For 85 percent of kids with acute lymphoblastic leukemia (ALL), chemotherapy is a cure—but not for Emily Whitehead, 6. Diagnosed at 5, she got an infection from her first round of chemo and nearly lost her legs. Then the cancer came back; she was put into remission and scheduled for a bone marrow transplant. As she waited, the cancer returned yet again. There was nothing else to try.
Nothing except a crazy experimental treatment never before given to a child: Blood was taken out of Emily’s body, passed through a machine to remove white cells and put back in. Scientists at the University of Pennsylvania used a modified HIV virus to genetically reprogram the white cells so that they would attack her cancer, and reinjected them.
But the cells attacked her body, too. Within days Emily was so feverish she had to be hospitalised. She was sent to the intensive care unit and put on a ventilator. A doctor told her family that there was only a one-in-1,000 chance she would survive the night. Then the miracle breakthrough: Doctors gave Emily a rheumatoid arthritis drug that stopped the immune system storm—without protecting the cancer. Emily awoke on her 7th birthday and slowly recovered. A week later her bone marrow was checked.
Emily’s father remembers getting the call from her doctor, Stephan Grupp: “It worked. She’s cancer free.”
She still is, two years later—taking piano lessons, wrestling with her dog and loving school, which she couldn’t attend while sick. “I’ve been an oncologist for 20 years,” says Grupp, “and I have never, ever seen anything like this.” Emily has become the poster child for a radical new treatment that Novartis, the third-biggest drug company on the Forbes Global 2000, is making one of the top priorities in its $9.9 billion R&D budget. “I’ve told the team that resources are not an issue. Speed is the issue,” says Novartis Chief Executive Joseph Jimenez, 54. “I want to hear what it takes to run this phase III trial and to get this to market. You’re talking about patients who are about to die. The pain of having to turn patients away is such that we are going as fast as we can and not letting resources get in the way.”
A successful trial would prove a milestone in the fight against the cancer. Coupled with the exploding capabilities of DNA-sequencing machines that can unlock genetic code, recent drugs have delivered stunning results in lung cancer, melanoma and other deadly tumours, sometimes making them disappear entirely—albeit temporarily. Just last year the Food & Drug Administration approved nine targeted cancer drugs. It’s big business, too. According to data provider IMS Health, spending on oncology drugs was $91 billion last year, triple what it was in 2003.
But the developments at Penn point, tantalisingly, to something more, something that would rank among the great milestones in the history of mankind: A true cure. Of 25 children and five adults with ALL, 27 had a complete remission, in which cancer becomes undetectable. “It’s a stunning breakthrough,” says Sally Church, of drug development advisor Icarus Consultants. Says Crystal Mackall, who is developing similar treatments at the National Cancer Institute: “It really is a revolution. This is going to open the door for all sorts of cell-based and gene therapy for all kinds of disease because it’s going to demonstrate that it’s economically viable.”
There are still huge hurdles ahead: Novartis has to run clinical trials in both kids and adults around the world, ready a plant to create individualised treatments and figure out how to limit side effects. But Novartis forecasts all that work will be done by 2016, when it files with the FDA.
Progress like this explains why Jimenez is focusing his pharmaceutical giant on a simple mission: Cure cancer. Cancer drugs represent $11.2 billion of Novartis’ $58 billion in annual sales, but he says he’s “doubling down” on the cancer business. In April he did a deal that essentially traded Novartis’ unprofitable vaccine and consumer businesses and up to $9 billion in cash to GlaxoSmithKline in return for Glaxo’s cancer drugs, which generate $1.6 billion in sales but which Jimenez says include three pills he can turn into $1 billion sellers. The same day, he sold his veterinary business to Eli Lilly. He calls it “precision M&A”—bartering for the divisions you want, instead of bidding $100 billion for an other rival, as Pfizer is doing with AstraZeneca. Jimenez’s move, which he terms “the antithesis of megamergers”, will drop Novartis’ 2016 sales 5 percent but boost earnings per share (before extraordinary items) by 10 percent, according to investment bank Jefferies.
Jimenez has competition: Seattle-based Juno Therapeutics, which counts Amazon CEO Jeff Bezos among its backers. But that’s to be expected when the potential is so staggering—and tangible. “Anybody that gets associated with this technology and sees what this technology has been able to do really believes they’re participating in something that’s historic,” says Jimenez. “I look at it and think about the potential breakthrough that it could be. You could be looking at a transformation of the treatment of cancer over the next 20 to 30 years.”
Olson’s result was published, without his name, in the New England Journal of Medicine in August 2011. Data from two other patients were published at the same time in Science Translational Medicine. “The phone started ringing off the hook with people wanting to start companies and all sorts of venture capitalists and all that,” says Carl June, the Penn researcher whose team developed the therapy and who three years before couldn’t raise money around it. “Then we had three Big Pharmas coming. It was incredible how it happened.”
(This story appears in the 13 June, 2014 issue of Forbes India. To visit our Archives, click here.)