A new experimental therapy for a rare blood disorder is showing tremendous promise as a treatment.
According to a Forbes report the new gene therapy from Bluebird Bio has helped two patients suffering from beta-thalassemia. The disorder requires blood transfusions as treatment. The gene therapy eliminated those transfusions within a 12-day period of being administered.
"Thalessemia major patients are very sick," Marina Cavazzana, M.D., Ph.D., of Paris Descartes University, France told Forbes. "They have to receive transfusions regularly for all of their life. Because of this huge number of transfusions they suffer from a huge iron overload. To set up a treatment that can make them free of any transfusion treatment should be a great step toward curative treatment."
Bluebird's treatment is known as LentiGlobin and the company is assessing it as a potential treatment for sickle cell anemia as well.
LentiGlobin inserts a fully functional human beta-globin gene into patient stem cells. Bluebird treated its first patient with Lenti-D in 2010 and initiated a phase 1-2 study in the U.S. back in March.
This is not the first time the treatment has been reviewed as a potential medical option. A study in the journal Nature four years ago revealed that the treatment helped one patient avoid blood transfusions for six years.
According to Bluebird there are 288,000 patients diagnosed with beta-thalassemia and 15,000 live either in Europe or the United States and Europe. The disorder is a genetic disease that impacts the beta chain of hemoglobin.
According to Forbes Bluebird is planning to add another five patients to its study trial as part of its effort to get the treatment approved by the U.S. Food and Drug Administration.
"If we continue to see patients who look like this, then you're going to have a conversation with the agency," Nick Leschly, Bluebird's chief executive, told Forbes.
