June 9, 2016.
The European Medicines Agency (EMA) has cleared the pharmaceutical company GlaxoSmithKline to sell Strimvelis, a medicine for children with alymphocytosis, a genetic immune disease. A great first.
Therapy for “bubble babies”
The first gene therapy for children, Strimvelis, developed by Italian scientists and the pharmaceutical company GlaxoSmithKline, has received its Marketing Authorization (Marketing Authorization) by the European Medicines Agency.
This therapy, based on stem cells, will be delivered to children who suffer from a rare disease of the immune system. : severe combined immunodeficiency by adenosine deaminase deficiency (ADA-SCID), for which no matching bone marrow donor is available. About 15 children are born each year with this disease and rare are those who survive beyond 2 years, despite being locked in a bubble for their immune protection.
Treat other rare diseases in the future
Concretely, how does this treatment work? Stem cells are taken from the child’s spinal cord, the DNA in these cells is changed and injected back. The missing protein, adenosine deaminase, can then be expressed and fulfill its role of immune protection. The 12 children who were able to benefit from this treatment are still alive 3 years later.
” We are at the start of a new chapter in the treatment of rare genetic diseases, and we hope that this therapeutic approach could be used to treat other rare diseases in the future », Said Martin Andrew, head of the rare diseases unit at GlaxoSmithKline, in the columns of the Daily Mail.
Currently, only one center is authorized to manufacture Strimvelis and it is in Milan.
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