'Personalised medicines made up 21% of new drugs in 2014. In 2018 the proportion was twice that
, which the nerve cells that control the body’s muscles need to survive. Zolgensma uses an empty virus-like particle that recognises nerve cells to deliver working copies of the gene to where it is needed. Priced at $2.1m per patient, it is the most expensive drug ever brought to market. That dubious accolade might not last long. BioMarin, another biotech firm, is considering charging as much as $3m for a forthcoming gene therapy for haemophilia.
A world in which markets shrink, drug development gets costlier and new unmet needs are ceaselessly discovered is a long way from the utopian future envisaged by the governments and charities that paid for the sequencing of all those genomes and the establishment of the world’s biobanks.
Daphne Koller, boss of Insitro, a biotech company based in San Francisco, shares Dr Scannell’s scepticism about the way drug discovery has been done. A lot of candidate drugs fail, she says, because they aim for targets that are not actually relevant to the biology of the condition involved.
One more reason for optimism is that drugs developed around a known molecule that marks out a disease—a molecular marker—appear to be more successful in trials. The approval process for cancer therapies aimed at the markers of specific mutations is often much shorter now than it used to be. Tagrisso , an incredibly specialised drug, targets a mutation known to occur only in patients already treated for lung cancer with an older drug.
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