Discovery of biopharmaceuticals

The discovery of virtually all the biopharmaceuticals discussed in this text was a knowledge-based one. Continuing advances in the molecular sciences have deepened our understanding of the molecular mechanisms that underline health and disease. An understanding at the molecular level of how the body functions in health and of the deviations that characterize the development of a disease often renders obvious potential strategies likely to cure/control that disease. Simple examples illustrating this include the use of insulin to treat diabetes and the use of GH to treat certain forms of dwarfism (Chapter 11). The underlining causes of these types of disease are relatively straightforward, in that they are essentially promoted by the deficiency/absence of a single regulatory molecule. Other diseases, however, may be multifactorial and, hence, more complex. Examples include cancer and inflammation. Nevertheless, cytokines, such as interferons and in-terleukins, known to stimulate the immune response/regulate inflammation, have proven to be therapeutically useful in treating several such complex diseases (Chapters 8 and 9).

An understanding, at the molecular level, of the actions of various regulatory proteins, or the progression of a specific disease does not, however, automatically translate into pinpointing an effective treatment strategy. The physiological responses induced by the potential biopharmaceu-tical in vitro (or in animal models) may not accurately predict the physiological responses seen when the product is administered to a diseased human. For example, many of the most promising biopharmaceutical therapeutic agents (e.g. virtually all the cytokines, Chapter 8), display multiple activities on different cell populations. This makes it difficult, if not impossible, to predict what the overall effect administration of any biopharmaceutical will have on the whole body, hence the requirement for clinical trials.

In other cases, the widespread application of a biopharmaceutical may be hindered by the occurrence of relatively toxic side effects (as is the case with tumour necrosis factor a (TNF-a, Chapter 9). Finally, some biomolecules have been discovered and purified because of a characteristic biological activity that, subsequently, was found not to be the molecule's primary biological activity. TNF-a again serves as an example. It was first noted because of its cytotoxic effects on some cancer cell types in vitro. Subsequently, trials assessing its therapeutic application in cancer proved disappointing due not only to its toxic side effects, but also to its moderate, at best, cytotoxic effect on many cancer cell types in vivo. TNF's major biological activity in vivo is now known to be as a regulator of the inflammatory response.

In summary, the 'discovery' of biopharmaceuticals, in most cases, merely relates to the logical application of our rapidly increasing knowledge of the biochemical basis of how the body functions. These substances could be accurately described as being the body's own pharmaceuticals. Moreover, rapidly expanding areas of research, such as genomics and proteomics, will likely hasten the discovery of many more such products, as discussed below.

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