Comments: NaDCA showed significant improvements, and it is advised that all patients with mitochondrial enzyme deficits utilize it. NaDCA is recommended for usage in almost all conditions, yet this never actually happens. All of it is about the money! Human agony and life do not factor into the choice.
This illness has no known treatment. Ibdah et al. (1999) discovered that 67% of the affected children in their study were alive and receiving dietary treatment at the most recent follow-up, and the majority were able to attend school, despite the fact that the mortality rate among children with LCHAD deficiency or complete deficiency of the trifunctional protein had been reported to be 75 to 90%. Children with abnormalities of fatty acid oxidation receiving dietary treatment saw a significant decrease in morbidity and mortality.
Based on these findings from 1998, why isn’t NaDCA being used for these kids?
Three kids with mitochondrial encephalomyelopathy received sodium dichloroacetate (DCA), which lowers the levels of circulating lactate and pyruvate by promoting the activity of the pyruvate dehydrogenase complex (PDHC). Following DCA therapy (about 30 mg/kg per day, divided into three doses), significant clinical, biochemical, and radiologic improvements were attained. The non-pyruvate dehydrogenase complex (PDHC) deficiencies were present in all three patients; two of them displayed Leigh syndrome (complex I deficiency with unknown etiology), and the third patient had abnormal myelination (multienzyme deficiency), as shown by magnetic resonance imaging. The oral DCA therapy dramatically reduced the serum and cerebrospinal fluid (CSF) levels of lactic and pyruvic acid. Along with the CSF level, the lactic acid peak on the MR spectroscopy also significantly fell. Additionally, all patients’ MRI-detected brain lesions improved. None of the patients who have been monitored for more than 21 months after receiving DCA therapy experienced an aggravation. These findings imply that DCA therapy ought to be taken into account for all patients who have mitochondria-related enzyme deficiencies.