Following the identification of a patient with Kearns-Sayre syndrome who reported significant clinical improvements while taking tadalafil, a Hungarian-American research team studied the efficacy of this molecule and sildenafil, either of which was administered to six patients with primary mitochondrial diseases (MELAS, Kearns-Sayre syndrome, etc.) off-label.
- Analysis of patient fibroblasts incubated with tadalafil or sildenafil showed, in both cases, an improvement in mitochondrial respiratory function and normalisation of cellular metabolites.
- Patients examined after several months of treatment showed lasting clinical improvement and/or stabilisation of symptoms (reduction in dystonia, improved motor skills, better coordination, greater muscle strength, etc.).
- No adverse effects were reported.
- These effects are positive regardless of the clinical form of the patients treated, indicating good potential for thetherapeutic repositioning of tadalafil and sildenafil in mitochondrial diseases.
