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A pediatric brain cancer with no cure
Every morning, I trace my fingers over the photograph of my son’s radiant smile. A smile frozen in time when DIPG, a monster masquerading as a brain tumor, stole him at the age of six. My Neev – a vibrant boy silenced by DIPG in November 2021– embodies the devastating reality of pediatric brain cancer and the urgent need for policy reform. Neev endured 50 days of radiation bolted to a table by a mesh mask– my baby’s wide brown eyes screaming silently through the mesh. He went through four brain surgeries and four Phase 1 clinical trials with catastrophic treatment toxicities that left him paralyzed but still fighting to blink “yes” through tears. I learned what true helplessness feels like. The standard of care for DIPG hasn’t changed since 1962, when Neil Armstrong’s daughter died from it over 60 years ago!
This highlights the extraordinary challenges facing children and families. In the United States, approximately 47 children are diagnosed with cancer each day, nearly one classroom full of children. Pediatric brain tumors are the leading cause of death by disease in children. One in 5 children diagnosed will die of it.
More than half of children with DIPG, or Diffuse Intrinsic Pontine Glioma, the deadliest of them all, survive less than 1 year from diagnosis, and less than 10% survive 2 years from diagnosis.
Funding cuts stymie research & care
DIPG is a rare form of incurable pediatric brain cancer. Finding a cure requires consistent research over a long time, making funding harder to come by. Without government support, researching hard-to-cure diseases is nearly impossible. This year alone, the government has slashed over a billion dollars in funding to the National Institutes of Health. Slashing NIH funding doesn’t “trim fat”—it turns off lights, admin, IT support, among many other essentials for conducting research.
Neev’s body was ravaged by treatments designed for adults because pediatric research is an afterthought. Without NIH support, research stalls and trials collapse. Without trials, parents plan funerals instead of birthdays.
The consequences of funding cuts in biomedical research are vast. With less and inconsistent funding, the pace of research into pediatric brain tumors could slow dramatically, delaying the development of new treatments and potentially life-saving therapies.
Additionally, as Congress weighs a proposed $715 billion cut to Medicaid over the next decade, the consequences for children with cancer could be devastating. These cuts would affect an estimated 8.6 million people, disproportionately impacting vulnerable populations who depend on Medicaid for life-saving care. Medicaid currently insures nearly 1 in 3 children diagnosed with cancer in the U.S., providing critical access to pediatric oncology services, clinical trials, and out-of-state specialty care often denied by private insurance. For families battling aggressive diseases like DIPG, Medicaid can be the only bridge to urgent, experimental treatment. Leading advocacy organizations, including the American Cancer Society Cancer Action Network (ACS CAN) have warned that these proposed cuts threaten to unravel a vital safety net and widen existing health disparities.
How can you help?
Turning the tide on federal funding may not be in our hands, but our voices are ours to use, and as voters, our voices matter. This February, as a Rare Disease Legislative Advisory Committee Member representing the EveryLife Foundation For Rare Disease and as a Pediatric Cancer Patient Advocate with the Alliance For Childhood Cancer, I traveled to Washington, D.C., to meet with members of the House of Representatives and urge them to support or co-sponsor several critical bills introduced in the 119th Congress. These legislative asks are imperative to advance better treatment therapies, create meaningful incentives for pharmaceutical companies to develop drugs specifically for pediatric cancers, and to improve access to timely treatment. Please call your Senators and Representatives and let them know that accelerating cures and care for children with cancer is a priority for you. Urge them to support the following:
- Preserving the Give Kids a Chance Act (H.R. 1262 and S. 932). The umbrella Act combines two critical initiatives.
a. Give Kids a Chance Act: This authorizes the FDA to direct companies to study combinations of cancer drugs in children, ensuring they have access to the same innovative treatments as adults.
b. Creating Hope Reauthorization Act. This reauthorizes the Pediatric Priority Review Voucher (PPRV) program, which incentivizes drug companies to develop drugs for rare pediatric diseases. The PPRV program offers a voucher to companies that successfully develop treatments for rare pediatric diseases. This voucher allows for a faster FDA review (six months instead of 10) for another drug in the company’s pipeline, which can be a significant financial incentive. Alternatively, the voucher can be sold, providing companies with additional revenue. This market-driven approach encourages innovation without taxpayer expense. Since its inception, the PPRV program has led to the development of 65 new treatment options for rare pediatric diseases, of which only 4 are pediatric cancer drugs. This pragmatic nudge could lead to faster development of treatments for pediatric cancers like DIPG.
2. Accelerating Kids’ Access to Care Act (H.R 1509 and S. 752)
The Act will create a new pathway for pediatric providers to enroll in Medicaid programs in multiple states if they meet certain criteria. To qualify, the provider must be enrolled in their home state Medicaid program and also be in the lowest category for potential program integrity issues (have a clean track record). This would significantly reduce the time and resources currently spent on the cumbersome process of enrolling in Medicaid programs in multiple states.
Children with conditions like brain cancer often need to travel out of state for specialized care not available locally. The current requirement for out-of-state providers to undergo screening and enrollment in the child’s home state Medicaid program can cause dangerous delays in care, potentially worsening the child’s condition. This legislation aims to streamline insurance approvals, ensuring that no parent hears, “The trial starts tomorrow, but your paperwork takes 6 weeks.” Neev’s trial in New York required a catheter to be surgically implanted in his brainstem. We had days, not weeks, to decide.
I urge lawmakers to sit beside a child learning to swallow again after radiation burns their throat. To watch a 5-year-old cry as nurses search for a vein that hasn’t collapsed, and then explain why these bills can wait while defence contracts get prioritized. Neev’s last words, spelled through blinks, were “Love you infinity.” His legacy demands more than thoughts and prayers. My son’s heartbeat now exists in the laws that we shape.
“Unless someone like you cares a whole awful lot, nothing is going to get better. It’s not.” — Dr. Seuss