UCLA gene therapy trial for sickle cell disease: Evie’s story

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 stemcell.ucla.edu/Evie

[@TheValiantZero]

This is what the future looks like. You might not recognize it right now, but you are the medical pioneers of your time. You are at the forefront of medicine. Continue making these incredible advancements and get yourself in the history books! 100% of the human population can benefit from gene therapy.

[@lydiaedzordzinam9503]

IM SO HAPPY FOR HIM. I HOPE MY SON RECEIVES A GENE THERAPY SOON ENOUGH SO I DON'T LOSE HIM. I FEEL SO GUILTY.

[@YoungHaveNot]

Guilty for what? Its no one's fault.

[@michealmclaughlin429]

why them type of feeling. its a real feeling. not everyone looks at things with 'probabilities'

[@user-km5wq7if5m]

Good step,when will it be ready to use

[@florencetitie1025]

also my tried with the plan

[@magus7246]

I have sickle cell but I don't want to do chemo, so I'll be waiting for something else. There must be some way of doing this without putting me inside a microwave LOL

[@YoungHaveNot]

You dont think it's worth it tho

[@jemtopaz8306]

Wow great! Yay!

[@jemtopaz8306]

Just wondering if there is chemotherapy involved?

[@uclastemcell]

Yes, Evie went through a week of chemotherapy prior to receiving the transfusion of his genetically corrected blood stem cells.

[@jemtopaz8306]

@@uclastemcell thank for responding🙏🏽

[@magus7246]

@@uclastemcell However, I am interested in getting the cure. I'm reluctant to do it because of the chemotherapy though. How and why is that a necessary part of it? Do you have to weaken the immune system to a point for the new stem cells to be accepted? I don't understand...

[@YoungHaveNot]

@@magus7246 its what makes the process possible

[@simonmufor1951]

@@uclastemcell please how much is the treatment?

[@THEanovah]

Evie looks healthier and more muscular than many 'healthy' people out there

[@shritandi5242]

How old are you

[@dailydoseofjamaica9263]

Will the edited cells stick around forever?

[@javionriley8739]

Yes , it’s identical to taking out salt & adding in sugar to your food while preparing it

[@ebegwendolyn9204]

Also try plant stem cells you will thank God