IN JUST FIVE YEARS, TGEN AND DELL have turned weeks into hours, giving months and years back to children with cancer.
"In the first four years, we were looking at relapsed and refractory cancer, and they were incurable, but we've seen that change," explained Dr. Giselle Sholler, Chair of the Beat Childhood Cancer, formerly Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC). "We've even had some patients graduate from hospice. We've been able to take what we've learned, and now we will be able to treat them properly at diagnosis."
The NMTRC is a group of more than 40 universities, children's hospitals and research institutes worldwide working with TGen to develop new therapies for children with these rare and deadly cancers. This is the world's first precision medicine clinical trial for pediatric cancer.
Dell has provided more than $15 million in funding, employee engagement and computing resources in support of the effort, enabling Beat Childhood Cancer to expand the trials to Europe and the Middle East in 2015.
THE ART OF SEQUENCING
TGen recently sequenced the 200th genome from this cohort of young patients, and the scope of the project has expanded into osteosarcoma, other sarcoma subtypes and even more rare pediatric cancers.
"For many of these tumors, and particularly a swath of solid tumors that are higher risk and more aggressive, we don't yet have good treatments," explained Dr. Will Hendricks, Assistant Professor in the Integrated Cancer Genomics Division at TGen. "This is an area that is historically underfunded and understudied, in part because they tend to be more rare than adult cancers."
About 15,700 children under age 19 are diagnosed with any form of cancer each year, which is less than 1 percent of all cancer diagnoses, according to the National Cancer Institute. By comparison, more than 87,000 women and men will be diagnosed with melanoma this year.
Cancer is the second-leading cause of death in children behind accidents, and one of the challenges in treating pediatric cancer is that children are not "little adults."
"Adult cancers are caused by the environment or by us harming our bodies with things like smoking, and adult cancers have a lot of mutations," Dr. Sholler explained. "In children there are fewer mutations, and understanding the biology of the cancer and how it develops is different."
FINDING WHAT DRIVES THE CANCER
Based on initial findings from this study, Dr. Sholler predicted that in the future, children's cancers may not be treated based on the tissue biology - like lymphoma or neuroblastoma - but instead on the pathways and what drives the cancer growth.
Because of TGen's comprehensive approach - analyzing DNA, RNA and proteins - scientists and clinicians can identify new features of these tumors to help guide current treatment. They are also assembling a 'genomic landscape' across all the tumors that have been profiled at all phases that will serve as a powerful resource for the broader scientific community. Genetic profiling of these children's tumors has even guided diagnosis in some cases, empowering physicians to try different treatments.
"That's what's motivating and inspiring behind these studies," Dr. Hendricks said. "As genomicists, as research bench and computer scientists, we get to use our knowledge directly to help patients. We're seeing responses, seeing tumors shrink, seeing extended survival for some of these patients, and we're making a difference for these kids who don't have other options."
Initially, patients came into Beat Childhood Cancer because they had relapsed under the standard of care. Their tumors would be biopsied and sent to TGen for sequencing and analysis, then a tumor board - the team of scientists and oncologists - would assemble to discuss results and make treatment recommendations based on the patient's genetic profile. At first, the process could take a month or more from biopsy to treatment, but now it takes less than two weeks. Thanks to the collaboration with Dell, the computer processing time alone has shrunk from 10 days to six hours.
When Beat Childhood Cancer first began, the tumor board met perhaps once a month, but now, they are having two or three tumor boards per week.
PRECISION MEDICINE AS A FIRST LINE OF DEFENSE
The next phase of this project is to introduce this precision medicine process on the front-line when a child is first diagnosed with cancer, instead of after they have relapsed under the standard of care. This expansion has been made possible through the continued support of Dell and its Powering the Possible initiative.
To recognize their contributions TGen presented Dell with the 2017 John S. McCain Leadership Award, presented annually at its Founders Dinner to recognize individuals and organizations whose leadership and dedication have made a significant impact in the fight against disease and helping patients worldwide. Karen Quintos, Chief Customer Officer for Dell accepted the award.
"Technology is changing the world," said Karen Quintos, Chief Customer Officer for Dell. "Things that you thought were five to 10 years away are now in the here and now, and we don't want to wait another 10 years. We want to take that and figure out how to give that opportunity to the world."
