James Allison, Ph.D., the chairman of Immunology at MD Anderson Cancer Center, has won the Lasker-DeBakey Clinical Medical Research Award for 2015.
The annual Lasker awards, now in their 70th year, are known as "America's Nobels." Allison is being honored for his discovery of a drug that uses the body's immune cells — specifically the T cells — to destroy cancer.
His eureka moment came as he studied how T cells get going– they have surface proteins that act as both engines and brakes. The "engines" marshal the T cells and spur them to attack foreign substances like bacteria and abnormally-developing cells that could become cancer. The "brakes" work by reining in the powerful T cells, which otherwise would proliferate and attack the body's own tissues.
Other scientists had tried to rev the engines of the T cells to attack cancer. But Allison focused on the brakes. He realized that cancer cells were successfully tricking the brakes on T cells, and he knew he had to release the brakes to allow T cells to fight.
"So I had the idea just to eliminate the brakes temporarily," he said. "And maybe it would eliminate cancer."
Allison tested the idea in mice in the 1990s, and then pushed for years to get a drug company to take a chance on clinical trials. The new drug he developed, called Yervoy (ipilimumab), has shown astounding results in patients with advanced melanoma. Previously, most of those patients died in a matter of months.
Yervoy extended the survival of 22 percent of those patients to four or more years.
When Yervoy is combined with a second immunotherapy drug that works on another T cell "brake," almost half of the advanced melanoma patients had a significant response. (Long-term survival of that drug combination is still being studied.)
"It was a very radical approach to cancer, because basically it said ignore cancer," Allison said. "We're not treating the cancer cell, we're treating the patient's immune system."
"And secondly, we're not trying to say: ‘Oh, we know what the right thing to attack is.' We said: ‘We'll take the brakes off, and just let the immune system do what it would do, anyway.' And so for those reasons, people thought it was kind of nutty for a long time."
Immunotherapy has also shown success in lung cancer and is being tested against other types too.
Allison will accept his Lasker prize next week in New York City. Eighty-six recipients of the award have later gone on to get a Nobel Prize.