MEMBERSHIP IN ELITE CONSORTIUM OFFERS MANY BENEFITS TO RESEARCHERS, PATIENTS
Children’s of Alabama has become the only institution in the state – and among an elite group nationally and internationally – to be accepted into a unique clinical trials consortium focusing on personalized therapy approaches for children with malignant brain tumors.
Accepted into the Pacific Pediatric Neuro-Oncology Consortium (PNOC) in December 2020, Children’s membership in this distinguished group offers many advantages to researchers, clinicians, and ultimately to young patients, said Girish Dhall, M.D., director of the Division of Pediatric Hematology, Oncology, and Blood & Marrow Transplantation.
The consortium is comprised of about two dozen sites across the United States, Canada, Europe and Australia. Unlike other consortia, PNOC’s clinical trial portfolio includes neurosurgery trials with techniques such as convection-enhanced delivery, fluorescent agents and advanced imaging compounds.
“The only way to get access to certain cutting-edge or state-of-the-art clinical trials is to be invited by a drug company working on a multi-institution trial or in trials run by these consortia,” Dhall explained. “This means we have access to a multitude of open trials and our patients don’t have to go out of state to be enrolled in trials testing promising experimental drugs that are not yet FDA-approved.”
In addition to prestigious trial access, PNOC enables basic and translational scientists at Children’s and University of Alabama at Birmingham (UAB) to propose new trials of candidate drugs or therapies and fosters collaboration among them and researchers at other institutions, said Dhall, who was a founding member of PNOC about eight years ago when based in Los Angeles.
PNOC is unusual because of its focus on personalized therapies for children with brain tumors that aims to improve survival while reducing toxic, treatment-related side effects, he noted.
“Traditionally we use chemotherapy or radiation therapy for these cancers, but both just kill rapidly dividing cells and don’t discriminate between cells inside the cancer and cells that might normally be rapidly dividing inside the body,” he said. “The focus has shifted to understanding specifically what makes these cancer cells grow and divide by studying the DNA, RNA and protein inside the cancer cells to develop therapies that improve the efficacy but reduce toxicity to normal cells at the same time.”
Of PNOC’s current open clinical trials, Children’s will launch its participation in two: One in children with a uniformly fatal tumor called diffuse intrinsic pontine glioma, or DIPG; and another that combines two drugs for children with refractory or recurrent low-grade gliomas, “a population in which this is like a chronic disease and they progress from one therapy to another, so it’s important for them to have multiple options,” Dhall said.