According to a story from empr.com, a recent study revealed that gene therapy could allow patients with beta thalessemia to stop having to get regular blood transfusions. The gene therapy that was used to treat these patients is called LentiGlobin. The positive results came from Phase 1 and Phase 2 studies of the investigational therapy. If approved, LentiGlobin could significantly improve the quality of life of beta thalessemia patients.
Thalessemias are a group of rare blood disorders. Of the two main types, alpha and beta, beta thalessemia is the less common. Beta thalessemia causes abnormalities in the creation of hemoglobin, the substance in blood that transports oxygen. Beta thalessemia is caused by a genetic mutation; the severity of the disorder varies greatly. Some people may experience no symptoms, whereas others may have severe anemia, or a lack of blood cells. Symptoms include poor growth and skeletal abnormalities; patients are also vulnerable to spleen complications, which is often removed. They are also more likely to get gallstones. Severe untreated beta thalessemia can be lethal, causing heart failure. Patients may require frequent blood transfusions, which can cause significant issues such as iron toxicity. To learn more about beta thalessemia, click here.
In the study a total of 22 patients were involved. After a one time treatment with LentiGlobin, only seven patients still had to receive blood transfusions. The majority of those that had to continue receiving transfusions were able to reduce the frequency of transfusions and the volume of blood that was necessary. These patients were monitored in the study for period of no less than two years.