Background: Arresting the gradual decline of muscle function has been the goal of disease modifying treatments (DMTs) for Duchenne Muscular Dystrophy (DMD), by controlling inflammation, reducing fibrosis, thereby allowing muscle regeneration. However, with DMTs that include steroids, there are associated side effects. After establishing the safety, anti-inflammatory, anti-fibrotic effects apart from muscle regeneration of a Aureobasidium pullulans N-163 strain produced 1,3-1,6 ß-glucan (Neu-REFIX) in pre-clinical studies of mdx mice and proving the safety, improvement in muscle function in clinical studies, we herein report the effects of Neu-REFIX as a standalone agent on muscle function in mdx mice.
Methods: Thirty mice divided into three groups, 10 each; Gr. 1 normal mice (control), Gr.2 mdx mice as vehicle, and Gr.3 mdx mice administered Neu-REFIX for 45 days. Fatigue score was evaluated using fore-limb grip test on day 41 and day 45.
Results: Fatigue score was analysed by measuring the decrement between the first and the last pull of fore-limb grip test. The fatigue score (mean ± SD) decreased in mdx-Neu-REFIX (9.67 ± 17.24) compared to that of mdx-Control group (25.13 ± 31.97) on both day 41 (p-value = 0.6) and day 45 significantly: mdx-Neu-REFIX (7.2 ± 13.3) compared to mdx-Control (16.25 ± 11.23) group (p-value = 0.04), showing that mdx-Neu-REFIX mice have greater resistance to fatigue compared to mdx-control though improvement in forelimb grip strength was only modest.
Conclusion: Given the reduction in fibrosis, inflammation, and enhanced muscle regeneration in 45 days earlier, as well as significant evidence of comparative arrest in muscle fatigue decline in such a short duration of standalone administration of Neu-REFIX, large multi-centric clinical studies with Neu-REFIX beta glucans as a standalone agent or adjuvant to other therapies in slowing the progression of DMD are recommended.