So much of medical progress is accomplished through the science of research bolstered by the art of timing. At Children's Healthcare of Atlanta, the Ian's Friends Foundation Brain Tumor Biorepository is just one example of this. In less than 2 years, it has become a powerful weapon against brain cancer because of the serendipity of the right resources, people and, especially, timing.
The biorepository-established and funded through Ian's Friends Foundation in 2014-is designed to store fresh cancer cells for use at Children's Healthcare of Atlanta and other research facilities across the nation. The goal is to facilitate the sharing of these cells and support new opportunities for research.
"We need new treatments that will not only help children survive their illness, but survive it well," said Tobey MacDonald, MD, Director and Aflac Chair for the Neuro-Oncology Program at the Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta. "This allows us to break new ground-where we are not only armed with technology, but with a much more precise level of knowledge."
Limited Resources
Before the biorepository's existence, researchers often depended on other repositories at national facilities, which stored a very limited number of frozen cells that sometimes were categorized incorrectly or had been replicated so much that their usefulness was questionable.
In some cases, a researcher may have been able to obtain cells only to discover that "one tumor cell line is contaminated with another, or even worse, is actually not the cell line it should be at all," said Beverly Rogers, MD, Chief of Pathology at Children's.
"After years of supporting research projects, we found that the lack of available cells for research remains a major impediment to finding new cures for childhood tumors," emphasized Phil Yagoda, founder of Ian's Friend's Foundation.
But now, Children's Healthcare of Atlanta-which performs some of the largest volumes of pediatric brain tumor surgeries in the nation-can harvest fresh cells, catalogue and audit what was collected, and be a central clearinghouse for researchers seeking cures. In addition, it can collect tumors specifically tied to children, since scientists now know that the same cancer can sometimes present and act differently in children compared to adults.
Building the Infrastructure
Creating such a center might seem intuitive, but with the daily demands focused on bedside care, this project could have been delayed or scuttled if not for the persistence of Yagoda, who seeded the effort with $360,000-and whose own son, Ian, was diagnosed with an inoperable lesion in his brain stem.
"Honestly, this would not have necessarily been the first on my to-do list," said Rogers. "But suddenly, when you get the funding, and you realize how all these complex pieces have started coming together, it makes so much sense."
MacDonald agreed. "I'm not sure this was a project that would have flown at any other point other than right now-with the perfect timing of the right infrastructure, right resources, and right doctors," he noted.
Nationally-recognized neuropathologist Matthew Schniederjan, MD, has played a crucial role in the biorepository's development and success. By having him available to obtain, identify, and register the cancer cells, there's far less room for error when scientists ask to use the cells in their research.
The process is beguiling in its simplicity: Schniederjan checks surgery schedules and then reminds surgeons to collect fresh tissue for the repository. The repository then has a top-notch staff who grow, prepare, identify, and audit the cells for authenticity.
"I like what I do every day," Schniederjan said. "It feels that we're breaking new ground. It's a little surprising that it's taken so long for the medical field as a whole to realize how important this is."
The infrastructure of the biorepository is key to its success-making sure that each link in the chain is unbroken. That continues as more scientists inquire about taking samples. Through seamless legal approval, Children's is able to lend out the cells without patent, tissue engineering or intellectual property issues, which have also bogged down other similar ventures.
The result: sharing and fostering additional collaboration between Children's and other medical institutions, universities, and hospitals around the world.
Growing Impact
Researchers are already making good use of the cells available through the biorepository. For instance, Michelle Monje, MD, Assistant Professor of Neurology at Stanford University, published her findings about childhood pontine gliomas in Nature Medicine (2015;21:555-9).
The positive impact of the biorepository at Children's is a small, sweet victory for those who have seen so many battles lost against the worst of brain cancers.
"It gives you hope. It reminds you how research can make a difference," Rogers said. "I think about leukemia and how we now have a 90 percent survival rate. Just 40 years ago, the kids were dying from it.
This has potential; this sheds light," she concluded. "And with the right material, the right thought, and the right research, we can change the outcome of pediatric cancer."