April 2014 – Will This Man Cure Cancer ?
For 85% of kids with a terrible cancer called acute lymphoblastic leukemia, chemotherapy is a cure–but not for Emily Whitehead. Diagnosed at 5, she suffered an infection from her first round of chemo and nearly lost her legs. Then the cancer came back; she was put into remission once more 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 6-year-old Emily’s body, passed through a machine to remove her white cells and put back in. Then scientists at the University of Pennsylvania used a modified HIV virus to genetically reprogram those white cells so that they would attack her cancer, and re-injected them.
But the cells attacked her body, too. Within days Emily was so feverish she had to be hospitalized. Hallucinating, she asked her father, “Why is there a pond in my room?” 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, an electrical lineman named Tom Whitehead, 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 research and development 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 demon that has plagued living things since dinosaurs roamed the Earth. Coupled with the exploding capabilities of DNA-sequencing machines that can unlock the genetic code, recent drugs have delivered stunning results in lung cancer, melanoma and other deadly tumors, 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, tantalizingly, to something more, something that would rank among the great milestones in the history of mankind: a true cure. Of 25 children and 5 adults with Emily’s disease, 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 at hospitals around the world, ready a manufacturing plant to create individualized treatments for patients and figure out how to limit the side effects that nearly killed Emily. 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. Already 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 currently 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 another rival, as Pfizer is doing with AstraZeneca. Jimenez’s move, which he terms “the antithesis of mega-mergers,” will drop Novartis’ 2016 sales 5% but boost earnings per share (before extraordinary items) by 10%, according to investment bank Jefferies.