Buying a Cure.(Kathy Giusti's Multiple Myeloma Research Foundation)

Last May, Kathy Giusti was in midtown Manhattan pitching her current venture to an audience of potential investors. Giusti, a forty-eight-year-old Harvard Business School graduate and former pharmaceutical executive, believes that medical breakthroughs shouldn't be left to chance. In 1998, she created the Multiple Myeloma Research Foundation, a charitable organization dedicated to the lethal blood cancer, which afflicts more than fifty thousand Americans and has no known cure. Most medical charities focus on increasing public awareness and on raising money to distribute to researchers, in the hope that some of the work will lead to a new drug or a cure; Giusti runs hers as if it were a for-profit business, expecting high returns on the money she raises from "investors"--her term for philanthropists. Her staff includes four scientists, who track myeloma research at academic institutions around the world, advising her on which projects are most likely to lead to new therapies for patients, and are thus the best candidates for funding. (Drug companies have typically not been interested in developing drugs for myeloma, preferring to focus on diseases that affect large numbers of people, for which treatments are most profitable.) Researchers who receive money from Giusti's foundation are required to meet strict deadlines for demonstrating progress. "We try to get academics to work like businesspeople," she told her audience in Manhattan. "Money gives you power to drive people's behavior."

Giusti's organization maintains a tissue bank of more than fourteen hundred bone-marrow samples and nearly twelve hundred blood samples from patients with myeloma, which researchers use to test novel compounds, and it helps finance clinical trials of experimental myeloma drugs. Since 2001, twenty-one drugs derived in part from research funded by the foundation have entered clinical trials, and eight are currently in Phase II--a track record that pharmaceutical companies would envy.

Since Giusti established her foundation, medical philanthropies that apply business principles to their work have become increasingly common. In 2002, Scott Johnson, a former C.E.O. of several Silicon Valley startups who suffers from multiple sclerosis, created the Myelin Repair Foundation, with the goal of facilitating academic research into treatments for the damaged nerves characteristic of patients with the disease. Johnson identified five leading M.S. researchers and persuaded them to collaborate on a research program funded by his foundation. "The academic system is broken," Johnson told me. "Researchers focus on publishing to get tenure. It's all about credit for discoveries." (Johnson hopes to have a drug ready for lab testing in 2009.) Two years ago, Susan G. Komen for the Cure, the oldest and largest philanthropic organization dedicated to breast cancer, hired a new C.E.O., Hala Moddelmog, the former president of Church's Chicken, the fast-food franchise. (The organization's departing C.E.O. was a former health-care executive.) "We have followed a traditional paradigm," Moddelmog told me. "But we believe there is a smarter way to do things." This year, Komen is considering allocating thirty-five million dollars of its hundred-million-dollar research budget to support a new initiative called Promise Grants, which would require recipients to share data with one another and to observe strict guidelines. "We need flexibility and speed to discover new treatments and deliver them to patients," Moddelmog said.

Giusti told me that she has been contacted by several other new medical charities, including the Lance Armstrong Foundation, which is devoted to cancer; the Kirsch Foundation, which supports research into Waldenstrom's macroglobulinemia, a rare blood cancer; and ABC2, which was created in 2001 by Steve Case, the co-founder of AOL, and his brother Dan Case to combat brain cancer. (Dan Case died of the disease in 2002.) But Giusti's aggressive approach to myeloma research has been controversial among some academic scientists, who argue that it's unrealistic--even counterproductive--for patients and their advocates to think that with enough money and the right business model they can buy a cure.

Giusti's move from the pharmaceutical industry to the nonprofit world began in December, 1995, when she was thirty-seven years old and living in Lake Forest, Illinois. "I was on the fast track at Searle"--the pharmaceutical company--"for a leadership position," Giusti, who has short blond hair, intense blue eyes, and an energetic voice, told me. For four years, she had overseen the marketing of Searle's popular arthritis drugs, including Daypro, and she was in charge of developing the strategy for selling a new one, Celebrex. She travelled frequently, and as Christmas approached she was exhausted and losing weight. She had an eighteen-month-old daughter, and she and her husband, a real-estate developer, had been trying to conceive again. In late December, Giusti visited her doctor, who ordered some blood tests and referred her to a fertility specialist. "Two days after Christmas, the doctor called and said he wanted me to come back and get the tests done again," she recalled. She repeated the blood tests, and in early January she received a phone message from the physician, asking to meet with her and her husband.

"I was driving back to Lake Forest from a regional sales meeting and finally reached him," Giusti said. "I was on my car phone, and I was driving, and I said, 'Look, I know something is horribly wrong. My father was a doctor, and you wouldn't ask me to come in with my husband unless something was really bad.' And he said, 'Well, just come in and I will talk you then.' And I said, 'No. You need to tell me now.' I'm driving down the Kennedy Expressway in Chicago, and this guy was telling me that I likely have myeloma. I got home that night, and I just looked at my husband and said, 'I think that I have cancer, and we've got to go in tomorrow morning.' " The next day, on their way to the doctor's office, Giusti and her husband stopped at a bookstore, where Giusti consulted a copy of "Harrison's Principles of Internal Medicine," a textbook widely used in medical schools. "My husband and I were sprawled out in Borders reading this medical book, and, the more I read, I just had this huge knot in my stomach," Giusti recalled.

Myeloma is a cancer of plasma cells, which are normally found in bone marrow and produce antibodies that help the body fight infections. Under a microscope, plasma cells are easy to identify: they are robin's-egg blue and have a pale area behind the nucleus. When such cells become cancerous, they produce excessive amounts of a single kind of antibody, called a monoclonal protein, and release it into the blood. The malignant plasma cells can cause bone lesions, dangerously high levels of calcium in the blood, kidney failure, and nerve damage. Since 2003, several new drugs have been approved by the F.D.A. for treating the disease, including thalidomide, which was linked to severe birth defects after it was prescribed as a sedative to pregnant mothers in the nineteen-fifties and sixties, and Velcade, the first approved drug from a class of compounds that disable a structure in plasma cells called a proteasome, and by doing so kill the cell. Many myeloma patients are also given bone-marrow transplants, which are thought to extend the length of remissions. Even so, the cancer cells invariably return, evolving in such a way that they no longer respond to treatment.