MitoQ is a neuroprotective anti-oxidant targeted against oxidative damage in the mitochondria of living cells. It is uniquely targeted against oxidative stress developed in the mitochondria as a result of a plethora of metabolic reactions that go on within these organelles. This molecule is the first-of-its kind to be formulated for this purpose. According to latest research, oxidative stress and mitochondrial dysfunction contribute vitally to pathogenesis of Multiple Sclerosis (MS) in patients. This is mainly due to wear and tear of nerve fibres and myelin sheath as a result of free radicals released as metabolic by-products. Hence, the drug is currently being subjected to clinical development for treatment of patients with MS.
The protective effect of anti-oxidants on the cellular damage caused by free radicals has been a proven fact, and this is the main principle behind formulation of this drug. Previous formulations of the same class have been useful in managing conditions like Alzheimer’s Disease, Parkinson’s Disease and other neurodegenerative disorders. Preclinical trials involving groups of mice with experimental autoimmune encephalomyelitis (EAE) have been successfully demonstrated to show the possible neuroprotective effect exerted by MitoQ on MS. Studies have shown that mice who were pre-treated with MitoQ before contracting EAE showed the best results, followed by those who had been given the drug after getting EAE. Both sets showed not only an improvement in neuronal activity but also a considerable reduction in inflammatory markers, showing signs of demyelination as well. Hence its potential to not only reduce but also reverse signs and symptoms of MS have lead to development of human trials to test its pharmacokinetic, pharmacodynamic and toxicity profiles in human patients with the disease.
So far, trials to demonstrate the efficacy of the drug have been executed at the Oregon Health and Science University, with similar trials based on different anti-oxidants being carried out at the Johns Hopkins University as well. Results of human trials would pave the way for a better understanding of the drug’s role in neuroprotection and immunomodulation in MS. However, human trials (phase I and phase II) have been carried out for diseases like Parkinson’s Disease and Hepatitis C (both by New Zealand based Antipodean Pharmaceuticals) and the oral formulation has been declared safe to be used in humans.
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