A TED talk last week by geneticist and physician Francis Collins shed some light on a massive gulf that exists in the pharmaceutical world – that between the fundamental knowledge and the application of this knowledge. We currently know the molecular cause for 4000 diseases yet only have treatments for 250 of them. The main difficulty encountered on the path from discovery to the patient’s bedside is that of the lengthy ‘pipeline’ process which all drugs must follow if they are to gain approval. From a pool of sometimes tens of thousands of drug maybe 5 are chosen to enter the trials stage, then comes the FDA review, followed by the selection of the final drug. The procedure takes up to 14 years and can cost up to a billion dollars.

One way we have sped up this process in recent years is with our knowledge of the human genome. We now have an unprecedented ability to ‘unzip’ chromosomes, and decode the genetic ‘instruction book’ for life – and by extension – disease. The example given by Dr. Collins was in his treatment of the degenerative condition called Hutchinson Gilford Progeria Syndrome. The knowledge of molecular pathways gained through the human genome allowed Dr. Collins to discard the many compounds which may have been effective and cut straight the one that was, saving months if not years of painstaking work.

The problem here is not just the amount of time, however. Often the final drug isn’t even particularly effective for the intended treatment and often 14 years of work is retired to the freezer. The kicker here though is that many drugs which have been developed for treating one disease and have wound up being less than effective have often proven themselves highly effective at treating another condition.

A 2011 article entitled Could pharma open its drug freezers? explains how many initiatives based around the re-purposing of these ‘shelved’ drugs are already underway – with plenty of success stories already established. One such success story is that of thalidomide, a drug with a very shady past but one that has recently shown to be highly effective at treating leprosy and myeloma.

Not only is this a highly effective way of helping patients who are finding new treatments in ‘old’ drugs, but it is also already broadly pursued as a route to cost-effective drug development for compounds with known safety profiles: Pfizer’s Indication Discovery Unit (IDU) is dedicated to repositioning, biotechnology companies have built business models around the strategy, and academic groups have applied this approach to neglected diseases.

The talk is a real eye-opener, even by TED standards. Why not check it out for yourself below?

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About the author: Conor Hughes works as a Marketing Executive at Life Science Recruitment

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