Early low-dose chemotherapy appears to substantially improve short-term survival in patients with the most aggressive and common form of brain cancer, offering the first significant advance against the disease in decades.
Whether the treatment can help cure brain cancer remains to be seen, but the approach at least seems to slow the often rapid progression of the disease for some.
The treatment, tested in a form of brain cancer called glioblastoma multiforme, involves the drug Temodar. Until now, the medicine has typically been used only after radiation to shrink the tumor.
A major international study released Monday shows that giving low doses of the capsule at the very start - for six or seven weeks during and after radiation - doubles the chance of being alive two years later.
"This is the first trial that has been clearly positive in brain cancer in 30 years,'' said Dr. M.J. van den Bent of the Daniel den Hoed Oncology Center in Rotterdam, the Netherlands. "This is a great day.''
Radiation and surgery are the first-line treatments for glioblastomas, but even with them the disease usually kills within a year or less.
Intravenous chemotherapy available since the 1970s improves these odds only marginally and can have serious side effects.
Several doctors predicted that upfront Temodar will quickly become the new standard of care, routinely offered to all victims of this disease.
"To be able to tell people they may have two or three years of survival rather than nine months is pretty major,'' said Dr. Adam Mamelak of City of Hope National Medical Center in Duarte, Calif., who was not involved in the study.
The study was conducted and financed by the European Organization for Research and Treatment of Cancer and released in New Orleans at a meeting of the American Society of Clinical Oncology. It was done at more than 80 hospitals in Europe, Canada and Australia.
The study's director, Dr. Roger Stupp of University Hospital in Lausanne, Switzerland, said patients found the drug easy to take, and fatigue was the most common ill effect.
"We have started with the most malignant and devastating form'' of brain cancer, he said.
The next step will be to try the drug against less aggressive tumors and combine it with the newer, so-called targeted drugs designed to block cancer's internal growth signals.
Unlike most cancer, which kills by spreading through the body, glioblastomas grow quickly inside the head, destroying everything in their path.
They are the most aggressive of the 100 or so forms of cancer that originate in the brain, and they account for half or more of all cases. Around the world, 175,000 cases are diagnosed annually, killing 125,000.
In the new study, 573 patients were randomly given standard treatment with or without early Temodar. After two years, 26 percent receiving Temodar were still alive, compared with just 10 percent getting the usual care.
Even with the treatment, most patients died quickly. Nevertheless, doctors said doubling short-term survival is an important milestone in such a grim disease.
"Twenty-six percent survival is not that great in the large scheme of things. But it is still progress,'' said Dr. Frank Haluska of Massachusetts General Hospital.
Until now, chemotherapy has also not had an important role in treating prostate cancer, which is much more common.
At the meeting Monday, other researchers released details of studies that led the Food and Drug Administration last month to approve use of Taxotere, a standard chemotherapy drug, for men with advanced cases of this malignancy. Before these studies, no treatment had been found to improve survival in prostate cancer.
Spreading cancer can be suppressed with hormone treatment, but eventually this approach fails, and patients typically die within a year.
Two large studies released at the meeting show that Taxotere can improve survival in these men by a median of about two months, although some lived several years while on the drug.
"This is reason for celebration, because there is a survival advantage, and there is also reason for optimism, but we have a long way to go in these patients,'' said Dr. Mario Eisenberger of Johns Hopkins University, who headed one of the studies.