We are thrilled to share news about the next phase of the stem cell and gene therapy trial. This phase of the trial will bring us closer to a cure for all children and adults with cystinosis. The CYStem Phase I/II clinical trial is open to children aged 2 to 5 years with nephropathic cystinosis and was recently published on ClinicalTrials.gov. Details about the trial from Novartis can be found on our website at cystinosisresearch.org/current-clinical-trials/
Make no mistake, the road to the cure has required hard work, millions of dollars, and the unwavering commitment of families, patients, friends, and researchers. Without the early and continued support of CRF to Stéphanie Cherqui, PhD, this extraordinary news would not be a reality today. Since 2007, CRF has funded over $6.6 million in grants to Dr. Cherqui and because of our early financial support, other funding agencies including the California Institute for Regenerative Medicine (CIRM) and the National Institutes of Health (NIH) have supported Dr. Cherqui’s work leveraging CRF’s early research dollars.
Dr. Cherqui has dedicated her career to finding a cure for our children and adults with cystinosis. It is CRF’s partnership with Dr. Cherqui that has brought our wish for a cure from a possibility to reality. She has been our guiding light, our beacon of hope, and we are incredibly grateful.
A very special thanks to the five adults with cystinosis who pioneered this treatment. They risked so much on behalf of the entire community, and their participation and the successful data from their phase of the trial led to this important phase of the stem cell trial.
It is with gratitude that we thank Novartis and their cystinosis stem cell and gene therapy group for their commitment to our community, for listening to our voices about the burden of disease, quality of life, clinical trial design and most importantly, our plea to move quickly to ensure this treatment is approved for all those with cystinosis. We look forward to continuing to work with them during this next and critical phase of the stem cell trial.
