Novartis CYStem Stem Cell and Gene Therapy Trial Is Moving Forward and Recruiting!
The Novartis stem cell trial is moving forward! We are excited to inform you that one arm of the Novartis CYStem stem cell and gene therapy trial is officially recruiting! This arm of the study is for Cohort 0 which is the group of patients that will NOT be treated with the stem cells. Cohort […]
Stem Cell Gene Therapy Treatment for Cystinosis Potential Treatment for Alzheimer’s Disease
In 2003, the mission of the Cystinosis Research Foundation was to find better treatments and a cure for cystinosis. Since then, the research funded by CRF has resulted in significant advances in the field of cystinosis have been accomplished. This research has impacted other more prevalent diseases and disorders, including Danon disease and Friedreich’s Ataxia. […]
Natalie Stack 5th Patient Stem Cell and Gene Therapy Trial – March 29, 2022
Wow, what a year 2022 has been so far! I have a wonderful boyfriend in my life (we are getting a puppy soon), the beloved show, This is Us is ending and I was the 5th patient to receive a stem cell transplant! For those of you who don’t know the show, This is Us, the show’s message […]
Tyler Joynt – 4th Patient in the Stem Cell and Gene Therapy Trial on November 15, 2021
By Tyler Joynt, San Diego, California As a kid, I really only knew three things about cystinosis. Doctors, sickness, and the San Diego Zoo. From an early age I remember throwing up often, going to the ER regularly, and seeing doctors frequently. I was lucky enough to be diagnosed before I was two years old, […]
Stem Cell Trial Phase I/II Updates by Stéphanie Cherqui, PhD
Dr. Stéphanie Cherqui provided an update on the stem cell gene therapy trial at the 2022 Day of Hope Family Conference. Five patients have now received the transplant, and she has good follow-up data on two of them. White blood cell cystine levels in these patients have gone down to normal despite not being on […]
The Third Patient Was Transplanted November 16, 2020!
From Rocky Seachord, Jacob’s Mom: “He is in very good spirits and I think it is a good omen that he is in the very same room that Jordan was in… The transplant, considered Day 0, is Monday the 16th. This will be his new birthday. I can’t even imagine the possibilities in his life […]
