Rare Diseases & Cancer – Adjuvants

Duchenne Muscular Dystrophy (DMD) Rare Disease:

Neu-REFIX Beta 1,3-1,6 glucan is a novel immune-modulating biological response modifier beta glucan produced an exopolysaccharide by the N-163 strain of Aureobasidium pullulans. In Duchenne muscular dystrophy (DMD), it has demonstrated significant therapeutic potential, including stand-alone efficacy in reducing skeletal muscle inflammation and fibrosis in mdx mice, enhancement of muscle regeneration within 45 days, and a decline in fatigue scores in preclinical studies. It has been shown to be clinically safe as an adjuvant alongside standard-of-care treatments and has demonstrated improvement in plasma dystrophin levels in a 45-day clinical study. A six-month clinical trial indicated safe halting of disease progression, along with beneficial reconstitution of the gut microbiome in patients with DMD and clinical improvement in individuals with limb-girdle muscular dystrophy (LGMD). Additionally, it has been shown to reduce myocardial fibrosis in mdx mice. GN Corporation’s Neu-REFIX Beta glucan, manufactured in Japan, has received both Rare Pediatric Disease Designation (RPD) and Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of Duchenne muscular dystrophy.

(Image source: Front. Genet., 09 June 2025 Volume 16 – 2025 | https://doi.org/10.3389/fgene.2025.1569289)

Cancer:

Nichi Brite Beta 1,3-1,6 glucan, produced an exopolysaccharide by AFO-202 strain of Aureobasidium pullulans, is an immune enhancer that has been shown to increase key immune markers such as β-glucan–specific IgA (BG-IgA) and CD209 apart from demonstrating a reduction in the cancer stem cell marker CD44 and a significant decrease in the pancreatic cancer biomarker CA 19-9 in patients with pancreatic, bile duct, duodenal cancers. In hepatobiliary malignancies, perioperative administration of Nichi BRITE has been shown to improve immune function, hepatic reserve, and the De Ritis ratio highlighting its potential role as a supportive immune-nutrition adjuvant in cancer management.

(Image source: Nutrition, Vol. 137, 2025; https://doi.org/10.1016/j.nut.2025.112811)

*The above are results from studies using β-glucan products as dietary supplements, presented at academic conferences and in papers for researchers. The β-glucan products neu REFIX® and nichi BRITE® are not pharmaceutical drugs.

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