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Emory University is hopeful that a new study will lead to a breakthrough in childhood cancer treatment.

The university says new research from a partnership with Australia's QIMR Berghofer Medical Research Institute shows a potential new targeted treatment for brain cancer in children.

What we know:

In a recent paper published in the journal Nature Communications, the researchers say the novel drug CT-179 was shown to be effective in killing tumor cells in preclinical models using mice.

The drug specifically targeted tumor cells that are resistant to standard treatments, which could lead to more effective and less toxic treatments.

The researchers say the drug could possibly apply to other brain cancers as well.

What they're saying:

Emory University Professor Timothy Gershon was one of the study's lead researchers. He is also a pediatric neurologist at Children’s Healthcare of Atlanta and director of the Children’s Center for Neurosciences Research.

"Current treatments, including radiation and chemotherapy, often eliminate most of the tumor, but sometimes fail to eliminate cancer stem cells," Gershon said. "These cancer stem cells can regrow the tumor after treatment, causing fatal recurrence. We show that CT-179 treatment specifically disrupts cancer stem cells. Combining CT-179 with treatments such as radiation therapy treats the whole tumor more effectively, including both stem cells and tumor cells that are not stem cells. Adding CT-179 to combinations of treatments may bring new efficacy to brain tumor therapy." 

Professor Bryan Day, the leader of QIMR Berghofer Medical Research Institute's Sid Faithful Brain Cancer Laboratory, described the findings as a "breakthrough."

"Children with brain cancer urgently need more effective and less toxic treatments," Day said. "Our study demonstrated that the drug CT-179, used in combination with standard radiation therapy can cross the blood-brain barrier and penetrate the tumor. It prolonged survival in a range of preclinical medulloblastoma models, delayed recurrence of the disease, and increased the effectiveness of radiotherapy. Brain cancer is an incredibly tough puzzle to solve. As researchers, what gets us out of bed every day is trying to solve that puzzle. This global research could potentially lead to new combination therapies that improve outcomes for these young patients."

By the numbers:

According to the American Cancer Society, about 9,550 children in the United States under the age of 15 will be diagnosed with cancer this year. Cancer is the second leading cause of death in children between the ages of 1 and 14.

While childhood cancer rates have been declining in recent years, the number of deaths is still estimated to be over 1,000 annually.

What's next:

The next step in the research will be clinical trials to test the drug's effectiveness.

"We've been working with our collaborators, particularly in the U.S. and Australia, and we're very hopeful that the culmination of all this work has paved the way for the first in-human clinical testing of CT-179 in patients with brain cancer," Day said.