Chennai, July 11 (BPNS)
A team of doctors from Tamil Nadu, have developed a safe disease modifying treatment for Duchenne Muscular Dystrophy (DMD), jointly with scientists from Japan, using a novel immune modulating Beta 1,3-1,6 glucan (Neu-REFIX).
The results of their clinical trial published in IBRO Neuroscience Reports – the official journal of the International Brain Research Organization – has confirmed the safety and improvement of muscle strength in a short span of 45 days in those who consumed Neu-REFIX beta-glucans compared to the patients in the control group.
Dr. K. Raghavan, Dept of Paediatric Neurology, Jaicare Hospital, Madurai, said, “This is the first such treatment for DMD patients in the world that, without any side effects, offers improvement in the quality of life and increases life expectancy. This uses a food additive which increases the levels of dystrophin – a cytoplasmic protein which is responsible for muscle functions in the heart, limbs and blood vessels – in the blood to 32%. DMD patients have got very low levels of dystrophin and this new treatment addresses the disease at its fundamental level of muscle pathology.”
The collaboration was led by Dr K. Raghavan in India the Japanese side was headed by by Prof Nobunao Ikewaki (Kyushu Health and Welfare University, Miyazaki).The team of doctors from both the countries undertook basic research and pre-clinical studies in animal models using a novel Beta glucan produced as an exo-polysaccharide by N-163 strain of a yeast Aureobasidium Pullulans (Trade name: Neu REFIX) commercially available in Japan as a food additive.
Duchenne Muscular Dystrophy (DMD) is a rare genetic disease where the patient usually become wheelchair-bound by around the age of 12 due to muscle weakness and will then die prematurely. This affects 1 in 3500 male births worldwide and there are approximately 80,000 patients in India.
Disease-modifying treatment – the approach that slows down the progress of the disease – continues to be the main type of treatment for DMD. Two of the treatments – the Gene therapy that costs INR 26 crores and the Exon skipping therapies costing INR 1.2 to 2 crores – are not affordable for most people.
Prof. Nobunao Ikewaki, Dept. of Medical Life Science, Kyushu University of Health and Welfare, Japan, said, ”Our research showed the potential of Neu-REFIX in immune modulation and controlling the fibrosis which causes muscle weakness in DMD due to lack of dystrophin.
The collaboration included Dr Samuel JK Abraham, Centre for Advancing Clinical Research (CACR),University of Yamanashi – School of Medicine, Chuo, Japan and Head, R&D of GN Corporation, Japan as well as Prof Pushkala Subramaniam (Prof. of Immunology, TN MGR Medical Univ., Chennai).
“If myocardial fibrosis can also be controlled with Neu-REFIX, it could become a promising universal immune modulator and a drug adjuvant to many auto-immune and inflammatory diseases,” said Prof. Pushkala.
The research proved, in the MDX mice model, that NeuRefix beta-glucan can efficiently control inflammation, fibrosis and increased muscle strength. A longer duration human clinical study of six months has confirmed the efficacy in terms of improved muscle strength and walking capacity of patients with DMD as proven by six-minute walking test (6MWT), Medical Research Council (MRC) Grading and North Star Ambulatory Assessment (NSAA).