A photo of Nancy Ratner.

Co-Director, Rasopathy Program

Program Leader, Cancer Biology and Neural Tumors Program

Beatrice C. Lampkin Chair, Cancer Biology

Professor, UC Department of Pediatrics



Biography & Affiliation


Neurofibromatosis is a very common genetic disorder affecting 1 out of 3,000 children and adults. NF1 patients can develop brain and nerve tumors that can compromise brain and nerve structure and function. We aim to pursue cancer research, and primarily study nerve tumors, with the goal of improving the quality of life of >110,000 people in the U.S., and many more worldwide.

My research interests include glial cell biology, intracellular signaling, preclinical therapeutics, and peripheral nerve tumorigenesis. My laboratory team and I share the goal of finding innovative treatments for neurofibromatosis by utilizing our knowledge of developmental biology together and levels of ribonucleic acid (RNA) and proteins in neurofibroma tumor and immune cell types as guides for drug selection and testing.

Some of the notable discoveries my team and I have made in our lab include: 1) developing and producing transcriptome data for investigating NF1 cells and nerve tumors and 2) pioneering the use of mitogen-activated protein kinase (MEK) inhibitors in a new and original preclinical model of the peripheral nerve tumor. This research led clinicians to pursue clinical trials, and in 2020, the Food and Drug Administration (FDA) granted approval for use of MEK inhibitors as a therapy for plexiform neurofibromas.

I also recruit and mentor neuro-oncology and rasopathy faculty at the Cincinnati Children’s Hospital Medical Center, and mentor students and postdoctoral fellows in my laboratory.

Throughout my career, I received awards, including:

  • von Recklinghausen award, the highest honor of the Children’s Tumor Foundation, 2010
  • Jacob Javits Neuroscience Investigator Award from the National Institute of Neurologic Disease and Stroke (NINDS) at the National Institutes of Health, 2014 - 2021
  • Team Science award, Children’s Tumor Foundation, 2016

I joined the Cincinnati Children’s Hospital Medical Center team in 2006. I now have more than 30 years of experience in Schwann cell biology and cancer research. My research has been consistently funded by the National Institutes of Health, the Department of Defense Program on Neurofibromatosis and other agencies. My work has been published in numerous journals, including Cancer Cell, Cell Reports, Scientific Reports, Cancer Research, PNAS and Clinical Cancer Research.

Clinical Interests

Preclinical testing in neurofibromatosis

Research Interests

Genetic mutations in tumor suppressor genes; development and tumorigenesis of the nervous system; peripheral NF1, NF2

Academic Affiliation

Professor, UC Department of Pediatrics

Research Divisions

Experimental Hematology and Cancer Biology, Cancer and Blood Diseases

Blog Posts

Inhibiting MEK Protein Shrinks NF1 Plexiform Tumors


Inhibiting MEK Protein Shrinks NF1 Plexiform Tumors

Nancy Ratner, PhD6/30/2019


PhD: Indiana University, 1982.

BA: Brown University, 1975.

Fellowship: Washington University St. Louis, 1982-1987.


Purinergic signaling in peripheral nervous system glial cells. Patritti-Cram, J; Coover, RA; Jankowski, MP; Ratner, N. GLIA. 2021; 69:1837-1851.

WNT5A inhibition alters the malignant peripheral nerve sheath tumor microenvironment and enhances tumor growth. Thomson, CS; Pundavela, J; Perrino, MR; Coover, RA; Choi, K; Chaney, KE; Rizvi, TA; Largaespada, DA; Ratner, N. Oncogene. 2021; 40:4229-4241.

Neurofibromatosis in the Era of Precision Medicine: Development of MEK Inhibitors and Recent Successes with Selumetinib. Galvin, R; Watson, AL; Largaespada, DA; Ratner, N; Osum, S; Moertel, CL. Current Oncology Reports. 2021; 23.

Transposon Mutagenesis-Guided CRISPR/Cas9 Screening Strongly Implicates Dysregulation of Hippo/YAP Signaling in Malignant Peripheral Nerve Sheath Tumor Development. Vélez-Reyes, GL; Koes, N; Ryu, JH; Kaufmann, G; Berner, M; Weg, MT; Wolf, NK; Rathe, SK; Ratner, N; Moriarity, BS; et al. Cancers. 2021; 13.

NF106: A Neurofibromatosis Clinical Trials Consortium Phase II Trial of the MEK Inhibitor Mirdametinib (PD-0325901) in Adolescents and Adults With NF1-Related Plexiform Neurofibromas. Weiss, BD; Wolters, PL; Plotkin, SR; Widemann, BC; Tonsgard, JH; Blakeley, J; Allen, JC; Schorry, E; Korf, B; Robison, NJ; et al. Journal of Clinical Oncology. 2021; 39:797-806.

MicroRNA-155 contributes to plexiform neurofibroma growth downstream of MEK. Na, Y; Hall, A; Choi, K; Hu, L; Rose, J; Coover, RA; Miller, A; Hennigan, RF; Dombi, E; Kim, MO; et al. Oncogene. 2021; 40:951-963.

A molecular basis for neurofibroma-associated skeletal manifestations in NF1. Ma, Y; Gross, AM; Dombi, E; Pemov, A; Choi, K; Chaney, K; Rhodes, SD; Angus, SP; Sciaky, N; Clapp, DW; et al. Genetics in Medicine. 2020; 22:1786-1793.

Brain-wide structural and functional disruption in mice with oligodendrocyte-specific Nf1 deletion is rescued by inhibition of nitric oxide synthase. Asleh, J; Shofty, B; Cohen, N; Kavushansky, A; Lopez-Juarez, A; Constantini, S; Ratner, N; Kahn, I. Proceedings of the National Academy of Sciences of the United States of America. 2020; 117:22506-22513.

HuR/ELAVL1 drives malignant peripheral nerve sheath tumor growth and metastasis. Palomo-Irigoyen, M; Pérez-Andrés, E; Iruarrizaga-Lejarreta, M; Barreira-Manrique, A; Tamayo-Caro, M; Vila-Vecilla, L; Moreno-Cugnon, L; Beitia, N; Medrano, D; Fernández-Ramos, D; et al. Journal of Clinical Investigation. 2020; 130:3848-3864.

After Nf1 loss in Schwann cells, inflammation drives neurofibroma formation. Fletcher, JS; Pundavela, J; Ratner, N. Neuro-Oncology Advances. 2020; 2:i23-i32.