April 08, 2019
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Experts applaud $500 million proposal for pediatric cancer research, but say more needed

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Photo of Peter C. Adamson 
Peter C. Adamson
Photo of David Frazer 
David Frazer

President Donald J. Trump recognized pediatric cancer research as a national health priority during his State of the Union address in February, when he proposed $500 million over the next decade toward finding cures.

Experts on the subject expressed gratitude but said the White House proposal to cut the overall NCI budget may have a negative impact on pediatric cancer research that is unlikely to be offset by a $50 million annual directive fund.

Peter C. Adamson, MD, chair of Children’s Oncology Group, and the Alan R. Cohen endowed chair in the department of pediatrics at Children’s Hospital of Philadelphia, said the pediatric cancer community is grateful that the president addressed childhood cancer as a problem for the nation.

“This in and of itself will be impactful for the pediatric cancer community, as resources are still relatively limited and any significant additional resource is welcomed,” Adamson told HemOnc Today. “However, a concern that many of us share is that children with cancer benefit from a robust NCI budget. Growing, not cutting, the NCI budget will improve the outcome for children with cancer.”

David Frazer, CEO of the National Pediatric Cancer Foundation, said he applauds the focus of the president and NCI Director Norman “Ned” E. Sharpless, MD, on research for those most likely to benefit.

“Priority was placed on research that will impact children,” Frazer told HemOnc Today. “However, it is hard to place a set value on the effort to save one child’s life,” he added. “Theoretically, no amount of funding is enough until we find a cure for this horrible disease.”

‘Children are our future’

Cancer is the leading cause of disease-associated mortality in children aged younger than 19 years in the United States. Yet, only 4% of government funding for medical research is allocated to pediatric oncology.

“We know that one in every 285 children will be diagnosed with cancer by the time they are 20 years old. This equates to 43 children diagnosed daily with cancer and nearly 17,000 new cancer cases annually,” Frazer said. “We also know that 95% of those who survive childhood cancer treatments will have significant health-related issues by the time they are 45 years old. We need more funding to prevent cancer, and to find better treatments and cures for children to be able to live healthier lives.”

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Both the government and pharmaceutical sectors tend to base funding on the rates of disease mortality and morbidity, as well as on the more common cancer types, such as leukemia, breast cancer and prostate cancer.

“More adults than children are diagnosed with and die [of] cancer,” Frazer said. “However, there is an applied discrepancy that exists when studying the average age of diagnosis and the years of lost life to cancer. For example, the average age of an adult diagnosis is 67 years vs. 6 years for a child. Adults average 15 years of life lost to cancer vs. 70 years of life lost for a child. Children are our future and we need to invest more in saving them.”

Frazer drew comparisons between the cost of saving a child’s life and building a new highway or airplane.

“To put this all into perspective, the cost to build an F22 aircraft is $300 million and the cost to build a new, one-mile, concrete highway is $1 million,” Frazer said. “When we start to compare saving a child’s life to things like building an airplane or a highway, $50 million does not appear to be that much to save a child’s life. We need more funding for pediatric cancer research, and there should be a creative and strategic effort to find additional dollars to support the future lives of our children.”

Still, the proposed $500 million over the 10-year period has the potential to fund various investigative, translational and clinical trials, Adamson said.

“This is the very best time to increase the investment in childhood cancer research,” Adamson said. “We are at a remarkable point in medical history where the tools at hand for understanding the basis for childhood cancer and for dissecting pathways that may be amendable to treatment are now routine.”

Next steps

The next step is for Congress to accept the president’s budget proposal and increase the proposed funding for pediatric cancer research, Frazer said.

“Once funding is secured, the NIH/NCI needs to execute the stated vision to support areas with the potential to improve cancer prevention, detection and research,” Frazer said. “Specifically, there is a need for the development of new, more effective and safer, less toxic therapies for pediatric cancer, and we need to do this faster than the current norm.

“In addition, multiple systems need to be looked at to improve data management, the exploration of new therapies, and the reduction of what hospitals and research institutes charge for direct costs,” he added. “Lastly, Americans should increase their awareness of pediatric cancer and the lifelong impact on families and children. More support is needed for nonprofits that have been successful ‘gap fillers’ with valid ratings and missions that support pediatric cancer research to find a cure.”

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Pediatric cancer is talked about as if it were one disease, but the reality is that it is more than 100 diseases from a pathologic basis, Adamson said.

“As we begin to understand the molecular basis, these already-rare cancer cases are going to be divided into smaller and smaller subsets,” he said. “Because it is a constellation of rare and ultra-rare cancers, the economic return on developing a therapeutic for any one of these cancers is not always apparent, but the good news is that we have a remarkable example with larotrectinib.”

In November, the FDA granted larotrectinib (Vitrakvi; Bayer, Loxo Oncology) — an oral TRK inhibitor — accelerated approval for the treatment of adults and children with solid tumors that have a neurotrophic receptor tyrosine kinase gene fusion without a known acquired resistance mutation.

As previously reported in HemOnc Today, the FDA based the approval on pooled results of three trials that included a combined 55 adults and children with NTRK gene fusion cancers, including soft tissue sarcoma, salivary gland cancer, infantile fibrosarcoma, thyroid cancer, lung cancer, melanoma, colon cancer, gastrointestinal stromal tumor, appendix cancer, breast cancer and pancreatic cancer.

Results showed a 75% overall response rate, a 22% complete response rate and a 53% partial response rate across various tumor types. Of the 41 patients who achieved response, 73% remained in response for 6 months or longer at the time of data cutoff.

“The agent targets the NTRK pathway that is central to certain rare malignancies, including those that occur in infants,” Adamson said. “It is remarkably effective for cancers driven by NTRK in both children and adults. We hope this example will begin to set the stage in precision medicine that perhaps there are pathways for the private sector to begin to invest more in childhood cancer research.” – by Jennifer Southall

For more information:

Peter C. Adamson, MD, can be reached at Children’s Hospital of Philadelphia, 3401 Civic Center Blvd., Philadelphia, PA 19104; email: adamson@email.chop.edu.

David Frazer can be reached at National Pediatric Cancer Foundation, 5550 W. Executive Drive, Ste. 300, Tampa, FL 33609; email: dfrazer@nationalpcf.org.

Disclosures: Adamson and Frazer report no relevant financial disclosures.