CIRM Grants Nearly $6M to Promising Sickle Cell Research

May 15, 2018 | by Michael Easterling

Thanks to a $5.74 million award from the California Institute for Regenerative Medicine (CIRM), Joseph Rosenthal, M.D., and his team can advance their research into finding a cure for sickle cell disease (SCD).
 
The research involves the use of mixed chimerism — transplanting stem cells from a half-matched family member into a patient who has received a less toxic form of chemotherapy. Prior to the transplant, the donor stem cells are depleted of CD4+ T cells, which provides a more hospitable environment within the patient and reduces his or her chances of contracting graft-versus-host disease (GVHD), a common side effect of transplantation.
 
“This CIRM funding allows us to conduct a phase 1 trial in six adult patients with severe SCD,” said Rosenthal, chief of the Division of Pediatric Hematology/Oncology and the Barron Hilton Chair in Pediatrics.
 
We believe we can improve the quality of life in these patients, and reduce the risk of GVHD and other transplant-related complications. Our hope is that this treatment can be eventually offered to SCD patients as a curative therapy.”
 
Rosenthal is the principal investigator of the phase 1 trial.
Rosenthal-Joseph Joseph Rosenthal, M.D.
 
Defu Zeng, professor of diabetes immunology and diabetes complications and metabolism in the Diabetes & Metabolism Research Institute at City of Hope, is the co-principal investigator of the trial. He developed the mixed chimerism induction process in early preclinical tests. His lab studies with animal models have also shown that transplants with half-matched donors can potentially cure autoimmune diseases like type 1 diabetes, systemic lupus and multiple sclerosis. As such, City of Hope’s Wanek Family Project for Type 1 Diabetes, established last year, is also supporting the SCD research.
 
SCD is an inherited blood disorder in which a gene mutation causes normal red blood cells to take on a crescent shape, resembling a sickle. This abnormal shape makes it difficult for red blood cells to move easily and carry oxygen through the body. Sickle cell disease affects around 100,000 people in the United States, most of whom are African-American.
Zeng-Defu Defu Zeng
City of Hope’s sickle cell disease program offers several unique features that allow patients to receive treatment in a more efficient and less invasive manner. Because City of Hope has its own donor apheresis center and blood bank, patients there don't have to travel to another hospital for transfusions and, most times, the transfusion process can be completed in one day.
 
Traditional treatment options for SCD include medication, blood transfusions and, more recently, stem cell transplantation, which has become the treatment standard. In addition to transplants for SCD patients, City of Hope has also developed preclinical gene therapy trials and a new approach for gene therapy using non-nuclease technologies.
 
“City of Hope is at the forefront of developing bolder gene therapy options for patients with severe sickle cell disease,” Rosenthal said. “Our research and clinical trials are creating hope for patients with this devastating disease.
 
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