Use of Dichloroacetate for Leigh's patients

[Guest guest]

I have a 10 month old son who has been diagnosed with

Leigh's desease. A doctor at Children's Hospital in

Boston would like him to try an experimental drug

called Dichloroacetate (DCA). It is supposed to help

lower his levels of lactic acid. This drug while not

yet approved has been successful in trials with other

patients. There are some possible side effects,

including nerve damage. Does anyone on this list

have any advice on the use of this drug. I would be

interested in any feedback both positive and negative

on the use of DCA for children with Mitochondrial

deseases.

Thanks,

Mark

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[Guest guest]

Mark,

our daughter used DCA when young for 1 year. She had lactic acidosis from Pyruvate dehydrogenase deficiency. The DCA did reduce her lactate levels, but she did not improve clinically whilst on the drug. I think lots of other things were happening at the time too. She took thiamine along with the DCA to help minimize the risk of peripheral neuropathy, otherwise sh sufferred no ill effects.

ne

(in Australia). Mum to Gaby 6 years, mito disorder, ketogenic diet, G Tube, microcephaly, global delays, and the prettiest smile.

-----Original Message-----From: Mark Munzer Sent: Monday, 24 September 2001 10:46To: mito Subject: Use of Dichloroacetate for Leigh's patientsI have a 10 month old son who has been diagnosed withLeigh's desease. A doctor at Children's Hospital inBoston would like him to try an experimental drugcalled Dichloroacetate (DCA). It is supposed to helplower his levels of lactic acid. This drug while notyet approved has been successful in trials with otherpatients. There are some possible side effects,including nerve damage. Does anyone on this listhave any advice on the use of this drug. I would beinterested in any feedback both positive and negativeon the use of DCA for children with Mitochondrialdeseases.Thanks,Mark__________________________________________________