Orphan drugs discovery

Tang Herbal, 147 Target-based drug discovery biological targets, 180-184 chemical end point in, 180 chemical tools, 178-179 definition of, 5 description of, 175-177 linear approach, 176 orphan receptors, 180 preclinical process in, 176-177 random variation in gene expression, 178... 

Stadel, J. M., Wilson, S., and Bergsma, D. J., Orphan G-protein-coupled receptors a neglected opportunity for pioneer drug discovery, Trends Pharmacol. Sci., 18(11), 430 437, 1997. 

Xie, W., Uppal, H., Saini, S.P., Mu, Y., Little, J.M., Radominska-Pandya, A. and Zemaitis, M.A. (2004) Orphan nuclear receptor-mediated xenobiotic regulation in drug metabolism. Drug Discovery Today, 9, 442-449. 

Dowell SJ. Understanding GPCRs—from orphan receptors to novel drugs. Drug Discovery Today 6 884-886 (2002). 

Willson, T. etal. The PPARs from orphan discovery to drug discovery, J. Med. Chem., 43, 527, 2000. 

Willson, T. M., Brown, P.J., Sternbach, D.D., Henke, B.R. The PPARs from orphan receptors to drug discovery. 

Occasionally, discoveries cannot be patented. For example, if you discover something novel, such as lithium ameliorating bipolar disorder, there is a problem. Lithium is a chemical element, not a new chemical compound that can be patented per se. Since the industry could not make much money making lithium tablets, there was less incentive to establish a use patent. Nowadays, for some cases such as this, there is an orphan drug statute in the United States, which, since 1983, gives a company a seven-year exclusivity and enables it to develop a drug treatment and still help patients. But this is a very slow and very difficult process. It is much better to patent, and let the patent drop should no one develop it into a coiranercial product, than not to patent at all. 

Shi Y. Orphan nuclear receptors, excellent targets of drug discovery. Comb. Chem. High Throughput Screen. 2006 9 683-689. 

Drug Discovery Using Adopted Orphan Receptors... 

The new paradigm for orphan receptor-based drug discovery is shown diagrammatically (Fig. 5). A first step toward ligand identification for a given orphan... 

DISCOVERY, DEVELOPMENT, MARKETING AND DISTRIBUTION OF ORPHAN DRUGS... 

Discovery, development, marketing and distribution of orphan drugs... 

The largest GPCR subfamily is known as class A. It comprises approximately 90 % of all GPCRs [1], Members of this class of GPCRs have been studied at both the molecular and the structural levels [1], Identification of the properties of class A receptors has resulted in the identification of orphan receptors [4, 5] that have become reagents for drug discovery in drug screens [5],... 

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