Dragon Master Foundation has announced plans to provide $300,000 in funding for the first clinical trial that will make patient data available live in the cloud while the trial is underway.
The clinical trial is an initiative by the Pacific Pediatric Neuro-Oncology Consortium (PNOC) in partnership with the Children's Brain Tumor Tissue Consortium (CBTTC) to accelerate the process of testing new therapies tailored to the cellular pathways or mutations of each child's specific tumor. Samples will be gathered and analyzed quickly enough to use them for guidance in personalized treatments for each patient on the trial. The trial will be for children with High Grade Gliomas (HGG), in particular Diffuse Intrinsic Pontine Gliomas (DIPGs).
The DIPG trial is the first to use such large-scale, next-generation sequencing to inform an individualized treatment plan in children and young adults with newly diagnosed DIPG and utilize an open access research platform to further discovery in real-time. Clinicians can take advantage of advances in gene expression and sequencing technologies to devise a plan using Food and Drug Administration (FDA)-approved drugs, based on each child's tumor characteristics.
"We have shown it's feasible to collect and analyze samples in a timely fashion, and because not one single tumor looked like another, we've demonstrated the need for individualized, precision medicine approaches," says Sabine Mueller, MD, PhD, MAS, the trial's principal investigator.
"No longer will we have to rely on one-size-fits-all medicine. These patients will get treatments customized to their specific mutations to the full extent of our medical knowledge. It's truly precision medicine, and it is being enabled first for the most vulnerable of patients, those children with DIPG who so desperately need answers before their time runs out," says Amanda Haddock, President of Dragon Master Foundation.
The trial is set to launch in late summer 2017. Dragon Master Foundation's efforts to raise money for the trial have already begun.