A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.
Grant Award Details
Grant Number:
CLIN2SCD-11674
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$600,000
Status:
Closed
Grant Application Details
Application Title:
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.
Public Abstract:
Therapeutic Candidate or Device
ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients.
Indication
Severe Sickle Cell Disease
Therapeutic Mechanism
Hematopoietic stem cell transplantation is the only cure for severe sickle cell disease. The ECT-001 expanded cord blood cells will replace the patient's sickling red blood cells with healthy cells.
Unmet Medical Need
African-Americans are the most affected by sickle cell disease. Unfortunately, donor availability for this specific population is very limited for standard bone marrow transplantation. Using cord blood unit, with sufficent cell doses, would eliminate the donor availability problem.
Project Objective
Phase 1 Trial completed
Major Proposed Activities
ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients.
Indication
Severe Sickle Cell Disease
Therapeutic Mechanism
Hematopoietic stem cell transplantation is the only cure for severe sickle cell disease. The ECT-001 expanded cord blood cells will replace the patient's sickling red blood cells with healthy cells.
Unmet Medical Need
African-Americans are the most affected by sickle cell disease. Unfortunately, donor availability for this specific population is very limited for standard bone marrow transplantation. Using cord blood unit, with sufficent cell doses, would eliminate the donor availability problem.
Project Objective
Phase 1 Trial completed
Major Proposed Activities
- Manufacture product to supply the proposed trial
- Assess clinical safety and efficacy
Statement of Benefit to California:
Our innovative technology has the potential to greatly increase the quality of life and prevent early mortality for patients with blood disorders, such as leukemia, myeloma and sickle cell disease. 12 California patients will be the first to benefit of this curative technology, at very low costs. Also, Californian clinical sites involved in the trial will develop the expertise on ECT-001 grafts, and will therefore be the first to benefit from it once ECT-001 is approved.