Medicine development is hunting magic bullets
Medicines are becoming increasingly precise and innovative, but at the same time increasingly expensive. With their innovations, it is up to universities to increase competition, thus causing prices to drop. This is what newly appointed Professor of Biomolecular Analysis Hubertus Irth argues. His inaugural lecture is on 4 December.
It was one of the most lauded discoveries in the life sciences: the complete mapping of the human genome in 2003. ‘Knowledge of all the human genes opened up a host of possibilities for discovering the cause of diseases,’ says Hubertus Irth. He is also scientific director of the Leiden Academic Centre for Drug Research (LACDR), and for decades already has been involved in research into new screening methods for medicine development. ‘The hope was that we would discover precise targets in these genes for fighting complicated diseases such as cancer and Alzheimer’s.’
Unfortunately, it proved more complicated in practice. ‘In pharmaceutical research, we are always on the hunt for magic bullets: that one substance that precisely “hits” the cause of the disease and thus cures the patient. Both scientists and the public hoped that the knowledge of the genome would result in a huge quantity of magic bullets, but the body proved more complex than the small number of 20,000 genes that were sequenced.’ However, the first successful precision medicines have been developed. Orkambi, a drug for patients with cystic fibrosis, is a good example of this. ‘This medicine focuses precisely on the one protein in the lung cells of these patients that doesn’t function properly, and improves its function,’ Irth explains.
However, Orkambi touches a hot potato in medicine development: the extremely high cost of innovative medication in particular. The production costs of this drug are negligible compared to the market price, Irth contends. ‘But despite all the technological advances, drug development continues to be long, risky and expensive process.’ It can take 10 to 12 years to develop a medicine. Many do not make the finish line or fail at a very late stage. ‘Drugs undergo, and quite rightly, very strict checks before they can be brought to market. But the manufacturer must be able to recuperate the cost of such a lengthy process.’
There is also discussion about high costs in relation to another important pharmaceutical development: personalised medicines. ‘We are gaining increasing access to technological resources that allow you to customise a therapy to a patient. For instance, by administering a particular drug to a patient on the basis of the presence of certain genes or by adjusting the dose.’ Leiden is at the forefront of this field of personalised diagnosis and possible therapy, together with its partners in the Medical Delta, says Irth. But these innovations involve high investments, which must then be recuperated from a smaller group of patients.
Job for universities
Irth is pleased this the subject public debate of public debate. ‘It is a difficult decision: on the one hand the enormous costs for society and on the other the health of patients. This is the ethical side of drug development.’ Irth believes that more competition in the pharmaceutical world could keep the price of innovative medication down. ‘Universities and medical centres also bear a great responsibility here: more so than in the past, we must ensure that our innovations reach the market. Our task is not only to develop academic technologies but to translate these into a professional, commercial environment.’