Beneficial immune-modulatory effects of the N-163 strain of Aureobasidium pullulans-produced 1,3-1,6 Beta glucans in Duchenne muscular dystrophy: Results of an open-label, prospective, exploratory case-control clinical study

• Published in: IBRO neuroscience reports Vol. 15; pp. 90 - 99
• Main Authors: Raghavan, Kadalraja, Dedeepiya, Vidyasagar Devaprasad, Srinivasan, Subramaniam, Pushkala, Subramanian, Bharatidasan, Sudhakar S., Ikewaki, Nobunao, Iwasaki, Masaru, Senthilkumar, Rajappa, Preethy, Senthilkumar, Abraham, Samuel J.K.
• Format: Journal Article
• Language: English
• Published: Elsevier Ltd 01.12.2023; Elsevier
• Subjects: Beta glucan; Disease modifying agent; Duchenne muscular dystrophy; Dystrophin; Fibrosis; Inflammation; N-163; Short Communication; NSAA; Beta glucan; CK; Inflammation; LeCR; NASH; 6MWT; N-163; LGMD; TNF-α; NLR; Disease modifying agent; Duchenne muscular dystrophy; Fibrosis; DMD; LCR; Dystrophin; MRC
• ISSN: 2667-2421; 2667-2421
• DOI: 10.1016/j.ibneur.2023.06.007

This exploratory case-control study is to evaluate the effects of supplementation of Aureobasidium pullulans-N-163 strain produced 1,3–1,− 6 beta glucan in young patients with Duchenne muscular dystrophy (DMD). Twenty-seven male subjects aged 5–19 years with DMD were included, nine in the control arm and 18 in the treatment arm to receive N-163 beta glucan along with conventional therapies for 45 days. While performing the analysis, steroid usage was also taken into consideration, those not administered steroids (Steroid -ve) (Control, n = 5; treatment, n = 9), those administered steroids (Steroid +ve) (Control, n = 4; treatment, n = 9). IL-6 showed a significant decrease in the treatment groups, especially the N-163 Steroid -ve group. IL-13 decreased in both treatment groups and TGF-β levels showed a significant decrease in the treatment groups, especially the N-163 Steroid –ve group, (p < 0.05). Dystrophin levels increased by up to 32% in the treatment groups compared to the control. Medical research council (MRC) grading showed slight improvement in muscle strength improvement in 12 out of 18 patients (67%) in the treatment group and four out of nine (44%) subjects in the control group. Supplementation with the N-163 beta glucan food supplement produced beneficial effects: a significant decrease in inflammation and fibrosis markers, increase in serum dystrophin and slight improvement in muscle strength in DMD subjects over 45 days, thus making this a potential adjunct treatment for DMD after validation. The study was registered in Clinical trials registry of India, CTRI/2021/05/033346. Registered on 5th May, 2021. •Clinical study to evaluate the safety and efficacy of N-163 beta glucan in patients with Duchenne muscular dystrophy (DMD).•After N-163 supplementation, there was decrease in inflammatory and fibrosis markers IL-6, IL-13 and TGF-β levels.•Dystrophin levels in plasma increased by up to 32% in the N-163 group.•Medical research council (MRC) grading showed muscle strength improvement in 67% of the patients.•N-163 beta 1,3-1,6 glucan food supplement is a potential disease-modifying adjunct treatment for DMD.