When there is no chance of rescue, the parents of terminally ill patients keep on fighting. First for your own child, then for other children.
American doctors have made a potentially groundbreaking discovery in the treatment of one of the most deadly brain cancers, and their achievements would not have been possible without the generosity of parents who lost their own children in the fight against this disease. New perspectives will soon emerge for young patients being treated for diffuse intrinsic pontine glioma, also known as DIPG, which has always led to the death of a child.
Cancer develops in the part of the brain that is largely responsible for breathing and swallowing. As the tumor grows, it attacks more and more parts of the brainstem, and until now doctors could only watch it helplessly, notes Amar Gajjar, head of brain cancer research at St. Jude Children’s Research Hospital in Memphis. Since this type of cancer is inoperable, it is very difficult to treat. Moreover, since surgeries to remove the lesion are not practiced at all, doctors could not take tumor samples for testing beforehand, adds Gajjar.
It is for this reason that medicine has been helpless in the face of childhood glioblastoma of the brainstem so far, and its cure rates have not flickered for three decades. That’s why doctors recently asked parents of children with DIPG, a disease usually diagnosed between the ages of 5 and 10, for a special favor: they needed permission to collect brain cancer samples for autopsy within hours of the patient’s death, says Suzanne Baker. one of the research program managers from St. Jude’s Hospital.
The researchers were unsure how the parents would respond to such a request. “It’s such a debilitating and very aggressive type of cancer,” notes Gajjar. – When a child is ill, parents are usually shocked, from start to finish.
The doctors had the most ungrateful of tasks: they were to talk about the child’s autopsy with the parents who were just getting ready for a painful loss. Yet more than 50 families agreed, Baker notes. This is a truly remarkable response, considering that no more than 200 to 300 children a year develop [in the US] brainstem glioma.
For Lenore Wyant, the choice was obvious. Long time ago, both her husband and she entered the list of organ donors. And now that the opportunity had arisen to be of service to scientists seeking help for other children, this gift of the heart would be the Wyant family’s only relief for their son’s death. Foster passed away in 2011 at the age of only six. – He was our light – says the mother. – He was a wonderful child, a wonderful friend. He liked to tell jokes and to cheer others up.
Lenore Wyant took his cells for research purposes as a sign that he was still here somewhere with us, alive. Maybe as a star in the sky, she winks at me and says, ‘I’m still here, mom.’
For Howard Scott, in turn, handing over the tumor to scientists of his son, Tyler, created the possibility of at least temporarily taking control of the situation. The boy died in 2006, at the age of five, just 10 days after diagnosis. – At St. Jude told us they didn’t know where these tumors actually came from, says Scott, a Charlotte resident of North Carolina. – To which I replied: How could it be changed? As the father of a seriously ill child, I felt completely powerless. You are expected to be able to protect your own children and I have failed my son.
To honor the memory of Tyler, Scott founded the Tyler’s Treehouse Foundation, which has raised 520 people so far. dollars for research. – We are counting on the fact that one day we will help find a medicine for sick children – says the father. “My wife and I imagine that this is exactly what Tyler would expect of us. We have to help. We have to do something.
This kind of hope combined with compassion for others is common to many families of children affected by cancer, doctors say. If only three percent of adult cancer patients participate in clinical trials, this rises to 60 percent of childhood cancer patients, notes Peter Adamson, chairman of the Children’s Oncology Group. This attitude of parents contributed to the fact that the five-year cancer survival rate in children increased from 10% to in the 60s, to over 80 percent Nowadays.
Wyant and Scott are happy to know that someone else will be treated effectively in the future with tumor samples from their children. Tissues from deceased patients allowed scientists at St. Jude Hospital making important observations: 80 percent. of DIPG cases, several genetic mutations were found. The researchers from Germany and Canada, acting independently, came to the same conclusions as the Americans. Since these mutations are so common, it can be suspected that they directly affect tumor development. Knowing about it is a real breakthrough, especially as it concerns cancer, which until recently was a complete mystery to scientists and doctors, adds Baker. – This is a milestone – announces the researcher from St. Jude’s Hospital. – We feel very excited about this discovery, because until recently our knowledge was very scarce.
Listing the mutations present in diffuse brainstem glioma is also important because of their location, adds Malcolm Smith, a pediatric oncologist at the National Cancer Institute. The abnormalities in the genetic material concern genes involved in the formation of histones, proteins that affect the packaging of DNA in the cell nucleus. Histones and their associated chemicals make up the system that scientists have dubbed the epigenome, which determines which genes come to the fore and which genes go off, related to cell growth. DIPG is the first known type of cancer whose histones contain multiple genetic mutations, notes Smith. If we examine them, we will probably find out how brain stem glioblastoma develops. And, most importantly, now scientists will be able to initiate work on new, more effective drugs, says Baker. Although Gajjar is cooling her enthusiasm: it will take many years to patent the therapy.
Because even if doctors manage to select one of the existing, although not yet patented pharmacological agents for the treatment of DIPG cancer (companies have many of them in their laboratories), it will be a long time before these agents can be administered to sick children, even in experimental studies, adds Gajjar. . (…) Medicines must first undergo numerous tests, first in the laboratory, on animals, and then in the phase of clinical trials, in humans. “We’ve only just entered the path that could lead us to effective treatment of brain stem glioma, and it won’t be easy,” says Adamson, head of clinical pharmacology at Children’s Hospital in Philadelphia.
Assuming that most little DIPG patients die within a year of diagnosis, it is unlikely that any of today’s children have a chance to benefit from a discovery made by the St. Jude’s. “We’re in the early stages,” emphasizes Smith. “But finally we have an idea of where to find answers to our questions.”
Moreover, the new data came at a time when interest in DIPG’s cancer created hope for a better future, notes Katherine Warren in Frontiers in Oncology. More texts than ever before have been written about brain-stem glioma in the past five years. Successful scientists give hope to parents like Wyant and Scott. “It’s as if Foster’s life is taking on a new meaning,” comments his mother. Six-year-old Gabrielle Wyant is now playing in the room that once belonged to her older brother. Foster was exactly her age when he died in October 2011. Many of his belongings can still be found in the room, but now Gabrielle is king.
Researchers from St. The Jude Hospital was so concerned about the parents’ reaction to the proposal to autopsy their children after death that they conducted a detailed study of 33 families, the results of which were published last year in the Journal of Pediatrics. It turns out that all families today believe in the rightness of their decision, we read. None of the parents regretted this step, and 91 percent. stated that in similar circumstances it would have done the same again. “I can’t imagine going this road without faith, without hoping to see each other again,” Wyant says today. – These moments of joy, this joint laughter – there will come a time when we regain it, although it will be in a different world.
Foster’s mother says that even if the research on her son’s sample were to help only one family, all that effort would not be wasted. “Let it be at least one kid whose mommy won’t have to cry over him, but enjoy his growing up,” Wyant says hopefully.
Tekst: Liz Szabo