Neuroblastoma is the the most common solid tumor of early childhood. It is generally diagnosed only in advanced stages and even after intensive treatment only few children survive.
Researchers at the Children's Cancer Institute (CCIA) have developed a drug called CBL0137, which holds the key to fight neuroblastoma and pave the way for possible prevention strategies.
‘Neuroblastoma is regarded as the worst childhood tumors as it is mostly diagnosed only during advanced stages and mortality rate is almost 50%.’
The new drug prevents cancer cells from repairing themselves, ensuring they are killed off after chemotherapy. The drug is to be taken for clinical trial in children by the Children's Oncology Group of the United States. The findings were published in the international journal Science Translational Medicine
Professor Glenn Marshall, director of the Kids Cancer Centre at Sydney Children's Hospital and an author of the study, said the drug was used in conjunction with other treatments.
"The drug works in concert with the existing chemotherapy in synergy, so the two drugs together are very potent in killing the neuroblastoma cells," he said.
"What highlights how exciting this is that I first presented our research findings in 2013 when we had the very preliminary data showing really that in our laboratory models of neuroblastoma that the tumors were virtually melting away — results that we'd never seen before," said Dr.Michelle Haber, CCIA executive director.
"It's the first type of experiment of its kind and hopefully will lead to more experiments and discoveries that could eventually lead to prevention, because in cancer prevention is much better than cure," said Dr.Marshall.
"Our hope is we can have greater and more rapid shrinkage of the tumor with the new drug and combination, and that we have more durable remissions and hopefully fewer patients relapsing," he added.