By Luke Timmerman
April 13 (Bloomberg) -- Children have a higher risk of dying from brain cancer if they have excess amounts of a protein driven by a tumor-growth gene, researchers said.
The protein, ODC1, was linked to shorter remissions and survival times in a study of 209 children with neuroblastoma, researchers said today at a medical meeting in San Diego. A separate study of mice with amplified versions of ODC1 found a drug that blocks the protein was able to help extend remission times, researchers said.
Neuroblastoma is the most common form of cancer in infants and the fourth-most prevalent in children, according to the Atlanta-based American Cancer Society. About 650 new cases are diagnosed each year in the U.S., according to the cancer group. The standard treatments are surgery, radiation and chemotherapy, according to the National Cancer Institute.
``These kids with a high-risk disease, they don't respond to our best available treatments, so there's an urgent need,'' said Michelle Haber, executive director of Children's Cancer Institute Australia in Sydney, in an interview. Haber is the study's lead author. ``ODC should be a good target for new drugs.''
The high amounts of the ODC1 protein were driven by a tumor growth gene called MYCN, researchers said. Data on the neuroblastoma study was presented today at the American Association for Cancer Research meeting in San Diego.
To test the role of ODC1 in neuroblastoma, researchers gave mice with the disease a dose of Sanofi-Aventis SA's Eflornithine, a drug found to be effective for a parasitic infection called African Sleeping Sickness. The drug, known chemically as difluoromethylornithine, or DFMO, blocks the protein responsible for poor prognosis in neuroblastoma, researchers said.
It failed in various cancer studies as an independent therapy in the 1980s, and many researchers gave up on it, Haber said.
Reviving a Treatment
This time, researchers tried the drug in combination with cisplatin, a chemotherapy agent. Mice in a study had their tumors disappear for a longer time than those on cisplatin alone, researchers said.
The next steps would be to test the drug in combination with other chemotherapies that might be more effective, and in different variations of neuroblastoma, Haber said.
``Because DFMO has been shown to be quite safe for use in humans, we would hope that we can proceed rapidly to clinical trials,'' Haber said in a statement.
The drug can be taken as a pill, or in a powder form in drinking water, making it a good candidate for small children, Haber said.
To contact the reporter on this story: Luke Timmerman in San Francisco at ltimmerman@bloomberg.net
Last Updated: April 13, 2008 15:00 EDT
