A Tale of Two Worlds

Abstract

This book describes exciting opportunities for drug development for patients with autoimmune illnesses. The opportunities include targeted delivery, genome-based drugs, nanomedicine, microparticles, metabolic targets, microbiome, nervous system control, biomarkers, and stem cells. Answers in any one of these areas may lead to dramatic changes in the care of ill humans. The chapters present the points of view of scientists engaged in this enterprise. Clinicians see the problems differently. The starting point for all clinical research and treatment trials—but not necessarily for clinicians—is correct diagnosis. To achieve this, investigators study only patients who meet well-defined inclusion and exclusion criteria—those with “pure” diagnoses—and they assume that information from the “pure” group will apply to excluded patients as well. Clinicians, however, treat patients who have “pure” and “impure” diagnoses. Neither investigators nor clinicians know whether the same mechanisms apply to both groups or whether the treatment algorithms should be the same. Investigators and clinicians rarely ask: Is there a gold standard for the concept, correct diagnosis? How precise must it be? For how long does a diagnosis remain valid? While investigators require clear definitions of diagnoses, clinicians do not. For clinicians, disease symptoms and actionable mechanisms are important. External variables that influence a patient’s outcome are important. Diagnosis names are not.