The UK is the first in the world to approve a revolutionary method and authorize gene therapy for the treatment of sickle cell anemia and β-thalassemia in patients over 12 years of age.
State like reports The MHRA, the UK’s Medicines and Healthcare Products Regulatory Agency, has officially approved this treatment. Genetic diseases are caused by errors in the hemoglobin genes, which red blood cells use to carry oxygen throughout the body. Julian Beach, acting director of the MHRA, explains:
“Both sickle cell disease and β-thalassemia are serious lifelong diseases that can even be fatal in some cases.”
According to the publication, the British regulatory body has approved an innovative treatment for these diseases with the drug Casgevy, which works by “editing” the gene that causes these ailments. The drug became the world’s first licensed drug using a gene editing tool. In 2020, its inventors won the Nobel Prize.
Clinical trials have shown that Casgevy restores healthy hemoglobin production in most participants with sickle cell disease and transfusion-dependent β-thalassemia, relieving symptoms of the disease, Beach noted. However, during the tests no serious problems with the safety of the drug were found.
Scientists who study genetic diseases hope that Casgevy will eliminate the need for bone marrow transplants for people with these diseases. Previously, the only possible treatment was such transplants, even though the body could reject the donor bone marrow. Patients also require regular blood transfusions to treat anemia.
The Guardian recalls that in the UK there are about 15,000 patients with sickle cell anemia, and almost 1,000 patients have β-thalassemia.
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