When a child is diagnosed with a life-threatening cancer, you’d think the hardest battle would be the disease itself. But for families like the Bitzers, the fight for survival stretches far beyond hospital walls, into bureaucracies, bank accounts, and GoFundMe pages. In 2023, three-year-old James Bitzer was diagnosed with stage IV neuroblastoma, an aggressive pediatric cancer with a relentless course. Two years and multiple relapses later, his family is navigating a painful truth: the most promising treatments, cutting-edge immunotherapies, and advanced clinical trials often remain out of reach because of where you live, what insurance you have, or how much you can raise from strangers online. This isn’t just James’s reality. It’s a devastating pattern. Across the country, families confronting high-risk pediatric cancers are forced to become full-time fundraisers just to keep their children alive. While science continues to deliver new hope, through breakthroughs like GD2-directed therapy and CAR-T, access to that hope remains deeply unequal. This article explores how grassroots philanthropy has become a default safety net for families in crisis, why that model is untenable, and what needs to change if we’re serious about closing the gap between innovation and impact.
A Family’s Fight for Access: James Bitzer’s Story
In the winter of 2023, James Bitzer, then just three years old, was diagnosed with Stage IV neuroblastoma, a rare and aggressive pediatric cancer. Now five, he is enduring his second relapse, after surviving multiple rounds of chemotherapy, surgery, and chemotherapy, surgery, radiation, numerous blood transfusions, emergency overnight hospitalizations, immunotherapy, chemo-immunotherapy, anti-GD2 vaccine trial, and DFMO maintenance therapy.
As they prepare for what could be the most critical phase of James’s care, his parents, Jason and Beta, are navigating the painful reality of pediatric cancer in America: the science may be promising, but the system is not. They’re pursuing access to advanced therapies, including clinical trials, but much of what could offer James a fighting chance isn’t covered by insurance. It’s up to them to cover travel, housing, and experimental tests out of pocket.
Some of these costs are staggering. Jason is currently paying over $1,000 a week just to support ongoing stool sampling that tracks how James’s microbiome shifts during immunotherapy. This is part of a broader effort to study potential markers, like Bifidobacterium, that may help unlock the effectiveness of emerging treatments. But until a trial is approved or sponsored, the burden falls on them.
“I will go broke and give everything to save my son,” Jason said. “I don’t care about that, but I have to keep a roof over my family’s heads.”
Like so many parents in their position, the Bitzers have turned to their community, and strangers online, for help. They’re fundraising not just for James’s treatment, but for the right to even pursue it.
James’s story is deeply personal, but it is not unique. Families battling high-risk pediatric cancers are forced to fight on two fronts: against the disease, and against the barriers that determine who gets access to hope. Even in an age of unprecedented discovery, survival can still depend on something as arbitrary as a zip code or whether your story goes viral.
Sidebar 1: Neuroblastoma Primer
What Is Neuroblastoma?
Neuroblastoma is a rare pediatric cancer arising from immature nerve cells, typically in the adrenal glands, neck, chest, or spine. It is the most common extracranial solid tumor in children and accounts for 15% of pediatric cancer deaths despite representing just 6-10% of diagnoses.
High-Risk Neuroblastoma:
Roughly 50% of children are diagnosed with high-risk neuroblastoma, often defined by:
Stage 4/metastatic disease
MYCN amplification (a poor prognostic marker)
Diagnosis after 18 months of age
Unfavorable histology or chromosome 1p/11q deletions
Standard treatment includes:
Induction chemotherapy
Surgical resection
High-dose chemotherapy with autologous stem cell rescue
Radiation therapy
Immunotherapy with anti-GD2 antibody (e.g., dinutuximab)
Maintenance therapy with isotretinoin
Anti GD2 vaccine trial; DFMO
Challenges at relapse:
Relapsed or refractory neuroblastoma remains notoriously difficult to treat, with survival dropping below 15% in second-relapse patients. Access to experimental therapies and clinical trials becomes critical, but availability, eligibility, and cost barriers often limit options.
Financial and Geographic Barriers to Pediatric Cancer Innovation
James Bitzer’s journey isn’t only a medical story; it’s a systems failure playing out in real time.
While the campaign does not provide exhaustive details on specific expenses, it is clear that the family is navigating a system where insurance alone is not enough to meet the demands of advanced cancer treatment. Like many families in similar situations, they have turned to public fundraising, not for experimental luxuries but for the basic means to pursue the next step in their son’s care.
James’s story is one of thousands like it. Across the United States and abroad, families dealing with relapsed or high-risk pediatric cancers routinely use platforms like GoFundMe to raise money for care-related expenses, including travel, housing, and access to clinical trials. Some families, especially those seeking newer therapies like CAR-T or investigational antibody treatments, have had to raise hundreds of thousands of dollars just to receive care abroad or outside of their home network.
These experiences point to a broader systemic gap. Despite significant advances in pediatric oncology, funding and access remain uneven. According to the National Cancer Institute, less than 4% of its annual budget is allocated to pediatric cancer research, a figure that has remained relatively stagnant despite advocacy efforts. As a result, many promising therapies are slow to reach clinical use, and access to existing trials remains concentrated in just a handful of major academic centers.
For families pursuing those trials, especially after relapse, the burden of participation can be immense, even when the science offers hope. Costs related to screening, travel, temporary relocation, and uncovered services can add up quickly. Without financial support networks, even the most well-informed and motivated families can find themselves locked out of potentially life-saving opportunities.
This raises uncomfortable but necessary questions: Why must so many families rely on donations to pursue medical care? Why are trial opportunities so limited in geographic scope? And what can stakeholders across biopharma, government, and advocacy communities do to lower the logistical and financial barriers that separate patients from progress?
James’s family isn’t asking for special treatment. They are asking for a fair shot: a chance to pursue the same care any child deserves, without having to fight both a disease and a system.
GD2 Therapies, CAR-T, and the Innovation-Access Divide
As James Bitzer’s family explores options following a second relapse, they find themselves in a narrowing landscape, not because promising science is absent but because access remains elusive.
For children with relapsed stage IV neuroblastoma, the treatment playbook is unforgiving. Standard-of-care options offer limited benefit once the disease returns. This is where emerging immunotherapies and cell therapies, particularly those targeting GD2, offer new hope.
GD2, a disialoganglioside expressed abundantly on neuroblastoma cells, has been a leading target in recent years. FDA-approved anti-GD2 monoclonal antibodies, such as dinutuximab and naxitamab, have already improved outcomes for patients at high risk. But when neuroblastoma recurs after these agents, more advanced, experimental tools, including CAR-T therapies and next-generation bispecific antibodies, become the next frontier.
One of the most inspiring examples of patient-driven scientific progress in this space comes from the nonprofit Band of Parents. In the mid-2000s, a group of families whose children were treated at MSK banded together to fund early-stage trials of Hu3F8, a humanized anti-GD2 monoclonal antibody. Their efforts directly supported research that helped save a child named James Goebel, who remains in long-term remission. The organization has since funded dozens of trials and research efforts in collaboration with MSK and other institutions, a model of grassroots-driven innovation.
Today, families like the Bitzers are looking for similar next-generation therapies to buy back time. CAR-T cell therapies that target GD2, such as GD2-CART, GPC2-CAR, or even dual-antigen approaches, have shown promise in early trials, but most remain available only at a handful of elite centers. These trials are often limited in enrollment, geographically inaccessible, or off-limits due to eligibility constraints, especially for patients who have already received extensive treatment.
Even when trials exist, navigating them is a full-time job. Protocols vary. Insurance may not cover screening. Families must uproot to participate. And in a second relapse setting, delays can make the difference between eligibility and exclusion.
The Bitzers are now exploring trial options, most recently a new study that focuses on resetting the immune system through gut health to aid in their fight against cancer. But like many families, they must weigh not only the science but the logistics, financial viability, and feasibility of pursuing treatment far from home.
This disconnect between scientific potential and patient access represents one of the most urgent challenges in pediatric oncology. As innovation accelerates, the infrastructure to deliver it equitably must keep pace. Otherwise, the most advanced therapies will remain out of reach for too many children like James.
Emerging Therapies and Clinical Trials for High-Risk Neuroblastoma
Anti-GD2 Antibodies:
Dinutuximab (Unituxin): FDA-approved; improves event-free survival in frontline maintenance therapy.
Naxitamab (Danyelza): Approved for relapsed/refractory disease in bone or marrow with limited soft-tissue involvement.
Next-Generation Immunotherapies:
GD2-CAR T cells: Engineered T cells targeting GD2; early trials show promise but face toxicity and persistence challenges.
GPC2-targeted CAR T: Alternative surface target under investigation at CHOP and Stanford.
Bispecific T-cell engagers (BiTEs) and immunocytokines (e.g., hu14.18-IL2) are in early-phase testing.
Targeted Agents:
ALK inhibitors (e.g., crizotinib, lorlatinib, ceritinib): For ALK-mutated tumors (~10% of cases).
B7-H3, PHOX2B, and MAGE-A4 targets: In development for immune or peptide-based therapies.
Clinical Trial Networks:
Pediatric MATCH (NCI)
Beat Childhood Cancer Consortium
St. Jude’s NBL relapsed/refractory platform
MSK Kids, CHOP, and Seattle Children’s CAR-T programs
Why Pediatric Cancer Care Depends on Philanthropy and Biopharma Support
For families like the Bitzers, funding is not a distant policy issue; it is the barrier between hope and despair. With each relapse, each new treatment option, and each promising trial comes a new price tag: not just for the therapy itself, but for travel, lodging, unpaid time off work, out-of-pocket diagnostics, and in some cases, compassionate-use access to unapproved drugs.
Increasingly, philanthropic fundraising has become the informal safety net for pediatric cancer families. GoFundMe campaigns, like the one created to support James Bitzer’s treatment journey, serve as both a financial lifeline and a window into a sobering reality: for many children with high-risk cancers, charitable giving is the only viable path to access emerging therapies.
In James’s case, community giving may determine whether he can enroll in a cutting-edge trial, a path that could be his last meaningful option. This model of reliance on grassroots philanthropy is unsustainable and inherently inequitable. It raises an urgent question: What responsibility does the life sciences industry have in closing the gap?
To its credit, the pharmaceutical industry is increasingly engaging in pediatric oncology, particularly in high-need areas like neuroblastoma. Some of the most significant recent advances, including dinutuximab, naxitamab, and ongoing GD2-CAR-T candidates, have been developed in close collaboration with academic institutions and supported by industry co-funding models, pediatric priority review vouchers, and philanthropic matching.
Programs like the NCI Pediatric MATCH Trial, which matches children to therapies based on tumor genetics, reflect public-private partnerships in action. So do manufacturing support initiatives that help scale cell therapies for pediatric populations, often too small to attract full commercial investment but too urgent to ignore. A growing number of companies are also funding “trial access subsidies,” which cover travel and housing costs for pediatric patients enrolled in their studies, a small but vital step toward broader trial equity.
What’s needed now is a more systemic approach to pediatric trial funding and access, combining:
Pharma-supported subsidy programs for non-clinical costs like lodging and transportation
Shared manufacturing platforms to reduce the cost of cell therapies for small patient populations
Expansion of pediatric-specific trial networks in underserved regions
And more widespread use of adaptive trial designs and real-world data to lower trial entry barriers for relapsed and refractory patients.
James’s story is a powerful reminder that philanthropy can fill urgent gaps, but it is no substitute for systemic solutions. The more that biopharma companies partner with families, advocacy groups, and clinical researchers, the more likely it is that the next generation of therapies will be not only effective, but accessible to every child who needs them.
Pediatric Cancer Cost Breakdown: What Families Actually Pay
The Hidden Cost of Survival
Even with insurance, families of children with high-risk neuroblastoma face staggering costs:
$800,000+: Estimated total cost of standard first-line treatment over 18-24 months
$30,000-$100,000: Out-of-pocket costs in year one (travel, lodging, lost income, co-pays)
$250,000+: Estimated costs for relapse or trial-based care, including non-covered therapies or international access
Relocation Burden:
Many families must relocate temporarily for trial access, often without housing support. Cities like New York and Philadelphia, home to top GD2/CAR-T centers, are among the most expensive in the U.S.
Global Inequities:
In Australia and the UK, families regularly raise $300K–$500K to access CAR-T or GD2-directed therapies only available in the United States
Only a fraction of children in low- and middle-income countries receive even basic neuroblastoma treatment due to cost and infrastructure gaps.
A Strategic Call for Investor and Industry Action
James Bitzer’s story is not just a portrait of perseverance; it’s a case study in the urgent need for collaborative, cross-sector solutions in pediatric oncology. As the biopharmaceutical industry continues to innovate at the frontiers of immunotherapy and precision medicine, the question is no longer whether breakthroughs are possible, but whether they will reach the children who need them most.
Pharma companies, nonprofit organizations, and academic centers each hold part of the answer. But it is only through intentional partnership that these pieces can come together into a cohesive system of care.
Organizations like Band of Parents and the Neuroblastoma Alliance UK have already demonstrated the power of nonprofit-led research acceleration, funding early-stage antibody trials, enabling compassionate use access, and supporting long-term survivors. The next step is to scale those efforts in coordination with sponsors and regulators. Pharma companies can:
Expand trial eligibility criteria in collaboration with advocacy groups, making more relapsed patients eligible for advanced therapies;
Fund infrastructure grants that help smaller hospitals partner with major centers like MSK, CHOP, or St. Jude to share trial access more broadly;
Support travel and treatment subsidies for trial participants through direct sponsorship or foundations;
And proactively offer compassionate use and expanded access programs, especially for patients with limited standard-of-care options.
These aren’t just altruistic initiatives but smart, strategic investments in the future of oncology. High-risk neuroblastoma is among the most biologically complex and treatment-resistant pediatric solid tumors. But that complexity also makes it a powerful proving ground for emerging platforms like CAR-T, bispecific antibodies, immunocytokines, and personalized cell therapies.
The long-term payoffs are immense. Success in this space won’t only improve survival rates for neuroblastoma. It will also accelerate our understanding of tumor microenvironments, immune evasion in children, and the manufacturing and delivery of advanced therapeutics at scale. Every innovation brought to bear here has the potential to ripple outward, improving therapies for sarcomas, gliomas, and even adult cancers.
But it starts with access. With intentionality. With the recognition that every delay in equitable access is a missed opportunity not just for one child, but for the field at large.
James’s story calls us to act, not just in admiration of one family’s strength but in determination to ensure that stories like his become less common, less desperate, and more hopeful. The tools exist. The science is moving. Now, it’s time for systems, funding, policy, access, and industry alignment to keep pace.
Donate to support James’s fight here
