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(Circulation. 1957;15:391.)
© 1957 American Heart Association, Inc.

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Failure of Alpha-Phenylbutyrate and Beta-Phenylvalerate in Treatment of Hypercholesterolemia

DONALD S. FREDRICKSON M.D.¹ DANIEL STEINBERG M.D., PH.D.¹

¹ From the Laboratory of Cellular Physiology and Metabolism, National Heart Institute, National Institutes of Health, U.S. Public Health Service.

Sodium alpha-phenylbutyrate and sodium beta-phenyl-n-valerate have been administered to a carefully controlled group of hypercholesterolemic patients. Prior reports that these compounds are effective in the treatment of hypercholesterolemia are not confirmed. Two patients developed toxic reactions requiring cessation of treatment. The ineffectiveness of these drugs, despite their inhibitory effect on the synthesis of cholesterol from acetate, is discussed in terms of recent biochemical information concerning their site of action.

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