US-based cancer charity, Dragon Master Foundation, is providing $300,000 to help fund an effort to make pediatric patients' data live in the cloud and available to researchers during a clinical trial.
The trial - which is being run by the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and Children's Brain Tumor Tissue Consortium (CBTTC) - is aiming to accelerate the process of testing new therapies tailored to the cellular pathways or mutations of brain cancers in individual children.
The organisations claim this is the first time live patient data is being made available this way.
Samples will be gathered and analysed quickly enough to use them for guidance in personalised treatments for each patient on the trial, which is for children with high grade gliomas, in particular diffuse pontine gliomas (DIPGs), Dragon Master said.
The DIPG trial is the first to use next-generation sequencing to inform an individualised treatment plan in children and young adults with newly-diagnosed DIPGs and use an open access research platform to further discovery.
Clinicians can take advantage of advances in gene expression and sequencing technologies to devise a plan using FDA-approved drugs based on each child's tumour characteristics.
"We have shown it feasible to collect and analyse samples in a timely fashion - and because not one single tumour looked like another, we've demonstrated the need for individualised, precision medicine approaches," said Sabine Mueller, MD and the trial's principal investigator.
"No longer will we have to rely on one-size-fits-all medicine. These patients will get treatments customised to their specific mutations to the full extent of our medical knowledge," Dragon Master president Amanda Haddock said.
"It is 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."
The trial will launch in late winter.
Since 2013, Dragon Master's founders, Amanda Haddock and her husband Richard, have been on a mission to create a database containing at least 50,000 human genomes to find better treatments and cures for cancers. In 2012, Amanda and Richard lost their 16-year-old son to the highly aggressive glioblastoma multiforme brain tumour.
In early 2015, the organisation partnered with five specialist hospitals in the United States to build awareness around the disease, and gather more biological specimens and other data necessary for ongoing research.
Genomic data associated with cancer is growing rapidly, but for rare childhood brain tumours there has been a lack of advocacy around the need to use genetic and demographic information to develop new treatments and a potential cure.
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