CHARLESTON, S.C. (WCBD) – June 19 is World Sickle Cell Day.

Sickle cell disease is a painful and debilitating genetic blood disorder that has limited treatment options.

But a clinical trial for a new gene therapy that Cleveland Clinic is taking part in is showing promising results.

“We are seeing very encouraging results where the patients involved haven’t had a pain crisis from sickle cell after undergoing the gene therapy. Other colleagues who are participating in this clinical trial are seeing similar results for their patients as well,” explained Rabi Hanna, MD, the principal investigator of the study at Cleveland Clinic Children’s.

Dr. Hanna said the trial involves an experimental gene editing cell therapy that modifies a patient’s own stem cells to correct the mutation responsible for sickle cell disease.

Two of the four patients involved in the multicenter trial so far were treated at Cleveland Clinic Children’s.

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 a functional cure. It wouldn’t reverse the damage sickle cell disease has already caused. It would help prevent any future damage,” Dr. Hanna said.

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.

These results were recently presented at the European Hematology Association Hybrid Congress in Frankfurt, Germany, on June 10, 2023.