UC San Diego researchers receive nearly $8 million grant for new stem cell-based AML treatments

The Independent Citizens Oversight Committee of the California Institute for Regenerative Medicine (CIRM) unanimously approved yesterday two grants worth a total of almost $8 million to University of California San Diego School of Medicine researchers investigating novel stem cell-based treatments for acute myeloid leukemia or AML.

In 2017, there will be more than 62,000 new cases of leukemia (of all kinds) in the U.S. and 24,500 deaths, according to the American Cancer Society. AML will account for approximately one-third of new leukemia cases and almost half of all leukemia-related deaths. AML primarily afflicts adults, with an average patient age of 67. It is uncommon before the age of 45. Standard chemotherapy is effective in two out of three AML patients, but much less so for older patients, and recurrence is common.

The first grant for $5.15 million was awarded to Dan Kaufman, MD, PhD, professor of medicine in the Division of Regenerative Medicine, to advance clinical translation of natural killer (NK) cells derived from human embryonic stem cells into a standardized treatment for treating, and possibly curing, AML and other leukemias. NK cells are a type of immune system cell critical to rapid response to infections and tumor formation. They get their name from the ability to attack tumor cells without requiring patient-specific triggering markers.

The second grant for $2.7 million was awarded to Catriona Jamieson, MD, PhD, deputy director of the Sanford Stem Cell Clinical Center and director of stem cell research at UC San Diego Moores Cancer Center, in collaboration with Michael Burkart, PhD, professor in the UC San Diego Department of Chemistry and Biochemistry, and Leslie Crews, PhD, assistant professor in the Division of Regenerative Medicine at UC San Diego School of Medicine. The funding will support testing of a novel therapeutic splicing modulator approach targeting cancer stem cells in AML. These cells, which evade or become resistant to cancer treatment, are believed to be the cause of the high relapse rate in AML and other cancers.

"This research is critically important in advancing our knowledge of stem cells and are the foundation for future therapeutic candidates and treatments," said Maria Millan, MD, president and CEO of CIRM. "Exploring and testing new ideas increases the chances of finding treatments for patients with unmet medical needs. Without CIRM's support, many of these projects might never get off the ground. That's why our ability to fund research, particularly at the earliest stage, is so important to the field as a whole."