Orphan G protein-coupled receptors: a neglected opportunity for pioneer drug discovery

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Abstract

Access to DNA databases has introduced an exciting new dimension to the way biomedical research is conducted. `Genomic research' offers tremendous opportunity for accelerating the identification of the cause of disease at the molecular level and thereby foster the discovery of more selective medicines to improve human health and longevity. The current challenge is to close the gap rapidly between gene identification and clinical development of efficacious therapeutics. In the present review, Jeffrey Stadel, Shelagh Wilson and Derk Bergsma outline the rationale and describe strategies for converting one large class of novel genes, orphan G protein-coupled receptors (GPCRs), into therapeutic targets. Historically, the superfamily of GPCRs has proven to be among the most successful drug targets and consequently these newly isolated orphan receptors have great potential for pioneer drug discovery.

Section snippets

Reverse molecular pharmacology

Until recently, research into the identification of GPCRs as targets for drug discovery has been conducted using the traditional approach illustrated in Fig. 2. For this strategy, the starting point is functional activity, which forms the basis of an assay by which a ligand is identified through purification from biological fluids, cell supernatants or tissue extracts. One example of the success of this strategy is the discovery of the potent vasoconstricting peptide endothelin[39]. Once

Screening strategy

Fig. 3 illustrates the generic strategy that we use for our reverse molecular pharmacological approach. In addition to the EST approach, which has yielded the majority of our collection of orphan receptors, we have also used a number of more traditional approaches such as low-stringency screening, using portions of known GPCRs as hybridization probes, as well as PCR-based methods. By these techniques we have succeeded in identifying more than 70 orphan receptors in addition to those already

Concluding remarks and future challenges

Although orphan GPCRs have been around for over ten years, very few companies have, until recently, been willing to risk their resources to explore opportunities among this category of receptors. However, the environment for the pharmaceutical industry has changed due to the confluence of several major technological advances. The conversion of gene sequences encoding GPCRs to drug targets is substantially aided by the development of combinatorial chemistry methods and miniaturized

Acknowledgements

The authors wish to thank Drs Robert Ruffolo, Christine Debouck, Paul England and George Livi for their critical comments, as well as their continued encouragement and support.

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