Just what the patient ordered

摘要

In the face of mortal illness, patience is not necessarily a virtue. When Kathy Giusti was diagnosed with multiple myeloma, a form of cancer, doctors gave her just a few years to live. Like many patients, Ms Giusti took to the internet to learn as much as she could about her illness. But that meant precious little when it came to treatments for her disease. At the time of her diagnosis, in 1996, there were no exciting new drugs in development for multiple myeloma; in fact, there were no new drugs at all. "The medicines they recommended for me were the ones they used on my grandfather, years before," says Ms Giusti. With only 50,000 sufferers in America, multiple myeloma is one of thousands of "orphan" diseases that are simply not common enough to interest most drug-makers: there are too few sufferers to justify the cost of developing a new treatment. Although special legislation exists in Europe and America to encourage drugmakers to tackle orphan conditions, the combination of tax breaks and market exclusivity on offer are simply not enough for many firms to attract them to the field. So Ms Giusti, a former pharmaceutical executive, set out to do something about it-and she has succeeded. In 1998, she and her twin sister founded the Multiple Myeloma Research Foundation (MMRF) with the aim of raising money and awareness to "jumpstart" researchers' bright ideas into treatments. Just a few years later, MMRF has garnered more than $50m for research, there are now roughly no drugs in various stages of development for the disease, and Ms Giusti has proven her doctors' prognosis wrong.