Novel Therapeutics:
Targeted Disruption of Cancer Metabolism and Ribosome Biogenesis through Small Molecule Precision Therapeutics
Dr. Deepak Nijhawan, M.D., Ph.D.
- Associate Professor, Departments of Internal Medicine (Hematology/Oncology) and Biochemistry
Dr. Jef De Brabander, Ph.D.
- Professor of Biochemistry, UT Southwestern Medical Center
This Discovery Track combines two first-in-class small molecule platforM.S. designed to attack the core vulnerabilities of cancer cells. One program targets NVL, an essential ribosome assembly protein, selectively triggering p53-dependent cell death in cancers like AML and colorectal carcinoma without causing DNA damage. The second program inhibits lanosterol synthase (LSS) to force glioma stem-like cells into metabolic collapse via toxic epoxycholesterol accumulation, offering a new therapeutic strategy for glioblastoma, where treatment options remain limited. Both prograM.S. arise from a powerful phenotype-to-target discovery engine that couples medicinal chemistry, resistance mapping, and cryo-EM structural biology. Supported by Harrington and CPRIT grants, this dual platform is now advancing through lead optimization and preclinical validation with defined biomarkers and path-to-IND studies.
