MOSCOW , 1 Mar. Scientists of the National Research University «BelSU» as part of an international scientific team have shown the effectiveness of the treatment of amyotrophic lateral sclerosis (ALS) using a bioavailable derivative of vitamin B1. Experiments on transgenic mice confirmed that the treatment reduces the level of inflammatory signaling molecules in the spinal cord. The results are published in Biomedicine & Pharmacotherapy.
Amyotrophic lateral sclerosis is a fatal neurological disorder characterized by rapid degeneration of motor neurons. The most famous patient suffering from this disease was the British physicist Stephen Hawking.
The frequency of occurrence of this disease, according to WHO, is 2-5 per 100 thousand people, that is, in Russia there are supposedly about 7.5 thousand such patients. There are hereditary forms of the disease, when offspring inherit a «broken» gene from their parents. There is also a non-hereditary form of the disease, in which the risk of developing it increases with age and, according to some estimates, is 1/300 (the highest risk in men, military men, smokers, handball athletes).
Researchers at the Belgorod State National Research University (NRU «BelGU») said that the drugs that are currently used to treat ALS prolong the life of patients by only two to three months.
As part of an international team of scientists, they conducted research on an animal model of amyotrophic lateral sclerosis (FUS) transgenic mice created in Russia. The fact is that these mice develop symptoms of the disease, like humans, and without treatment they die of paralysis at the age of four months. The model created by scientists allows us to study promising drugs without injuring patients.
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“We have found that the studied bioavailable derivative of vitamin B1 (O,S-dibenzoylthiamine), which is a powerful antioxidant, showed efficacy against ALS in transgenic mice. Greater mobility, less weight loss, less brain damage were shown in treated mice,” said Aleksey Deikin, Associate Professor of the Department of Pharmacology and Clinical Pharmacology of the National Research University “BelSU”, Director of the Joint Center for Genetic Technologies.
The scientists also found that when the test substance was used, the body reacted at the molecular level — in the spinal cord, the level of inflammatory signaling molecules (glycogen synthase kinase-3β (GSK-3β) and interleukin IL-1β) decreased. A nuclear magnetic resonance study showed a specific metabolome of transgenic mice, which, according to the researchers, makes it possible to track the dynamics of the development of the disease.
According to the scientists of the National Research University «BelSU», their work paves the way for the search for new approaches for the diagnosis and treatment of amyotrophic lateral sclerosis .
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As Aleksey Deikin said, the work of the international team would not have been possible without the participation of Sechenov University Professor Tatiana Strekalova. cooperation with the University of Oxford, the First Moscow State Medical University. Sechenov, the Institute of General Pathology and Pathophysiology, the University of Liege, the Institute of Physiologically Active Compounds of the Russian Academy of Sciences and the University of Maastricht.
The work is carried out within the framework of the Priority 2030 development program, the Gene Therapy Clinic project and grant within the framework of the FSTP for the development of genetic technologies on the topic «Development of genetic modeling technologies in the field of biomedical research and gene therapy for neuromuscular diseases».