Background: Duchenne muscular dystrophy (DMD), is a debilitating genetic neuromuscular condition that leads to progressive muscle weakness and early mortality. Supportive care with disease modifying agents are widely used despite complications, while definitive gene therapies are in various stages of progress. Following an earlier 45 days clinical pilot study proving safety and some efficacy, and a pre-clinical study in MDX mice yielding anti-fibrotic and anti-inflammatory effects of β-1, 3-1,6-Glucan produced by N-163 strain of Aureobasidium pullulans, when orally consumed, we conducted a linear six-month clinical study to evaluate the efficacy of this novel Beta glucan, commercially available as Neu-REFIX as a food supplement on muscle function in young boys diagnosed with DMD.
Methods: 24 patients with DMD were enrolled; 12 ambulatory, 12 non-ambulatory, all orally consumed ß-glucan food supplement along with the standard of care regimen, including steroids. At baseline and after six-months, participants’ muscle function was evaluated by Medical Research Council (MRC) score, Six-minute walk-test (6MWT) and North Star Ambulatory Assessment (NSAA).
Results: In the ambulatory group (n=12), MRC-score improved in 11, with 7.09% average improvement. The 6MWT distance improved in 9, with 29.5 metres average improvement. The NSAA improved by 1 unit in 3 patients and declined by 1 unit in 1 patient. In the remaining patients there was no change. In the 12 non-ambulatory patients, the MRC score improved in 8 patients by 9.27% and declined in 2 patients. No adverse effect of any kind was observed.
Conclusion: This safe and allergen free biological response modifier glucan food supplement has yielded an efficacious outcome in terms of improvement of MRC in 91.6% of patients, 6MWT in 75 % and NSAA in 25 % within six months, unravelling its potential as a disease modifying drug adjuvant in DMD. Having proven its anti-inflammatory and anti-fibrotic efficacy by muscle biopsy in mdx mice, and improvement of plasma dystrophin and IL-12 beside beneficial regulation of the gut microbiome in an earlier clinical pilot study, we recommend a global multi-centric clinical study for its validation as a drug-adjuvant in safely slowing down the progress of DMD.