Gene therapy for joints

The domestic development of a unique drug can become a revolution in the treatment of autoimmune diseases

Minister of Health of the Russian Federation Veronika Skvortsova presented information about the development of a unique Russian medicinal product.

"As part of the work of the scientific and educational cluster of directed innovative development of the Ministry of Health of the Russian Federation, Russian scientists have managed to achieve significant success in the development of a new generation genetically engineered drug for the treatment of autoimmune diseases. The research team of the N.I. Pirogov RNIMU under the leadership of the outstanding scientist Academician of the Russian Academy of Sciences Sergey Lukyanov and Doctor of Biological Sciences Dmitry Chudakov has created a unique drug that has no analogues in the world for the treatment of ankylosing spondylitis (Bekhterev's disease) and psoriatic arthritis. The revolutionary domestic development based on the most modern bioinformatic, genetically engineered technologies has a targeted effect only on the pathological clone of cells of the immune system, which includes cells that cause autoimmune damage, while significantly not depressing the overall immune background."

The stage of preclinical studies has now been completed, during the tests the drug showed high efficacy and safety not only on models of laboratory small animals, but also on primates (monkeys).

The new domestic drug has a number of serious advantages over the anti-cytokine monoclonal antibodies used today. It provides not only relief of the systemic inflammatory reaction, but also eliminates the root cause of the disease for a long time. It is important that there is no addiction to the drug and its effectiveness does not decrease during use. The expected frequency of use of a new drug is once every 3-5 years (existing drugs need to be used weekly or monthly).

As a result, the drug stops the progression of the disease in animals, leads to a regression of the processes of destruction of joints, prevents disruption of joint functions during its use. Without reducing the overall immune background, the drug does not lead to the development of tuberculosis and other immunosuppressive complications.

According to Veronika Skvortsova, "the data obtained will allow us to proceed to clinical trials of the drug this year, and if its effectiveness is confirmed in patients, it will be a world-class breakthrough in the treatment of autoimmune diseases of various origins. The medicine will be a salvation for millions of people suffering from Ankylosing spondylitis and psoriatic arthritis (10 to 15 million people in the world need such treatment today). Scientists of the N.I. Pirogov RNIMU have filed applications for patents, including international ones, fixing the priority of the Russian Federation."

Successful preclinical trials of the drug made it possible to connect one of the domestic manufacturers of innovative medicines to the development of experimental batches, which will make it possible to quickly begin conducting its clinical trials and subsequently begin its release as soon as possible after state registration. Clinical trials of the first phase are scheduled for autumn 2019.

Reference Autoimmune diseases (type 1 diabetes mellitus, psoriasis, ankylosing spondylitis, rheumatoid arthritis, etc.) are a significant problem for healthcare today.
Unfortunately, the existing methods of treating such diseases are aimed, in fact, at eliminating the consequences, and not the causes of the attack of the immune system cells on the body's own cells. Immunotherapeutic drugs that have recently appeared, acting on mediators of the autoimmune response, also do not eliminate the cause of the disease, when the cells of the immune system mistakenly recognize as "foreign" certain types of cells of their own body. In addition, such drugs require periodic administration and, unfortunately, lose their effectiveness over time, while acting systematically, they cause a general weakening of immunity. Therefore, the treatment, as a result of which the very cause of the disease will be eliminated, will become truly revolutionary, while allowing you to avoid general immunosuppression due to its targeted action.

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