Disease-modifying immune-modulatory effects of the N-163 strain of Aureobasidium pullulans-produced 1,3-1,6 Beta glucans in young boys with Duchenne muscular dystrophy: Results of an open-label, prospective, randomized, comparative clinical study

BackgroundThis trial 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). MethodsTwenty-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, patients were stratified into: those not administered steroids (Steroid -ve) (Control, n=5; treatment, n=9), those administered steroids (Steroid +ve) (Control, n=4; treatment, n=9), which was not pre-specified. ResultsIL-6 showed a significant decrease in the N-163 Steroid -ve group, from 7.2 {+/-} 1.2 pg/ml to {+/-} 0.03 pg/ml. IL-13 decreased in both treatment groups--from 157.76 {+/-} 148.68 pg/ml to 114.08 {+/-} 81.5 pg/ml (N-163 Steroid -ve) and from 289.56 {+/-} 232.88 pg/ml to 255.56 {+/-} 214.13 pg/ml (N-163 Steroid +ve). TGF-{beta} levels showed a significant decrease in the N-163 Steroid -ve group. Dystrophin levels increased by up to 32% in both treatment groups. 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. ConclusionSupplementation 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. Trial registrationThe study was registered in Clinical trials registry of India, CTRI/2021/05/033346. Registered on 5th May, 2021.