The Fight Against Childhood Cancer at UH Leaves No Stone Unturned

The terrain of childhood cancer is constantly shifting, with so many different types to consider. With all the complexity, it can be hard to know whether it’s possible to even declare partial victory.

However, there are unmistakable rays of hope. Data from around the country show that pediatric oncologists, researchers and all the caregivers who support them can be justifiably proud of the progress they’ve made for their young cancer patients in the past 50 years in this life-or-death struggle. The American Cancer Society reports that five-year survival rates for kids with cancer are now at 85% -- up from just 58% in the mid-1970s. At the same time, the number of children developing cancer in the first place has stabilized since 2015, after several decades of increases. Despite the formidable nature of childhood cancer, we do make progress every day.

‌However, one stark fact remains: Cancer continues to be the leading cause of disease death among American children, second only to accidents.

Here at UH Rainbow Babies & Children’s Hospital and our Angie Fowler Institute for Adolescent and Young Adult Cancer, we’re all-in for this fight. We’re looking in unusual places to find new tools for treating particularly deadly and virulent childhood cancers. We’re doing the basic animal research that makes later clinical trials possible – trials that can benefit both our patients and so many others. And we’re seeking ways to make the cancer treatments we give our young patients less harmful to them as they grow and develop. As the odds of surviving childhood cancer have changed, we clinicians, too, must change and develop new options for how our young patients can grow and make a successful transition to adulthood.

‌It's a packed agenda, but one we’re eagerly pursuing with energy, intellect and laser-focus.

‌For example, our pediatric oncologists Alex Huang, MD, PhD, Theresia G. and Stuart F. Kline Family Foundation Chair in Pediatric Oncology, and Kristen VanHeyst, DO, are tackling bone cancer that has spread beyond the original tumor, known as metastatic osteosarcoma. Between 10% and 20% of children, teens and young adults diagnosed with osteosarcoma have this type of disease spread when first diagnosed, most commonly in the lungs. The consequences are often grave.

Newer and more direct approaches are desperately needed, they tell me. To make that happen, they’re broadening the search to find new agents that are more effective. They’ve done the painstaking basic research, for example, to identify a pair of molecules in the lungs that appear to be important in osteosarcoma that spreads there. Experiments with mice show that when one of these two molecules is absent, osteosarcoma doesn’t spread. Taking this to the next level, the dynamic research team has found a potential solution for blocking this interaction in an unusual source – an existing FDA-approved therapy for multiple sclerosis and inflammatory bowel disease called natalizumab. The result of this brilliant work? A Phase 1 clinical trial is now open at UH, attracting patients from across the country.

Not content with just repurposed medicines as potential treatments, Dr. Huang and local colleagues have also continued to think out of the box. His collaborator has identified the lowly shell of oat bran – and an important molecule within it -- as a possible anti-cancer powerhouse. This molecule seems to rally the anti-tumor response of the immune system. Instead of an immune system that is suppressed, this molecule seems to stimulate it to fight the cancer. The team has shown that it can even stop tumor growth in mouse models of osteosarcoma, melanoma and pancreatic cancer. Research is ongoing, but promising. The end goal is to develop a new, safe treatment based on this molecule that could help enhance immunotherapy for cancers like osteosarcoma and so many others.

Pediatric oncologist John Letterio, MD, Division Chief of Pediatric Hematology & Oncology at UH Rainbow and the Jane and Lee Seidman Chair in Pediatric Cancer Innovation, is also vigorously engaged in the childhood cancer fight – but from a different, also invaluable perspective. He’s spent three decades looking for an agent that can help protect young brains and bodies from the ravages of cancer treatment. It’s well known that treatment aimed at eradicating cancer in a young patient often has harmful “late effects” that show up years later, affecting the developing brain and other organs, even with the most modern protocols. One study, for example, documented rates of hearing loss, abnormal cholesterol levels, hormonal dysfunction and abnormal lung function at more than 60% among adult survivors of childhood cancer, to say nothing of cancer-induced cognitive impairment.

“We need strategies to mitigate the late effects of our lifesaving treatments,” he tells me.

Now, one of the key projects Dr. Letterio has worked on for decades to address this critical issue has reached a crucial new stage. He and colleagues are working with funding from the National Cancer Institute to test a promising new drug candidate they’ve identified. Its target? The brain aging process thought to be caused by chemotherapy and radiation therapy, resulting in “late effects.”

According to Dr. Letterio, research shows that cancer treatment in children creates what amounts to a state of premature aging in the brain. Accumulations of certain damaging cells can cause chronic inflammation, like what you’d expect with Alzheimer’s disease or Parkinson’s. Fortunately, however, studies also show that certain agents called triterpenoids can reduce activation of these cells and improve therapy-related cognitive problems.

That’s where the research stands – with more developments sure to come.

“This will pave the way to the clinic for other drugs in this family we’re studying,” Dr. Letterio says.

Of course, this is by no means an exhaustive list of the stellar cancer research and outstanding patient care we’re known for at UH Rainbow Babies & Children’s Hospital and UH Rainbow Angie Fowler Adolescent & Young Adult Cancer Institute. But these few glimpses give you a sense of how we’re approaching this fight.

Dedication and drive are the watchwords. Solutions can take years, but we’re undaunted.

The battle against childhood cancer is one we must win. In the end, one thing is clear: Growing up is hard enough without a life-or-death fight to confront you every day.

At UH, we’re dedicated to helping create that shining day when this is no longer the case -- when kids can just be kids.