Meet the Innovators Rethinking Childhood Cancer Treatment

For many childhood cancers, modern medicine has transformed what was once a devastating diagnosis into a survivable disease. Today, it’s estimated that around 90% of children diagnosed with certain forms of leukemia will survive. But that success tells only part of the story. For children diagnosed with aggressive brain tumors, recurrent bone cancers, and other rare solid tumors, treatment options remain limited, with many patients still receiving therapies that have changed little over the past decades. Families are often faced with repeated rounds of chemotherapy, radiation, and surgery, knowing that these treatments may only buy time rather than offer a cure.

This reality has inspired a new generation of scientists to ask a different question. Rather than attacking cancer directly with increasingly aggressive treatments, what if we could teach the body's own immune system to recognize and destroy it? That idea sits at the heart of cancer immunotherapy, which is one of the fastest-growing areas of cancer research. Two biotechnology companies supported by our partner Music Beats Cancer, Merlin Biotech and OX2 Therapeutics, are exploring two different ways to make that vision a reality for children facing some of the hardest-to-treat cancers.

One reason many cancers are so difficult to treat is that they can evade the body's natural immune response. Rather than recognizing cancer cells as a threat, the immune system may fail to mount a strong enough attack, allowing the disease to grow unchecked.

Merlin Biotech is developing an experimental treatment designed to change that. Using messenger RNA (mRNA), which is the same technology that became widely known through COVID-19 vaccines, the company aims to deliver instructions directly into a tumor that encourage the immune system to notice what it previously overlooked. Their lead therapy, called Starlight, introduces a naturally occurring protein known as USP6 into tumors.

In simple terms, the treatment is designed to "switch on the lights", helping immune cells recognize that the cancer does not belong there. Instead of attacking the tumor itself, the therapy helps the immune system do what it was designed to do. For children with cancers such as recurrent Ewing sarcoma, where treatment options become extremely limited once the disease returns, this approach could eventually offer a completely new strategy.

Merlin's CEO, Dr. Randall Hyer, who previously served as Senior Vice President of Global Medical Affairs at Moderna, now leads the company to pursue the mission to develop innovative immunotherapies for childhood cancer. Merlin is advancing Starlight through preclinical development, anticipating its first patient studies within the coming year. Hyer believes that bold ideas are needed if outcomes for children with difficult cancers are ever going to improve.

While Merlin focuses on making tumours more visible, OX2 Therapeutics is taking another approach: training the immune system to actively hunt down cancer cells. OX2 Therapeutics is a biotech spinoff run by University of Minnesota researchers Michael Olin and Dr. Chris Moertel. The company is developing an experimental therapeutic vaccine for children with devastating brain cancers such as diffuse midline glioma (DMG). Unlike preventive vaccines, cancer vaccines are designed to help the immune system recognize an existing tumor and attack it. The treatment works alongside a carefully timed dose of radiation. Radiation exposes proteins on the surface of the tumor, making it easier for the immune system to identify it as abnormal. The vaccine then helps "teach" immune cells to target those cancer cells more effectively.

While Merlin focuses on making tumours more visible, OX2 Therapeutics is taking another approach: training the immune system to actively hunt down cancer cells. OX2 Therapeutics is a biotech spinoff run by University of Minnesota researchers Michael Olin and Dr. Chris Moertel. The company is developing an experimental therapeutic vaccine for children with devastating brain cancers such as diffuse midline glioma (DMG). Unlike preventive vaccines, cancer vaccines are designed to help the immune system recognize an existing tumor and attack it. The treatment works alongside a carefully timed dose of radiation. Radiation exposes proteins on the surface of the tumor, making it easier for the immune system to identify it as abnormal. The vaccine then helps "teach" immune cells to target those cancer cells more effectively.

For diseases like DMG, this represents a new direction. Current treatments remain very limited. In many cases, doctors must tell families that available therapies are unlikely to stop the cancer and are intended mainly to prolong life. As pediatric neuro-oncologist Dr. Anne Bendel explains, brain tumors present a unique challenge because the brain is protected by the blood-brain barrier, making it difficult for many chemotherapy drugs to reach the tumor in sufficient amounts. After years of disappointing results, researchers are increasingly looking to immunotherapy as a more promising path forward.

Early experiences with the therapeutic vaccine have offered encouraging signs. One child diagnosed with DMG in late 2024 was initially given only months to live. After receiving the experimental treatment, the disease had not progressed 20 months later. While individual cases cannot prove that a treatment works, they provide researchers with important reasons to continue carefully testing the therapy in clinical trials.

Stories like these naturally raise an important question: if these ideas are so promising, why aren't they already available? The answer lies in one of the biggest challenges facing childhood cancer research. Developing a new medicine is a long, expensive process. Before a treatment can become widely available, it must pass through multiple phases of clinical trials to demonstrate both safety and effectiveness. For childhood cancers, that process is especially difficult.

Because these cancers are relatively rare, finding enough patients for clinical trials can take years. Companies must also raise significant funding to manufacture treatments, monitor patients, collect safety data, and satisfy strict regulatory requirements. For OX2 Therapeutics alone, providing one year of treatment for a child in its current clinical programme costs around US$40,000, with larger trials requiring many more participants before researchers can determine whether the therapy truly works. The science may be promising, but without sustained support, many innovative treatments struggle to reach the children who need them most.

The future of childhood cancer treatment is unlikely to depend on a single breakthrough. Instead, progress will come from many different ideas being explored simultaneously. Merlin Biotech and OX2 Therapeutics represent two of those ideas and they both share the same ambition: helping the immune system fight cancers that have resisted conventional treatments for decades.

Their work also highlights that behind every promising discovery are years of research, countless clinical trials, and communities willing to invest in hope long before success is guaranteed. That is why initiatives like Music Beats Cancer matter. By supporting pioneering childhood cancer research and helping early-stage innovations move closer to the clinic, they play a vital role in giving families genuine reasons to believe that better treatments may be on the horizon.

This mission comes to life through Music Beats Cancer's upcoming Thumbs Up for A Cure Hamptons campaign, which brings together families affected by childhood cancer, biotech entrepreneurs, biopharma leaders, clinicians, artists, philanthropists, and local community supporters who share a common goal: improving outcomes for children with cancer. By connecting the people driving scientific innovation with those committed to supporting it, the campaign helps build the momentum needed to turn promising research into tomorrow's treatments. The event is happening on September 5th in East Hampton, with the support of partners like Tough2Gether, giving young voices a platform to ensure that patients and families affected most by these diseases remain at the heart of innovation.

Every breakthrough begins with bold ideas, but it also depends on the communities willing to champion them. Together, they offer hope that the next generation of children facing cancer will have access to safer, more effective treatments than ever before.

This article was written in collaboration with Music Beats Cancer, a non-profit organization focused on fundraising for cancer research.