Gene therapy of HGB

Gene therapy has cured people with a rare blood disease

Svetlana Maslova, Hi-tech+

Treatment is based on the modification of their own stem cells with mutations that lead to the development of the disease.

An international team of scientists reported that six patients with a rare hereditary disease – X-linked chronic granulomatous disease – are in remission after a clinical trial of a new gene therapy. In this disease, the antimicrobial activity of phagocytes decreases in patients. Such people are very susceptible to infections from birth and get sick almost continuously.

In total, nine people participated in the study, six of them stopped other types of concomitant treatment, according to the website of the Center for the Study of Regenerative Medicine and Table Cells of the University of California at Los Angeles.

People with chronic granulomatous disease have mutations in one of the five genes that help white blood cells attack pathogens. X-linked CGB is the most common form of the disease. The only treatment option for CGB remains bone marrow cell transplantation from healthy donors. At the same time, it is very difficult to find a compatible donor, and when transplanting such cells, there is a high risk of their rejection by the body.

A new method of gene therapy uses the own hematopoietic table cells of patients with CGB and corrects mutations in them.

Previously collected cells with the mutation are modified in the laboratory, and then returned to the patient.

The treatment was tested on nine patients aged 2 to 27 years. Remission was achieved by seven patients, six of whom completely stopped taking prophylactic antibiotics. Two patients died due to an infection that developed in them before the start of gene therapy, the authors said.

On the left – white blood cells (red) of one of the participants in the clinical trial before gene therapy. On the right – after gene therapy, the leukocytes of the same patient show the presence of chemicals (blue) necessary to attack and destroy bacteria and fungi. A drawing from the UCLA – VM press release.

Scientists have not recorded complications after treatment. The team is now planning a larger clinical trial. Confirmation of the effectiveness of experimental therapy will soon help to obtain approval of the method for implementation into medical practice, the authors hope.

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