Pediatric Brain Tumors: Advances in Low-grade Glioma Care and Research

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Thea developed nystagmus when she was only 4 months old. Her parents first noted this on a Monday. By Saturday, she wasn’t sleeping, and was spitting up and screaming in pain. “I was in our local ER, watching them put an IV in my baby’s arm,” recalls her mom, Trisha. “Next thing I know, they are telling me, ‘You have to get to Children’s Hospital of Philadelphia.’”

Thea had hydrocephalus caused by a huge mass in her brain — a low-grade glioma of her optic (visual) pathway. Pediatric low-grade glioma is the most common type of brain tumor in children. Low-grade glioma is survivable, but patients nevertheless must undergo multiple different forms of therapy that may lead to long-term neurological deficits.

Thea had emergency surgery performed by Phillip “Jay” Storm, MD, Chief of the Division of Neurosurgery at CHOP who specializes in pediatric brain tumors. Recalls her mom, “Thea had almost more tumor than brain. It was surreal.”

Low-grade gliomas are usually slow-growing tumors and don’t spread, but as the brain has limited space in the skull and the glioma is typically embedded in sensitive areas, they can be difficult to remove without causing damage. Surgery is just the first step. Complex, comprehensive treatment to manage the tumor is needed, such as what Thea — who’s now 11 years old — received from CHOP’s Pediatric Neuro-Oncology Program. In conjunction with the program’s clinicians, CHOP researchers are pursuing improving survival and functional outcomes for patients living long-term with tumors. Ultimately, a cure for brain tumors lies in a better understanding of their biology.

“Thea’s tumor is located in a difficult spot, between major blood vessels and the optic nerve,” explains Peter Phillips, MD, Thea’s neuro-oncologist and former chief of the section. “She’s had many different forms of chemotherapy,” the most recent of which was doses of Avastin® (bevacizumab) every 4 weeks for several years.

“She’s tolerated it quite well,” says Phillips. “It’s difficult to get off of, though, without the tumor coming back — but we’ve been able to do that. She’s doing magnificently.”

Multiple specialties on the care team

Thea’s care has involved multiple specialty groups at CHOP. She’s had several more surgeries, some to place shunts. During her major surgery she experienced a stroke, not uncommon when a tumor is wrapped around a blood vessel; she therefore is followed by CHOP’s Pediatric Stroke Program. She has an aversion to eating, and so needs a gastric tube for feeding and consultations with the Clinical Nutrition Department. Team members from the Division of Ophthalmology and the Center for Rehabilitation help her with visual and motor deficits. “Her case doesn’t fit neatly into any one medical specialty,” says Dr. Phillips.

CHOP’s Neuro-Oncology team has wide expertise in optic pathway gliomas. “We’ve recently written updated guidelines for their management,” says Michael Fisher, MD, Chief of the Section of Neuro-Oncology and Director of the Neurofibromatosis Program at CHOP. “We’re conducting studies and planning clinical trials that incorporate functional outcomes and quality of life, such as preserving vision and hormonal function. And we’re looking at molecular features that predict their occurrence and behavior.”

The Neuro-Oncology clinical program at CHOP encompasses brain and spinal cord tumors, neurofibromatosis (NF), retinoblastoma, vascular tumors, proton radiation therapy, survivorship, and the neurologic complications that develop in children and adolescents with cancer. In 2017, the team cared for more than 1,400 unique patients.

Robust research program

CHOP Neuro-Oncology has a very active clinical research program with over 30 open clinical trials, providing patient access to cutting-edge therapies. CHOP also has a team of bench scientists who are conducting research on such brain tumor types as low-grade glioma, high-grade glioma, diffuse intrinsic pontine glioma, and medulloblastoma, in search of novel therapeutics that can hopefully one day be brought into the clinic.

Regarding specifically pediatric low-grade gliomas (PLGG) like Thea’s, CHOP researchers are leading the way to unlocking new discoveries that could further advance treatments for all pediatric brain tumors. The CHOP team has discovered key biological differences in how mutated genes combine with other genes to drive PLGG development and growth, and are working to identify new treatment targets. CHOP is not only digging into the molecular properties of these tumors, but is leading the largest data collection effort of biospecimens for PLGG across the world. What’s learned about PLGG could lead to discoveries about other types of brain tumors.

Adam Resnick, PhD, is a research scientist based in the Division of Neurosurgery at CHOP, Director of Data Driven Discovery in Biomedicine (D3b), and scientific chair for several consortia-based efforts, including the Children’s Brain Tumor Tissue Consortium (CBTTC), led by CHOP. “We’re driving this revolution, using data to promote shared understanding to leverage new technologies, cloud computing, and sophisticated algorithms for collaborative discovery to develop improved, less toxic therapies,” says Resnick. “Our goal is to develop an analytic infrastructure so physicians can deliver the right therapy to precisely target tumors, at the right time, to individual patients.”

CHOP is working to achieve that goal by integrating neuro-oncology, neurosurgery, research and big data.

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