New Research Published
New research published on potential mannosamine therapy for HIBM (GNE myopathy) and kidney glomerular disease
“Oral monosaccharide therapies to reverse renal and muscle hyposialylation in a mouse model of GNE myopathy”, T. K. Niethamer, et al. Molecular Genetics and Metabolism, 2012, http://dx.doi.org/10.1016/j.ymgme.2012.10.011.
This recent publication provides new information on the prospects of using N-acetylmannosamine (ManNAc, DEX-M74 ) and its derivative, mannosamine hydrochloride, to treat the adult onset muscular dystrophy HIBM (hereditary inclusion body myopathy), also called GNE myopathy and possibly certain kidney glomerular diseases.
The scientists demonstrated that mannosamine hydrochloride, but not sialic acid, mannose, galactose or glucosamine administered to pregnant mice had a similar prophylactic effect on renal hyposialylation, pathology and neonatal survival of mutant off-spring, as previously shown for DEX-M74 therapy. Because GNE myopathy patients will likely require treatment in adulthood after onset of symptoms, the scientists also demonstrated that oral administration of DEX-M74 to adult mutant GNE myopathy mice markedly improved the muscle and renal hyposialylation. These preclinical data strongly support further evaluation of oral DEX-M74 as therapy for GNE myopathy and conceivably for certain glomerular diseases with hyposialylation.
The U.S. National Institutes of Health (NIH) is currently conducting a Phase I clinical trial of DEX-M74 (ManNAc) in patients with GNE myopathy (HIBM). This study is a collaboration between two NIH Institutes, the National Human Genome Research Institute (NHGRI) and the NIH Center for Advancing Translational Sciences (NCATS)’s Therapeutics for Rare and Neglected Diseases (TRND) program along with New Zealand Pharmaceuticals Ltd (NZP). The purpose of this clinical trial is to study the safety of ManNAc and to understand how the body handles it to determine dosing schedules. Future studies to test the efficacy of ManNAc are being planned.
For more information about this study visit: www.clinicaltrials.gov (NCT01634750) or the NIH Clinical Center website: http://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2012-HG-0207.html.