Rare but deadly, infiltrating brainstem glioma is one of the deadliest forms of childhood brain cancer. One child, however, seems to defy the statistics: his treatment, unique in the world, raises great hopes for research today.
Despite advances in the treatment of pediatric cancer, the tumor, which affects 50 to 100 children and teenagers every year in France, represents a challenge for the medical world, at the center of World Day on Thursday.
Although the five-year survival rate for pediatric cancers now approaches 85%, some of them, including brain stem infiltrating gliomas, remain with a poor prognosis.
Inoperable, these cancers are usually treated with radiotherapy, which sometimes helps slow the disease but whose effects are short-lived. No drug has yet demonstrated its effectiveness.
Progression is very rapid indeed and the outcome is usually fatal within 9 to 12 months after the tumor is discovered.
A Belgian child, Lucas, who is now 13 years old, has defied all predictions: diagnosed with this supposedly incurable disease at the age of 6, he is now considered cured, his brain no longer showing signs of a tumor.
“Lucas has exploded all the counters of life,” his doctor, Jacques Grill, the pilot of the brain tumor program in the pediatric cancer department of the Gustave-Roussy anti-cancer center south of Paris, rejoiced to AFP.
– “Biological Features” –
The pediatrician remembers with emotion announcing to Lucas’ parents seven years ago that their son was going to die.
At the time, his family came to France for treatment, where the little boy was one of the first patients to join a clinical trial to test the new drug, a targeted therapy.
From the beginning, Lucas responded very well to the treatment. “During the MRIs, I saw that the tumor had completely disappeared,” Dr. says Grill, who, despite these miraculous results, does not dare to decide to stop the drug. Until he found out that a year and a half ago, the child himself had stopped taking it.
“Such a case, I don’t know of anyone else in the world,” confides the doctor, whose team started researching this cancer about fifteen years ago.
What remains to be understood is why Lucas recovered and how his medical case could bring hope to hundreds of young people in the future.
A dozen other children included in the same clinical trial saw their life expectancy increase and were still alive many years after diagnosis — but their cancer didn’t completely disappear.
“The biological characteristics of their tumor” undoubtedly led to increased life expectancy, which explains their better response to treatment compared to other patients undergoing the same therapy, Dr Grill underlined.
– “Long term work”-
“Lucas’ tumor had a very rare mutation and we believe it was this mutation that made his tumor cells more sensitive to the drug,” adds the pediatrician, also a researcher at Insurm.
In an ongoing trial (Biomed), which compares the drug discovered by Lucas with a promising new treatment, the Gustave-Rousseau researchers are not only studying the genetic abnormalities of all patients’ tumors but also creating tumor organoids (3D copies of the patient). Tumors are studied in the laboratory to understand their biology and their sensitivity to drugs.
“Lucas’ case opens up real hope: we will try to reproduce in vitro the changes we have identified in his cells,” Marie-Anne Debilly, the teacher-researcher who supervised the work, explained to AFP.
Specifically, medical teams want to find out whether the DNA changes Lucas introduced, once “reproduced” in other patients, also result in their tumors shrinking.
If this is the case, “the next step will be to find a drug that affects the tumor cells in a similar way to these cellular changes”, suggests Mary-Ann Debilly.
Enthusiastic about this new “therapeutic avenue,” doctors warn that it will take years to find a potentially curative treatment.
“It takes an average of 10 or 15 years between track and medicine, it’s a long-term job,” recalls Jacques Grill.
Published at 8:46 pm on February 13, AFP
