Clinical trials begin this week at Wake Forest University on a cancer therapy that has completely cured the disease in every mouse tested over the past few years.

The therapy involves the transfusion of white blood cells from cancer-resistant donors into cancer patients, letting loose a uniquely qualified army of disease fighters to attack the invading tumor.

Some scientists are skeptical about the move from mice to humans, but others are excited about the possibility of success.

Dr. Zheng Cui, the lead investigator, and his team at the Wake Forest University School of Medicine announced the move to human clinical trials Saturday at the Understanding Aging Conference in Los Angeles. The team recently won approval for human trials from the Food and Drug Administration.

"This is the first time that such aggressive cancer in mice has been eradicated like this," Cui said. "This is a very dramatic result."

The result is especially dramatic considering its discovery stemmed from a series of accidents, starting with one extraordinary mouse.

In the late 1990s, Cui and his team were using mice as experimental cancer patients for their research, injecting them with malignant cells. Within three to four weeks, as expected, all the injected mice developed tumors and died.

But in 1999, for some reason, one mouse didn't develop tumors and didn't die.

Dr. Lloyd Old of the Ludwig Institute for Cancer Research, who was collaborating in the research, later said that if Cui had been trained as an immunologist, he would have thrown out the mouse right then. But Cui was trained as a medical doctor, and his curiosity led him to continue testing the oddball mouse, injecting it with higher and higher lethal doses of carcinogens.

No matter how many times the researchers tried to give the mouse cancer, it didn't develop a tumor, and it didn't die.

The mouse was immune to cancer.

Making sure

As cautious scientists, Cui and his team decided to breed the mouse and test its offspring for cancer immunity.

"We knew that if we hadn't made a mistake, something very dramatic was happening, but we had to know we weren't making a mistake," he said.

It wasn't a mistake. Three of the mouse's seven grandchildren didn't get cancer, either. Whatever was causing the cancer resistance was built into the mouse's family genes. News of the finding created a stir.

"Our lives were suddenly overtaken by an unexpected media frenzy," Cui wrote in 2003. Headlines proclaimed a cure for cancer -- albeit in mice.

"People got very excited for a reason," he said. "It was exciting. We had direct evidence for cancer immunity that we could reproduce at will. It was a very profound result, and it was not subtle. I don't think people could have overreacted."

The next step was to figure out how to transfer that cancer immunity from the special mice to mice that were dying of cancer. The solution is apparently hidden in the mice's white blood cells, which are like a tiny biological army. They are carried in the bloodstream to fight infection and disease throughout the body.

For some reason -- Cui and his team don't know why -- the white blood cells from the immune mice could defeat the cancer every time, while the other mice's white blood cells were unable to stave off the infection.

The majority of contemporary cancer research focuses on these cellular soldiers. But most research seeks to isolate certain parts of the cells and stimulate them in test tubes, a complex process.

Cui's procedure is simple.

"We don't have to do anything to manipulate the white blood cells," Cui said. All he and his team did was transfuse the immune mouse cells into the sick mice, and the tumors melted away.

Next page >