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Gene Therapy to reduce blood transfusions need in Thalassemia : NEJM
Beta-thalassemia is a genetic disorder characterized by reduced or negligible production of hemoglobin's beta chains. People suffering from severe form of beta-thalassemia require blood transfusions on a monthly basis for replenishment of their red blood cells supply.Professor Philippe Leboulch at Harvard Medical School and colleagues conducted a study for evaluating the effectiveness of gene therapy for beta-thalassemia patients. The researchers found that gene therapy is safe and effective for treatment of beta-thalassemia. Scientists say they are "excited" by the results of this trial for the inherited blood disorder beta-thalassaemia, which will reduce the need for blood transfusions.The study was published in The New England Journal of Medicine.
"It was always our hope to bring our research findings to patients," said co-corresponding author Leboulch, whose primary appointment has transitioned to the University of Paris as a Professor of medicine and Institute Director. "We have taken our work from the lab, through preclinical models, and past the proof-of-principle stage, and are now able to gauge its effectiveness in patients with this disease. It is immensely gratifying."
As a postdoctoral fellow at MIT, Leboulch began researching a therapeutic approach to compensate for the genetic mutations that lead to both sickle-cell disease and beta-thalassemia. Leboulch joined the Brigham's Division of Genetics in 1996, where he continued his work as an HMS associate professor in medicine to develop a viral carrier, or vector, that could insert genetic instructions into a patient's own blood stem cells and restore hemoglobin production. Leboulch and colleagues hoped that re-introducing the altered cells back into people would allow them to make enough hemoglobin, eliminating the need for blood transfusions. At the Brigham and HMS, Leboulch and colleagues studied the vector, known as "LentiGlobin," in pre-clinical models, publishing results from mouse studies in Science. In 2010, Leboulch and his collaborator, Marina Cavazzana of University Paris-Descartes, published a paper in Nature detailing the success of using Leboulch's LentiGlobin to genetically correct cells and transplant them back into a single beta-thalassemia patient. Last year, they published in the NEJM on a successful gene therapy of the first sickle-cell anemia patient using the same vector.
In the newly published NEJM study, Leboulch, Cavazzana, and their colleagues teamed up with the second group of U.S. and international clinical investigators in Australia and Thailand to share data and results from their respective phase II clinical trials. In total, the two teams treated 22 patients at six different sites around the world. Among nine patients with the most severe form of beta-thalassemia, the one-time treatment reduced the need for red-blood-cell transfusions by 73 percent. Three of the nine subsequently discontinued transfusions altogether. Twelve of the 13 patients with a slightly less severe form of the disease no longer needed any blood transfusions after treatment. The team reports no safety concerns - treatment-related adverse effects were typical of those seen in patients who receive cell transplants of their own stem cells.
"When you have an anecdote of a single patient, you never know if it will be confirmed. Here, with a multi-center trial in a larger number of patients, we see a convergence of results, and we can measure the magnitude of the therapeutic effect," said Leboulch. "There is room for improvement, as we'd like to see the elimination of dependency on transfusion even for patients with the most severe form of the disease; but there is also hope with protocol modifications we have introduced in our phase III trials."
Based on these results, two pre-drug marketing phase III clinical trials have begun.
For more details click on the link: http://dx.doi.org/10.1056/NEJMoa1705342
beta chainsBeta thalassemiablood disordersblood transfusionblood transfusionsgene therapygenetic disorderHemoglobinPhilippe Leboulchred blood cellred blood cell transfusionred blood cellssickle cellStem Cellstransplantation
Source : With inputs from NEJMNext Story
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