Falling through the cracks: why young cancer patients need specialised research and care

In this exclusive interview, world-renowned paediatric neuro-oncologist Dr. Eric Bouffet, Emeritus Professor at the University of Toronto and former SIOP President (International Society of Paediatric Oncology), explains how targeted research, personalised guidance and international advocacy are rewriting the story for young people facing cancer.

At Anticancer Fund, our commitment is simple yet profound: turning evidence into care. Nowhere is this mission more critical than for Adolescents and Young Adults (AYAs) diagnosed with cancer. Straddling the line between paediatric and adult medicine, AYAs frequently fall through systemic cracks.

This feature is part of our Too Young To Stop campaign, dedicated to shedding light on the urgent, unmet needs of teenagers and young adults, and pushing for treatments tailored to their realities.

Can you explain what are AYAs and what are the main challenges they face?

"Adolescents and Young Adults (AYAs) fall into a gap between paediatric and adult medicine. Because by law, hospitals strictly must divide patients by age, these young people often miss out on specialised care.

It’s a very complex situation. We have identified that, in the context of leukemia, adolescent and young adults who were treated with adult protocol for leukaemia had a much lower survival than adolescent and young adults treated with paediatric protocols.

And in the context of brain tumours, which is my area of focus, you have paediatric brain tumours in adults. Sadly, many of these patients are treated like adult brain tumours with massive doses of radiation. These people will die sooner than if they had a paediatric type of treatment.

This is why raising awareness about AYAs is so critical: we urgently need to advocate for funding, build bridges between paediatric and adult medicine, and train specialists with expertise in both worlds."

Research

You are the investigator of a trial we co-fund, focusing on AYAs, called U-R-Immune Glioma. Can you tell us why this is an important study?

"It's a long story. It started with research conducted by Doctor Uri Tabori and myself. We were in what we call a twinning programme in countries where the level of consanguinity is relatively high, like Jordan or Pakistan. It was very intriguing because we were presented cases of patients who had a very aggressive tumour with what we call café au lait spots.

In the Western world, café au lait spots are associated with a condition called neurofibromatosis type 1, leading to the development of a benign tumour. But here, Doctor Tabori studied that the tumour was indeed malignant and was associated with specific gene mutations.

So, in 2015, we treat the first patients with immune checkpoint inhibitors, drugs used in lung cancer or in melanoma where the tumour has a high number of mutations. To our surprise, and it made us cry, we saw dramatic responses in kids who had failed treatment with surgery, radiotherapy and chemotherapy..

We developed a phase II trial and showed that in the context of recurrent high-grade glioma (tumours in the brain) where normally there is no survivors, we had a 40% survival rate after four years.

U-R-Immune Glioma is the next generation. It’s a trial for children with a condition called CMMRD or Constitutional Mismatch Repair Deficiency and is focused on brain cancer: patients are treated with immune checkpoint inhibitors upfront, and we try to avoid radiation or to delay radiation.

The study has started in Jordan and India. I don't have the results yet, but it's very exciting because this would be the first time that we can demonstrate that a high-grade glioma in a child can be treated successfully without the use of radiation and even without the use of chemotherapy. Only immune checkpoint inhibitors."

Because you can avoid radiation or delay it, there are fewer side effects? So, this is like a major improvement?

"Radiation is never good to the brain because it makes your brain aging much faster: you have less memory, you have less learning capacity. Math becomes a big challenge and learning a second language is nearly impossible for an irradiated child.

And of course, you avoid all the side effect of chemotherapy, which is immune suppression, low blood count, and risk of infection."

My Cancer Navigator

Anticancer Fund supports this trial, but it doesn’t stop there. We, alongside your work as a consultant for My Cancer Navigator, also guide AYAs towards the best treatment available.

"Certainly. And let me explain why this is important with a living example.

A week ago, I got an e-mail: "Can you help?" And I say, "Of course, what's the problem?"

The patient was in Poland, 19 years old with a paediatric tumour, so just falling through the cracks. She was initially seen in an adult hospital mid-June where she had a scan. They gave her steroids, said they would not do any biopsy and decided to observe.

Mid-July the patient started to deteriorate. The doctors decided to do a biopsy, and after that, they didn't do anything because they didn't know what to do.

By mid-August, she continued to deteriorate, and the neurosurgeon sent her to a radiation oncologist. But the radiation oncologist didn’t want to treat her because her condition was too bad. She was sent home undergoing palliative care.

And that's when they contacted me.

I was talking to the family with an interpreter, and I asked who decided not to give radiation, as this is what she needed. I sent an e-mail to the radiation oncologist asking him to reconsider. He never responded. I then contacted a paediatric oncologist and said, "Can you please take care of this patient?" He told me, "No, she's an adult."

So, I took my phone, my e-mail, my WhatsApp, and today the patient is in the Czech Republic and she's starting her treatment. That's just two hours away from where she lives.

She was stuck in a system where nobody was really caring about her because of the system itself: it's so rigid that nobody takes responsibility. For two months and a half, she went through a nightmare because people had no expertise and they were not communicating. That's a typical example of an AYA patient falling through the cracks. Organisations like Anticancer Fund can help to find a solution, which in this case was a treatment abroad."

She was falling through the cracks and losing precious time. How does My Cancer Navigator help prevent this?

"Of course! How many patients may die in this context because they knock on the wrong door, or they were referred to the wrong people, or nobody knew?

And it happens all the time. This case was in Poland, but it happens everywhere. Teenagers are sent to adult facilities and are treated inappropriately.

When a request comes in, you don't care about the age. You look at the situation, and you try to find what's the best for the patient. You have the knowledge about how to treat them, so of course you can help and provide a good service to the patient.

I'm always impressed with the work done ahead of these requests by the My Cancer Navigator team. It’s amazing and helps me a lot."

Message for AYAs

If you could share one positive or encouraging message with an AYA patient diagnosed today, what would you tell?

"It is very important to continue to promote AYA as a unique specialty. With the threat on finances and budgets, people are trying to run medicine as a business and want to decrease costs. And when you have a specialty like AYA oncology, it becomes a big issue because people don't want to invest here. So, I think it’s critical to advocate and to promote the development of national AYA programs and multinational collaboration.

To patients, I want to send a message of hope. Patients often feel lost in a maze when they have a tumour that doesn't fit the expertise of the local team, but there is always knowledge around, particularly in the AYA field.

It's all a matter of who you meet. Sometimes you feel obliged to stay with the same person because you say, "No, he's nice," or "She's nice with me." But it's not about being nice; it's about expertise. People should not have a feeling of betrayal when they seek a second opinion. This is normal. More and more people seek second opinions from Chat GPT, so why don't you seek a second opinion from expert human beings?"

Biography

Eric Bouffet, MD, PhD - Paediatric Neuro-Oncologist

Dr. Eric Bouffet is an internationally recognized paediatric neuro-oncologist, Emeritus Professor at the University of Toronto, and former Head of Neuro-Oncology at Toronto’s Hospital for Sick Children. After completing his medical education in France and clinical appointments in the UK, he led the Paediatric Neuro-Oncology Program at The Hospital for Sick Children (SickKids) in Toronto for over two decades.

A globally respected voice in oncology, he served as President of the International Society of Paediatric Oncology (SIOP) from 2016 to 2019 and was named among The 100 Most Influential People in Oncology in 2025. His primary research focuses on novel therapeutics, innovative clinical trials for paediatric brain tumours, and improving access to care in low- and middle-income countries. Dr. Bouffet has authored over 400 peer-reviewed studies dedicated to advancing treatment, rehabilitation, and advocacy for children, adolescents, and young adults.