[Science] Many cancer drugs can be co-opted to treat different types of tumour – AI

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[Science] Many cancer drugs can be co-opted to treat different types of tumour – AI


By Michael Le Page Hero Images Inc. / Alamy Stock Photo If standard treatments for cancers fail, doctors sometimes prescribe drugs that haven’t been approved for that particular cancer type. In the Netherlands, this is now being done as part of a new kind of trial, so we can get a better idea of which drugs work for what cancers – and which don’t. The results from the first 215 people show that a third of them did get some benefit from the “off-label” use of drugs. One or two had a complete remission, says Emile Voest of the Netherlands Cancer Institute in Amsterdam. Knowing about failures is just as important, because then we can prevent people who may not have long to live from being given drugs that won’t help them and could have nasty side effects. A new drug goes through an approval process for treating a specific disease, such as a certain kind of breast cancer. Once a drug is approved for one purpose, doctors can prescribe it for another purpose, although often health insurers won’t pay for off-label use. Advertisement Off-label This is typically done on an ad-hoc basis. One doctor will decide what drug might help a patient who has run out of other options, and there is no systematic way of reporting the outcomes to help other doctors facing similar dilemmas. “If you have a success, you cannot share it and if you have a failure you cannot share it,” says Voest. This off-label approach can help some people, but it can also go horribly wrong. In the 1980s, some heart drugs were widely used off-label in the US. Later trials suggest that this caused 50,000 premature deaths. So Voest and his colleagues have set up a more rational way of using off-label drugs for cancers. The starting point is to sequence the whole genomes of tumours in people for whom standard treatments have failed, and to use that information to identify drugs that might help them. For example, several drugs have been approved to treat breast cancers with a mutation that makes them produce excessive amounts of a growth factor called HER2. Some other cancer types also produce lots of HER2 so these drugs might work for them too. In the trial, a person with a particular tumour type is assigned get a drug that might help. Similar patients are assigned the same drug, until eight people have tried it. If none of them benefit, no more people with that tumour type will be given that drug. If at least one benefits, more patients are enrolled to see if others benefit too. Read more: Medical roulette: Dicing with death In the Netherlands, the trial has been set up with the help of hospitals, charities and pharmaceutical companies, which are donating the drugs free of charge. The number of patients is now over 1000. “It’s a national effort,” says Voest. Several other countries are adopting the protocol, including Canada, Denmark and Italy. Mathew Garnett at the UK Wellcome Sanger Institute thinks the UK should too. “This is a smart and flexible trial design,” says Garnett. “An international effort would help to increase the number of patients across a wider number of tumour types.” The findings of the trial should still be checked by proper randomised controlled trials, says Voest. But they have already led to health insurers in the Netherlands agreeing to pay for an off-label use of one drug even though this hasn’t been approved by the European Medicines Agency. Voest says the scheme has also made doctors more aware of the benefits of sequencing cancers. “It has really propelled precision medicine forward in the Netherlands,” he says. Journal reference: Nature, DOI: 10.1038/s41586-019-1600-x More on these topics: cancer

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