Palliative and Causative Acting Drugs

By far the largest fraction of today's pharmacopoeia does not target disease at its cause - as these causes are largely unknown - but by modulating a pathway that affects the disease-relevant phenotype or function. We refer to such drugs as symptomatic or palliative agents. The pathways they target are known from more than a century of physiological, biochemical, and pharmacological research. The pathways they modulate are disease phenotype-relevant (albeit not disease cause-relevant), and while they are not dysfunctional, their modulation can effectively be used to counterbalance the effect of a dysfunctional, disease-causing pathway. Thus signs and symptoms of the disease can be alleviated, often with striking success, notwithstanding the fact that the real cause of the disease remains untouched. A classical example of such an approach is the acute treatment of thyrotoxicity with b-adrenergic-blocking agents: even though in this case the sympathetic nervous system does not contribute causally to tachycardia and hypertension, dampening even its baseline tonus through this class of rapidly acting drugs can quickly and successfully relieve the cardiovascular symptoms and signs of this condition, and may well prevent a heart attack if the patient has underlying coronary disease, before the causal treatment (in this case, available through partial chemical ablation of the hyperactive thyroid gland) can take effect.

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