Dr. Hanna Mikkola, professor of molecular, cell and developmental biology at UCLA and co-director of the Epigenomics, RNA and Gene Regulation Program at the UCLA Health Jonsson Comprehensive Cancer Center, has received a $1 million grant from The V Foundation for Cancer Research to study myeloid leukemia of Down syndrome (ML-DS) and develop safer, more effective therapies.
Children born with Down syndrome, also called trisomy 21, are at greatly increased risk of developing myeloid leukemia of Down syndrome, a cancer that often begins before birth in a pre-leukemia stage called transient abnormal myelopoiesis. While these abnormal cells can disappear on their own, in as many as 30% of cases, the disease returns within a few years as leukemia.
Although myeloid leukemia of Down syndrome typically responds to chemotherapy, the treatment can be especially harsh for children with Down syndrome, and if the cancer comes back, survival rates are low.
The grant will support Mikkola, who is also a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and her team’s efforts to uncover the hidden mechanisms driving this leukemia using patient samples that are collected from the womb or at birth through leukemia diagnosis, treatment, and relapse to map the disease’s evolution at the single-cell level. The team will look for rare leukemia “starter” cells that can survive after transient abnormal myelopoiesis appears to resolve, identify biomarkers to detect them early, and investigate why some leukemia cells resist treatment or return after therapy.
“By using cutting-edge molecular methods that allow us to study unique patient samples at single cell resolution, we hope to uncover the root causes of this leukemia and find less toxic treatments that improve survival and quality of life for children with Down syndrome,” said Mikkola.
