The Novartis CYStem Clinical Trial is Open at Stanford University for Cohort 1!
This is another milestone, another giant step forward for our small community. We have worked together, stayed united in our effort to find a cure and we have driven the research to ensure that this day would be a reality for our community. Now, some of our youngest patients will be able to participate in this clinical trial. Today we are one step closer to a cure for every person with cystinosis!
We are excited to announce that the stem cell and gene therapy trial is recruiting for Cohort 1 at Stanford University. The trial is open to children aged 2 to 5 years with nephropathic cystinosis. If you are interested, please contact: Research Nurse (650) 725-9032 or email scgt_clinical_trials_office@lists.stanford.edu.
It took many years and the collaboration of all stakeholders in our community to realize this historical day. We would not be here today without the dedication and commitment of Dr. Stephanie Cherqui, Professor at UC San Diego. Since 2007, she has worked to find a cure for cystinosis, and now the cure is within reach. She has been our guiding light, our beacon of hope, and we are incredibly grateful.
We thank CRF donors, families, friends, and the patient community for your unwavering support. We also thank CIRM and the NIH for the millions of dollars that were invested in research and early clinical trials so that our children and adults could dream of a life without cystinosis.
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 working with them during this next phase of the stem cell trial.
CRF’s mission is a cure for all people with cystinosis – no one will ever be left behind. We will work tirelessly to advocate for accelerated approval so that the treatment is a reality as soon as possible.
If you are interested in the clinical trial, the eligibility criteria are listed on the website. Please view the trial details here: https://clinicaltrials.gov/study/NCT06910813?cond=cystinosis&aggFilters=status:not&rank=1#study-overview