Severe Combined Immunodeficiency (ADA-SCID) is a rare genetic disorder that affects around 15 patients per year in Europe.
It is caused by a faulty gene which leaves the body defenseless against viruses and bacteria, thus making it extremely fatal.
Now a special gene therapy treatment called Strimvelis promises to completely remove this disease by altering the genetic makeup of the patient itself.
Under this treatment, the doctors remove the stem cells from a patient’s bone marrow. These cells are then treated in a test tube to replace the faulty gene, essentially altering the basic building blocks of life.
These treated cells are then reintroduced into the body where the restart the production of the Adenosine Deaminase (ADA) Protein, which is required to produce white blood cells called lymphocytes.
The treatment has so far been tested in clinical trails, where it was 100% effective as a permanent solution for the disease.
By approving this gene therapy treatment for children, the European Commission has created a landmark moment for scientific research and treatment.
This will become a great precedent for encouraging future research into other life threatening diseases and possible genetic treatments, thereby saving millions of lives.