A new treatment developed by Bluebird Bio could significantly improve the health and quality of life for people who suffer from beta-thalassemia, a rare genetic blood disorder.
The genetic disorder is caused by faulty links in the beta chain of hemoglobin, which transports oxygen throughout the body. People who suffer from beta-thalassemia have to regularly receive blood transfusions for their entire lives.
However, two patients who were given the experimental gene therapy treatment no longer needed blood transfusions twelve days after starting treatment. In 2010, a patient was successfully treated with a similar treatment, but it had taken longer to be effective, and the patient had needed blood transfusions for the first year after starting treatment.
Bluebird Bio's experimental new treatment uses lentivirus, a modified HIV virus to replace the faulty beta globulin gene in stem cells. A doctor who was involved in researching the treatment says that it has relatively few side effects.
