Michael C. Milone, MD, PhD

Associate Professor of Pathology and Laboratory Medicine at the Hospital of the University of Pennsylvania
Associate Director, Toxicology Laboratory
University of Pennsylvania Perelman School of Medicine

Contact Information3400 Spruce Street 7103 Founders Pavilion
Philadelphia, PA 19104
Office: 215-662-6575
Fax: 215-662-7529


Specialty Division

Laboratory Medicine, Immunobiology and Experimental Pathology

Research Expertise

Research in my laboratory is focused on T cell biology and applications of genetically-engineered T cells to the treatment of disease. Active research projects in my laboratory include:

1) Developing chimeric antigen receptors (CARs) for adoptive immunotherapy of cancer with enhanced function and improved safety.

2) Developing and applying synthetic immunoreceptors to the treatment of immune-mediated disease.

3) Exploring the role of co-stimulatory signals in directing T cell metabolism and the way this metabolism affects T cell function within tumors.


Clinical Expertise

Clinical Laboratory Toxicology, Therapeutic Drug Monitoring, Clinical Chemistry, Blood Banking/Transfusion Medicine, Therapeutic Apheresis

Itmat Expertise

My laboratory is focused upon developing methods to use the immune system to treat cancer. Active research includes 1) exploring the mechanical properties of artificial adhesion substrates used in T cell culture and their influence on T cell biology, 2) development of artificial antigen receptors and signaling systems for control of T cell specificity

Graduate Groups

Cell and Molecular Biology


B.S (Chemical Biology), Stevens Institute of Technology, 1993
M.D. (Medical Doctor), UMDNJ- New Jersey Medical School, 1999
Ph.D (Experimental Pathology/ Immunology), UMDNJ - Graduate School of Biomedical Sciences, 1999

Specialty Certification

Clinical Pathology, American Board of Pathology, 2002

Postgraduate Training

Internship, Internal Medicine, Hospital of the University of Pennsylvania, 1999-2000
Resident, Clinical Pathology, Hospital of the University of Pennsylvania, 2000-2002
Chief Resident, Division of Laboratory Medicine, Hospital of the University of Pennsylvania, 2001-2002
Clinical Fellowship, Blood Banking and Transfusion Medicine, Hospital of the University of Pennsylvania, 2002-2003
Postdoctoral. Research Fellow, Translational Research Program, Abramson Family Cancer Institute, University of Pennsylvania School of Medicine, 2003-2007
Clinical Fellow, Clinical Chemistry and Laboratory Toxicology, Hospital of the University of Pennsylvania, 2004-2007

Awards and Honors

Stanley S. Bergen Award for Academic Excellence, UMDNJ, 1996
Faculty Organization Award, UMDNJ-New Jersey Medical School, 1999
Stanely S. Bergen Medal of Excellence, UMDNJ, 1999
The Peter C. Nowell Teaching Award, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, 2011
Top Ten Clinical Research Achievement Award, Clinical Research Forum, 2017
Top 20 Clinical Research Papers for 2019, Clinical Research Forum, 2019
Though Leader Award, Agilent Technologies, 2020

Memberships and Professional Organizations

American Society of Clinical Pharmacology and Therapeutics, 2007 - 2010
American Association of Clinical Chemistry, 2007 - Present
College of American Pathologists, 2008 - Present
Academy of Clinical Laboratory Physicians and Scientists, 2008 - 2015
American Society of Hematology, 2008 - Present
International Association of Therapeutic Drug Monitoring and Clinical Toxicology, 2009 - Present
American Society of Gene and Cell Therapy, 2009 - Present
American Association of Immunologists, 2009 - Present
American Society of Clinical Pathology, 2015 - Present
National Institute for innovation in Manufacturing Biopharmaceuticals (NIIMBL), 2017 - Present

Web Links

Selected Publications

Efficacy and Safety of Hydroxychloroquine vs Placebo for Pre-exposure SARS-CoV-2 Prophylaxis Among Health Care Workers: A Randomized Clinical Trial

Abella BS, Jolkovsky EL, Biney BT, Uspal JE, Hyman MC, Frank I, Hensley SE, Gill S, Vogl DT, Maillard I, Babushok DV, Huang AC, Nasta SD, Walsh JC, Wiletyo EP, Gimotty PA, Milone MC, Amaravadi RK; Prevention and Treatment of COVID-19 With Hydroxychloroquine (PATCH) Investigators., JAMA Intern Med 181(): 195-202, 2021, PMID:33001138

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Pharmacology of Chimeric Antigen Receptor-Modified T Cells

Song EZ, Milone MC., Annu Rev Pharmacol Toxicol 61(): 805-829, 2021, PMID:33035447

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Adoptive T cell immunotherapy for medullary thyroid carcinoma targeting GDNF family receptor alpha 4

Bhoj VG, Li L, Parvathaneni K, Zhang Z, Kacir S, Arhontoulis D, Zhou K, McGettigan-Croce B, Nunez-Cruz S, Gulendran G, Boesteanu AC, Johnson L, Feldman MD, Radaelli E, Mansfield K, Nasrallah M, Goydel RS, Peng H, Rader C, Milone MC, Siegel DL., Mol Ther Oncolytics 20(): 387-398, 2021, PMID:33614919

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Antigen-specific B cell depletion for precision therapy of mucosal pemphigus vulgaris

Lee J, Lundgren DK, Mao X, Manfredo-Vieira S, Nunez-Cruz S, Williams EF, Assenmacher CA, Radaelli E, Oh S, Wang B, Ellebrecht CT, Fraietta JA, Milone MC, Payne AS., J Clin Invest 130(): 6317-6324, 2020, PMID:32817591

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Itacitinib (INCB039110), a JAK1 Inhibitor, Reduces Cytokines Associated with Cytokine Release Syndrome Induced by CAR T-cell Therapy

Huarte E, O'Connor RS, Peel MT, Nunez-Cruz S, Leferovich J, Juvekar A, Yang YO, Truong L, Huang T, Naim A, Milone MC, Smith PA., Clin Cancer Res 26(): 6299-6309, 2020, PMID:32998963

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Stable HLA antibodies following sustained CD19+ cell depletion implicate a long-lived plasma cell source

Zhang Z, Schuster SJ, Lacey SF, Milone MC, Monos D, Bhoj VG., Blood Adv 4(): 4292-4295, 2020, PMID:32915973

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Enhancing Chimeric Antigen Receptor T Cell Anti-tumor Function through Advanced Media Design

Ghassemi S, Martinez-Becerra FJ, Master AM, Richman SA, Heo D, Leferovich J, Tu Y, García-Cañaveras JC, Ayari A, Lu Y, Wang A, Rabinowitz JD, Milone MC, June CH, O'Connor RS., Mol Ther Methods Clin Dev 18(): 595-606, 2020, PMID:32775494

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Ligand-Induced Degradation of a CAR Permits Reversible Remote Control of CAR T Cell Activity In Vitro and In Vivo

Richman, S. A., Wang, L. C., Khire, U. R., Albelda, S. M., Milone, M. C., Mol Ther, 2020, PMID:32559430

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4-1BB costimulation promotes CAR T cell survival through noncanonical NF-κB signaling.

Philipson Benjamin I, O'Connor Roddy S, May Michael J, June Carl H, Albelda Steven M, Milone Michael C, Science signaling 13(625): , 2020, PMID:32234960

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Imaging CAR T Cell Trafficking with eDHFR as a PET Reporter Gene.

Sellmyer Mark A, Richman Sarah A, Lohith Katheryn, Hou Catherine, Weng Chi-Chang, Mach Robert H, O'Connor Roddy S, Milone Michael C, Farwell Michael D, Molecular therapy : the journal of the American Society of Gene Therapy 28(1): 42-51, 2020, PMID:31668558

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