Successful clinical response achieved in beta-thalassemia patient with single treatment of Bluebird Bio's lentiviral gene therapy
Bluebird bio ( formerly Genetix Pharmaceuticals ), an emerging leader in the development of innovative gene therapies for severe genetic disorders, has announced publication of its promising phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.
The patient, who had been transfusion dependent since early childhood, has become transfusion independent for the past 21 months, more than two years after treatment with the LentiGlobin vector.
The study also identified a subset of cells with the corrected beta-globin gene that overexpressed a truncated form of a gene called HMGA2. The patient has not experienced any adverse events.
The data show that while early on, the HMGA2 clone was a significant portion of the corrected cells, the clone levels had declined at the time the paper was prepared, and further follow up indicates the decline is continuing.
Although based on the first treated patient, these results are impressive and illustrate for the first time the significant potential for treatment of beta hemoglobinopathies using lentiviral beta-globin gene transfer in the context of autologous stem cell transplant. For beta-thalassemia, the researchers have worked intensely for almost 20 years to design, develop and manufacture LentiGlobin to provide a sustained high level hemoglobin production, resulting in a major clinical benefit.
For the first time, a patient with severe beta-thalassemia is living without the need for transfusions over a sustained period of time. These results are not only important due to the tremendous medical need that exists for thalassemia patients around the world, but also represents a significant step forward for the field of autologous stem cell therapy as an emerging therapeutic modality.
Source: Nature, 2010