The government approved a new treatment for the nation's second-leading cancer killer Monday — after a record-setting seven-week review — a last-ditch chemotherapy for advanced-stage patients who have exhausted today's standard drugs.
Eloxatin's effects at first glance seem small: When taking it together with older drugs, about 9 percent of treated colon cancer patients saw their tumors shrink significantly and they gained about two extra months before the cancer started growing again.
But until now these incredibly hard-to-treat patients have had no other alternative once standard treatment failed — and the FDA is anticipating soon data that could prove whether Eloxatin works better when given in earlier stages of the disease.
"We felt it was important for the American public to have access to this drug," said FDA cancer drugs chief, Dr. Richard Pazdur. "These patients don't have any treatment options available to them."
The drug, made by the French pharmaceutical company Sanofi-Synthelabo, was approved faster than any other cancer treatment; only AIDS drugs have passed through the FDA faster.
It marks the first good news for patients with advanced colorectal cancer since the implosion of the biotechnology company ImClone, which had touted its own colon cancer drug Erbitux as a miraculous treatment right up until the FDA rejected it last December.
The FDA has told Congress that ImClone performed shoddy science that failed to tell if Erbitux has any effect, leaving patients in limbo. ImClone's former chief executive pleaded innocent Monday to charges of insider stock trading.
FDA's Pazdur pointedly told the pharmaceutical industry to follow Sanofi's example — not ImClone's — in testing cancer drugs.
"We want to send a message," he said, saying FDA employees worked overtime and canceled vacations to speedily review Eloxatin because the science behind the drug was so strong. "We're willing to do that if we think the drug is worth that."
Sanofi followed FDA's advice in designing a 463-patient study that promised, and delivered, some of the cancer industry's most statistically solid results. Participants were divided into thirds, getting either standard therapy, Eloxatin alone or a combination.
Combining Eloxatin with the standard drugs 5FU and leucovorin made it work much better, something scientists may never have known had Sanofi performed a cheaper but less rigorous study. The latter is the kind of research that frustrated FDA officials have recently seen "a rash of" as companies have tried to rush medications to the market, Pazdur said.
Eloxatin was approved under a special FDA program that allows promising cancer drugs to be sold before there's proof they actually prevent or delay death. But Pazdur said FDA should soon evaluate a study presented at a recent cancer meeting suggesting Eloxatin can help survival when given earlier in the disease.
Colorectal cancer strikes about 150,000 Americans a year, and kills about 56,000 of them.
Eloxatin, known chemically as oxaliplatin, will be available by the end of the month. Sanofi refused to reveal a price, but said it will be comparable to other chemotherapies.
The injected therapy, given once every two weeks, does cause side effects. About 75 percent of patients suffer some degree of peripheral neuropathy, a numbness and tingling of the hands and feet as nerve endings there are damaged, said FDA medical reviewer Amna Ibrahim. The condition, worsened by cold temperatures, typically improves once treatment is ended.
Very rarely, patients suffer a life-threatening allergic reaction. Like other chemotherapies, it also can cause vomiting, diarrhea and anemia.