Clinical Radiological improvements in mitochondrial encephalomyelopathy following Sodium Dichloroacetate therapy.
Comments; Significant improvements achieved with NaDCA, and a suggestion that NaDCA be used in all patients with Mitochondria-related enzyme deficiencies. Pretty much every condition NaDCA is tested on carries the recommendation for use, however it never happens. It’s all about the money! Human life and suffering does not form part of the decision.
This is what Wikipedia says regarding treatment.
There is no known cure for this disease. Although the mortality rate among children with deficiency of LCHAD or complete deficiency of the trifunctional protein had been reported to be 75 to 90%, Ibdah et al. (1999) found that 67% of the affected children in their study were alive and receiving dietary treatment at the most recent follow-up, and most were able to attend school. Dietary treatment of children with fatty acid oxidation disorders dramatically reduced morbidity and mortality.
Why is NaDCA not being used for these children, based on these results from 1998?
- Seiji Kimura,
- Noriyuki Ohtuki,
- Atuo Nezu,
- Miyabi Tanaka,
- Saoko Takeshita
- Department of Pediatrics, Urafune Hospital, Yokohama City University, 3-46, Urafune-cho, Minami-ku, Yokohama 232, Kanagawa, Japan
- Received 22 April 1997. Revised 19 August 1997. Accepted 19 August 1997. Available online 19 February 1998.
We administered sodium dichloroacetate (DCA), which reduces the circulating lactate and pyruvate concentrations by stimulating the activity of the pyruvate dehydrogenase complex (PDHC), to three children with mitochondrial encephalomyelopathy. Significant clinical, biochemical and radiologic improvements were obtained following DCA therapy (approximately 30 mg/kg per day, divided into three doses). All three patients had non-pyruvate dehydrogenase complex (PDHC) deficiencies: two exhibited Leigh syndrome (complex I deficiency and unknown etiology), and one abnormal myelination (multienzyme deficiency), demonstrated on magnetic resonance imaging (MRI). The lactic and pyruvic acid concentrations in serum and cerebrospinal fluid (CSF) were decreased significantly by the oral DCA treatment. The lactic acid peak on MR spectroscopy also markedly decreased in parallel with the CSF level. In addition, the brain lesions observed on MRI were improved in all patients. No exacerbation was observed in any of the patients, who have been followed-up more than 21 months following the DCA therapy. These results suggest that DCA therapy should be considered in all patients with a mitochondria-related enzyme deficiency.