Grant Award Details
- Objective of this study is to assess sequential T cell receptor/CD19+ B cell depleted hematopoietic stem cell transplant in pediatric and adolescent and young adults with SIOD, FSGS, Cystinosis and SLE patients allowing immunosuppression free kidney transplant from the same donor
Grant Application Details
- Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant
Therapeutic Candidate or Device
Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells
Renal failure due to one of four genetic and/or immunological diseases
1. By using haploidentical parents, we will expand the number of potential living donors, and 2. with pre-HSCT immune ablation we can cure the underlying immune associated disease reducing the risk of disease recurrence in the newly engrafted kidney, and 3. with the establishment of a full donor lymphoid engraftment, we eliminate the risk of kidney rejection and the need of lifelong immunosuppression. Therefore, the likelihood of a second kidney transplant will be reduced, if not eliminated.
Unmet Medical Need
Our same donor sequential αβdepleted-HSCT kidney transplantation strategy addresses the urgent unmet medical need to abrogate the need for post-kidney transplant lifelong immunosuppression, the risk of chronic rejection, and, ultimately, the need for repeated transplantation.
Phase 1b trial completed
Major Proposed Activities
- Perform αβdepleted-HSCT and achieve complete donor engraftment without severe GvHD
- Perform post-αβdepleted-HSCT kidney transplantation and discontinuation of the immunesuppression within 90 days post-KT
- Develop a closed system for production of αβdepleted-HSC to expand the number of facilities able to provide αβdepleted-HSC manufacturing
California performs ~ 100 pediatric KT/year of which ~50 patients experience rejection. These patients enter a cycle of dialysis and waiting for a new kidney creating a quality of life and healthcare burden. Sequential same donor αβdepleted-HSCT/KT can 1) eliminate the need for lifelong immunosuppression, 2) reduce the need for a 2nd KT, and 3) cure the underlying disease, resulting in improved lives of patients, decreased societal costs and enormous socio-economic benefit to California.