In this phenomenal memoir, Dr David Fajgenbaum relates his diagnostic, therapeutic, and scientific odyssey with the rare disorder of Castleman’s disease, and does so from the perspective of a patient, a medical student, and a physician‐scientist. A memoir may seem like an odd choice for a Clinical and Translational Science book review, but, in fact, this is a quintessential translational medicine story. Translational medicine is often described as an intrinsically inspirational field, because a core goal is bench‐to‐bedside research, often with direct patient impact, but Fajgenbaum inspires to the next degree. You need read no further than the key words in the title―chasing, cure, race, hope, action―to realize the excitement and motivational impact of this work.
Fajgenbaum went from a fit, totally healthy medical student to near death, experiencing multisystem organ failure in the intensive care unit of the very same hospital of his medical school. To make matters worse, his diagnosis―and treatment plan―were initially mysteries. The book details Fajgenbaum’s diagnostic, therapeutic, and ultimately scientific journey. The first two components―a diagnostic and therapeutic odyssey―have much in common with the experience of many patients suffering from a rare disease. But Fajgenbaum also directly confronts his condition with a scientific investigation of what was ultimately diagnosed as Castleman’s disease. Like many rare diseases, the etiology of this disease is poorly understood, and the truly unique aspect of the story is the patient‐centered translational medicine and scientific detective work involved in unraveling the etiology―and a novel treatment―of Castleman’s disease. No story can be more patient‐centered than doing medical research on your own disease, and saving your own life.
Overall, the book is moving, impactful, heartfelt, and inspiring. Fajgenbaum discovers the importance of making every moment count in both life and science. He writes, “Every second of my life was now filled with three possibilities―triumph, failure, or just getting by. Surprisingly, living in overtime had liberated me to be my best self.” That sense of urgency translates to his community of family, friends, fellow patients, and researchers as well. It is clear that Fajgenbaum powers through his diagnostic and treatment odyssey with the help of a strong community of friends and family. At one point in the book, Fajgenbaum is introduced to another patient with Castleman’s disease, who was in remission, and helps to kindle his hope. Noting that the Castleman’s research community had abundant silos, he cofounded the Castleman Disease Collaborative Network, in part to create a research environment in which silos can be minimized through collaboration and sharing.
The heart of the book is about turning hope into action through curiosity and innovation. The scientific and related therapeutic journey had a number of turns, and disappointments, along the path. Spoiler alert: After a series of hypotheses and blind alleys, Fajgenbaum discovers the connection between the vascular endothelial growth factor (VEGF) pathway, T cells, and Castleman’s disease leading to a therapeutic advance with a repurposed mammalian target‐of‐rapamycin inhibitor, sirolimus. This frameshift from traditional thinking about Castleman’s disease required hard work and great collaborations. Fajgenbaum notes, “But there is a hard truth about epiphanies: They don’t materialize out of thin air.” Early on in Fajgenbaum’s diagnostic and therapeutic journey, he developed blood moles coincident with the presentation of his disease. As a medical student at the time, he thought this finding must be part of the picture, but his doctors advised him to ignore them, presumably because he had bigger medical problems. However, his unrelenting curiosity was not abated, and these blood moles proved to be one of the connections to VEGF that ultimately helped unravel the mystery. Furthermore, Fajgenbaum believes his experience does not have to be unique, “Innovation isn’t an art. Innovation, like hope itself, is a force. Innovations are most effectively made with the following systematic approach: Inventorying all possible ideas from a wide range of stakeholders, systematically evaluating and prioritizing them, recruiting the very best people in the world, and then working like mad to execute.” The field of translational medicine would do well to take his prescription for innovation as a guiding principle.