Stories of Hope: Liver Disease
Rosie Barrero was 26 years old and pregnant with twins when she was diagnosed with Retinitis Pigmentosa (RP) a rare degenerative disorder that attacks the light sensing cells in the eye. “It was absolutely devastating to know that I would be a mom, and that I would eventually go blind.”
RP has no cure, and no effective treatment. The first symptom is usually diminished night vision. It becomes more difficult to see in dimly lit or dark places. People with RP also slowly lose their peripheral vision and eventually end up with tunnel vision, or no vision at all.
Over the next 14 years Rosie steadily lost her vision and became legally blind. In 2016 she took part in a clinical trial funded by CIRM. She had an injection of stem cells called retinal progenitors in one eye. The hope was that these cells would repair or replace cells already damaged by the disease. Rosie says that over the course of several months her vision slowly improved, and she went from complete darkness to being able to see shapes and colors, and finally the faces of her children.
“My dream was to see my kids. I always saw them with my heart, but now I can see them with my eyes. Seeing their faces is truly a miracle.”
Other patients in the trial also benefitted and experienced improved vision.
In 2017, the company behind the therapy, jCyte, was granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US Food and Drug Administration (FDA). This could pave the way for accelerated approval of this stem cell therapy for patients with RP.