A drug initially developed to treat Alzheimer’s Disease may be a promising option for patients with Duchenne muscular dystrophy, a small phase III study in The Lancet suggests.
The placebo controlled study of 66 patients with Duchenne muscular dystrophy (DMD) who were not taking glucocorticoids showed significantly less decline in standard respiratory function measures including peak expiratory flow (PEF) with 12 months of treatment with the oral antioxidant idebenone.
Idebenone significantly attenuated the fall in PEF % from baseline to week 52 by 3.05 points (95% CI -7.07 to 0.97) compared with a decrease of 9.01 points (95% CI -13.18 to -4.84, P=0.0001) in patients randomised to placebo, the researchers reported in The Lancet.
“Idebenone also had a significant effect on PEF (L/min), weekly home-based PEF, FVC and FEV1,” they wrote.
Treatment with idebenone was safe and well tolerated with adverse event rates similar in both groups, they said.
Writing in an accompanying editorial Eugenio Mercuri, MD, of Catholic University in Rome, and Francesco Muntoni, MD, of University College London said the results were promising because of the favorable safety profile of idebenone but also show the challenges that exist for studies in people with DMD.
Recently proposed outcome measures that were not available at the time the current study was designed should be investigated.
“They might be even more relevant in young patients on the verge of losing, or who have recently lost, ambulation,” they said.
The study was funded by Santhera Pharmaceuticals. Some co-authors are company employees.