Right to risk it
No one wants to see someone die of a terrible disease, especially if a drug exists that could potentially save them. But what if the drug is unproven? Does a terminally ill patient have the right to take it? Controversy erupted after Penelope London, a four-year-old American girl suffering from a neuroblastoma, a rare cancer of the nervous system, died in 2007 after being refused a potentially life-saving treatment.
The treatment Penelope's parents tried to obtain had only been tested in six people. The company developing the drug refused to make it available, saying that the risks were too high for Penelope and for its own commercial success.
Commentators also pointed out that the whole point of drug testing is to ensure that drugs are safe and effective for critically ill people. Once those rules are bent, the whole system is undermined. And it would open the door to unscrupulous people selling bogus potential cures to vulnerable people.
Nevertheless, setting society's needs against the incredible pain of a family with a dying child must be one of medicine's toughest ethical dilemmas.
A gamble with nothing to lose?
Still, critically ill patients, or their families, may decide they want to gamble on an unproven drug. The father of a 19-year-old victim of vCJD - the human form of mad cow disease - took an NHS trust to court to secure an untested treatment for his son.
After a long battle, the courts allowed the blood-thinning drug pentosan polysulphate to be infused into the teenager's brain. Although he is the longest-surviving person with vCJD, his condition has not improved. A recent review of vCJD patients treated with the drug found no evidence that loss of brain tissue had been halted. But with small numbers and no control group, it is difficult to draw any firm conclusions.
A slightly different but equally heart-rending tale centres on dichloroacetate, a simple chemical similar to acetic acid. It is being trialled as a possible treatment for rare mitochondrial disorders, and researchers in Canada wondered whether it might also work in cancer. A trial in rats was highly encouraging - it was highly effective and had few side-effects.
But dichloroacetate is not new and cannot be patented, so drugs companies have shown little interest taking it forward. Step forward Jim Tassano, owner of a pest-control and marketing company in Sonora, California. He was looking for therapies to help his ballroom-dance instructor, who was dying of cancer.
Remarkably, Tassano ordered dichloroacetate from a chemical supply company and with a chemist friend worked out how to synthesise it chemically. They sold it via a website (labelled 'for veterinary use' to keep things legal - although the Food and Drug Administration still moved to stop web sales in July 2007).
While sympathy must be with the patients clutching at straws, the case again raises difficult questions. Dichloroacetate could well have harmful effects in people. Because the patients are not in a clinical trial no useful data are being gathered that might help others in the future. And if patients die while taking dichloroacetate, it might harm the chances of proper clinical trials being organised in the future.
Untested drugs are extremely risky. They could trigger unpleasant side-effects, and even hasten death. The fact is that eight out of ten drugs that enter clinical testing are dropped because they either are too toxic or simply don't work.
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