A woman with a rare cancer was cured. Now scientists have discovered why – science and health

Researchers from the National Center for Oncology Research (CNIO), in Spain, were able to show that the rare mutations to which she suffered enhanced the effect of the treatment she was subjected to, using the drug temsirolimus.

It was oncologist Jose Pablo Maroto who decided to use temsirolimus for this treatment, but little did he know that the results would be so positive.

The results of this research will now allow other kidney cancer patients with the same mutation to take temsirolimus or other drugs of the same family soon after diagnosis, because until now this type of drug is used intravenously only after other methods have not shown results – The so-called last line drugs.

to the worldCristina Rodriguez-Antona, researcher in CNIO’s Human Cancer Genetics Program, explained: “At the moment, temsirolimus-family drugs are not the first choice in the treatment of kidney cancer, but this finding suggests that they should be so in certain patients because they work well.” very “.

To reach this answer, scientists needed several years of investigation because it was necessary to find many patients in the same situation. The results indicate very rare mutations in the USP9X protein, which is responsible for regulating the cellular processes responsible for tumor growth. These mutations eliminate USP9X function so that the cell can no longer recycle its waste and dies.

“To understand the impact of USP9X mutations, we developed cellular models and performed tests indicating that cancer cells without USP9X have impaired cellular autophagy,” explains Cristina Rodriguez-Antona. By using Temsirolimus, it is possible to alter autophagy, the process by which cells recycle their waste products, due to their “synergistic effect” so that “tumors respond better to treatment.”