Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients
Grant Award Details
Grant Type:
Grant Number:
CLIN2-14315
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$10,642,420
Status:
Active
Grant Application Details
Application Title:
Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients
Public Abstract:
Therapeutic Candidate or Device
αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191
Indication
Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias
Therapeutic Mechanism
Fanconi anemia (FA) patients undergoing HCT have heightened sensitivity to current conditioning regimens and short and long-term sensitivity to graft versus host disease (GvHD). The combined product attmpts to address both issues. JSP191 confers non-genotoxic host HSC removal and thus will be tested as a replacement for TBI. αβdepleted-HSCT is stem cell graft source that has been shown to have lower frequencies of GvHD than alternative HSC sources and faster immune reconstitution.
Unmet Medical Need
Current conditioning regimens for HCT are toxic for FA patients. FA patients are also very susceptible to GvHD post-HSCT. HSCT approaches that diminish pre-transplant toxicity and post-transplant GvHD could improve outcomes for FA patients and become a new global standard of care.
Project Objective
Complete Phase 1b/2a
Major Proposed Activities
αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191
Indication
Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias
Therapeutic Mechanism
Fanconi anemia (FA) patients undergoing HCT have heightened sensitivity to current conditioning regimens and short and long-term sensitivity to graft versus host disease (GvHD). The combined product attmpts to address both issues. JSP191 confers non-genotoxic host HSC removal and thus will be tested as a replacement for TBI. αβdepleted-HSCT is stem cell graft source that has been shown to have lower frequencies of GvHD than alternative HSC sources and faster immune reconstitution.
Unmet Medical Need
Current conditioning regimens for HCT are toxic for FA patients. FA patients are also very susceptible to GvHD post-HSCT. HSCT approaches that diminish pre-transplant toxicity and post-transplant GvHD could improve outcomes for FA patients and become a new global standard of care.
Project Objective
Complete Phase 1b/2a
Major Proposed Activities
- Manufacture αβdepleted-HSC graft product for proposed trial
- Clinical Testing of reduced intensity JSP191-based conditioning before αβdepleted-HSC graft transplant in FA patients
- Complete evaluation of primary, secondary and correlative objectives
Statement of Benefit to California:
A safe and durable cure for FA patients can provide a great economic benefit to the state. A successful outcome of our trial will significantly reduce the length of hospitalization, thus reducing patient care costs. Thus, the benefit to California is the improved lives of its citizens (both patients and family members) while simultaneously decreasing societal costs. It would bring further pre-eminence to California as somewhere that develops patient-centric safe and effective HSC therapies.