Genetically engineered immune cells are saving the lives of cancer patients. That may be just the start.
Cellectis began developing the treatment in 2011 after doctors in New York and Philadelphia reported that they’d found a way to gain control over T cells, the so-called killer cells of the immune system. They had shown that they could take T cells from a person’s bloodstream and, using a virus, add new DNA instructions to aim them at the type of blood cell that goes awry in leukemia. The technique has now been tested in more than 300 patients, with spectacular results, often resulting in complete remission. A dozen drug firms and biotechnology companies are now working to bring such a treatment to market.
The search to expand immune therapy now involves not only the world’s largest drug companies but also tech firms. Sharp says that last year Google held two summits at MIT of top immune oncologists and bioengineers to determine what parts of the problem could be “Googlified.” Attendees say the search giant paid special attention to new research techniques that fingerprint cells from a tumor biopsy in rapid-fire fashion. These methods might generate big data about what immune system cells are actually doing inside a tumor, and new clues about how to influence them. So far, Google’s life science unit, named Verily, hasn’t revealed its plans in cancer immunotherapy. But in New York’s Union Square, I met Jeffrey Hammerbacher, a former Facebook employee who now runs a lab that is part of Mount Sinai, the hospital and medical school. With 12 programmers in a light-soaked loft—the nearest thing to blood and guts is a photo of an exhausted surgeon on the wall—he’s also spending time on T cells. He’s developing software to interpret the DNA sequence in a patient’s cancer and predict from it how to goose the response of killer T cells.
A clinical trial by Mount Sinai should start this year. The patients receive a dose of abnormal protein fragments that Hammerbacher’s software predicts will train T cells to attack the cancer. “What was fun was that what we submitted to the [U.S. Food and Drug Administration] was not a molecule but an algorithm,” he says. “It might be one of the first times the output of a program is the therapy.”
In January, Juno Therapeutics (see “Biotech’s Coming Cancer Cure”) paid $125 million to acquire AbVitro, a Boston-area company that specializes in sequencing the DNA inside single T cells. Now Juno is trying to locate T cells that are active inside cancers and study their receptors. Juno’s chief scientist, Hyam Levitsky, says an experiment that used to take seven months now takes seven days. And data is piling up: an average experiment generates 100 gigabytes of information. “A lot of what is happening is technology-driven,” he says. “The questions have been there for a while, but there was no way to get at the answers. Now we’re visualizing them with new technology in ways we never could before.”
In March Pfizer appointed John Lin to head its San Francisco biotech unit, which develops cancer drugs and recently started making engineered T cells. He says the company had been negotiating with Cellectis well before the news of Layla’s treatment and that no one there was even aware the girl had been treated before it hit the news. “The publicity was a big surprise,” he says.
Lin says years of scientific work have finally resulted in a level of mastery that makes therapeutic products seem practical. He thinks the treatments will go beyond leukemia, and beyond cancer. “We think that this fundamental principle, engineering human cells, could have broad implications,” he says, “and the immune system will be the most convenient vehicle for it, because they can move and migrate and play such important roles.”
Researchers are already working on autoimmune disorders, like diabetes, multiple sclerosis, and lupus. Infectious disease is also in the sights of T-cell engineers. Edward Berger, a virologist at the National Institutes of Health who helped discover how HIV enters human cells, thinks it may be possible to permanently keep the virus in check, a so-called “functional cure.” In February, he says, he will start giving monkeys T cells genetically programmed to find and destroy any cell in which the simian version of HIV is replicating.