Pier Paolo Pandolfi, MD, PhD, is Director of the Cancer Center and Cancer Research Institute at BIDMC. He holds appointments in the Departments of Pathology and Medicine at BIDMC and is Victor J. Aresty Professor of Medicine at Harvard Medical School.
Pandolfi is noted for his research on the molecular mechanisms and genetics underlying the pathogenesis of leukemias and solid tumors. In particular, the Pandolfi laboratory elucidated fusion oncoproteins and genes involved in the chromosomal translocations of acute promyelocytic leukemia (APL). These insights led to the development of effective therapies and APL is now considered curable.
The laboratory's research also yielded novel insights into the role of tumor suppressor genes in the development of human cancer. They characterized the genetics of major tumor suppressors such as PTEN and p53, and cancer genes such as POKEMON and INPP4B. Dr. Pandolfi's extensive modeling of cancer in the mouse resulted in new discovery platforms that are used to evaluate drugs and predict the course of disease, treatment and resistance.
More recently, the Pandolfi Laboratory presented a theory describing how RNAs exert biological functions with profound implications for human genetics, cell biology and cancer biology. The laboratory demonstrated the critical role the vast and novel non-coding RNA genetic dimension plays in tumorigenesis. This work included the functional characterization of oncogenic pseudogenes, miRNAs and ceRNAs, lincRNAs, circRNAs, fusion circRNAs, and even small peptides encoded by lincRNAs.
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Selected Recent Publications
A.C. Bester, J.D. Lee, A. Chavez, Y.R. Lee, D. Nachmani, S. Vora, J. Victor, M. Sauvageau, J.L. Rinn, P. Provero, G.M. Church, , J.G. Clohessy, P.P. Pandolfi. An Integrated Genome Wide CRISPRa Approach to Study Drug Resistance Uncovers Coding and Noncoding Networks. Cell, 2018 Apr Cell. 2018 Apr 19;173(3):649-664.
M. Bezzi, N. Seitzer, T. Ishikawa, M. Reschke, M. Chen, G. Wang, C. Mitchell, C. Ng, J.M. Katon, A. Lunardi, S. Signoretti, J.G. Clohessy, J. Zhang, P.P. Pandolfi. “Diverse genetic-driven immune landscapes dictate tumor progression through distinct mechanisms.” Nature Medicine. 2018 Feb;24(2):165-175.
M. Chen, J. Zhang, K. Sampieri, J.G. Clohessy, L. Mendez, E. Gonzalez-Billalabeitia, X.S. Liu, Y. Lee, J. Fung, J.M. Katon, A. Venugopal Menon, K.A. Webster, C. Ng, M. Dilia Palumbieri, M.S. Diolombi, S.B. Breitkopf, J. Teruya-Feldstein, S. Signoretti, R.T. Bronson, J.M. Asara, M. Castillo-Martin, C. Cordon-Cardo, P.P. Pandolfi. “An aberrant SREBP-dependent lipogenic program promotes metastatic prostate cancer.” Nat Genetics 2018 Feb;50(2):206-218.
M. Chen, L. Wan, J. Zhang, J. Zhang, L. Mendez, JG. Clohessy, K. Berry, J. Victor, Q. Yuan, W. Wei and P.P. Pandolfi. “Deregulated PP1α phosphatase activity towards MAPK activation is antagonized by a tumor suppressive failsafe mechanism.” Nat Commun. 2018 Jan 15;9(1):159
J. Guarnerio, L.M. Mendez, N. Asada, A. Venugopal Menon, J. Fung, K. Berry, P.S. Frenette, K. Ito, P.P. Pandolfi. “A non-cell-autonomous role for Pml in the maintenance of leukemia from the niche.” Nat.Comm. 2018 Jan 4;9(1):66
A. Matsumoto, A. Pasut, M. Matsumoto, R. Yamashita, J. Fung, E. Monteleone, A. Saghatelian, K.I. Nakayama, J.G. Clohessy, P.P. Pandolfi. “mTORC1 and muscle regeneration are regulated by the LINC00961-encoded SPAR polypeptide.” Nature. 2017 Jan 12;541(7636):228-23.
J. Guarnerio, M. Bezzi, J.C. Jeong, S.V. Paffenholz, K. Berry, M.M. Naldini, F. Lo-Coco, Y. Tay, A.H. Beck, P.P. Pandolfi. “Oncogenic Role of Fusion-circRNAs Derived from Cancer-Associated Chromosomal Translocations”. Cell. 2016 Apr 7;165(2):289-302.
View more publications in PubMed