According to a story from The Motley Fool, The gene therapy development company bluebird bio has been working on a treatment for beta thalassemia, and it has the potential to dramatically improve the quality of life for patients with the condition. The drug is called LentiGlobin, and if it gains approval, it will be the first commercially successful venture for the company.
Beta thalassemia refers to a group of inherited blood disorders. The condition appears when the synthesis of beta chains in hemoglobin is either absent or significantly reduced. In some individuals, the condition does not cause major symptoms, but in some cases it can lead to severe anemia. The condition is caused be a genetic mutation. Symptoms of thalassemia include spleen issues (which often results in its removal), gallstones, poor growth, and skeletal abnormalities. When left untreated, beta thalassemia lead to death from heart failure. Family history of the condition is the primary risk factor; people from Italy, Greece, The Middle East, South Asia, and Africa have a higher likelihood of beta thalassemia. In most patients, the only resort for treatment is periodic blood transfusions in order to compensate for blocked beta chains. Unfortunately, this can lead to its own deadly side effects, such as iron toxicity. Excessive iron can put both the heart and liver at risk of damage, with cirrhosis and liver cancer being potential complications. To learn more about beta thalassemia, click here.
Clearly, there is a great need for more options so that getting treatment for beta thalassemia does not put the patient at risk for other severe diseases. As a genetically linked condition, gene therapies like LentiGlobin are a logical option for new therapies. The treatment works by replacing or supplementing the dysfunctional or absent beta chains that cause the beta thalassemia. In 12 of 13 patients with reduced hemoglobin, a single infusion of LentiGlobin allowed them to go around 27 months without a blood transfusion. Patients with that did not produce any hemoglobin also experienced benefit.