Introduction and Dedication: Liz’s Story
The Low sisters loved the Beatles. Elizabeth (Liz) Low liked to play George Harrison while her older sisters Cindy and Jan played Paul McCartney and Ringo. But who would be John Lennon? It was left to other children from the neighborhood, sometimes Bee-Barb Bennett, our next door neighbor—or in times of real desperation my sister Karen or I—to fill in. Liz’s guitar wept, but not so gently; she died at age 7 of leukemia.
Had Liz survived, she would have become my stepsister when her father and my mother got married in 1979. She died because in the 1960s, we were just learning how to treat childhood leukemia. In the 1950s, chances of surviving 5 years with acute lymphocytic leukemia (ALL) were around 1 percent.¹ When Liz died, that had improved to 1 in 3 chance of survival. By the 1980s, 5-year survival among children was at 80 percent. ALL went from being a death sentence in 1960 to expected cure by 1980 (with four children cured for each one who died).² The job is not over—twenty percent is still too many dying children—but it is still a remarkable story of science-driven progress. But the kinds of studies that make up that science were many and varied.
The survival difference between 1950 and 1990 was neither a new miracle drug nor scientific breakthrough. In fact, advances came mainly from using drugs available by the end of World War II, several of them initially developed as candidates for poison gas—ironically discovered in the quest for chemical weapons. Progress grew from a combination of rigorous clinical testing, methods of controlling bleeding and infection, knowledge about how to block cancer cells from proliferating, and incremental, systematic study of how children fared, with small improvements year-by-year.³ The intricacies of cellular metabolic pathways—how vital chemicals are processed by enzyme cascades—was an important source of ideas for several drugs, pioneered by Nobelists Gertrude Elion and Robert Hitchings. If there was a single scientific theme that proved most important, it was the growing rigor of systematic clinical trials. The story involved thousands of scientists, hundreds of clinicians, and many research centers.
More to the point, progress depended critically on most children with leukemia being taken care of in the context of clinical research, a system of care that captured information about treatment improvement from today’s patients to benefit tomorrow’s.4 The vast majority of children with cancer, over 80 percent, got (and still get) referred to child oncologists who participated in research. (Compare that to an estimated 3 percent of adults with cancer participating in research.) Improvement followed from cooperative, collective advance of knowledge through rigorous design, information-sharing, and focus on the goal of improved treatment survival.
Liz was engaged in clinical research. She died, but children with the same disease a few years later usually did not. Progress against childhood leukemia was a story of successful medical research—of scientists and clinicians collaborating to understand biology, and to translate new knowledge into clinical application—from laboratory bench to bedside to system of care. It was also a story of people participating in research, thousands of Lizes who lost their lives while contributing tiny bits of knowledge that added up to better medicine. This website is about the policies that might replicate that story.
³ John Laszlo tells the story of childhood leukemia from the perspective of the scientists and clinicians involved in The Cure of Childhood Leukemia: Into the Age of Miracles (Rutgers University Press, 1995).
4 Joseph V. Simone and Jane Lyons, Huntsman Cancer Institute, University of Utah, “Superior Cancer Survival in Children Compared to Adults: A Superior System of Cancer Care?” background paper prepared for the National Cancer Policy Board, Institute of Medicine and Division of Environmental and Life Sciences, National Research Council, Natoinal Academy of Sciences, http://www.iom.edu/iom/iomhome.nsf/pages/Background+Paper+Cancer+Survival+in+Children, accessed 28 January 2002.