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INTRODUCTION

The first edition of Goodman & Gilman, published in 1941, helped to organize the field of pharmacology, giving it intellectual validity and an academic identity.* That edition began: “The subject of pharmacology is a broad one and embraces the knowledge of the source, physical and chemical properties, compounding, physiological actions, absorption, fate, and excretion, and therapeutic uses of drugs. A drug may be broadly defined as any chemical agent that affects living protoplasm, and few substances would escape inclusion by this definition.” This General Principles section provides the underpinnings for these definitions by exploring the processes of drug invention, development, and regulation, followed by the basic properties of the interactions between the drug and biological systems: pharmacodynamics, pharmacokinetics (including drug transport and metabolism), and pharmacogenomics, with a brief foray into drug toxicity and poisoning. Subsequent sections deal with the use of drugs as therapeutic agents in human subjects.

Use of the term invention to describe the process by which a new drug is identified and brought to medical practice, rather than the more conventional term discovery, is intentional. Today, useful drugs are rarely discovered hiding somewhere waiting to be found. The term invention emphasizes the process by which drugs are sculpted and brought into being based on experimentation and optimization of many independent properties; there is little serendipity.

*Deceased, December 23, 2015. AGG served on the Board of Directors of Regeneron Pharmaceuticals, Inc., a potential conflict of interest.

ABBREVIATIONS

Abbreviations

ADME: absorption, distribution, metabolism, excretion

AHFS-DI: American Hospital Formulary Service-Drug Information

BLA: Biologics License Application

CDC: Centers for Disease Control and Prevention

CDER: Center for Drug Evaluation and Research

DHHS: U.S. Department of Health and Human Services

FDA: U.S. Food and Drug Administration

HCV: hepatitis C virus

HMG CoA: 3-hydroxy-3-methylglutaryl coenzyme A

IND: Investigational New Drug

LDL: low-density lipoprotein

NDA: New Drug Application

NIH: National Institutes of Health

NMEs: New Molecular Entities

NMR: nuclear magnetic resonance

PCSK9: proprotein convertase subtilisin/kexin type 9

PDUFA: Prescription Drug User Fee Act

PhRMA: Pharmaceutical Research and Manufacturers of America

R&D: research and development

SCHIP: State Children’s Health Insurance Program

siRNAs: small interfering RNAs

FROM EARLY EXPERIENCES WITH PLANTS TO MODERN CHEMISTRY

The human fascination—and sometimes infatuation—with chemicals that alter biological function is ancient and results from long experience with and dependence on plants. Because most plants are root bound, many of them produce harmful compounds for defense that animals have learned to avoid and humans to exploit (or abuse).

Earlier editions of this text described examples: the appreciation of coffee (caffeine) by the prior of an Arabian convent, who noted the behavior of goats that gamboled and frisked through the night after eating the berries of the coffee plant; the use of mushrooms and the deadly nightshade plant by professional poisoners; of ...

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