The vast majority of drugs are small molecules designed to bind, interact, and modulate the activity of specific biological receptors. Receptors are proteins that bind and interact with other molecules to perform the numerous functions required for the maintenance of life. They include an immense array of cell-surface receptors (hormone receptors, cell-signaling receptors, neurotransmitter receptors, etc.), enzymes, and other functional proteins. Due to genetic abnormalities, physiologic stressors, or some combination thereof, the function of specific receptors and enzymes may become altered to the point that our well-being is diminished. These alterations may manifest as minor physical symptoms, as in the case of a runny nose due to allergies, or as life threatening and debilitating events, such as sepsis or depression. The role of drugs is to correct the functioning of these receptors to remedy the resulting medical condition.
As an example, the highest grossing drug in 2000 was Prilosec, which earned $4.102 billion in sales. Prilosec is used to treat stomach ulcers and acid reflux disease. Prilosec targets a specific enzyme, the proton pump, which is located in the acid producing cells lining the stomach wall. This enzyme is responsible for the production of stomach acid. Due to genetic reasons, such as deficient enzymes that regulate acid secretion, or physiologic causes, such as stress, too much acid may be produced. This leads to ulceration of the stomach lining or acid reflux disease and heartburn. Prilosec binds to the proton pump and shuts it down, thereby diminishing the production of stomach acid and its associated symptoms.
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