Gene Therapy Shows Promise for Blood Disorder Patients

 In Hematology

For patients with a blood disorder, blood transfusions are a normal part of life. A biotech company called Bluebird Bio is working to change that.

Recently, they’ve published the results of two early clinical trials in The New England Journal of Medicine that show promising results for patients with severe beta-thalassemia – an inherited blood disorder that reduces the production of hemoglobin, which are the red blood cells that carry oxygen to cells throughout the body.

The new experimental gene therapy aims to reduce the number of blood transfusions a patient with the disorder. Currently, 22 patients have undergone the treatment. Two-thirds of the group experienced healthy levels of hemoglobin and did not need transfusions.

Another trial is underway, so more research can be done to determine the effects for younger patients. However, the current findings are exciting.

There are about 288,000 cases of beta-thalassemia in the world. The only cure is a bone marrow transplant from a sibling, but that’s not an available option for most. Patients must regularly receive blood transfusions – even starting as an infant – and will typically be dependent on transfusions the rest of their life.

Read more about the study on CNN.

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