The European Medicines Agency (EMEA) authorized the use of a revolutionary product for the treatment of severe combined immunodeficiency - the very "disease of the boy from the bladder", which gained fame due to the sad history of David Vetter (1971-1984).

A unique product called Strimvelis is intended for the treatment of severe combined immunodeficiency associated with adenosine deaminase deficiency (ADA-SCID).

Therapy involves removing the patient's stem cells and reprogramming the genetic defect that causes the deficiency of the enzyme.

Recall that ADA-SCID - is the most severe genetic disease of the immune system, in which a person disrupts the production of adenosine deaminase. As a result, a toxic substance deoxyadenosine accumulates, which destroys the lymphocyte cells and reduces resistance to infections to zero.

In addition to the fact that sick children have problems with growth and development, their hair falls out, the work of the liver and kidneys is disrupted; children with ADA-SCID usually die in the first two years of life because of severe infections and cancers.

For today in Europe and America for such patients there is one radical option of treatment - bone marrow transplantation. However, only 1 out of 4 children can find a completely matching bone marrow donor, and in the event of a mismatch, tragic consequences are possible.

In an interview with the Wall Street Journal, Dr. Bobby Gaspar, a professor of pediatrics and immunology from England, said that the introduction of a new product for such a rare and severe disease is a significant achievement that will save hundreds of children who are currently without adequate treatment.

"If the Strimvelis product is approved in the EU, it will be the world's first ex-vivo gene therapy (" outside the living body ") in children who have received official authorization," said Martin Andrews, Department of Rare Diseases of the pharmaceutical company GlaxoSmithKline.

Treatment with Strimvelis is only available in Milan, where this product was developed. First, doctors take stem cells from the bone marrow of the patient, insert a normal copy of the adenosine deaminase gene into their DNA, and inject them back to the child.

Before the injection, the child undergoes low-dose chemotherapy, so that the body "better" accepts new cells.

The results of treatment Strimvelis can not but rejoice: 100% of children have had a good treatment, and 92% (11 of 12) have been living for three years without signs of immunodeficiency.

After receiving the historic EMEA solution, treatment will be available to all children in Europe, although its long-term effectiveness and side effects will have to be closely monitored for many years.

medbe. en.

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