Grants - V Clinical Scholars - California - 2020

Alice Bertaina, MD, PhD

Funded by Lloyd Family Clinical Scholar Fund

Leukemia is a cancer that starts in blood-forming cells found in the bone marrow. It is the most common cancer in children and teenagers, accounting for almost 1 out of 3 cases. Despite recent advances in the treatment of childhood leukemia, a substantial proportion of patients are resistant to conventional treatments. For these children, the probability of cure is very low (<30-50%). The best treatment for leukemia patients, especially those who have not responded to other therapies, is stem cell transplant, but the application of this life-saving treatment has been traditionally limited by a lack of suitable donors. The lack of suitable donors is a particular problem in African American or mixed heritage populations because finding a matched donor is less likely in these populations. We have developed a stem cell transplant strategy that greatly increases the number of patients who can receive transplants. However, this strategy cannot provide the critical anti-leukemic and infection fighting functions required to kill all the leukemic cells and is therefore unable to give patients who receive transplants long term cancer-free outcomesIn this project we will perform three clinical trials designed to test the safety of three innovative cell therapies, which, when given in conjunction with our stem cell transplant strategy, have the potential to fight leukemia. Our ultimate goal is to identify the optimal anti-leukemic cell product that improve cancer-free outcomes for children with leukemia.