Scores of terminally ill cancer patients are being given fresh hope as part of a pioneering project to rescue potentially life-saving medicines from drug company vaults. The pharmaceutical industry has hundreds of new drugs sitting on company shelves, but most will never see the light of day.
Paul Watkins, 43, who has untreatable tumours in several organs, is the first person to benefit from a deal struck by a British cancer charity to unearth some of these potential blockbusters.
Cancer Research UK (CRUK) has borrowed a promising new chemical from GlaxoSmithKline (GSK) where it had been gathering dust while the company focused on more lucrative options. The charity will spend around £2m conducting the first human trials which will test how safe and effective it is in patients who have run out of other options. The innovative scheme could lead to a new cancer treatment for thousands within 10 years.
While venture capitalists already target shelved drugs in the hope of making millions from a new medical breakthrough, the charity's motives are altruistic. It will spend money only on research others deem too risky or complex to undertake.
It hopes to find drugs that will treat tumours affecting the pancreas, lungs or oesophagus, all of which have largely failed to attract interest or money from the pharmaceutical industry and university scientists. Consequently, there are fewer treatment options for such cancer sufferers. If the drug proves successful, any payback would be re-invested into more cancer research.
Other medical charities will be watching closely as CRUK is the first to negotiate such a deal in Britain. Five drugs have so far been rescued from drug and biotechnology companies and the charity is hoping to complete a sixth deal this year.
Nigel Blackburn, the director of CRUK's drug development office, said: "Choosing which compounds to take forward into trials is an art as well as a science, and drug companies do not always get it right. So there are, metaphorically speaking, test tubes of white powder compounds sitting on drug company shelves which could be new medicines. We will take more risks because our aim is to improve treatment options for cancers such as those affecting the pancreas and lung, not to make a profit for shareholders. We want to make stuff happen that otherwise would not."
Mr Watkins's survival has defied all expectations after several cycles of chemotherapy failed to halt the spread of a rare form of kidney cancer diagnosed in May 2008. The cancer has spread to his stomach, lungs, breast, spine and lymphatic system.
He leapt at the chance to participate in the trial despite a host of terrible side-effects, including permanent hearing loss and facial nerve damage, from earlier chemotherapy.
Mr Watkins, from Wakefield in West Yorkshire, said: "I'm not ready to die yet and this is my last chance. I'd sign 10 million disclaimers just to be allowed to continue on the drug, at whatever dose, just to see if it worked. I know there isn't a miracle cure but, if it would just stop the cancer spreading – I don't care if there are side-effects – it would be a price worth paying."
Scientists hope the new drug will hamper the enzyme, aurora kinase, which misbehaves in many cancers. Doctors will carry out targeted tests to check that the drug does in humans what it did in the lab.
If it passes these rigorous tests, GSK has the opportunity to take it back and develop it further. If it declines to do so, CRUK can offer it to other companies. Subsequent trials can be 30 times more expensive because they involve thousands of patients, making them too expensive for CRUK.
But if the drug succeeds and gets into the lucrative market, the charity will get a lump sum and a share of future royalties as reward.
In recent years, as profits have stalled, drug companies have become more open to collaborative ways of working. GSK has deals with universities, competitors, and now charities, in order to share costs and knowledge, and so improve their chances of uncovering the next wonder drug.
Mr Watkins said: "The only time I've cried was last November when the doctors told me there was nothing more they could do for me, and that I may only have another 20 days. But here I am; I feel so lucky to have been given this last chance. And even if it doesn't help me, I hope it can save others."