Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant
Grant Award Details
Grant Type:
Grant Number:
CLIN2-14068
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$9,054,216
Status:
Active
Grant Application Details
Application Title:
Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant
Public Abstract:
Therapeutic Candidate or Device
COH-MC-17: A minimally manipulated half-match donor blood stem cell transplant with a low-toxic conditioning regimen of the transplant host
Indication
Older (>40 yrs) Severe Aplastic Anemia (SAA) patients that are ineligible for the potentially curative standard stem cell transplant
Therapeutic Mechanism
Allogeneic stem cell transplant from full-match donor is the only cure for SAA currently. For the 20-30% SAA patients without a full-match donor, our product is designed to induce mixed chimerism and immune tolerance in these patients when transplanted with blood cells from half-match donor. Mixed chimerism is when both donor and host blood cells co-exist in the transplant host. The right mix of donor to host blood cells will allow SAA patients free of disease for a long time.
Unmet Medical Need
Our product will allow more SAA patients without full match donors assessible to the potentially curative stem cell transplant. Our method will allow patients to receive less-toxic conditioning regimen before receiving stem cell transplant from half match donors.
Project Objective
Complete a Phase I study with 6 evaluable patients
Major Proposed Activities
COH-MC-17: A minimally manipulated half-match donor blood stem cell transplant with a low-toxic conditioning regimen of the transplant host
Indication
Older (>40 yrs) Severe Aplastic Anemia (SAA) patients that are ineligible for the potentially curative standard stem cell transplant
Therapeutic Mechanism
Allogeneic stem cell transplant from full-match donor is the only cure for SAA currently. For the 20-30% SAA patients without a full-match donor, our product is designed to induce mixed chimerism and immune tolerance in these patients when transplanted with blood cells from half-match donor. Mixed chimerism is when both donor and host blood cells co-exist in the transplant host. The right mix of donor to host blood cells will allow SAA patients free of disease for a long time.
Unmet Medical Need
Our product will allow more SAA patients without full match donors assessible to the potentially curative stem cell transplant. Our method will allow patients to receive less-toxic conditioning regimen before receiving stem cell transplant from half match donors.
Project Objective
Complete a Phase I study with 6 evaluable patients
Major Proposed Activities
- Donor Cell Manufacturing and Release of Allogeneic Product
- Culture Sensitivity Training, Recruitment, Community Engagement and Clinical Trial (Treat 6 Patients)
- Assess Safety, feasibility of producing sufficient donor cells, and the ability to induce mixed chimerism
Statement of Benefit to California:
About 2000 people died of SAA in California between 1999-2016, most of them are 45 and older (CDC Wonder database). Our new product is designed to first provide previously excluded SAA patients assess to curative stem cell therapy, and secondly to induce immune tolerance in transplant host and improve the treatment success rate, and finally to improve the health and well-being of California citizens who suffer from this disease, and significantly reduce health care costs.