UCSF Benioff Children's Hospital and Research Center, Oakland
The Blood and Marrow Transplant program at CHRCO fosters the development and participation in clinical transplantation research studies for children with malignant and non-malignant hematological disorders, including:
-Co-leading a NIH-supported clinical trial to test bone marrow transplantation in young adults with sickle cell disease who have sibling or unrelated donors; this is the first study in the US to explore transplantation in older individuals with this disorder
-Participating in unrelated donor transplantation clinical trials for thalassemia and sickle cell disease to expand the availability of this curative therapy beyond those who have sibling donors
-Clinical team partner with bluebird bio Inc. to initiate a clinical trial of gene therapy for thalassemia major using a lentiviral vector for replacement gene transduction in hematopoietic stem cells. The bluebird bio Inc sponsored Northstar clinical trial, conducted under a FDA IND and funded in part by CIRM, is currently recruiting young adult patients with thalassemia major
-Clinical team partner in Sangamo’s Phase I clinical trial of high-efficiency targeted genome editing of hematopoietic stem cells through the zinc finger nuclease gene editing technology, funded by CIRM.
In collaboration with Bruce Ames lab, we have discovered alterations in the metabolomics profile in a mouse model of graft-versus-host disease that indicates the occurrence oxidative damage that precedes the organ damage characteristic of this condition. We are exploring therapeutic pathways that might mitigate the oxidative changes in target organs.
Initiated by Dr. Bert Lubin and extended by Dr. Mark Walters, the Sibling Donor Cord Blood Sibling Connection program proved that it is possible to collect high-quality umbilical cord blood units from families with sickle cell disease and thalassemia and to use these cryopreserved cells to support clinical transplantation. In a partnership with the ViaCord, this program continues to make this public resource available to eligible families, at no cost.
Recent publiations: F. Locatelli et al. Outcome of patients with haemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling, in press, Blood
Z Romero et al. Transduction of Human Bone Marrow CD34+ Cells by a Lentiviral Vector Carrying a Modified Human , in press, J Clin Invest.
NR Kamani, MC Walters et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of a phase II STUDY from the Blood and Marrow Transplant clinical trials network (BMT CTN), Biol Blood Marrow Transpl., 18:1265-72, 2012.
B Kanathezhath et al. Engraftment with minimal Graft-Versus-Host Disease after MHC-Mismatched Cord Blood Transplantation with Photochemically Treated Donor Lymphocytes, Exp Biol Med (Maywood) 236:492-504, 2011.
A Ruggeri et al. Umbilical Cord Blood Transplantation for Thalassemia and Sickle Cell Disease, Biol Blood Marrow Transpl, 17:1375-82, 2011.
M. Sabloff et al. HLA-matched Sibling Bone Marrow Transplantation for 117:1745-50, 2011.
MC Walters et al. Pulmonary, Gonadal and Central Nervous System Status after Bone Marrow Transplantation for Sickle Cell Disease, Biol Blood Marrow Transpl, 16:263-72, 2010.