CIRM-funded research at Sanford Burnham Prebys Medical Discovery Institute in San Diego is identifying compounds that could be clues to treat a deadly childhood cancer.

A rare brain cancer

The cancer is choroid plexus carcinoma (CPC), a rare brain tumor that occurs mainly in children. As it grows the tumor can affect nearby parts of the brain resulting in nausea, vomiting and headaches.

Treatment involves surgery to remove the tumor followed by chemotherapy and radiation. However, many of the children are too young to undergo radiation and only around 40 percent are still alive five years after being diagnosed. Even those who do survive often experience life-long consequences such as developmental disabilities.

A new mouse model

One obstacle to developing better therapies has been the lack of a good animal model to understanding of the disease. That’s where this later research, published in the journal Cancer Research, comes in.

The team at Sanford Burnham developed a new mouse model, by knocking out p53, a gene known to suppress tumor formation, and activating a gene called Myc, which is known to cause cancer.  

In a news release, Robert Wechsler‑Reya, the senior author, says this new model closely mirrors how CPC grows and develops in humans.

“This model is a valuable tool that will increase our understanding of the biology of the cancer,” Wechsler-Reya said. “This advance brings us one step closer to a future where every child survives—and thrives.”

The team tested nearly 8,000 compounds on the mouse tumor cells to see whether any could slow or stop the disease. They identified three that inhibited the cancer and spared healthy surrounding cells.

“These compounds are promising, much‑needed leads in the quest for an effective CPC treatment,” says Wechsler‑Reya. “Our laboratory plans to evaluate these and additional compounds that can effectively treat this cancer.”

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