TOKYO--(BUSINESS WIRE)--Skeletal muscle fibrosis which leads to locomotor dysfunction in Duchenne Muscular Dystrophy (DMD) could be significantly reduced by oral consumption of Neu-REFIX beta 1,3-1,6 glucans alone for 45 days without any other drugs, in MDX mice model of DMD. This research was undertaken jointly with the team of Dr Yoshitsugu Aoki, Head, Dept. of Molecular Therapy, National Center for Neurology and Psychiatry (NCNP), Tokyo, Japan, a pioneer in developing solutions to DMD such as exon skipping therapy. The findings published in Scientific Reports, a publication by Nature portfolio also reported a reduction in muscle inflammation, which precedes fibrosis and muscle dysfunction, leading gradually to respiratory muscle failure followed by lung dysfunction, cardiac fibrosis, heart failure and premature death.
This study followed an earlier clinical trial involving 27 patients in two groups in which safety along with reduction of inflammation and fibrosis and increase in plasma dystrophin were confirmed in those who consumed the same neu REFIX Beta-glucans along with standard of care medications, compared to the control group who took only the standard of care medications. An important break-through in this animal study is that the treatment-arm group MDX mice consumed only the Neu-REFIX Beta glucans without any drugs, unlike the clinical study. Currently, for DMD there are no definitive and efficacious remedies available. Exon skipping therapy is available only to less than 30% of the patients with specific exon deletions and gene therapy may not be applicable to patients in the advanced stages of DMD. Going by our clinical experience, Neu-REFIX β-glucans are worth validating clinically for longer duration, both as a stand-alone option and also as an adjuvant to standard of care medications, in different sets of appropriate age groups of patients, opined Dr Raghavan, who has recently completed another clinical study of six-months duration wherein Neu-REFIX yielded improvement in 6MWT, NSAA & MRC.
With additional experiments in MDX mice underway, as more data evolves, that may be value adding to recommend Neu REFIX β-glucan to DMD patients, said Dr Yoshitsugu Aoki. Reduction of myocardial fibrosis in MDX mice and beneficial reconstitution of gut microbiome in clinical studies give us a hope that this allergen-free orally consumable and safe food supplement could become a worthy adjuvant to disease modifying treatments in DMD, said researchers who presented the data in World Muscle Society WMS 2023 and World Congress of Neurology WCN 2023.