Long-term treatment of juvenile Huntington's chorea with dipropylacetic acid.
Since the proposed mode of action of dipropylacetic acid, an anticonvulsant, is to increase central nervous system gamma-aminobutyric acid levels, we used this agent to treat identical twins with juvenile Huntington's chorea. Their clinical status did not improve immediately after they received dipropylacetic acid. Furthermore, long-term administration (over a year) of high doses of the agent (up to 2,400 mg per day; 92 mg per kilogram per day) did not seem to alter the slow progression of their disease. Prior to treatment with dipropylacetic acid, the twins had normal cerebrospinal fluid gamma-aminobutyric acid levels. In addition, cerebrospinal fluid 5-hydroxyindoleacetic acid and homovanillic acid were determined before and after 18 hours of high-dose probenecid. The former showed a normal threefold to fourfold increase after probenecid administration, but homovanillic acid had a distinctly subnormal turnover after probenecid, with only a threefold rather than the normal tenfold increase.
D002648:Child; D004200:Diseases in Twins; D005260:Female; D006207:Half-Life; D006801:Humans; D006816:Huntington Disease / Q000175:diagnosis / Q000188:drug therapy*; D014631:Valerates / Q000627:therapeutic use*; D014635:Valproic Acid / Q000009:adverse effects / Q000378:metabolism / Q000627:therapeutic use*
D014631:Valerates; D014635:Valproic Acid