Gene therapy first approved for the treatment of diseases
Last reviewed: 23.04.2024
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In the medical history of Europe, an epoch-making event is about to occur - the first approval of the practical application of gene therapy for the treatment of hereditary disease.
The European Medical Agency recommended the use of this method for the treatment of a rare genetic disease that deprives patients of the ability to metabolize fats.
Based on this recommendation, the European Commission must make a final decision on the admissibility of this method of treatment.
The principle of gene therapy is simple: if a defect is found in some part of the human genetic code, it is replaced with a gene material with the desired properties created in the laboratory.
However, in reality, not everything is so simple. In clinical trials, the teenager Jesse Gelsinger died in the US, and other patients became ill with leukemia.
Currently, neither in Europe, nor in the US gene therapy methods are not applied.
The first gene drug
The Committee for Medical Products at the European Medical Commission reviewed the use of the drug Glybera for the treatment of Buerger-Grütz disease, a hereditary lipoprotein lipase deficiency.
This syndrome occurs in one of a million people. Patients have a damaged gene structure, which is responsible for the breakdown of fats in the digestive tract.
This leads to the accumulation of fats in the blood, inguinal pains and acute pancreatitis.
The only way to alleviate the condition of such patients so far was the strictest fat-free diet.
The new treatment is based on the use of a virus that infects muscle tissue and injects a copy of the intact gene into the body.
It is recommended for patients who suffer from acute inflammation of the pancreas, pancreatitis, and who do not respond to the diet.
Replacement of a defective gene
The manufacturer of the new drug, pharmaceutical company UniQure, said that this solution is a landmark event for patients and for medicine in general.
The head of the company, Jorn Aldag, said: "Patients with lipoprotein lipase deficiency are afraid to eat normal food, because this can lead to acute and extremely painful inflammation of the pancreas, which often results in hospitalization."
"Now - for the first time in history - a treatment method has been developed that not only reduces the risk of inflammation, but also has a long-term beneficial effect on the patient's condition," he added.
China became the first country in the world to officially authorize the use of gene therapy.
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