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In theory, the prescription of medication compensating the deregulation of molecular pathways should lead to better treatment of cancer. However, in a multicentre phase II study in France, this approach did not render longer periods of progression-free survival. The results, published in The Lancet, raise concerns about the implementation of personalised medicine in cancer.

In the study, 195 patients with metastatic cancer were included whose tumours showed deregulations in a molecular pathway for which specific treatments exist. Half of the group received personalised medication, even if the treatment was not approved for their type of cancer, while the other half was treated according to physician’s choice. All participants were informed about their medication allocation.

After a median follow-up of 11.3 months, no significant difference was found between the median progression-free survival of the experimental and control group (2.3 and 2 months, respectively). According to the lead author, oncologist Christophe Le Tourneau of the Institut Curie in France, the prescription of off-label medication based on molecular findings occurs quite often in practice, even though the efficacy of this approach has not been validated yet. “I understand why it happens: patients want to live and physicians want to offer help.”

These results, however, do not imply that personalised medicine will never be applied to the treatment of cancer, since the study has some caveats. For example, many patients had advanced disease and were unlikely to benefit from treatment in the first place and the matching of targeted treatments to cancer mutations relied on a simplistic approach, according to Apostolia Tsimberidou, an oncologist at the University of Texas, US. Nonetheless, prescription of off-label medication for cancer is not feasible in its current form, says Le Tourneau.

Sources: The Lancet, Nature

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