A Journey Into the Unknown With My Daughter’s Aggressive Brain Cancer

Walking into the hospital to select a cancer treatment plan, I knew we were stepping into uncharted territory. My daughter Chloe had been diagnosed with an embryonal tumor with multi-layered rosettes (ETMR), a rare and aggressive brain cancer that, because of its rarity and complexity, has no established standard of care. She was 3 years old at the time.

ETMR tumors are relatively new in the landscape of pediatric oncology, and their rapid growth rate and unique characteristics make them difficult to treat using typical brain tumor protocols. With so few cases, researchers haven’t had the time or data to develop a definitive, proven treatment approach. In the absence of a standard protocol, I was provided with two options by Chloe’s care team: ACNS0334, the default treatment for pediatric brain tumors, and Head Start IV, an intensive chemotherapy-based clinical trial.

ACNS0334 had a certain weight behind it. As the default protocol, it felt more established, like the “safe” option… or as close to safe as anything felt at the time. But it came with a heavy cost: radiation on Chloe’s developing brain, something that would leave permanent effects and carry its own lifelong risks.

Head Start IV, on the other hand, didn’t rely on radiation, offering instead a high-dose chemotherapy regimen that aimed to preserve cognitive function. Yet it was also a clinical trial — a word that, in my early days as a “cancer novice,” felt a little like the Wild West. Could a trial offer the same level of stability? Was I putting her at risk by stepping into something so experimental?

From the start, I had questions and fears about how to make the right choice. I wanted a treatment path that would give Chloe the best chance of survival without compromising her future. And, if I’m honest, I wasn’t sure a clinical trial would be the answer. The word itself implied the unknown, something less established, and I worried about whether decisions would be based more on research than on her individual needs. But as I would soon discover, Head Start IV was a trial built with Chloe’s unique needs in mind — and it became the best choice we could have made.

I was in an unusual situation. ETMR is so new that it doesn’t have its own standard of care. Chloe’s care team supplied two options, applying their standard of care for pediatric brain cancers, or a clinical trial. I mulled that over for a second before concluding “That means for ETMR, both options are essentially trials.” That thought gave me peace. It leveled the playing field and allowed me to simply look at the treatment plans as that: treatment plans.

ACNS0334 was developed for children with brain tumors generally, but it also meant heavy radiation. Head Start IV, in contrast, was designed to avoid radiation wherever possible, relying on a rigorous chemotherapy regimen instead. It felt like a leap, a journey into the unknown, but it also offered Chloe something invaluable: a chance to come through this without sacrificing her cognitive future.

Head Start IV wasn’t just another clinical trial. The program was developed by Dr. Jonathan Findlay, one of the foremost figures in pediatric neuro-oncology, whose life’s work has focused on sparing children the long-term impacts of radiation. Findlay’s reputation is monumental: he’s spent decades pushing boundaries, challenging norms and driving research that could give children with brain tumors a shot at survival without compromising their potential. The program had evolved through generations, each iteration building on the successes and learnings of the previous one, backed by extensive data and a community of experts who believed in his mission. Knowing that Chloe would be part of a trial created by someone so respected, with a focus on preserving not only life but quality of life, gave me the assurance I needed to make this choice.

Although we are deeply grateful for her doctors at the Shawn Jenkins Children’s Hospital, being part of a clinical trial added a level of security I hadn’t anticipated. This wasn’t just Chloe’s immediate team overseeing her care; by joining Head Start IV, she was connected to a network of specialists across the country and abroad, who would review her charts, track her MRI results and provide input on every development. With each new layer of oversight, I felt an added sense of support, like reinforcements arriving to join her fight. Every extra set of eyes on her case was another safeguard, another commitment to her survival.

My trust in clinical trials grew through the human touch we experienced along the way. During Head Start IV, there were moments when Chloe’s care needed to deviate from the protocol, and it was in those moments that I saw just how committed her doctors were to her individual well-being.

One particularly traumatic experience involved a spinal tap to confirm her cancer’s status. The procedure went awry, resulting in a “traumatic puncture” and leaving Chloe in pain. After witnessing her distress, her doctors petitioned the trial’s oversight board to allow MRIs alone to monitor her remission. The petition was approved, sparing her from additional spinal taps unless deemed medically necessary.

Then, during the stem cell transplants, Chloe’s body needed extra support to recover. After each of the first two transplants, her team administered a boost of stem cells to aid her regeneration. But by the time she received the second boost, her stem cell reserves were nearly depleted, and her care team made the decision to give her the remainder.

To ensure Chloe’s safety, her doctors chose to cancel the third round of chemotherapy and stem cell transplant entirely. Each adjustment showed me that Head Start IV was not a rigid framework — it was a partnership with flexibility and compassion at its core.

Throughout all of this, I reached out with questions and concerns to Findlay and Dr. Gupta, the neuro-oncologists who had helped shape the HS4 protocol. Despite their demanding schedules and the volume of cases they manage, they responded with care, often within hours. They didn’t just answer my questions; they took the time to explain the science, offered reassurance and treated me as a true partner in Chloe’s care. These doctors weren’t only committed to advancing their research, but they were deeply invested in the lives impacted by it. Each conversation reminded me that we weren’t just part of a trial. We were part of a community dedicated to seeing Chloe, and other children like her, through to a brighter future.

Ultimately, choosing Head Start IV wasn’t just about treatment; it was about stepping into a shared mission, a community dedicated to finding a way to fight this cancer without needless harm. I had entered this journey with doubts about clinical trials, wondering if they were too untested, too unknown. But what I found was a trial deeply rooted in care, backed by knowledge and guided by principles I believed in. And it was only the beginning of seeing just how personal and flexible a trial like Head Start IV could be and how it would prioritize Chloe’s well-being at every turn.

As Head Start IV drew to a close, a new option came onto the horizon: Difluoromethylornithine (DFMO), a clinical trial focused not on treating existing cancer, but on preventing its return. DFMO works by inhibiting tumor growth and recurrence, which, for families like ours, is a proactive defense against relapse. It’s a way to protect children who have fought cancer once from facing it again, giving Chloe a chance at a future unburdened by recurring disease. Proactive! I never thought I would hear that word when it came to Chloe’s treatment, but like an oasis in the desert, it is magnificent!

But DFMO’s impact reaches beyond Chloe alone; her participation is essential to the trial’s progress, helping researchers better understand how well DFMO works at reducing the risk of relapse for children with similar cancers.

Every scan, every test result and every milestone Chloe reaches contributes data to a growing body of evidence that is establishing DFMO as a standard preventive measure for pediatric cancer survivors. This trial benefits Chloe, but it also relies on her journey to inform the care of countless others. I was grateful Chloe could join Head Start IV because it had already undergone three iterations, and I am so thankful for those who had trialed the patients. The opportunity to join DFMO felt like a culmination of everything we’d come to understand about clinical trials. It also wasn’t just a treatment option, but a shared mission, a partnership where Chloe’s experience could drive research forward. Each trial has become a layer of protection for Chloe, each one bringing her closer to a life free from cancer’s shadow and offering the same hope to others like her.

So, when DFMO was offered as the next step, there was no hesitation. DFMO was the next stage in a journey we had begun with Head Start IV, a continuation of the same mission to protect Chloe without compromising her future. And just as with Head Start IV, DFMO connected us to another network of professionals at Levine Children’s Hospital, a team as dedicated to Chloe’s future as they were to advancing research. As with the heads of Head Start IV, the care team at Levine has been every bit as receptive, responsive and engaged in Chloe’s treatment as anyone.

By this point, I am a proponent of clinical trials, not only because they offered Chloe the best care possible but because they are constantly evolving. They are built on compassion, flexibility and the dedication of those who want to see children not only survive but thrive. For us, trials like Head Start IV and DFMO weren’t just options; they were lifelines supported by people willing to go the extra mile.

Looking back on our journey through Head Start IV and now DFMO, I realize that clinical trials have provided a foundation of hope built on research, resilience and the dedication of professionals who have stood by Chloe every step of the way. Chloe’s fight against ETMR has required an immense amount of strength from all of us, but it has also required trust in the people and protocols that sometimes felt like stepping into the unknown.

Through Head Start IV, we learned that clinical trials aren’t just research experiments. They’re commitments made by people who refuse to accept limitations, who push the boundaries to find safer, more effective ways to help children like Chloe. And when DFMO became an option, we embraced it without hesitation, knowing it was part of the same mission. It was to give Chloe not only a chance at survival but a future free from compromise.

Today, as Chloe continues her journey, I’m grateful for the research community and for the power of clinical trials to blend science with compassion. In a fight where certainty is scarce, trials like Head Start IV and DFMO have become our lifelines, allowing us to believe in Chloe’s tomorrow with the confidence that she is in the best possible hands. Clinical trials may still carry a sense of the unknown, but for us, they have become symbols of possibility, courage and an unwavering commitment to a brighter future.

This story was written and submitted by Mark Younce. The article reflects the views of Younce and not of CURE®. This is also not supposed to be intended as medical advice.

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