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New Quebec research offers hope for rare therapy for childhood cancer

New Quebec research offers hope for rare therapy for childhood cancer
New Quebec research offers hope for rare therapy for childhood cancer

A joint university study has found that a pathway to a therapy program against a rare and deadly brain tumor in children is now possible.

The study was led by scientists from the McGill University Health Centre (MUHC) Research Institute and Baylor College of Medicine in Texas.

Due to a lack of effective treatment, the tumors, called posterior fossa group A (PFA) ependymomas, have the highest recurrence rate and the worst prognosis of all childhood cancers.

Dr. Nada Jabado, senior scientist at MUHC, explains that available treatments so far consist of surgery and radiotherapy, but the cancer often comes back.

“This is a cancer that affects very young children, infants, and children up to six and seven years old. Sometimes a little later, but mostly infants,” Jabado said. “Almost 70 percent die within the first few years, and up to 90 percent later. Some survive, but it’s rare.”

The study examined interactions between regions of the genome that are unique to PFAs. They named these predictable sections TULIPs, or Type B Ultra-Long Interactions in PFAs.

“(TULIPs) are three-dimensional structures in the nucleus that are very specific to this tumor, only present in this tumor, which allows the cell to maintain a very undifferentiated and aggressive phenotype,” Jabado said. “By understanding what holds those structures together, we can provide a treatment, because if we can dissolve them, (TULIPs) are removed and the cells will mature better and we can give those kids a break.”

Lead author of the study, Marco Gallo, said in a statement that TULIPs interact with each other surprisingly robustly over long distances, for example from one end of a chromosome to the other.

“TULIPs on different chromosomes can also converge and interact strongly with each other,” Gallo said. “We also found that regions outside of TULIPs generally appear more relaxed. This is important because TULIPs are linked to cell function.”

By inhibiting specific proteins in TULIPs, the team observed “weaker interactions between TULIPs and reduced survival of PFA cells,” the statement said.

Their observations revealed that interactions between TULIPs “are important for PFA cell viability, providing new potential treatment targets.”

The research began about five years ago, and it could be another year or two before the treatment reaches clinical trials, “and I’m being very generous,” Jabado said. “The next step is to validate that if we use this inhibitor, the one we use in the paper, on a larger set of samples and in multiple labs and models, that we see similar results.”