Gene therapy for hemoglobinopathies

A list of innovative technologies that were selected by a committee of subject matter experts at the Cleveland Clinic, located in Cleveland, Ohio, was recently announced.

The list, led by Will Morris, MD and executive medical director of Cleveland Clinic Innovations, and Akhil Saklecha, MD and managing director of Cleveland Clinic Ventures, has 10 items that will make a difference in the world of medicine, including therapy genetics for hemoglobinopathies.

Hemoglobin disorders, or hemoglobinopathies, arise when an individual's beta-globin gene is dysfunctional. The most common hemoglobinopathies include sickle cell disease and thalassemia, which together affect about 330 million newborn children worldwide each year.

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There is currently no cure for sickle cell disease and thalassemia. In addition to blood and bone marrow transplantation, there are only treatments for symptom control, however, the latest research has brought experimental gene therapy for a cure in these patients, adding functional copies of a modified form of the beta-globin gene to the patient's hematopoietic stem cells. Gene therapy gives patients the potential to make functional hemoglobin molecules, therefore, functional red blood cells.

In several studies that are still ongoing, in patients with six or more months of follow-up after treatment for sickle cell disease, average levels of sickle hemoglobin have been reduced to 50% or less of total hemoglobin in the absence of blood transfusions. In thalassemia, therapy studies have found enough hemoglobin production to reduce or eliminate the need for transfusion support among patients who would otherwise need chronic blood transfusions.

These results earned the designation of advancing therapy from the FDA (the agency that regulates medicines and health products in the USA). As the first gene therapy for any of these conditions, medical researchers are about to approve a cure for these chronic blood disorders.

Source: Cleveland Clinic News Room