The U.S. Food and Drug Administration last week approved a genetic treatment for the blood disorder beta-thalassemia, marking the third U.S. gene therapy for a rare disease. www.science.org/content/article/news-glance-new-gene-therapy-europe-s-drought-and-black-hole-s-photon-ring?
The disorder causes low haemoglobin and severe anaemia, and the regular blood transfusions used to treat it can cause iron build-up that damages organs. The new treatment, Zynteglo, from manufacturer Bluebird Bio, relies on a virus to deliver a gene for haemoglobin into the patient’s bone marrow cells, grown in culture; the cells are then infused back into the body. In clinical trials, 89% of treated patients no longer required transfusions. Zynteglo won European approval in 2019 but was removed from the market after countries baulked over the high price; in the United States, it will cost $2.8 million per one-time treatment, making it one of the most expensive drugs ever. Bluebird is testing a different product that uses the same method for sickle cell anaemia, which is more common in the United States than thalassemia.
Lalita Panicker is consulting editor, views, Hindustan Times, New Delhi