New Treatment Brings Hope to Children with Cancer

Researchers at the University of British Columbia (UBC) Faculty of Medicine in Canada have developed a new targeted cancer therapy that can precisely seek out and destroy tumor cells, showing strong results in preclinical studies that bring the breakthrough closer to human clinical trials. The findings were published last Monday in the Cancer Discovery Journal. The therapy targets a protein called IL1RAP, which is found on the surface of certain cancer cells, but is largely absent from normal tissues. By linking a cancer-killing drug to an antibody that recognizes this protein, the team created an antibody-drug conjugate that delivers treatment directly to cancer cells while sparing healthy tissues. In multiple models of Ewing sarcoma — a rare and aggressive cancer affecting children and young adults — the treatment eliminated established tumors and dramatically reduced the spread of cancer. Similar effects were seen in other cancers, including lymphoma and tumors driven by specific genetic alterations (NTRK fusions). “This is exactly the kind of target we look for, something that’s present on cancer cells but largely absent from normal tissues,” said senior author Dr Poul Sorensen, professor of pathology and laboratory medicine at UBC’s Faculty of Medicine and a distinguished scientist at BC Cancer. “It allows us to deliver treatment with a high degree of precision.” “This isn’t a distant possibility,” said Sorensen, who also holds the Johal Endowed Chair in Childhood Cancer Research. “The data position this for clinical trials in the very near future, pending the next steps in development and regulatory approval.” The approach builds on earlier work by Sorensen and his team, including first author Dr Haifeng Zhang from the Sorensen laboratory, which identified IL1RAP as a key protein that helps cancer cells survive in the bloodstream and spread to other parts of the body. When cancer cells break away from a primary tumor, they must endure a hostile environment in the circulatory system before establishing new tumors elsewhere. IL1RAP appears to help them adapt and survive during this process. Metastatic disease, which occurs when cancer spreads throughout the body, is the single most powerful predictor of poor outcome for cancer patients of all ages and has been a difficult process for researchers to study or for clinicians to target. “We think of this protein almost like a protective shield,” said Zhang, a scientist in the Sorensen Lab. “It helps cancer cells withstand the stresses of travelling through the body and forming metastases. What we’ve done here is turn that shield into a target, using it to deliver a drug directly into those cells.” Crucially, the therapy demonstrated a strong safety profile in preclinical testing, supporting its readiness for clinical development — an important step toward human trials. The findings highlight a promising new strategy for targeting cancers driven by specific genetic alterations that expresses the IL1RAP protein, potentially opening the door to more precise, effective treatments for both pediatric and adult patients.