Breakthrough: The Quest for Life-Changing Medicines

In this stimulating debut report, Pao, the former chief development officer at Pfizer, takes readers inside the process for identifying effective pharmaceutical treatments and bringing them to market. Scientists start by using an automated robotic apparatus to quickly test millions of molecules against a targeted disease, selecting the most potent and least toxic substances for chemical refinement and further testing on animals, healthy individuals, and, finally, patients with the disease. Case studies detailing the development of eight treatments illustrate the many ways the process can go awry. For example, Pao recounts how the painkiller paracetamol (now available as Tylenol) remained unused for decades because in 1893, the respected physiologist Joseph von Mering mistakenly concluded that it caused blood toxicity problems. Other drugs rightly never make it to market, Pao notes, discussing how while searching for a new breast cancer treatment, molecular biologist Michel Maira had to abandon the compound BEZ235, which had shown success shrinking tumors in mice, because the effectiveness on humans varied so widely that it was impossible to determine dosing recommendations. The meticulous look inside the drug development process portrays biomedical scientists as near Sisyphean figures who must overcome daunting challenges (90% of all drugs fail human trials) to deliver life-changing treatments to patients. This will leave readers in awe of doctors’ ingenuity and determination. Agent: Euan Thorneycroft, A.M. Heath Literary. (Jan.)