Russia developing possible treatment for Tay–Sachs disease

Russia has officially announced that it has completed the development of a drug that could be recommended for the treatment of Tay–Sachs disease – a genetic disorder that results in the destruction of nerve cells in the brain and spinal cord and which, so far, has been considered as fatal.

According to developers, the new drug is based on altered stem cells, which help the body to produce the enzyme hexosaminidase A, which can stop the progression of the disease. To date, the drug has already been tested on laboratory animals, during which it has shown high efficacy and safety. Clinical trials will begin soon.

According to Albert Rizvanov, director of the Scientific and Clinical Center for Precision and Regenerative Medicine of the Kazan Federal University - a research center, which is the main developer of the new drug - during their work scientists added a special gene into stem cells taken from the fat tissue of the patent. That was carried out with the help of a carrier virus. As a result, cells received the ability to produce the enzyme hexosaminidase A, which can stop the progression of the disease.