First of all, we need to understand that cancer, at its core, is a genetic disease. In about 80% of cases, cancer arises sporadically. This is why cancer is often considered a disease of aging; the longer you live, the more time there is for genetic mutations to accumulate, increasing the likelihood of cancer, especially if exposed to mutagens such as tobacco, UV light, or certain viral infections such as HPV
Childhood cancers, however, are very different from adult cancers because they have much less time to develop. Some childhood cancers are even present at birth or diagnosed within the first few months of life. Biologically, childhood cancers often originate from different tissues than adult cancers. Typically, they arise from developing tissues that, during the organ formation, are rapidly growing, such as the brain, nervous system, or blood. Many of these tumors are restricted to a specific age timeframe in which the organ is developing and do not appear during adulthood. On the other hand, adult cancers more frequently originate in the epithelial tissues of organs such as the lungs, colon, or breast. Pediatric tumors also usually have fewer mutations, often just one or two drivers, and are generally more genomically stable than adult cancers. Also, the driver alterations in pediatric cancer are different from those in adults, such as the ETV6-RUNX1 fusion, typical in pediatric acute lymphoblastic leukemia but very rarely observed in adults.
In terms of diagnosis, it’s not so much that childhood cancers are harder to diagnose, but because they are rare, they may be mistaken for other common childhood illnesses, which can lead to delays in diagnosis, and access to healthcare plays a crucial role. In terms of treatment, drug approvals are highly specific to certain tumor types and patient populations, and because pediatric cancers are much rarer than adult cancers, conducting large-scale clinical trials for them is more difficult. This slows down the approval of new treatments for pediatric cancers, leading to fewer therapeutic options specifically designed for pediatric patients.
This creates inequalities in care. Pediatric patients often receive adult cancer treatments adapted for pediatric use, which might not be optimal for the patients. Even more, the quality of care a child receives can vary significantly depending on access to precision oncology diagnostics, the availability of targeted treatments, and regulatory approvals in different countries. Unfortunately, this means that some pediatric patients do not have the same treatment opportunities as others simply based on where they live.
Finally, another crucial consideration is the long-term effect of the treatment. Given the potential for long-term survival, minimizing treatment-related side effects is crucial in pediatric oncology. This focus aims to reduce the risk of late effects, such as secondary cancers or organ dysfunction, which can arise from aggressive treatments.
Diagnosing childhood cancers early is challenging, but I’m not sure if there’s anything specific to pediatric oncology that makes early diagnosis inherently different from adult cancers, except for the fact that routine screening tests for babies don’t typically cover oncology. Childhood cancer is rare, so widespread screening isn’t standard practice.
There are some exceptions, particularly for hereditary cancer syndromes. For example, retinoblastoma, which I focused on during my PhD, has a penetrance higher than 90%, meaning that if a parent has had it, there’s a very high chance their child will as well if they have received the mutated allele. In such cases, screening might not happen prenatally, but it would certainly be recommended to perform thorough and frequent examinations as soon as the baby is born. In general, late diagnosis in childhood cancer is often due to healthcare access rather than difficulty in identifying the disease. If a child has access to proper medical care, reaching a diagnosis is usually not the biggest barrier. However, if we’re talking about precision medicine diagnostics, such as next-generation sequencing, that’s a completely different issue. The availability of advanced molecular testing varies widely depending on resources and healthcare infrastructure, which can affect how quickly and accurately a child’s cancer is diagnosed.
What has definitely changed in the last 10 years is our understanding of the genomic landscape of pediatric tumors and how they differ from adult cancers. This has expanded our knowledge of what specific treatments these children need, rather than simply applying what works for adult cancers. Integrating genomic profiling into clinical practice has enabled more personalized treatment approaches.
In recent years, we’ve seen the development of targeted therapies and immunotherapies specifically for pediatric cancers, some of which don’t even exist in adult oncology. A particularly meaningful example for me is a case that’s close to my heart: my friend’s daughter had neuroblastoma and received a specific immunotherapy treatment called anti-GD2. This therapy targets a molecule that is overexpressed in neuroblastoma cells, a cancer that is almost exclusively pediatric and extremely rare in adults. The first antibody was approved in 2015 and the second one in 2020, and they have dramatically improved neuroblastoma patients’ survival. She is now, happily, cancer-free.
Of course, precision oncology has advanced across all fields, not just pediatrics. But, what has changed significantly in pediatric oncology is the growing awareness that it is not simply adult oncology in smaller patients. It is an entirely different field with its own biological and therapeutic challenges.
We’ve also enhanced the risk stratification of patients by identifying clinical and/or biological prognostic factors. This way, pediatric oncologists can define the best course of treatment based on each individual risk profile, improving treatment outcomes while also reducing potential toxicities.
I guess the example I just gave about neuroblastoma answers this in a way. Just as we have studied the mutational and expression landscapes of adult cancers, like breast cancer, where we can divide tumors into distinct subtypes or genomic landscape based on molecular characteristics. We have now begun to do the same in pediatric oncology. This kind of classification has evolved in pediatric cancer as well.
By understanding the genomic background of a tumor, we can better predict how it will behave. This not only helps in choosing more effective treatments - if a targeted therapy exists for a particular molecular alteration - but it also provides valuable insights into diagnosis and prognosis.
So, precision medicine is helping pediatric oncologists make more informed decisions, ultimately leading to better treatments and outcomes for their patients
That’s a tough one - what’s most promising is hard to say. Several advancements have transformed childhood cancer treatment. One example is liquid biopsies, which provide a minimally invasive method to monitor pediatric cancers. Also, there have been great innovations in radiation therapy, which can now target the tumor with high precision and minimal or no damage to the healthy tissue.
However, personally, I find immunotherapy treatments such as CAR-T cell therapy to be the most encouraging innovations in cancer vaccines.
In oncology, and especially in pediatric cases, one of the biggest challenges is tumor relapse. Even for cancers that were considered fatal just a few years ago, many patients now achieve full remission. But when the cancer returns, the chances of curing it drop significantly in many cases.
Cancer vaccines aim to address this by preventing relapse - stopping the tumor from reappearing. That’s their goal. This approach is still in its early stages, but we’re heading in the right direction. If we can successfully develop these vaccines, they could help cure many cancers where relapse remains a major challenge.
So, I wouldn’t say with certainty that cancer vaccines are the future, but I’m particularly eager to see how this field evolves in the coming years.
I think one of the biggest challenges in pediatric cancer is something that might seem obvious, but it’s important to say: the pediatric patient is not the one making the decisions. Parents carry the enormous burden of trying to understand everything that’s happening while making life-altering choices for their children. At the same time, they have to be emotionally present and strong for them.
Most importantly, parents need access to proper education so they can feel confident in their decisions, knowing they are not only prioritizing their child’s survival but also considering their future quality of life. For example, in retinoblastoma, a tumor of the retina, the safest option in many cases is to remove the affected eye. This procedure can save a child’s life. But many parents around the world refuse this option, not necessarily because they’re being irrational, but because they may not fully understand the long-term consequences of delaying or avoiding treatment.
This is very different from adult oncology, where patients typically rely on their oncologists’ expertise to make decisions for themselves. But when parents are making choices for someone so young, someone they love more than anything in the world, it becomes much more complex. That’s why advocacy efforts that provide family therapy, emotional support, and educational resources are so important. I’ve seen great initiatives where advocates create simple books for children, explaining what’s happening in a way they can understand.
And sometimes, advocacy isn’t just about big systemic changes - it’s about the little things that can make a child’s experience easier. I remember a time in the hospital when a young patient undergoing chemotherapy had lost her appetite. She was sick all the time, barely eating, and had lost a lot of weight. One day, she told her parents she wanted spaghetti with butter and cheese. But when they asked the hospital kitchen, they were told it wasn’t on the menu - only meals with meat were available. It seemed like such an unnecessary obstacle.
Thankfully, a kind nurse stepped in, made a call, and worked some magic. Eventually, the little girl got her spaghetti with butter and cheese. It was such a small thing, but it made all the difference. Seeing how happy her parents were when she finally ate was a reminder that sometimes, the simplest actions can change an entire experience.
For pediatric cancer research, funding is still largely dependent on donations. Since childhood cancers are rare, developing treatments for them is not always profitable. This means research often relies on the generosity of people around the world. While these donations have enabled incredible work, I believe there should be stronger international political support for pediatric oncology and rare diseases. Some countries have already introduced pediatric oncology laws to regulate and prioritize funding, but this isn’t the case everywhere. Governments should take more responsibility in funding research so that progress doesn’t depend solely on charitable contributions.
As for what the broader community can do, advocacy is key. Pushing for proper legal and financial support for pediatric oncology at an international level is one of the most impactful ways to help.
My message to the little ones: You are little superheroes! I hope you are full of life and that your treatments are as easy as possible, with the best outcome.
To parents, I want to say that your children are warriors. They are stronger than you think, and so are you. You will get through this. I hope you and your child have a beautiful life together and know that there are so many people working tirelessly so that, one day, no family will ever have to go through this.
And before we finish, I’d really like to take a moment to thank the medical community, the researchers, and, most importantly, the families who support these efforts. It’s important for them to know that there is a big community that truly cares and is working every day to make a difference.
With a background in bioengineering, a master’s in molecular oncology, and a PhD in pediatric oncology and bioinformatics (thesis pending), Daiana has trained in leading hospitals worldwide and witnessed firsthand the transformative impact of precision medicine in pediatric cancer.
