional
states and be amenable to miniaturization to 96-well plates
and beyond. The importance of, and requirements for,
assay miniaturization in drug discovery have been previ-
ously discussed
3,4
. Current formats for ion-channel assays
have been recently summarized in an excellent review
5
and will only be discussed briefly here.
The purpose of this article is to discuss the implemen-
tation of ion-channel assay technologies that combine
high-screening throughput with high-information content.
Although there are a variety of approaches being
explored, including automated patch-clamping, ion-chan-
nel measurements in lipid bilayers and viability assays
based on the toxicity of constitutively opened channels
6
, it
is not currently possible to use these methods for ion-
channel HTS. Therefore, the article will focus on platform
technologies that have a broad application to HTS, and in
particular, will discuss the efforts of Aurora Biosciences
Corporation (San Diego, CA, USA) to apply FRET-based
voltage sensing to ion-channel HTS.
Cell-based assays and
instrumentation for screening
ion-channel targets
Jesús E. González, Kahuku Oades, Yan Leychkis, Alec Harootunian
and Paul A. Negulescu
Jesús E. González, Kahuku Oades, Yan Leychkis, Alec Harootunian and Paul A. Negulescu*, Aurora Biosciences Corp, 11010
Torreyana Road, San Diego, CA 92121, USA. *tel: +1 619 452 5000, fax: +1 619 404 6719, e-mail: NegulescuP@aurorabio.com
Ion channels are an important class of drug targets.
They comprise the molecular basis for essential physio-
logical functions including fluid secretion, electrolyte
balance, bioenergetics and membrane excitability.
High-throughput screening for ion-channel function
requires sensitive, simple assays and instrumentation
that will report ion channel activity in living cells. This
article will review relevant assay technologies for ion
channels and describe voltage-sensitive probes and
instruments based on fluorescence resonance energy
transfer (FRET) that enable ion-channel drug discovery.