Right to risk it?

Does a terminally ill patient have the right to take potentially life-saving drugs that may not be safe?

No one wants to see someone die of a terrible disease, especially if a drug exists that could possibly save them. But what if the drug is unproven? Controversy erupted in 2013 after Josh Harvey, a seven-year-old American boy, was refused unlicensed drugs that could potentially cure a crippling viral infection. Josh had become infected while on immunosuppressant drugs after a bone marrow transplant to treat his cancer. The drugs weaken the immune system to prevent transplant rejection, making the patient extremely susceptible to any kind of infection.

A social media storm put the small biotechnology company Chimerix under immense pressure to supply Josh with the drugs. After initially refusing, claiming that it was too costly, Chimerix bowed to the pressure in March 2014. Josh was in hospital for several months but eventually left for home in July 2014.

Despite the success of this story, drug testing is in place to ensure that drugs are safe and effective for critically ill people. Some argue that once those rules are bent, the whole system is undermined, which 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?

Drugs that haven’t undergone substantial safety testing pose a much higher risk to the health of the patient. Still, critically ill people, or their families, may decide they want to gamble on an unproven drug. In 2003, 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 polysulfate 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 drug companies have shown little interest in 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 that might help others are being gathered. And if people die while taking dichloroacetate, it might harm the chances of proper clinical trials being organised in the future.

The film Dallas Buyers Club explored the ways in which HIV/AIDS sufferers used to import and distribute unlicensed drugs in the USA in the mid-1980s. In many cases, the imported drugs were incredibly successful. At the time, the taboo nature of the subject meant that only one drug was FDA approved, and often the patients knew better than the medical institutions what was good for them.

However, untested drugs are extremely risky. They could trigger unpleasant side-effects or even hasten death. The fact is that eight out of ten drugs that enter clinical testing are dropped because they are too toxic or simply don’t work. In recent years, expanded access regulations have made it easier for critically ill people to be treated using drugs that are still in development, but this remains a highly contentious issue.

About this resource

This resource was first published in ‘Drug Development’ in August 2014 and reviewed and updated in August 2014.

Drug Development
Education levels:
16–19, Continuing professional development