Gene-cell therapy
Diseases of the nervous system will be treated with modified blood cells
RNF Press Service
Scientists of Kazan State Medical University have created a drug based on genetically modified umbilical cord blood cells. In the future, it can be used to treat neurodegenerative diseases, including amyotrophic lateral sclerosis, ischemic stroke and neurotrauma. Research supported by a grant The Russian Science Foundation. The scientists' article was published in the journal Blood (Bashirov et al., Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy).
Neurodegenerative diseases are progressive diseases associated with the gradual death of nerve cells. These include, for example, Parkinson's and Alzheimer's diseases and amyotrophic lateral sclerosis, which the famous astrophysicist Stephen Hawking suffered from. Mass death of neurons also occurs after an ischemic stroke (due to insufficient blood supply) and neurotrauma. Patients who are diagnosed with such diseases receive treatment aimed at eliminating only the symptoms of the disease, but not its consequences.
Today, scientists consider the use of gene-cell technologies to be the most promising solution to the problem. They consist in various modifications of stem cells and embedding in them artificial DNA molecules that encode proteins that stimulate brain regeneration.
Scientists of Kazan State Medical University together with colleagues from the Scientific Center of Neurology and the N.F. Gamalei Research Center of Epidemiology and Microbiology have created a gene-cell drug and conducted its preclinical tests on rodents and mini-pigs.
The basis of the new drug was made up of umbilical cord blood cells. In them, scientists have introduced modified DNA molecules of viruses carrying human genes. In the area of degenerative changes in the brain, genetically modified umbilical cord blood cells are responsible for activating processes that prevent the death of neurons and for restoring their functions.
To test the effectiveness of the drug, scientists conducted preclinical tests on three different biological models: amyotrophic lateral sclerosis in mice, spinal cord contusion injury in rats and mini-pigs, and ischemic stroke in rats. It turned out that genetically modified umbilical cord blood cells can successfully transfer from the blood to the brain, reach the affected area and activate regeneration processes. So, when the drug was administered to a mini-pig with a contusion injury of the spinal cord, scientists noticed that the previously paralyzed hind limbs of the animal began to move.
"The need to develop technologies for obtaining genetically engineered medicines is due to several factors. Firstly, research in the field of gene-cell technologies for practical medicine abroad in most cases has not been brought to industrial production. Secondly, the technologies and know-how completed in this area will be extremely expensive. According to our calculations, in the long term, their cost will be significantly higher than the costs that are necessary today for the formation and development of this direction in Of Russia. We have already created the first domestic gene-cell drug for the treatment of neurodegenerative diseases and have tested it on animals. This drug has shown high efficacy in inhibiting the death of neurons in neurodegenerative diseases, ischemic stroke and neurotrauma. No serious side effects were detected," one of the authors of the work, Doctor of Medical Sciences, Professor, Head of the Department of Medical Biology and Genetics of Kazan State Medical University Rustem Islamov sums up.
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