Long-Term Phase 2 Trial Results

Published in Clinical Transplantation, August 2023

While kidney transplantation has traditionally required lifelong immunosuppression, an investigational stem cell therapy, FCR001, has been demonstrated to induce tolerance and eliminate the need for immunosuppression through the establishment of persistent mixed chimerism in a phase 2 clinical study. Real-world evidence demonstrated that kidney transplantation combined with non-myeloablative conditioning and FCR001 resulting in superior kidney function without increasing the risk of rejection, graft loss, or death among patients off immunosuppression.

Immune tolerance via FCR001 cell therapy compared with maintenance immunosuppression for kidney transplantation: Real-world evidence analysis of safety and efficacy

Phase 2 Clinical Trial Results

Published in Transplantation, June 2023

In the FCR001 cohort, a standardized measure of kidney function, estimated glomerular filtration rate (eGFR; measured by Modification of Diet in Renal Disease), among persistently chimeric participants improved over time compared with SOL control at all time points following 1 year post-transplant (Figure 4). BPAR occurred in 28.8% of SOC recipients at 2 years, 34.1% at 3 years, and 34.8% at 5 years post-transplant, whereas 0% of persistently chimeric participants in the FCR001 cohort off IS exhibited BPAR at all time points.

Evaluation of Immunocompetence and Biomarkers of Tolerance in Chimeric and Immunosuppression-Free Kidney Allograft Recipients

First Unrelated Delayed Tolerance Case

Published in Transplantation Proceedings, May 2021

By day ±20 after HCT, he demonstrated donor engraftment with 100% full donor chimerism in unsorted peripheral blood cells. His post-HCT course was rather uneventful, and he was discharged on day +21 after HCT. At his most recent follow-up 12 months after HCT, he remained in complete remission with normal renal function, no evidence of active acute or chronic graft-versus-host disease, and complete immunosuppression withdrawal 6 months after the HCT. He maintained 100% full donor chimerism at 57 and 131 days post-HCT.

Acute Myeloid Leukemia Presenting Less Than 3 Weeks After Living Donor Kidney Transplant: A Case Report

Elimination of Immunosuppressive Drugs from Transplant Patients

“76% of the patients treated in the phase 2 and 3 clinical trials were able to permanently stop taking immunosuppression medications—none ever needed to go back on immunosuppression medications.”
Dr. Joseph Leventhal

This chart represents the patients who have stopped taking immunosuppression medications in phase 2 and phase 3 trials. Starting with the first patient in phase 2 to be withdrawn from immunosuppression in 2009, all patients who were withdrawn from immunosuppression in both phases of the trial have continuously been off of immunosuppression medications.