Gene therapy of leprosy

Modified lymphocytes cured mice of leprosy

Sergey Vasiliev, N+1

The ancient companion of man leprosy (leprosy) still remains difficult to treat, and in the later stages leads to severe complications and disability. The disease is caused by mycobacteria that affect the skin, peripheral nerves, upper respiratory tract, feet and hands. Hundreds of thousands of people suffer from it every year – mainly in South America, Africa, Southeast Asia, and occasionally cases of leprosy are registered in Russia.

A way to completely get rid of the disease is offered by chimeric antigen receptor therapy (CAR) – a complex but surprisingly promising technology that promises to cure even oncological and autoimmune diseases. CAR-T therapy is already being used to treat some types of leukemia and lymphoma. At the same time, the patient receives artificially synthesized antigens that regulate the work of his T-lymphocytes, forcing them to attack cancer cells or B-lymphocytes that cause an autoimmune reaction.

Inhibition of T-lymphocyte activity against the background of excessive B-cell activity also occurs in patients with leprosy. At the same time, the usual means that stimulate the destruction of B-lymphocytes do not work for such patients. Therefore, Marko Radic and his colleagues from the University of Tennessee turned to CAR-T, testing the technology on experimental mice infected with leprosy. Scientists write about this in an article published in the journal Science Translational Medicine.

To begin with, the animals underwent harsh radiation treatment, which destroyed their "old" T cells (sick people also undergo radiotherapy before CAR-T). Then they were injected with modified T-lymphocytes: in more than 60 percent of mice, they completely or almost completely destroyed hyperactivated B-lymphocytes, bringing the immune system closer to normal.

According to scientists, the skin, kidneys, pancreas and other parts of the animal's body were completely cleansed and showed no signs of the disease. Almost all of them then lived for more than a year – a sufficient period for small rodents – while the control group mice that did not undergo CAR-T treatment died no later than 8-10 months.

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