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Book Review: The Journey to Drug Discovery and Development

Yale Medicine Magazine, Spring 2025 (issue 174) AI for Humanity in Medicineby Kenny Cheng

Contents

Physician-scientist William Pao ’97 PhD, ’98 MD, was 13 years old when his father died during a difficult surgery for colon cancer. The loss had a profound impact on Pao, inspiring a lifelong commitment to making a difference for patients like his father. Two decades later, he became a medical oncologist and translational science researcher, dedicating his career to advancing cancer treatment.

Now, after working as a researcher at Memorial Sloan Kettering Cancer Center, a professor and head of the personalized cancer medicine unit at Vanderbilt University, and an executive at Pfizer and Roche Pharmaceuticals, Pao has written a book titled Breakthrough: The Quest for Life-Changing Medicines. With a foreword by Nobel Prize winner Harold Varmus, MD, the book follows the journeys of scientists, physicians, and patients whose ingenuity and persistence led to the discovery and development of eight modern lifesaving drugs.

Among the eight stories, Pao explores how paracetamol (marketed in the United States under brand names including Tylenol®), now among the most widely used painkillers, remained overlooked for decades; how a mother’s relentless advocacy spurred a medical breakthrough for spinal muscular atrophy (one of the world’s most common rare diseases); and how countless hours of painstaking experiments with a reengineered immune molecule (antibody) transformed hemophilia treatment. Pao also reflects on his own experience in lung cancer research as a postdoc in Varmus’ lab, bringing a personal perspective that enriches each of the drug-discovery stories with insights into the world of academic science.

Pao offers readers a compelling, up-close look at the science, creativity, and innovation that has led to some of the most significant medical advances of recent decades. It is a fitting tribute to the power of team science to enhance human health.

—Kenny Cheng

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