British Approval of Gene Therapy for Blood Disorders Sparks Hope for Patients
Three years after being awarded the Nobel Prize for its development, gene therapy technology has taken a major step forward. The UK has become the first country to approve the use of gene scissors, also known as crispr technology, for the treatment of two serious blood disorders.
The groundbreaking gene therapy involves a process where patients are first given chemotherapy, followed by the extraction of stem cells from their bone marrow. These cells are then treated in a laboratory using crispr technology before being returned to the patient.
The treatment has been approved for patients over twelve years of age with sickle cell anemia and thalassemia. Both of these diseases are currently treated with complicated blood transfusions or bone marrow transplants, which come with severe side effects.
Helen O’Neill, a geneticist at University College London, expressed excitement about the approval, stating that the word ‘cure’ has so far been incompatible with sickle cell anemia and thalassemia. She believes that the approval represents a “historic, positive moment in history,” and predicts that the future of life-changing medicines lies in crispr-based technology.
The approval for sickle cell anemia is based on a study involving 29 patients, where 28 of them had no serious problems from the disease one year after the treatment. For thalassemia, 39 of the 42 patients in the study did not require any transfusions post-treatment.
Sickle cell disease is an inherited condition that leads to changes in the protein hemoglobin, resulting in deformed blood cells, increased blood viscosity, and various complications such as impaired blood flow, infections, kidney damage, and anemia. The disease affects an estimated 52,000 people in Europe.
Thalassemia, on the other hand, is a hereditary disease that leads to a lack of hemoglobin, causing anemia. In Sweden alone, there are approximately 100 people with the severe form of thalassemia that requires blood transfusions.
The approval of gene therapy for these blood disorders offers hope for patients and represents a major step forward in the treatment of these conditions.
Source: National Board of Health and Welfare