The First Cystinosis Patient was Transplanted on October 7, 2019!

Yesterday was a day that will forever be remembered as a historic day for adults and children with cystinosis and their families. It’s a day that could very well mark the beginning of a world without cystinosis.

Yesterday, Jordan Janz, age 20 from Consort, Alberta, was the first patient to receive a genetically modified autologous stem cell transplant for cystinosis.

The treatment developed by Stéphanie Cherqui, PhD at UC San Diego, was FDA approved last year. CRF is proud to have supported Dr. Cherqui and her groundbreaking research since 2007.  Thanks to the dedication and generosity of our CRF friends and family from around the globe,  CRF has funded over $5.4 million in grants to Dr. Cherqui.  It was the early grants from CRF that allowed Dr. Cherqui to pursue her stem cell and gene therapy work.  Dr. Cherqui has dedicated her career to finding a cure for cystinosis – she is beloved by all of us.

Jordan is a true pioneer in our community, and we are incredibly grateful to him for his courage to be the first cystinosis patient to undergo the treatment. Jordan’s bravery has paved the way for all future cystinosis patients who will be part of this trial.  We are all thankful to Jordan and his family for their gift of hope and promise to our community.  We still have many months to wait before we know how the treatment is working and if it will be a lasting cure, but we are hopeful and optimistic that it will be.  

The Cystinosis Research Foundation began with Natalie’s wish, “to have my disease go away forever.” The quest for the cure has been driven by parents and patients who were determined to find a cure. We were embraced by family and friends and together we started a movement. Thank you to every family, donor, researcher and scientist, for being part of our movement.  Thank you to every child and adult with cystinosis – you inspire us every day and teach us how to live a life full of joy, celebration and hope.  This day belongs to all of us – Natalie’s wish has come full circle.

We must be patient as we wait for updates on Jordan and the transplant.  We pray, hope and support Jordan and the next patients who will be part of the trial. We celebrate Jordan and his family, our research and scientific community, and our family and friends for their love, dedication and perseverance.

The Cystinosis Research Foundation set out to find a cure, and today, we are more hopeful and optimistic than ever that we have found it!