Saturday, December 8, 2018

Early clinical trial data show gene therapy reversing sickle cell anemia

The Cincinnati Children's Cancer and Blood Diseases Institute has been researching and developing a new gene therapy to combat the horribly inherited disease, sickle-cell anemia. After finally being able to treat two patients, and now a year and six months after treatment, both patients are seeing "remarkable improvement in the quality of life due to remarkable reduction in disease symptoms."

This therapy developed in Cincinnati uses "modified gamma globin lentivirus vector to transfer healthy fetal hemoglobin (HbF) gene into a patient's blood stem cells." If sufficient amounts of HbF are present in the human body, the blood cells cannot "sickle" and therefore are able to properly transport oxygen through the blood. First, cells are collected from the sickle-cell patient and genetically modified with the lentivirus. Then after a low dose of chemotherapy to the patient's bone marrow, the newly corrected cells are placed back into the body. The goal of such gene therapy is to make it most accessible and affordable in Central Africa, where sickle-cell is rampant. However, some 90,000 people in the United States are still plagued with the disease.

As mentioned, the patients who received gene therapy have been experience a better life after their treatment. After only 15 and 12 months, patient one had only one acute sickle event and the second has had no issue with blood cells becoming stuck in the bloodstream. Cincinnati Children's hopes to be able to further their research and continue to treat more patients with less harmful treatments so the quality of life for genetically sickle-cell patients continues to improve.

The research done in Cincinnati seems to have been successful for the sickle-cell community. I am sure that those who suffer from or have family members that suffer from this horrible disease would be glad to see treatment option soon available, with little harm and to their health, and seemingly quick relief of past symptoms.


  1. It seems that the treatment did not have any major complications. Hopefully this can be applied to every person out there suffering from sickle cell anemia

  2. The cure in process seems to show no adverse effects, and using it to cure sickle cell in places like Africa seems like a very realistic possibility

  3. Although this is a good treatment for people who suffer from sickle cell anemia, I do not think it is a good idea to eradicate it from Africa. Reason being is that sickle cell anemia has been found to prevent Malaria, and the reason why many people in Africa do have sickle cell. If you were to fix this mutation than it could potentially kill off entire communities. However, it is a great idea for people in communities that do not have to worry about Malaria.