Cancer treatments, these new allies in the fight against… rare diseases


Yasmina Kattou / Photo credit: JOE RAEDLE / GETTY IMAGES NORTH AMERICA / AFP

It is a hope for patients with a rare disease. In some cases, cancer treatments give miraculous results on patients affected by other pathologies. A diversion of use which may seem counter-intuitive at first glance but which nevertheless achieves feats.

At the Gustave-Roussy hospital, in the Paris region, at the height of his 11 years, Maxime is the first patient to have received an anti-cancer drug to fight against a rare genetic disease. Particularly affected mentally and physically, the pre-adolescent is not cured, but this treatment has turned his life upside down.

“He discovered life”

The young man thus began to walk alone, regaining muscle tone after a week of treatment. “It’s spectacular”, explains at the microphone of Europe 1, Stéphane, his father. A tablet every day in his favorite dessert cream has transformed him, he continues.

“Until the age of seven, it was as if he was in a deep coma. After this treatment, he discovered life. He is more focused and then it can also be seen in his eyes. Before, Maxime had more of a lost look. There, we realize that he is there, that he is present among us, “adds his father.

Big progress in perspective

Maxime’s blue eyes revived thanks to the drug, this is also what marked Doctor Birgit Gorger. The pediatric oncologist at Gustave Roussy had to fight so that the child could benefit from this therapy originally reserved for adults with blood cancer. The drug targeted the same genetic mutation responsible for Maxime’s rare disease. Thanks to an early authorization for trials, other children will soon be able to be treated.

“We will soon be able to have intrauterine detection to prevent children from having sequelae and to have the maximum effect on the development of the child’s intellect”, explains Birgit Gorger. A six-month-old Norwegian baby will soon receive the treatment. “That’s thanks to Maxime” rejoices Stéphane.



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