KATHRYN GIUSTI was driving home when her doctor called. She and her husband had been having trouble conceiving, and she was feeling constantly tired. Her doctor had the answer, but it was one that no one would want to hear. Giusti had multiple myeloma, a cancer that forms in the bone marrow and kills two-thirds of patients within five years.
鈥淚 was devastated,鈥� she says. 鈥淚 was 37 years old, at the top of my career and the mother of a 1-year-old girl. Then I was told I had three years to live.鈥�
That was in January 1996. Giusti is now in remission after a bone marrow transplant and drug treatment. But she has done more than beat the odds on her survival. In 1997 she and her sister founded the (MMRF), a patient advocacy group and funding body that has changed the face of multiple myeloma research. Over the past decade, the MMRF has raised tens of millions of dollars to fund clinical trials, and helped to bring several potentially life-saving treatments to market in a fraction of the usual time.
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In recent years, patient groups like the MMRF have become a powerful force in cancer research. No longer simply trusting the words of doctors and researchers, patients increasingly come armed with knowledge about the latest scientific developments and experimental treatments. Advocates have gained acceptance and respect from scientists and drug companies, and are invited to help set research priorities and comment on research proposals. What鈥檚 more, they ask difficult questions about why promising treatments take so long to reach patients.
At the time of her diagnosis, Giusti was a marketing executive with the pharmaceutical company Searle. She used this experience, combined with what she had previously learned at Harvard Business School, to launch the MMRF a year later. Using money raised from corporate sponsorship and events, the charity now spends $30 million a year on researching the condition 鈥� the same amount as the (NCI). The biggest myeloma research centres in North America now work with the foundation. Drug companies approach Giusti when they want to test their products and patients ask the MMRF about joining these tests. The result is a pipeline of potential treatments that is flowing fast enough for Giusti herself to have benefited.
鈥淪he has done an amazing job,鈥� says Beverly Laird, a breast-cancer survivor who advises the NCI and other cancer funding bodies. 鈥淕iusti has moved along science that would have taken years to complete.鈥�
Achieving this level of influence took more than a flair for fund-raising. 鈥淚 spent hours at the library poring over scientific journals, abstracts and anything else I could get my hands on,鈥� says Giusti. 鈥淭hen I reached out directly to researchers, clinicians and a handful of companies focusing on myeloma.鈥�
That research convinced Giusti that she needed to change the way science is carried out. For researchers outside the pharmaceutical industry, papers and grants, not treatments, are the measure of success and results are kept secret until publication. Giusti, however, wants treatments to emerge as quickly as possible, so one condition of receiving funding from the MMRF is that results must be shared. The foundation also holds researchers accountable when progress slows. 鈥淲e approach the drug development process with a sense of urgency,鈥� she says.
The process begins when a promising drug emerges from the lab. Researchers at 15 partner centres test the compound on the foundation鈥檚 blood and bone-marrow samples, and the results are posted on a shared database. If a treatment looks promising, the foundation will consider funding a clinical trial. Here, its link with patients comes into its own. The MMRF has a database of over 50,000 patients who can be alerted to new clinical trials that they may be eligible for. This can help speed up the time-consuming task of finding suitable subjects.
The process seems to be working. The foundation has run 16 clinical trials in just three years, many more than would usually be possible in that time. It also helped to find thousands of patients for a trial of lenalidomide, a drug which slows the division of cancer cells. Marketed as Revlimid, the drug was approved in 2006 and helped Giusti achieve remission.
While not all patient groups can boast this kind of success, smaller organisations can have a significant impact too. When Ginny Mason joined the in 2003, there was a shortage of tissue samples from patients with this condition, which accounts for no more than 5 per cent of all breast cancers. So Mason established a tissue bank which now contains over a hundred samples. 鈥淲e鈥檙e not just about raising money,鈥� says Mason. 鈥淲e want to be involved in the whole research process.鈥� Another bank of hard-to-obtain samples 鈥� 鈥� has been set up at the Indiana University Simon Cancer Center, Indianapolis, by advocates.
Advocates can also provide unique input into treatments. When a group of oncologists designed a study to test the combination of lenalidomide and the steroid dexamethasone, patient advocates objected to the proposed steroid dose as it can make users unpleasantly hyperactive. It鈥檚 a drug that has you 鈥渧acuuming at 3 am and snapping at your wife鈥�, says Michael Katz, a myeloma patient who examined the proposals. Reluctantly, the oncologists agreed to also trial a lower dose of the steroid 鈥� a decision that was vindicated when survival rates in the low-dose group proved to be significantly higher. 鈥淲hat was really fabulous was that this immediately changed the standard of care,鈥� says Katz.
Not everyone is in favour of this change in the balance of power, however. One charge against advocacy groups is that they focus on new therapies at the expense of improving existing ones. Another is that they take too much credit for advances that also required government funding and basic research into the disease. Despite these quibbles, cancer researchers who spoke to 麻豆传媒 credit advocates with bringing new money and focus to their field. In fact, most of complaints tend to come from those working in fields without strong advocate communities.
Bladder cancer is one such example. It is the sixth most common form of cancer in the US, killing around 14,000 people a year, yet there are only a handful of small advocacy groups that focus on the condition. This could help to explain why it attracts less than half the funding of multiple myeloma, which claims almost 4000 fewer lives per year but has a strong advocate community.
Lack of advocacy also means a lack of awareness, adds Mark Soloway, who treats bladder cancer at the University of Miami in Florida. Smokers are at a higher risk of the condition, so any smoker who develops symptoms such as blood in their urine should immediately be tested for it. Few smokers are aware of this, though, so they may delay before talking to their doctor. Since the window for life-saving treatment can be as short as three weeks, ignorance can be fatal. Some attention from well-informed advocates could be just what the field, and patients, need to help them beat the disease.
