CLEVELAND, OH (CLEVELAND CLINIC) - June 19th is World Sickle Cell Day. The disease is a painful and debilitating genetic blood disorder with few treatment options. But a clinical trial for a new gene therapy that Cleveland Clinic is taking part in is showing promising results.
Researchers say the trial involves an experimental gene-editing cell therapy that modifies a person's own stem cells to correct the mutation responsible for the disease. Along with relief from the pain sickle cell causes, initial results showed new white blood cells in all patients at about four weeks with no severe side effects. They also achieved a normal level of hemoglobin – which is the most important component of red blood cells that carry oxygen throughout the body.
"Our hope is to achieve functional cure. And what I mean, I mean by this functional cure is the ability to not have an impact on your life, because it wouldn't take away the sickle cell from your other organ. It wouldn't take away the impact of sickle cell that already happened. It will help prevent any future damage that would be caused by the sickle cell," said Dr. Rabi Hanna, Cleveland Clinic Children's.
The trial aims to enroll 40 adults, ages 18 to 50, with severe sickle cell disease. They will be monitored closely after treatment for up to two years.
