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Commercialization of Valproic Acid Expanded Cord Blood Stem Cells as Allogeneic Grafts for Adults with Refractory Hematological Malignancies

Stem cell transplantation represents the only potential cure for most patients with blood cancers that are refractory to chemotherapy, as well as patients with genetic disorders that involve blood cells (sickle cell anemia, thalassemia). Only 30% of patients who require a stem cell transplant will have a matched sibling donor. Although there are over 20 million adult volunteer unrelated donors in the National Marrow Donor Program and affiliated registries, many patients, especially individuals of diverse racial/ethnic backgrounds, will not have a donor identified. An alternate source of transplantable stem cells is umbilical cord blood (CB) that is present within the placenta of every child born. There are limited numbers of stem cells within CB collections, which leads to their being useful for children but unfortunately having limited use for adults. We developed a process to expand in the laboratory the number of stem cells within a single CB collection so as to allow safe transplant into adult patients. By treating CB-CD34+ cells in a serum free culture system containing a combination of cytokines and a histone deacetylase inhibitor, valproic acid (VPA), we have been able to increase 35-fold the number of stem cells. We will move this approach from the research laboratory to the clinic, which will allow adults who are transplant candidates without an appropriate stem cell donor to receive such expanded grafts.  Hopefully, this will provide them with a chance for a cure of their blood disorder.

 

Consortium Members

Investigator

Role

Icahn School of Medicine at Mount Sinai

Ronald Hoffman
Camelia Iancu-Rubin
Nina Bhardwaj
Marcia Meseck
Ian McNiece
Jeffrey Jhang
Alla Keyzner
John Levine
Christoph Schaniel
Umut Ozbek
Bartek Jablonski

PI
Co-PI
Collaborator
Collaborator
Consultant
Consultant
Collaborator
Collaborator
Collaborator
Collaborator
Project Leader

AllCells, LLC

Jay Tong

Co-I

Principal Investigatior: 
Ronald Hoffman
source: 
Icahn School of Medicine at Mount Sinai
value: 
$8 782 529