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The early 2000s marked a hopeful time for many cancer patients. Decades of basic research, growing understanding of the biology of cancer, and billions of dollars of government investment had finally yielded breakthroughs in treatments. Targeted therapies homed in on specific proteins in tumors, stopping them from growing and spreading without many of the debilitating side effects associated with traditional chemotherapy regimens. New drugs continued to come on the market. Growing numbers of people started wrestling with what it meant to live with cancer as a chronic health condition rather than as a death sentence. As a reporter on the health beat, I spent a year working on a series of articles for The Wall Street Journal chronicling the transformation in cancer patients lives. I came away from my reporting hopeful about what the new therapies could do.

Then, after finishing that series of articles in 2004, I got a very personal reminder of just how far we still had to go in finding effective treatments for cancer. In December of that year, my mother was rushed to the emergency room, doubled over in excruciating pain. Doctors diagnosed metastatic gallbladder cancer, a rare disease that, along with cancer of the nearby bile ducts, affects about 12,000 Americans each year. In comparison, more than 290,000 Americans are diagnosed with breast cancer every year.

I soon learned what that disparity meant. Those promising targeted therapies I had been writing about werent available for people like my mother. Thats because the pharmaceutical companies that run most of the clinical trials for new drugs werent working on developing new treatments for gallbladder cancer; the market was simply too small to justify the resources and investment required to identify, test, and get novel drugs approved. Even if they had been willing to try, studies indicated that the time from a promising idea in a lab to market approval was closing in on seventeen yearsfar too long to ever help my mother, since people with metastatic gallbladder cancer rarely live more than two years. The only available options were drugs that over the decades had exhibited only limited success; the odds of them helping now werent good.

At major cancer centers like the one where my mother underwent chemotherapy, there wasnt a lot of data available on how previous patients fared or if one drug seemed better than another. The number of patients with gallbladder cancer at individual institutions was in the single digits, too small to be statistically meaningful, and the researchers didnt pool data with all the other centers that also saw gallbladder cancer patients. Researchers who generated a hypothesis struggled to get funding from either the government or large private foundations to test their theories; as a result, promising ideas languished. Scientists interested in finding drugs for rare diseases often have to go it alone, with predictable consequences for patients like my mother. Each round of chemotherapy failed to stop or slow down the insidious progress of the disease. My mother died in May 2007, a few weeks after her sixty-fifth birthday.

After her death, a feeling of helplessness profoundly shook me. I had been reporting on promising new options for cancer treatments, but in my mothers case, we had none. Why? My mothers oncologist was thoughtful and compassionate but lacked any data upon which to base trying a different approach than one that had been failing for decades. A scientist I interviewed who was interested in pursuing novel research had collected gallbladder tumor samples, but they remained locked in a freezer because he couldnt get the time or funding to move the project forward. I felt I shared a portion of the blame too: Could I have done something different or something more to save my mothers life?

I started reading histories about successful patient advocacy movements started by patients with HIV and breast cancer who founded new organizations and confronted the medical establishment. I was struck by how many of the participants framed their efforts to accelerate drug development as part of a broader social movement fighting for social and political equality. In addition to helping drive the development of drugs in their own diseases, they tried to influence national health policy. HIV activists pushed the U.S. Food and Drug Administration (FDA) to think differently about the way it went about assessing if a drug was working. They wanted the agency, when weighing clinical trial results, to formally authorize the use of surrogate markers, data points that indicate patients are benefiting but dont definitively show they are living longer. The agency agreed and initiated the Accelerated Approval Program that over the years has allowed for faster approval of drugs for HIV and other life-threatening conditions. Breast cancer, disability and mental health activists, and other patient-driven groups also insisted that they should have the right to help shape policies surrounding clinical trials, including determining how much risk they were willing to take when trying experimental drugs. As a result of their activism, hospitals and academic centers routinely include community participants as members on institutional review boards that ensure the protection of the rights of people participating in research. Patients serve on various government advisory committees, offering their views on proposed projects or weighing in on some funding decisions.