The world’s first cure of a child with brainstem glioma, one of the deadliest forms of brain cancer in children, is giving hope to French scientists. Despite advances in the treatment of childhood cancer, this tumor remains widespread, affecting between 50 and 100 children and adolescents In France it is a real challenge for doctors every year.
Although the 5-year survival rate for childhood cancers is 85%, some of these diseases, such as: B. Brainstem gliomas, a very poor prognosis. This inoperable cancer is usually treated with radiation therapy. This can sometimes stop the progression of the disease, but the effect is only temporary. There is still no drug that is completely effective.
The development is very rapid and the fatal outcome often occurs between 9 and 12 months after the tumor is discovered. A Belgian boy, Lucas, who is currently 13 years old, broke all of these predictions: After being diagnosed with this virtually incurable disease at the age of 6, he is now considered cured and his brain shows no signs of the tumor.
“Lucas has destroyed all the life indicators,” explains his enthusiastic doctor Jacques Grill, pilot of the brain tumor program in the pediatric oncology department of the Gustave Roussy Center in the south of Paris.
The pediatrician still remembers with emotion the moment he told Lucas’ parents seven years ago that their son was dying. The family had traveled to France to treat him and the boy was one of the first patients to take part in a study Clinic to try out a new drug, a targeted therapy.
Lucas responded very well to the treatment right from the start. “During the entire MRI scan, I saw that the tumor had completely disappeared,” explains Dr. Grill, who, despite these extraordinary results, did not dare to stop the treatment. Until a year and a half ago he saw that the child had stopped taking it himself.
“I don’t know of any case like yours in the world,” says the doctor, whose team began researching this cancer about 15 years ago.
What remains to be understood is why Lucas was cured and how his case could bring hope to hundreds of sick young people. A dozen other children in the clinical trial had life expectancies that exceeded statistics and are still alive several years after diagnosis. But his cancer isn’t completely gone.
The higher life expectancy is undoubtedly due to the “biological peculiarities of his tumor,” explains Dr. Grill his better response to treatment.
“Lucas’ tumor had an extremely rare mutation and we believe it is this mutation that made his tumor cells much more sensitive to the drug,” the pediatrician added.
In an ongoing study (Biomède) comparing the drugs Lucas received with a new treatment, lThe Gustave Roussy researchers don’t just investigate the genetic abnormalities of tumors of all patients, but also create tumor organoids, three-dimensional copies of patients’ tumors created in the laboratory to analyze their biology and response to treatments.
“The Lucas case offers real hope: we will try to reproduce in vitro the changes we detected in his cells,” explains Marie-Anne Debily, who is overseeing these studies.
The medical teams want to find out whether the DNA changes presented by Lucas, once “reproduced” in other patients, also lead to a reduction in his tumor. If this is the case, “the next step will be to find the drug that has the same effect on tumor cells as these cellular changes,” explains Debily.
Doctors are excited about this new “therapeutic clue” but warn that it will be years before a possible effective treatment is found.
“It takes an average of 10 to 15 years between the racetrack and the medication, it’s a long-term job,” remembers Grill.
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