The Pills of Last Resort: How Dying Patients Get Access to Experimental Drugs

Darshak M. Sanghavi in The New York Times:

DrugIt was shortly after the breadbasket arrived last year at the Temple Bar near Harvard Square that Sarah Broom first told me about the last-ditch plan to save her own life. Broom’s mere presence that evening was something of a miracle. Several years earlier, in 2008, while pregnant with her third child, she received a harrowing diagnosis. A 35-year-old English lecturer and poet living in New Zealand, Broom developed a persistent cough. She saw doctors in Auckland repeatedly over the course of a few months, but they didn’t want to do an X-ray on a pregnant woman. Finally, her shortness of breath became so severe that they relented, and 29 weeks into her pregnancy, she was found to have a large mass on her lung. She underwent a cesarean section — her daughter was born almost three months early — and a biopsy. Broom had advanced-stage lung cancer. “I was told nothing could be done to cure the cancer, but that various treatments could give me time,” she recalled. Less than 1 percent of patients live more than five years. She endured chemotherapy for weeks but then developed severe pelvic pain. To her horror, tests showed there was a new plum-size tumor on her ovary that wasn’t present at her C-section. The cancer was spreading relentlessly. Broom’s doctors predicted she had only a few months to live. She worried about her two sons, Daniel and Christopher, ages 5 and 2; her husband, Michael, whom she’d been with since they were teenagers; and her premature baby, Amelia, who was still hospitalized in the newborn unit. “My determination was to live, to live a long time — what else could I do, with three little kids depending on me — but at this time, it was clear that the situation looked pretty dire indeed,” Broom told me.

In desperation, she called friends around the world, including Meghan O’Sullivan, a former deputy national security adviser in the Bush administration, who contacted Bruce Chabner, the director of clinical research at Massachusetts General Hospital Cancer Center in Boston. Chabner asked Broom to send him her biopsy, so he could analyze its DNA. Her tumor had a mutation in a gene called anaplastic lymphoma kinase, or ALK, which occurs in about 5 percent of lung cancers. A Japanese group discovered it only a year earlier. Chabner knew that the drug company Pfizer was developing a new compound called crizotinib that might treat this mutation. The trouble was, it had been given to only two ALK-positive people before, and one died anyway. Still, it was Broom’s only hope, and Pfizer agreed to enroll her in a trial in Australia. Incredibly, the tumors shrank by half, and Broom led an almost normal life for two years.

More here.