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It's time drugs regulation caught up with cancer science

Posted on 18 February, 2014 by Professor Alan Ashworth
Cancer therapies have traditionally emphasised the importance of the organ in which the tumour occurs. Drugs have been developed for lung cancer, for instance, or for bowel cancer. However, our analysis of cancer genomes is now giving insights which paint a more complex picture. Increasingly, mutations in the same gene may be found in quite different cancers, suggesting that they may share an underlying molecular similarity. This in turn could indicate that a drug directed against a particular molecular defect and developed for one cancer might also work for another.

The same situation may hold true when comparing adult and childhood cancers. For example, a molecular alteration in the ALK gene that occurs in adult lung cancer is also seen in cancer in children, not in lung cancer but in a nerve tumour called neuroblastoma. We urgently need new treatments for neuroblastoma -- less than two-thirds of children survive the disease, and there is often little that doctors can do for those with the most aggressive form. Therefore, it seems logical that drugs that target this altered ALK gene which are licensed to treat adult lung cancer might be worth trialling in children with neuroblastoma. This is where a problem arises that we highlighted at a recent press briefing, a story picked up by TV, radio and many national newspapers.

We discussed a loophole in EU roles allowing pharma companies to avoid running a paediatric trial simply because the adult cancer a drug was designed to treat doesn’t occur in children -- even when the drug looks like it could work in children’s cancers because of their molecular make-up. This loophole has been widely exploited: in recent years, exemptions for having to run paediatric trials have been granted for 14 out of 26 drugs where there was evidence that the agent was worth testing in children.

The treatment for children’s cancers has improved immensely over the past few years. Now 80% are cured. But if we are to push up this number further and reduce the burden of very serious lifelong side-effects suffered by many of those who do survive, we urgently need to gain fair and timely access to new drugs.
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