Fabiana Perna, MD, PhD

Cancer Focus:
Hodgkin Lymphoma, Leukemia, Multiple Myeloma-Plasma Cell Tumor, Myelodysplastic Syndromes (MDS), Non-Hodgkin Lymphoma

 Dr. Perna received her MD degree from Federico II University, Naples, Italy, where she also completed residency and clinical fellowship. Dr. Perna completed PhD program and Post-doctoral Research Fellowships under the mentorship of Dr. Stephen Nimer (2008-2012) and Michel Sadelain (2013-2019) at Memorial Sloan Kettering Cancer Center, New York City, New York. She joins us from Indiana University School of Medicine, where she completed additional clinical fellowship and joined the faculty as an Associate Professor in the Division of Hematology/Oncology. Dr. Perna’s research and clinical interests are on the pathogenesis and treatment of hematological malignancies including Chimeric Antigen Receptor (CAR) T cell therapy. Her laboratory investigates how essential genetic and epigenetic determinants of cancer shape the cell surfaceome, thus providing targets for promoting carcinogenesis and use of precision immunotherapy. She has published extensively on the translational aspects of CAR T cell therapy, including first and senior authorship of key manuscripts in Cancer Cell, Blood, Cell Reports Medicine, and Molecular Therapy. Her extensive training and expertise include creation of new CAR T cells and clinical translation of these research discoveries through Investigational New Drug (IND) applications at the FDA. The Perna lab is funded by multiple grants from the National Cancer Institute and licensing agreements to translate novel CAR T cell treatments into clinical trials for patients with hematologic malignancies. 

Education & Training

Fellowship:

• Memorial Sloan Kettering Cancer Center, New York, NY -
• Memorial Sloan Kettering Cancer Center, New York, NY -

Residency:

• Federico II University, Naples, Italy -

Medical School:

• Federico II University, Naples, Italy - MD

• Weber E, Bleakley M, Cronk JC, Shah NN, Perna F. Alternative immune effector cells picking up speed. Mol Ther. 2024 Mar.32(3):563-564. Pubmedid: 38340733. Pmcid: PMC10928136.
• Kale B, Khosravi-Maharlooei M, Perna F. Shifting gears with CAR T cells for autoimmune diseases. Mol Ther. 2024 Feb.32(2):261-263. Pubmedid: 38244538. Pmcid: PMC10862003.
• Marino S, Petrusca DN, Bishop RT, Anderson JL, Sabol HM, Ashby C, Layer JH, Cesarano A, Davé UP, Perna F, Delgado-Calle J, Chirgwin JM, Roodman GD. Pharmacologic targeting of the p62 ZZ domain enhances both anti-tumor and bone-anabolic effects of bortezomib in multiple myeloma. Haematologica. 2023 Nov. Pubmedid: 37981834.
• Ravn-Boess N, Roy N, Hattori T, Bready D, Donaldson H, Lawson C, Lapierre C, Korman A, Rodrick T, Liu E, Frenster JD, Stephan G, Wilcox J, Corrado AD, Cai J, Ronnen R, Wang S, Haddock S, Sabio Ortiz J, Mishkit O, Khodadadi-Jamayran A, Tsirigos A, Fenyö D, Zagzag D, Drube J, Hoffmann C, Perna F, Jones DR, Possemato R, Koide A, Koide S, Park CY, Placantonakis DG. The expression profile and tumorigenic mechanisms of CD97 (ADGRE5) in glioblastoma render it a targetable vulnerability. Cell Rep. 2023 Nov.42(11):113374. Pubmedid: 37938973. Pmcid: PMC10841603.
• Santomasso BD, Gust J, Perna F. How I treat unique and difficult-to-manage cases of CAR T-cell therapy-associated neurotoxicity. Blood. 2023 May.141(20):2443-2451. Pubmedid: 36877916. Pmcid: PMC10329188.
• Schorr C, Forindez J, Espinoza-Gutarra M, Mehta R, Grover N, Perna F. Thrombotic Events Are Unusual Toxicities of Chimeric Antigen Receptor T-Cell Therapies. Int J Mol Sci. 2023 May.24(9). Pubmedid: 37176053. Pmcid: PMC10179014.
• Palam LR, Ramdas B, Pickerell K, Pasupuleti SK, Kanumuri R, Cesarano A, Szymanski M, Selman B, Dave UP, Sandusky G, Perna F, Paczesny S, Kapur R. Loss of Dnmt3a impairs hematopoietic homeostasis and myeloid cell skewing via the PI3Kinase pathway. JCI Insight. 2023 May.8(9). Pubmedid: 36976647. Pmcid: PMC10243813.
• Schorr C, Perna F. Tailoring CAR T cell technology for T cell leukemia. Mol Ther. 2023 Mar.31(3):603-604. Pubmedid: 36803952. Pmcid: PMC10014257.
• Di Meo F, Iyer A, Akama K, Cheng R, Yu C, Cesarano A, Kurihara N, Tenshin H, Aljoufi A, Marino S, Soni RK, Roda J, Sissons J, Vu LP, Guzman M, Huang K, Laskowski T, Broxmeyer HE, Roodman DG, Perna F. A target discovery pipeline identified ILT3 as a target for immunotherapy of multiple myeloma. Cell Rep Med. 2023 Jul.4(7):101110. Pubmedid: 37467717. Pmcid: PMC10394163.
• Ardeshir-Larijani F, Schneider BP, Althouse SK, Radovich M, Masood A, Perna F, Salman H, Loehrer PJ. Clinicogenomic Landscape of Metastatic Thymic Epithelial Tumors. JCO Precis Oncol. 2023 Feb.7:e2200465. Pubmedid: 36787505. Pmcid: PMC10309539.
• Nguyen HP, Le AQ, Liu E, Cesarano A, DiMeo F, Perna F, Kapur R, Walker BA, Tran NT. Protein arginine methyltransferase 1 is a therapeutic vulnerability in multiple myeloma. Front Immunol. 2023 Aug.14:1239614. Pubmedid: 37600810. Pmcid: PMC10436492.
• Battistello E, Hixon KA, Comstock DE, Collings CK, Chen X, Rodriguez Hernaez J, Lee S, Cervantes KS, Hinkley MM, Ntatsoulis K, Cesarano A, Hockemeyer K, Haining WN, Witkowski MT, Qi J, Tsirigos A, Perna F, Aifantis I, Kadoch C. Stepwise activities of mSWI/SNF family chromatin remodeling complexes direct T cell activation and exhaustion. Mol Cell. 2023 Apr.83(8):1216-1236.e12. Pubmedid: 36944333. Pmcid: PMC10121856.
• Perna F, Espinoza-Gutarra MR, Bombaci G, Farag SS, Schwartz JE. Immune-Based Therapeutic Interventions for Acute Myeloid Leukemia. Cancer Treat Res. 2022 May.183:225-254. Pubmedid: 35551662.
• Zhang J, Luo J, Weng X, Zhu Y, Goyal G, Perna F, Espinoza-Gutarra M, Jiang L, Chen L, Mi JQ. A case report of the metagenomics next-generation sequencing for early detection of central nervous system mucormycosis with successful rescue in patient with recurrent chronic lymphocytic leukemia. Ann Transl Med. 2022 Jun.10(12):722. Pubmedid: 35845522. Pmcid: PMC9279813.
• Atallah-Yunes SA, Robertson MJ, Davé UP, Ghione P, Perna F. Novel Immune-Based treatments for Diffuse Large B-Cell Lymphoma: The Post-CAR T Cell Era. Front Immunol. 2022 Jun.13:901365. Pubmedid: 35720352. Pmcid: PMC9198279.
• Schorr C, Perna F. Targets for chimeric antigen receptor T-cell therapy of acute myeloid leukemia. Front Immunol. 2022 Dec.13:1085978. Pubmedid: 36605213. Pmcid: PMC9809466.
• Dong C, Cesarano A, Bombaci G, Reiter JL, Yu CY, Wang Y, Jiang Z, Zaid MA, Huang K, Lu X, Walker BA, Perna F, Liu Y. Intron retention-induced neoantigen load correlates with unfavorable prognosis in multiple myeloma. Oncogene. 2021 Oct.40(42):6130-6138. Pubmedid: 34504297. Pmcid: PMC8426332.
• Perna F. Safety Starts with Selecting the Targets. Mol Ther. 2021 Feb.29(2):424-425. Pubmedid: 33472030. Pmcid: PMC7854345.
• Haubner S, Perna F, Köhnke T, Schmidt C, Berman S, Augsberger C, Schnorfeil FM, Krupka C, Lichtenegger FS, Liu X, Kerbs P, Schneider S, Metzeler KH, Spiekermann K, Hiddemann W, Greif PA, Herold T, Sadelain M, Subklewe M. Coexpression profile of leukemic stem cell markers for combinatorial targeted therapy in AML. Leukemia. 2019 Jan.33(1):64-74. Pubmedid: 29946192. Pmcid: PMC6326956.
• Boulad F, Shore T, van Besien K, Minniti C, Barbu-Stevanovic M, Fedus SW, Perna F, Greenberg J, Guarneri D, Nandi V, Mauguen A, Yazdanbakhsh K, Sadelain M, Shi PA. Safety and efficacy of plerixafor dose escalation for the mobilization of CD34+ hematopoietic progenitor cells in patients with sickle cell disease: interim results. Haematologica. 2018 Sep.103(9):1577. Pubmedid: 30171018. Pmcid: PMC6119146.
• Boulad F, Shore T, van Besien K, Minniti C, Barbu-Stevanovic M, Fedus SW, Perna F, Greenberg J, Guarneri D, Nandi V, Mauguen A, Yazdanbakhsh K, Sadelain M, Shi PA. Safety and efficacy of plerixafor dose escalation for the mobilization of CD34+ hematopoietic progenitor cells in patients with sickle cell disease: interim results. Haematologica. 2018 May.103(5):770-777. Pubmedid: 29419425. Pmcid: PMC5927989.
• Perna F, Berman SH, Soni RK, Mansilla-Soto J, Eyquem J, Hamieh M, Hendrickson RC, Brennan CW, Sadelain M. Integrating Proteomics and Transcriptomics for Systematic Combinatorial Chimeric Antigen Receptor Therapy of AML. Cancer Cell. 2017 Oct.32(4):506-519.e5. Pubmedid: 29017060. Pmcid: PMC7025434.
• Ghosh A, Smith M, James SE, Davila ML, Velardi E, Argyropoulos KV, Gunset G, Perna F, Kreines FM, Levy ER, Lieberman S, Jay HV, Tuckett AZ, Zakrzewski JL, Tan L, Young LF, Takvorian K, Dudakov JA, Jenq RR, Hanash AM, Motta AC, Murphy GF, Liu C, Schietinger A, Sadelain M, van den Brink MR. Donor CD19 CAR T cells exert potent graft-versus-lymphoma activity with diminished graft-versus-host activity. Nat Med. 2017 Feb.23(2):242-249. Pubmedid: 28067900. Pmcid: PMC5528161.
• Sansone P, Berishaj M, Rajasekhar VK, Ceccarelli C, Chang Q, Strillacci A, Savini C, Shapiro L, Bowman RL, Mastroleo C, De Carolis S, Daly L, Benito-Martin A, Perna F, Fabbri N, Healey JH, Spisni E, Cricca M, Lyden D, Bonafé M, Bromberg J. Evolution of Cancer Stem-like Cells in Endocrine-Resistant Metastatic Breast Cancers Is Mediated by Stromal Microvesicles. Cancer Res. 2017 Apr.77(8):1927-1941. Pubmedid: 28202520. Pmcid: PMC5392366.
• Liu F, Wang L, Perna F, Nimer SD. Beyond transcription factors: how oncogenic signalling reshapes the epigenetic landscape. Nat Rev Cancer. 2016 Jun.16(6):359-372. Pubmedid: 27220480. Pmcid: PMC5548460.
• Sansone P, Ceccarelli C, Berishaj M, Chang Q, Rajasekhar VK, Perna F, Bowman RL, Vidone M, Daly L, Nnoli J, Santini D, Taffurelli M, Shih NN, Feldman M, Mao JJ, Colameco C, Chen J, DeMichele A, Fabbri N, Healey JH, Cricca M, Gasparre G, Lyden D, Bonafé M, Bromberg J. Self-renewal of CD133(hi) cells by IL6/Notch3 signalling regulates endocrine resistance in metastatic breast cancer. Nat Commun. 2016 Feb.7:10442. Pubmedid: 26858125. Pmcid: PMC4748123.
• Perna F, Sadelain M. Myeloid leukemia switch as immune escape from CD19 chimeric antigen receptor (CAR) therapy. Transl Cancer Res. 2016 Aug.5(Suppl 2):S221-S225. Pubmedid: 28824851. Pmcid: PMC5558892.
• Liu F, Cheng G, Hamard PJ, Greenblatt S, Wang L, Man N, Perna F, Xu H, Tadi M, Luciani L, Nimer SD. Arginine methyltransferase PRMT5 is essential for sustaining normal adult hematopoiesis. J Clin Invest. 2015 Sep.125(9):3532-3544. Pubmedid: 26258414. Pmcid: PMC4588241.
• Zhao Z, Condomines M, van der Stegen SJC, Perna F, Kloss CC, Gunset G, Plotkin J, Sadelain M. Structural Design of Engineered Costimulation Determines Tumor Rejection Kinetics and Persistence of CAR T Cells. Cancer Cell. 2015 Oct.28(4):415-428. Pubmedid: 26461090. Pmcid: PMC5003056.
• Kotini AG, Chang CJ, Boussaad I, Delrow JJ, Dolezal EK, Nagulapally AB, Perna F, Fishbein GA, Klimek VM, Hawkins RD, Huangfu D, Murry CE, Graubert T, Nimer SD, Papapetrou EP. Functional analysis of a chromosomal deletion associated with myelodysplastic syndromes using isogenic human induced pluripotent stem cells. Nat Biotechnol. 2015 Jun.33(6):646-655. Pubmedid: 25798938. Pmcid: PMC4464949.
• Perna F, Vu LP, Themeli M, Kriks S, Hoya-Arias R, Khanin R, Hricik T, Mansilla-Soto J, Papapetrou EP, Levine RL, Studer L, Sadelain M, Nimer SD. The polycomb group protein L3MBTL1 represses a SMAD5-mediated hematopoietic transcriptional program in human pluripotent stem cells. Stem Cell Reports. 2015 Apr.4(4):658-669. Pubmedid: 25754204. Pmcid: PMC4400644.
• Masarone M, De Renzo A, La Mura V, Sasso FC, Romano M, Signoriello G, Rosato V, Perna F, Pane F, Persico M. Management of the HBV reactivation in isolated HBcAb positive patients affected with Non Hodgkin Lymphoma. BMC Gastroenterol. 2014 Feb.14:31. Pubmedid: 24533834. Pmcid: PMC3938973.
• Themeli M, Kloss CC, Ciriello G, Fedorov VD, Perna F, Gonen M, Sadelain M. Generation of tumor-targeted human T lymphocytes from induced pluripotent stem cells for cancer therapy. Nat Biotechnol. 2013 Oct.31(10):928-933. Pubmedid: 23934177. Pmcid: PMC5722218.
• Vu LP, Perna F, Wang L, Voza F, Figueroa ME, Tempst P, Erdjument-Bromage H, Gao R, Chen S, Paietta E, Deblasio T, Melnick A, Liu Y, Zhao X, Nimer SD. PRMT4 blocks myeloid differentiation by assembling a methyl-RUNX1-dependent repressor complex. Cell Rep. 2013 Dec.5(6):1625-1638. Pubmedid: 24332853. Pmcid: PMC4073674.
• Bazzoli E, Pulvirenti T, Oberstadt MC, Perna F, Wee B, Schultz N, Huse JT, Fomchenko EI, Voza F, Tabar V, Brennan CW, DeAngelis LM, Nimer SD, Holland EC, Squatrito M. MEF promotes stemness in the pathogenesis of gliomas. Cell Stem Cell. 2012 Dec.11(6):836-844. Pubmedid: 23217424. Pmcid: PMC3812924.
• Battipaglia G, Avilia S, Morelli E, Caranci F, Perna F, Camera A. Posterior reversible encephalopathy syndrome (PRES) during induction chemotherapy for acute myeloblastic leukemia (AML). Ann Hematol. 2012 Aug.91(8):1327-1328. Pubmedid: 22237936.
• Abdel-Wahab O, Adli M, LaFave LM, Gao J, Hricik T, Shih AH, Pandey S, Patel JP, Chung YR, Koche R, Perna F, Zhao X, Taylor JE, Park CY, Carroll M, Melnick A, Nimer SD, Jaffe JD, Aifantis I, Bernstein BE, Levine RL. ASXL1 mutations promote myeloid transformation through loss of PRC2-mediated gene repression. Cancer Cell. 2012 Aug.22(2):180-193. Pubmedid: 22897849. Pmcid: PMC3422511.
• Moulick K, Ahn JH, Zong H, Rodina A, Cerchietti L, Gomes DaGama EM, Caldas-Lopes E, Beebe K, Perna F, Hatzi K, Vu LP, Zhao X, Zatorska D, Taldone T, Smith-Jones P, Alpaugh M, Gross SS, Pillarsetty N, Ku T, Lewis JS, Larson SM, Levine R, Erdjument-Bromage H, Guzman ML, Nimer SD, Melnick A, Neckers L, Chiosis G. Affinity-based proteomics reveal cancer-specific networks coordinated by Hsp90. Nat Chem Biol. 2011 Sep.7(11):818-826. Pubmedid: 21946277. Pmcid: PMC3265389.
• Hoya-Arias R, Tomishima M, Perna F, Voza F, Nimer SD. L3MBTL1 deficiency directs the differentiation of human embryonic stem cells toward trophectoderm. Stem Cells Dev. 2011 Nov.20(11):1889-1900. Pubmedid: 21341991. Pmcid: PMC3202892.
• Risitano AM, Perna F, Selleri C. Achievements and limitations of complement inhibition by eculizumab in paroxysmal nocturnal hemoglobinuria: the role of complement component 3. Mini Rev Med Chem. 2011 Jun.11(6):528-535. Pubmedid: 21561403.
• Risitano AM, Perna F. Aplastic anemia: immunosuppressive therapy in 2010. Pediatr Rep. 2011 Jun.3 Suppl 2(Suppl 2):e7. Pubmedid: 22053283. Pmcid: PMC3206528.
• Moran-Crusio K, Reavie L, Shih A, Abdel-Wahab O, Ndiaye-Lobry D, Lobry C, Figueroa ME, Vasanthakumar A, Patel J, Zhao X, Perna F, Pandey S, Madzo J, Song C, Dai Q, He C, Ibrahim S, Beran M, Zavadil J, Nimer SD, Melnick A, Godley LA, Aifantis I, Levine RL. Tet2 loss leads to increased hematopoietic stem cell self-renewal and myeloid transformation. Cancer Cell. 2011 Jul.20(1):11-24. Pubmedid: 21723200. Pmcid: PMC3194039.
• Liu F, Zhao X, Perna F, Wang L, Koppikar P, Abdel-Wahab O, Harr MW, Levine RL, Xu H, Tefferi A, Deblasio A, Hatlen M, Menendez S, Nimer SD. JAK2V617F-mediated phosphorylation of PRMT5 downregulates its methyltransferase activity and promotes myeloproliferation. Cancer Cell. 2011 Feb.19(2):283-294. Pubmedid: 21316606. Pmcid: PMC4687747.
• Perna F, Abdel-Wahab O, Levine RL, Jhanwar SC, Imada K, Nimer SD. ETV6-ABL1-positive "chronic myeloid leukemia": clinical and molecular response to tyrosine kinase inhibition. Haematologica. 2011 Feb.96(2):342-343. Pubmedid: 21193423. Pmcid: PMC3031707.
• Wang L, Gural A, Sun XJ, Zhao X, Perna F, Huang G, Hatlen MA, Vu L, Liu F, Xu H, Asai T, Xu H, Deblasio T, Menendez S, Voza F, Jiang Y, Cole PA, Zhang J, Melnick A, Roeder RG, Nimer SD. The leukemogenicity of AML1-ETO is dependent on site-specific lysine acetylation. Science. 2011 Aug.333(6043):765-769. Pubmedid: 21764752. Pmcid: PMC3251012.
• Strianese D, Tranfa F, Finelli M, De Renzo A, Staibano S, Schiemer R, Cardone D, Pacelli R, Perna F, Mascolo M, De Rosa G, Bonavolontà G. Hepatitis C virus infection in ocular adnexal lymphomas. Arch Ophthalmol. 2010 Oct.128(10):1295-1299. Pubmedid: 20937999.
• Perna F, Gurvich N, Hoya-Arias R, Abdel-Wahab O, Levine RL, Asai T, Voza F, Menendez S, Wang L, Liu F, Zhao X, Nimer SD. Depletion of L3MBTL1 promotes the erythroid differentiation of human hematopoietic progenitor cells: possible role in 20q- polycythemia vera. Blood. 2010 Oct.116(15):2812-2821. Pubmedid: 20585043. Pmcid: PMC2974589.
• Storto G, De Renzo A, Pellegrino T, Perna F, De Falco T, Erra P, Nardelli A, Speranza A, Klain M, Rotoli B, Pace L. Assessment of metabolic response to radioimmunotherapy with 90Y-ibritumomab tiuxetan in patients with relapsed or refractory B-cell non-Hodgkin lymphoma. Radiology. 2010 Jan.254(1):245-252. Pubmedid: 20032155.
• Gurvich N, Perna F, Farina A, Voza F, Menendez S, Hurwitz J, Nimer SD. L3MBTL1 polycomb protein, a candidate tumor suppressor in del(20q12) myeloid disorders, is essential for genome stability. Proc Natl Acad Sci U S A. 2010 Dec.107(52):22552-22557. Pubmedid: 21149733. Pmcid: PMC3012470.
• La Mura V, De Renzo A, Perna F, D'Agostino D, Masarone M, Romano M, Bruno S, Torella R, Persico M. Antiviral therapy after complete response to chemotherapy could be efficacious in HCV-positive non-Hodgkin's lymphoma. J Hepatol. 2008 Oct.49(4):557-563. Pubmedid: 18678434.
• De Renzo A, Perna F, Persico M, Notaro R, Mainolfi C, de Sio I, Ciancia G, Picardi M, Del Vecchio L, Pane F, Rotoli B. Excellent prognosis and prevalence of HCV infection of primary hepatic and splenic non-Hodgkin's lymphoma. Eur J Haematol. 2008 Jul.81(1):51-57. Pubmedid: 18397390.
• De Renzo A, Perna F, Persico M, Mainolfi C, Pace L. 18F-fluorodeoxyglucose positron emission tomography/computed tomography in the evaluation of early response in a primary hepatic lymphoma. Brit J Haematol. 2006 Jun.133(6):580. Pubmedid: 16704431.

Title: Alternatively spliced cell surface proteins as drivers of leukemogenesis and targets for immunotherapy
Sponsor: Nat Institutes of Health
PI: Perna, F.