Insertion of fetal hemoglobin gene reduces symptoms of sickle cell disease
Two patients receiving gene therapy have reported significant improvement in the quality of life.
A pilot phase of a clinical trial that started one and a half year ago brought positive, preliminary results.
Individuals with severe sickle cell disease received procedures similar to popular CAR-T therapy: their cells were collected from the body, genetically modified in a laboratory, and then infused back to the bloodstream. In this specific case, the modification relied on insertion of the fetal hemoglobin gene into hematopoietic stem cells.
After 15 and 12 months of treatment, two patients were free from chronic pain and sickling events and gave up the use of opioid drugs.
The trial will be continued to enroll a total of 10 individuals.
More: Clinical trial NCT02186418, https://clinicaltrials.gov/ct2/show/NCT02186418.