title: Isoniazid therapy of Huntington disease.
abstract: We describe clinical and biochemical changes in seven patients with Huntington disease given isoniazid (INH) in dosages three to five greater than normally used in tuberculosis. Because INH inhibits the enzyme gamma-aminobutyric acid aminotransferase (GABA-T), and increases GABA content in the brains of experimental animals, it might correct the brain GABA deficiency characteristic of Huntington disease. Of six patients treated long enough to be clinically evaluated, one showed marked and two others showed signifciant improvement. High-dose INH therapy carries serious toxic risks, which are influenced by patients' acetylator phenotypes. Nevertheless, results are sufficiently promising to warrant further controlled trials of INH or other GABA-T inhibitors in Huntington disease.
mesh_terms: D000612:4-Aminobutyrate Transaminase / Q000037:antagonists & inhibitors; D000328:Adult; D001921:Brain / Q000378:metabolism; D002986:Clinical Trials as Topic; D005260:Female; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy* / Q000201:enzymology / Q000378:metabolism; D007538:Isoniazid / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D010919:Placebos; D005680:gamma-Aminobutyric Acid / Q000378:metabolism
chemical_list: D010919:Placebos; D005680:gamma-Aminobutyric Acid; D000612:4-Aminobutyrate Transaminase; D007538:Isoniazid