Failures are stepping stones to…

Research. In this very interesting article in BioMed Central, Gregory Petsko writes about three phases of drug testing during the development of newer drugs. The first phase is for the assessment of toxicity of the drug to humans and is tested on healthy population. The second phase is for assessing how well the drug works with patients with the disease. The third phase tests are for assessing the effectiveness of the drug in comparison to what is available at the moment in the market.

Apparently, 19 out of 20 drug trails fail. And, most of them fail at phase two. And, the interesting point that Petsko makes is:

My main point is that the Phase II failures represent an enormous, untapped resource for the biomedical sciences – a resource that could go a long way towards solving the problem of low productivity, in terms of cures, that plagues both industry and academic medicine.

You see, the Phase II failures have all passed Phase I, so they have been shown to be safe in humans. They failed for efficacy. They failed because they did not effectively treat the disease they were intended to treat, even though they showed biological activity in assays and model systems. There are hundreds of them – perhaps more than a thousand. I don’t know the number because drug companies bury those failures. They don’t want to release a lot of information about the molecules in question because, among other things, they fear that will give their competitors too much of an insight into what they are working on. But here’s the question I would like you – and them – to ponder. What if those drugs were not tried on the right disease?

We now know that many quite different diseases share common pathways and processes in the cell. Cancer is a disease of abnormal cell survival; in Alzheimer’s disease the survival pathways have failed. Alzheimer’s patients have significantly lower risk of many cancers. What if the cure for Alzheimer’s disease is sitting on some drug company’s shelf, as a potential cancer drug that failed in Phase II? (A biotech company called Link Medicines is currently testing one such failure to find out.) Gaucher disease and Parkinson’s disease both involve lysosomal damage and display aggregates of a protein called alpha-synuclein; Gaucher carriers are at elevated risk for Parkinson’s. What if a drug intended to cure Gaucher disease, one that failed in Phase II, is actually a treatment for Parkinson’s? (Another biotech company, Amicus Therapeutics, is beginning to investigate that possibility.) Recent studies show that people diagnosed with psoriasis are at greater risk of developing heart disease; in fact, in patients with severe psoriasis who are younger than 50 years old, the risk is comparable to that seen in diabetes. How many Phase II-failed psoriasis drugs have ever been tested in heart disease clinical trials?

A very interesting piece!

PS: While you are at it, these two pieces on mammalian pheremones are interesting too:

On the scent of sexual attraction

Darcin: a male pheromone that stimulates female memory and sexual attraction to an individual male’s odour

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