Miracle Cure or Media Hype?

Last week's BioNews reported on the development of new drug called PTC124, which offers hope to patients with Duchenne muscular dystrophy (DMD), as well as cystic fibrosis (CF) and perhaps a number of other genetic disorders.

The drug is currently at the stage of clinical trials after successful trials on mice. PTC124 appears to have the potential to compensate for 'nonsense mutations' responsible for some genetic conditions. Some cases of DMD, along with other inheritable conditions such as CF, haemophilia and beta thalassaemia, are genetic disorders that can be caused by nonsense mutations - a single mutation that causes protein manufacture to stop early, resulting in the production of a faulty protein. The drug PTC124 works by binding to the cell's ribosome (where the gene is translated into protein) allowing it to ignore nonsense mutations. The journal Nature recently reported that studies on mice had shown the drug was able to restore muscle function normally lost in muscular dystrophy. Trials have already begun in humans although it will be a few years before the full results of the clinical trials can be established.

Following articles in the New Scientist and Nature on the 22 April, the story was reported by four major national news organisations, amongst others. The Times announced a 'Daily pill to beat genetic diseases'; the BBC proclaimed that 'Drug 'mends' muscular dystrophy'. '"Superpill" could treat 2000 genetic diseases" declared the Daily Mail and the Telegraph said that the 'Drug could treat 2000 hereditary disorders'.

Nonsense mutations are one type of mutation that cause single gene disorders. However, conditions including DMD and CF are caused by a number of different types of mutations. In actual fact only 10 per cent of CF in the UK is caused by nonsense mutations, and only 15 per cent of boys with DMD have a nonsense mutation. Therefore even if PTC124 did prove successful, it would only benefit a small number of patients. Whilst the Telegraph and the BBC do highlight this caveat early in their articles, the Daily Mail only vaguely refers to this point and does not make clear the low percentage of patients who could potentially benefit. In addition the drug trial is still in it's early stages, with two more phases to come before any possibility of the drug being licensed, an issue which none of the headlines addressed. Newspapers must use punchy headlines (often not written by the journalist who wrote the story) to capture the reader's interest, yet the complex process of drug development is slow and stringent. Although the results so far provide encouraging news and hope to patients, the reality is that it is highly unlikely that we will ever have 'a daily pill to beat genetic diseases' or a 'superpill'.

So how do patient groups feel about how developments such as these are reported? I spoke to Nick Catlin, the CEO of Parent Project UK, the DMD support group. He said they had had many calls from families looking for clarification after reading the reports on these developments. 'They [newspapers] did not make it clear that it's gene specific', Nick said, referring to the low percentages of cases that might actually be treatable with the new drug. Upon being asked which he would prefer, a powerful front page article raising expectations, or a small accurate article hidden in the middle of the paper, Nick replied, 'It's good that it's raised the profile of DMD. It would be nicer if the reports were clearer and less emotional, but we would rather have the front page even if it does raise expectations. That's not necessarily a bad thing'. Nick also highlighted the future work that would be necessary if this drug was to become licensed. 'In the past DMD has been diagnosed with a blood test rather than a genetic test so a lot of work will have to be done to identify which boys would have a treatable mutation'. His group have set up a DMD registry (DMD Registry Website) to collect gene variation information.

Progress in science should be congratulated and it is always encouraging to read of developments in research that will benefit mankind. We have to recognise the difficulties of reporting health and science stories, it is difficult to strike a balance between the newspaper's need to entertain the reader and the responsibility of accurate reporting. For patient groups it's a two edged sword - they benefit through gaining attention which can help raise money and the condition's profile, but the risk is that people lose trust in the veracity of reported information. We must keep a sense of proportion and allow people to be optimistic whilst being realistic.