Targeted Therapy for Leukemia and Other Cancers
The Sakamoto laboratory is developing novel drugs that inhibit the interaction between transcription factor CREB and its binding partner CBP by collaborating with experts in structural biology (Wakatsuki lab), computer modeling (Dror lab), and medicinal chemistry (Mark Smith, ChEM-H). We are also screening small molecule libraries to identify CREB inhibitors. We are repurposing drugs for Phase I clinical trials for relapsed/refractory AML.
Signaling Pathways in Bone Marrow Failure Syndromes
Diamond Blackfan Anemia (DBA) and myelodysplastic syndromes (MDS) are bone marrow failure syndromes that are associated with ribosomal defects. The Sakamoto laboratory is studying signal transduction pathways that contribute to abnormal erythropoiesis and anemia associated with DBA and MDS. A serine, threonine kinase known as Nemo-like Kinase (NLK) is hyperactive in cell models of DBA and MDS. Sakamoto and colleagues are studying transcriptomes downstream of NLK and developing novel drugs to inhibit NLK.
Transcriptional Regulation in Leukemogenesis
The Sakamoto laboratory is investigating transcriptomes in pediatric chronic myeloid leukemia. Her group has identified genes and pathways that are unique to pediatric patients compared to adult patients. Future work will focus on single cell RNA-seq to understand the clonal heterogeneity of pediatric CML stem cells.