When the FDA approved a gene-editing treatment for sickle cell disease for some patients last year, it marked a major step for medical research, and now the final results from the clinical trial that led to the drug’s approval have been published in the New England Journal of Medicine.
The paper was coauthored by Damiano Rondelli, MD, the chief of the division of hematology/oncology at the University of Illinois College of Medicine Chicago, who treats patients at UI Health and is a member of the University of Illinois Cancer Center.
The findings show the therapy reduces symptoms of the disease that can cause pain severe enough to require hospitalization. The paper reports data from the two-year clinical trial that took place at 16 sites in Europe and North America, including the University of Illinois Chicago (UIC).