05 April 2016

In the EU, TKID gene therapy was allowed

In Europe, gene therapy for children was allowed

Ekaterina Makarova, Vademecum

The European Medicines Agency (EMA) has approved the use of gene therapy for the treatment of children with severe combined immunodeficiency (ADA-TKID), for whom it was not possible to find a suitable bone marrow donor (New gene therapy for the treatment of children with ultra-rare immune disorder recommended for approval – VM).

Every year in Europe, about 15 children are born with the diagnosis of "ADA-TKID". The disease is a consequence of a genetic mutation that leads to disorders of the enzyme adenosine deaminase. Enzyme deficiency leads to disorders of the thymus (thymus gland), which is responsible for the development of cells of the immune system, in particular, lymphocytes. Children with ADA-TKID are practically immune and often die from any infection in the first year of their life. It is no coincidence that such patients are called "children in a bubble": only a sterile environment can guarantee their safe existence.

David Vetter, the most famous “bubble-boy", lived in a sterile bubble for 12 years – VM.

The solution to the problem for patients with AD-TKID today is bone marrow transplantation, but it is not always possible to find the necessary donor.

Strimvelis gene therapy, developed by GlaxoSmithKline, is designed for such cases, according to Reuters. Strimvelis is a stem cell gene therapy that is created for each individual patient based on his own cells. The principle of such therapy is as follows: a normal copy of the adenosine deaminase gene is inserted into the DNA of previously removed affected bone marrow stem cells of the patient, and then the corrected cells are injected back into the child's body. In order for the genetically modified cells to take root better, he undergoes low-dose chemotherapy before the procedure.

In the future, GSK plans to obtain a license to use gene therapy for the treatment of other types of severe combined immunodeficiency, including metachromatic leukodystrophy and Wiskott–Aldrich syndrome.

Gene therapy is gaining momentum all over the world. In mid-June last year, Bluebird Bio announced the successful implementation of gene therapy for a French teenager with a severe hereditary blood disease.

Portal "Eternal youth" http://vechnayamolodost.ru  05.04.2016

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