Liang Cao, Ph.D.

Liang  Cao, Ph.D.
Staff Scientist
Head, Molecular Targets Core

Dr. Cao’s current research is on the investigations of tumor biomarkers and their roles in cancer drug development. He did some early work on homologous recombination and tumor suppressor gene functions, both at Harvard and Harvard Medical School. He was an Associate Professor at the Medical School of University of Hong Kong with a research interest on viral oncogenesis. He also led research and development programs in biotech and biopharmaceutical companies, and is an inventor of more than 30 US patents on therapeutics, diagnostics, and bio-assay technologies. At NCI, he was initially involved in using the NGS technology to investigate the pathogenic mechanism of a fusion oncoprotein of in a childhood sarcoma. More recently, his group is focused on biomarkers for early stage drug development, on novel protein assays for treatment assessment, and on circulating tumor DNA analysis for patient selection and monitoring.

Areas of Expertise

1. Biomarkers studies in early drug development

2. Development of novel tumor antigen tests and protein biomarker analysis in clinical trials

3. Circulating tumor DNA analytical technologies in clinical trials

Contact Info

Liang Cao, Ph.D.
Center for Cancer Research
National Cancer Institute
Building 37, Room 6134
Bethesda, MD 20892-1906
Ph: 240-760-6893

1) Biomarkers studies in early drug development to cover areas including pharmacokinetics, pharmacodynamics, mechanism of action, dose-response relationship, and proof of therapeutic concept on novel cancer therapeutic agents. We have successful experiences with PK/PD analyses of various targeted and immunotherapy agents, including a first-in-class and first-in-human immune checkpoint inhibitor developed by EMD, Serono, and Merck group. 

2) Development of novel tumor antigen tests, including assay development, analytical and clinical validation, as well as generation of clinical data for intended clinical uses during drug development. We recently developed a new assay for the treatment assessment and progression evaluation of mesothelioma. We are engaged in multiple NCI intramural and NCI-sponsored extramural studies to evaluate the effectiveness of the assay for anti-mesothelin immunotherapies. 

3) Circulating tumor DNA analytic technologies in clinical trials, including digital PCR and digital NGS for exploratory research and for translation of circulating tumor DNA biomarkers into diagnostics. Our recent work has been examining cell-free DNA (cfDNA) in patients with HPV-associated cancer. Our work demonstrated the potential of cfDNA analysis in disease monitoring, and in providing the proof-of-concept data on a novel immunotherapy. Our results suggest the feasibility of cfDNA based patient genotyping for a T cell immunotherapy and the assay is being evaluated as a potential companion diagnostic. 


NIH Scientific Focus Areas:
Cancer Biology, Clinical Research, Genetics and Genomics, Immunology, Molecular Pharmacology

Selected Recent Publications

  1. Cao L, Alani E, Kleckner N
    Cell. 61: Cell Press 1089-1101, 1990. [ Journal Article ]
  2. Cao L, Faha B, Dembski M, Tsai LH, Harlow E, Dyson N
    Nature. 335: Nature Press 176-179, 1992.
  3. Cao L, Yu Y, Bilke S, Walker RL, Mayeenuddin LH, Pineda M, Azorsa DO, Yang F, Helman LJ, Meltzer PS
    Cancer Res. 70: American Association for Cancer Research 6497-6508, 2010. [ Journal Article ]
  4. Kang Z, Stevanović S, Hinrichs CS, Cao L
    Clin Cancer Res. 23: American Association for Cancer Research 6856-6862, 2017.
  5. Megakaryocyte potentiating factor as a predictive biomarker for therapies against malignant mesothelioma.
    Cao L, Yu Y, Thomas A, Zhang J, Onda M, Meltzer P, Hassan R, Pastan I.
    J Clin Oncol, Prec Oncol. 2: American Society of Clinical Oncology DOI: 10.1200/PO.17.00220, 2018.

Dr. Cao received his Ph.D. from the Department of Biochemistry and Molecular Biology at Harvard University. Subsequently, he was a Damon Runyon Cancer Research Fellow at the Cancer Center of Massachusetts General Hospital. He has managed cancer research, and drug discovery and development operations in academic and industry labs for 25 years. He is an inventor of over 30 US patents on assay technologies, diagnostics, and therapeutic targets and molecules.

Name Position
Zhigang Kang M.D., Ph.D. Postdoctoral Fellow (Contr)
Yunkai Yu M.S. Research Biologist