Researchers at City of Hope were awarded $32.3 million from the California Institute for Regenerative Medicine (CIRM) to support three novel phase I clinical trials evaluating innovative cell and gene therapies for patients with HIV, acute myeloid leukemia (AML), and severe aplastic anemia. Beckman Research Institute of City of Hope was awarded two-thirds of the $50 million in this latest round of CIRM awards, the highest of any entity in the state.
“City of Hope made a major advancement when our transplant team helped Paul Edmonds, globally known as the ‘City of Hope patient,’ go into remission for both HIV and leukemia,” said John A. Zaia, MD, the Aaron D. Miller and Edith Miller Chair for Gene Therapy at City of Hope. “We are hoping to evaluate a [chimeric antigen receptor T-cell] therapy for HIV in a phase I clinical trial, so one day more people with HIV might be able to achieve long-term HIV remission.” CIRM awarded Dr. Zaia $11.3 million for the clinical trial.
A Difficult-to-Treat Blood Cancer
CIRM awarded a City of Hope team led by Elizabeth Budde, MD, PhD, nearly $12 million to develop and conduct a first-in-human phase I immunotherapy trial in AML. “It is critical to find more effective therapies for patients whose AML has relapsed,” said Dr. Budde, Associate Professor in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope. “We are hopeful this novel therapy will eventually enable City of Hope and other institutions to offer a new treatment for patients whose AML has returned.”
Finding a Better Treatment for Severe Aplastic Anemia
A City of Hope research team received $9.05 million from CIRM to start a clinical trial using a novel blood stem cell transplantation procedure for severe aplastic anemia. If successful, the treatment could also be used for other autoimmune diseases, such as treatment-resistant type 1 diabetes. The phase I trial is expected to start later this year and will enroll up to six patients.
Defu Zeng, PhD, Professor in the Department of Immunology & Theranostics within the Arthur Riggs Diabetes & Metabolism Research Institute at City of Hope, has led research on the potential new therapy, which involves induction of mixed chimerism in adults.
“City of Hope has extensive expertise performing bone marrow and stem cell transplants for patients with blood cancers and other diseases, and we are consistently looking for ways to improve the process and effectively treat more patients with transplants, regardless of any barriers,” said Ryotaro Nakamur, MD, the Jan & Mace Siegel Professor in Hematology & Hematopoietic Cell Transplantation in the Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, at City of Hope, and the trial’s principal investigator. “We are hopeful this will provide another treatment option for patients with this devastating disease and thankful for CIRM’s funding.”
