From boos to hope: Challenging the dogma about deadly brain stem gliomas

Hailey Olson, seen here with Susan Chi, MD, and Mark Kieran, MD, PhD, is the first patient in a trial challenging the conventional wisdom about rare brain stem tumor. (Image: Sam Ogden, DFCI)

Hilary Olson had no reason to suspect that her daughter Hailey might have a brain tumor.

“Her smile was starting to droop a little, and one of her eyes was a little jumpy,” says the 6-year-old’s mother. “We took her to see a neurologist, and he thought she might have pinched a nerve.

“But when he sent us to Boston Children’s Hospital for an MRI,” she continues, “the radiologists sent us straight down to the emergency room.”

Hailey’s diagnosis came as a huge jolt: a rare, almost always fatal tumor called diffuse intrinsic pontine glioma (DIPG). “The doctors were shocked by the size of the tumor,” Hilary recalls.

DIPGs are so fatal because doctors haven’t had any success at treating them—and in fact haven’t known where to start. The problem has to do with where they occur. DIPGs nestle among the nerves in a portion of the brain stem called the pons, which controls our breathing, blood pressure and heart rate.

The pons is the area of the brain that regulates breathing. DIPGs are found in this area.

“For 40 years, we didn’t have the surgical techniques to safely take a biopsy of a DIPG,” say Mark Kieran, MD, PhD, director of the Brain Tumor Program at Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC), a partnership between Dana-Farber Cancer Institute and Boston Children’s focused on treating children with cancer. “In fact, it’s still part of the dogma taught to every oncologist—’Don’t biopsy brain stem gliomas’—because of fears about the risk of severe or fatal damage. And because we couldn’t biopsy DIPGs, we couldn’t study them to learn what makes them tick.”

Looking to turn that dogma on its head, Kieran has launched a clinical trial that takes advantage of the last 40 years’ worth of advances in neurosurgery and in our understanding of the biology of cancer to target and personalize DIPG treatment. Hailey is the first patient.

“At first the doctors told us that Hailey’s tumor was inoperable, but in that first week that we got the call telling us about this new clinical trial,” says Hilary. “It had been approved two days after Hailey was diagnosed.”

Kieran’s team is working with Boston Children’s neurosurgeons Liliana Goumnerova, MD, and R. Michael Scott, MD, to carry out surgical biopsies on children newly diagnosed with DIPG. All of the patients in the trial receive the current standard of radiation therapy, along with bevacizumab, a drug that stops the growth of blood vessels to and within tumors. They then receive other drugs based on the analysis of their tumor, including erlotinib—which zeroes in on a certain genetic mutation found in some tumors—and/or temozolomide—which attacks tumor cells directly.

In part about putting the trial together, Kieran says, was challenging the surgical dogma about DIPG. “A colleague, neuro-oncologist Michael Prados, MD, from the University of California, San Francisco (UCSF) and I advocated for a decade, saying that the time was ripe to take a fresh look at surgical sampling and analysis of DIPGs. We were booed off the stage of every conference we spoke at,” he says.

Kieran’s team knew the opportunity was there, though, because surgeons in France had started operating on DIPGs, taking biopsies from more than 20 children with DIPG with no ill effects. “The French blazed the trail, but they didn’t have access to the kinds of molecular tools we have here for finding out which treatments might work best for each individual tumor,” he says.

Mark Kieran, MD, PhD

Their break came three years ago when neurosurgeon Nalin Gupta, MD, PhD, from UCSF, spoke at a neurosurgical conference on Prados and Kieran’s behalf. Suddenly, everyone’s heads started nodding. “The difference was that we had a neurosurgeon, not an oncologist, speaking to neurosurgeons.”

The trial—currently open only at DF/CHCC, though another 19 centers will soon start participating—brings the hope that, in the future, more children will be able to survive this largely mysterious tumor. Kieran hopes to recruit between 25 and 100 children.

“For the first time, we should be able to give children with DIPG like Hailey personalized treatment options based on the makeup of their individual tumor,” Kieran says. “We have the opportunity to look within DIPG and understand why it differs so greatly from other tumors, which will help us map out better strategies for the future.”

When asked why she signed Hailey up for the trial, Hilary responds, “It was an easy decision. Knowing Hailey’s odds with DIPG, we had nothing to lose, and we know that what Dr. Kieran and his team learn from her will help other children in the future.

“Everyone has been really supportive,” she adds, “and the best part is that Hailey’s done really well so far. Her smile is back.”

6 thoughts on “From boos to hope: Challenging the dogma about deadly brain stem gliomas

  1. As a cancer survivor, I am very appreciative of the all the cancer survivors who went before me, especially those who participated in clinical trials.  Because of what we learned from them, the survivabilty for my cancer has increased from 30% to 70% – within my lifetime!  Thank you and best wishes for Hailey and your family.

    1. that is why we signed on for the study, she was the first and we’re glad we can help in any way we can and help little Hailey as well! thank you for the wishes, she’s a strong little lady!

      1. Hello! My nephew was just diagnosed with dipg. We need help in deciding what to do. He is already symptomatic and would like to contact anyone involved with this horrific cancer tumor. Please conatact me.
        Dawn VanVladricken

      2. I am so sorry to hear this horrific cancer has affected another child. My nephew was just diagnosed with dpig. I wish there was some way to get in touch with somone in this trial. My sister is trying to make a decision and it is so difficult without support. He is already symptomatic and time is of the essence here. Please reply here. It looks like I can’t leave my contact information, but anyone please help.

  2. My son was diagnosed Nov 2011 with D.I.P.G and is currently being treated at the jimmy fund. Now hearing this brightned my day. I will be asking his Dra about this study and it will benefit him in anyway

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