It’s a parent’s worst nightmare: taking your child to the doctor and receiving a life-changing diagnosis. It only adds to the heartbreak when they realize there is no effective treatment, and all they can do is hope for the best.
Few diagnoses strike fear into the hearts of parents and doctors more than a cancer called diffuse intrinsic pontine glioma, or DIPG. Primarily found in children, DIPG is a highly aggressive brain tumor that is uniformly fatal, Less than 10 percent of children Live longer than two years after diagnosis. Tumors grow quickly and in very important areas such as the spine and brain stem, making them exceptionally difficult to remove. Although young patients have been treated with radiation, chemotherapy and surgery, none have been cured of the deadly cancer.
However, for the first time, a 13-year-old boy named Lucas Zemelzanova from Belgium has defeated this challenge.
Various brain scans. Photo credit:
Diagnosed with DIPG at age six, Lucas’ doctor, Jack Grill, told Lucas’ parents, Cedric and Oleja, that he was unlikely to live long. Instead of giving up hope, Cedric and Oleja Lucas went to France to participate in a clinical trial called BIOMEDE, which tested new potential drugs against DIPG.
Lucas was randomly assigned a drug called everolimus in a clinical trial, a chemotherapy drug that works by blocking a protein called mTOR. mTOR helps cancer cells divide and grow new blood vessels, while everolimus reduces the blood supply to tumor cells and prevents cancer cells from reproducing. EverolimusThe tablet, which is taken once a day, has been approved in the UK and US to treat cancers in the breast, kidney, stomach, pancreas and others — but until the BIOMEDE clinical trial, it had never before been used to treat DIPG.
Although doctors weren’t sure how Lucas would respond to the medication, it soon became clear that the results were good.
“In a series of MRI scans, I saw that the tumor had completely disappeared,” Grill said In an interview. Even more remarkable, the tumor has not returned. Lucas, who is now thirteen years old, is officially considered cured of DIPG.
Even after the tumor was gone, Grill Lucas, head of the brain tumor program in the Department of Pediatric and Adolescent Oncology at the Gustave Roussy Cancer Research Hospital in Paris, was hesitant to stop treatment. Until about a year and a half ago, Lucas was taking everolimus once a day.
“I didn’t know when to stop, or how, because there was no other context in the world,” Grill said.
While Lucas is the only one in the clinical trial whose tumor has completely disappeared, the other seven children have been considered “long responders” to everolimus, meaning their tumors have not progressed more than three years after starting treatment.
So why did everolimus work so well for Lucas? Doctors think an extremely rare genetic mutation in Lucas’ tumor “made his cells very sensitive to the drug,” Grill said, while the drug worked well in other children because of the “biological characteristics” of their tumors.
While everolimus is by no means a cure, the trial offers real hope for parents and families of children diagnosed with DIPG. Doctors must now work to better understand why Lucas’ tumor responded so well to the drug and how they can replicate those results in tumor “organoids.” After that, “the next step is to find a drug that also works on tumor cells,” said Marie-Anne Debilly, a researcher at the BioMed trial.
A recent clinical trial tested a new immunotherapy treatment in young DIPG patients and showed promising results. Many patients’ tumors shrank and many participants saw functional improvements in their symptoms and daily life. But only one of the 11 patients has seen success that rivals Lucas’ — a young man known as Drew, who has been tumor-free more than four years after receiving treatment.
Once considered a certain death sentence, there is real hope for the first time. But there is much research and work to be done. Until then, however, Lucas’ doctors are thrilled.
“Lucas’ case offers real hope,” said DeBilli.
This article originally appeared two years ago. It has been updated.
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