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A medical breakthrough: How does gene therapy change the fate of patients with β-thalassemia?
Beta thalassemia is a hereditary blood disease. Due to the reduced or missing synthesis of beta globulin, patients have different manifestations ranging from severe anemia to asymptomatic. The global annual incidence rate is about 1 per 100,000 people. The condition affects millions of people around the world, with many patients requiring regular blood transfusions to maintain adequate hemoglobin levels and the resulting iron overload, which puts them at greater risk to their health.
In patients with beta thalassemia, the body cannot produce new beta globin chains, resulting in insufficient production of adult hemoglobin.
In beta thalassemia, the severity of the condition depends on the nature of the genetic mutation. There are three main forms of β-thalassemia: β-thalassemia minor, β-thalassemia intermedia, and β-thalassemia major. Severe cases usually develop symptoms of severe anemia soon after birth, while mild cases often go unnoticed due to subtle blood changes. These different disease types therefore have different treatment needs and health risks.
Due to the complexity of the disease, a confirmed diagnosis often requires adequate genetic testing to understand which variants affect the patient's health. With the advancement of science and technology, gene therapy for β-thalassemia is gradually showing new hope. The core idea of gene therapy is to solve the problem of hemoglobin synthesis from the source by correcting the disease-causing mutations.
Scientists at Weill Cornell Medical College have developed a gene therapy strategy that may treat both beta thalassemia and sickle cell disease.
In June 2022, a U.S. federal advisory panel recommended that the U.S. Food and Drug Administration (FDA) approve a gene therapy program called Zynteglo, developed by Bluebird Bio and costing up to $2.8 million. This therapy marks a milestone in the development of gene therapy. Advances in commercialization of treatments. Not only that, with the development of CRISPR technology, Vertex Pharmaceuticals and CRISPR Therapeutics have also launched the gene editing therapy Exagamglogene autotemcel. This therapy is engineered using the patient's own hematopoietic stem cells and has shown significant efficacy in the treatment of beta thalassemia.
However, the promotion of these treatment options also faces a series of challenges, including high financial requirements, patient acceptance, and health insurance coverage. Whether gene therapy can be a savior for patients with β-thalassemia still requires further clinical trials and long-term observation.
Will the development of gene therapy make beta thalassemia a thing of the past?
Objectively speaking, many patients with beta thalassemia still undergo conventional treatments, such as blood transfusions and iron chelation therapy, in the hope of reducing the complications of iron overload. Even so, the emergence of gene therapy provides these patients with new treatment options and hope. With in-depth research on genetic technology and support from relevant policies, more treatment options for β-thalassemia may emerge in the future.
So, for these patients, how to choose the most appropriate treatment plan under the existing medical system has become a new challenge they face?
