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Precision Medicine Aids 20% Drop in Pediatric Cancer Deaths

Between 1999 and 2014, pediatric cancer death rates dropped by 20 percent, suggesting that precision medicine techniques have led to treatment advances in certain types of cancer.

By Nathan Boroyan

- The pediatric cancer death rate declined by 20 percent between 1999 and 2014, according to new data from the National Center for Health Statistics (NCHS), indicating that precision medicine techniques and an emphasis on personalized care may already be showing an impact.

Precision medicine and personalized care for pediatric cancer patients

The NCHS data separated the pediatric cancer population into four different age groups (1-4, 5-9, 10-14 and 15-19). During the 15-year period, cancer death rates dropped for every group, ranging from 14 percent (5-9 year olds) to 26 percent (1-4 year olds).

Adolescents aged 15-19 had the highest rate of cancer deaths of all groups in 1999, 2006, and 2014. However, the cancer death rate for this group overall declined by 22 percent between 1999 and 2014.

In 1999, leukemia (29.7%) accounted for more cancer related deaths among children and adolescents than brain cancer (23.7%). By 2014, these rates flipped, with brain cancer (29.9%) becoming the leading cause of cancer deaths among 1-19 year olds.

Overall, leukemia and brain cancer were deadliest forms of the disease among children and adolescents, accounting for 53.4 percent of deaths in 1999 and 54.8 percent in 2014.

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David F. Arons, JD, Chief Executive Officer of the National Brain Tumor Society, said the NCHS data serves as "a stark reminder" of the need for a nationwide push towards developing "new and more effective treatments" for children with brain cancer, including those currently in development at precision medicine research centers around the country.

"Pediatric brain tumors have not become deadlier over the years – survival rates for these patients have stayed relatively flat for decades," Arons said. "The reason these patients now face the highest mortality rates is because while other areas of oncology have made great strides in recent years, pediatric brain tumor research has not generated advances that have translated into meaningful clinical benefit for the most vulnerable patients."

Other common cancer sites causing deaths among children and adolescents between 1999 and 2014 were bone and articular cartilage (10.1% in 2014), thyroid and other endocrine glands (9%), and mesothelial and soft tissue (7.7%).

The overall decline in the cancer death rate of children and adolescents may be attributed to "major therapeutic advances in treating some forms of cancer, particularly leukemia," the NCHS said. However, advances in treatment for other specific cancers have been inconsistent, added a new report from the American Cancer Society.

“We have seen significant progress when it comes to developing effective treatments for a variety of pediatric cancer sites and ultimately saving lives," said Katherine Sharpe, senior vice president of patient and caregiver support for the American Cancer Society. "But when we expand our view to look at all pediatric cancers, as well as long term health and survival, it becomes clear that there are still areas of research where progress is urgently needed.”

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Overall, the five-year survival rate for children diagnosed with all forms of cancer rose from 64 percent in 1975 to 84 percent in 2011, according to data from the American Cancer Society and Alliance for Childhood Cancer. However, the five-year survival rate is lower for diseases such as neuroblastoma.

The five-year survival rate for neuroblastoma is currently 78 percent, but that can dip to about 40 or 50 percent  for high-risk cases. For cancers such as diffuse intrinsic pontine glioma (DIPG), the five-year survival rate is practically zero.

Pediatric cancer research efforts are hampered by a variety of hurdles, says the ACS report.

For example, many pediatric cancers are unique to children and require separate research. According to the report, "this would require child-specific tumor banks and unique animal models for pediatric cancer drug testing."

Another hurdle is a lack of market incentives to draw industry investments in childhood cancer research and drug development, which puts the pressure on government and philanthropies to fund such initiatives. "Yet, historically, even combined, government and philanthropic funds have been insufficient to fill the gap," Arons said.

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Additionally, the small number of children diagnosed with any type of cancer can make clinical trials more challenging, making the coordination of research critical to ensure competition for the same patients is avoided.

“Recognizing that progress is still needed in childhood cancer, this report aims to describe the process by which childhood cancer drugs are developed," said Michael Link, M.D., co-chair of the Alliance for Childhood Cancer. “Describing the pediatric cancer research landscape and identifying barriers to progress allows us as a community to work collectively on ways to overcome these challenges.”

Increased investment, along with more education and outreach surrounding opportunities for clinical trial involvement, may help the precision medicine and research communities continue to lower the mortality rate in pediatric patients.

"Children with pediatric brain tumors and their parents are raising their hands and volunteering to be involved in clinical trials at a rate that far surpasses adult cancer patients, but unfortunately their courage has not been met with equal investment and progress in research and drug development," said Arons.

A recent report by the Blue Ribbon Panel of the Cancer Moonshot Initiative suggests improvements may be on the way. The panel would like to develop big data analytics and data sharing infrastructure to make it easier find and participate in clinical trials so patients are more likely to contribute information on their cancers. The panel also recommended an increase in funding to support pediatric cancer research.

A new collaborative arrangement may also bolster the use of personalized medicine and genomics for pediatric patients. Funded by philanthropist Denny Sanford, the Sanford Children's Genomic Medicine Consortium will bring together precision medicine researchers, genomics leaders, and big data analysts from five children's hospitals to develop ways to integrate genomics and personalized medicine more deeply into pediatric care.

A proposed $34.1 budget for the National Institutes of Health could also provide some relief. The budget represents a $2 billion increase over 2016 levels, which would help fund – among other programs and activities – precision medicine research.

The Senate Labor, Health and Human Services, and Education Appropriations Subcommittee approved the $34.1 billion budget in June. However, Congress has yet to greenlight the funding.


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