We the Scientists

Starred review from February 15, 2023
The story of a painful but inspiring search for a cure for a fatal disease. Dockser Marcus, a Pulitzer Prize-winning Wall Street Journal reporter, had finished a series on advances in cancer therapy when she endured the death of her mother to a rare cancer for which no treatment existed. The author's research revealed that activists had often pressured the Food and Drug Administration to pay attention to diseases such as AIDS and allow community participants on hospital and government advisory committees. It was an effective approach, but the author found that the scientists called the shots. They were the experts who would design experiments and work at their own, careful pace. This lugubrious system has worked miracles, but it's too slow if a loved one is sick. Dockser Marcus discovered others with the same experience who had organized to work with and even guide researchers. They called themselves "citizen scientists." The author concentrates on the fight against the rare genetic defect Niemann-Pick disease type C, which affects only about 200 individuals in the U.S. and 500 globally. Often healthy at birth, affected children "progressively" lose the ability to walk, talk, and eat; most die by age 19. Dockser Marcus introduces us to the families, children, physicians, researchers, and FDA officials. Almost all are sympathetic. Rather than merely lobbying or raising money, parents have come together to search for treatments and suggest lines of research. Fiercely motivated, they have educated themselves, devouring medical journals so obsessively that some have written articles for these same journals. They have convinced researchers and government agencies to launch studies and then cooperated closely, not only volunteering their children, but also gathering far more data than the usual parent. The author ends her expert mixture of reportage and storytelling on a somewhat hopeful note. Promising treatments are in the pipeline, but there have been numerous bitter disappointments, and many affected children have suffered serious complications during trials. A moving argument for a more focused, humane, and efficient system for conducting medical research.

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