For Evie Junior, living with sickle cell disease has been like running a marathon.
“But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said. “It gets harder as you get older. Things start to fail and all you can think about is how much worse it’s going to get down the road.”
In sickle cell disease, a genetic mutation causes the blood-forming stem cells — which give rise to all blood and immune cells — to produce hard, sickle-shaped red blood cells. These misshapen cells die early, leaving an insufficient number of red blood cells to carry oxygen throughout the body. Because of their sickle shape, these cells also get stuck in blood vessels, blocking blood flow and resulting in excruciating bouts of pain that come on with no warning and can leave patients hospitalized for days.
In July 2019, Junior enrolled in a clinical trial for an experimental stem cell gene therapy for sickle cell disease. The study is led by UCLA Broad Stem Cell Research Center physician-scientists Dr. Donald Kohn and Dr. Gary Schiller and funded by the California Institute for Regenerative Medicine. The therapy, developed by Kohn over the past 10 years, is intended to correct the mutation in patients’ blood-forming stem cells to allow them to produce healthy red blood cells.
Learn more at https://stemcell.ucla.edu/Evie