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Pier Paolo Pandolfi, MD, PhD

Director, Cancer Center
Director, Cancer Research Institute
Chief, Division of Genetics
Beth Israel Deaconess Medical Center
Reisman Endowed Chair of Medicine
Professor of Medicine and Pathology
Harvard Medical School
Boston, Massachusetts

Current Research

Goal: To develop new treatment strategies for metastatic breast cancer (MBC).

Impact: Dr. Pandolfi is developing molecules to block the development of invasive and metastatic breast cancer. After years of laboratory research that has led to the discovery of this target, his work is close to moving into clinical trials, where it can benefit patients with MBC.

What’s next: He and his colleagues have developed laboratory models to further validate their findings and hope to move to a clinical trial in the near future.

MBC is breast cancer that has spread beyond the breast to other locations in the body. While it can be treated for a period of time, MBC is an incurable disease and is the leading cause of breast cancer deaths. Dr. Pandolfi’s research is focused on a small molecule called miR-22, which he and his team have shown to drive aggressive metastatic disease. They have since developed laboratory models of MBC and are now using them to test a molecule that may reverse the effects of miR-22.

Full Research Summary

Research area: Identifying new targets and therapies in metastatic breast cancer.

Impact: Metastatic breast cancer is an incurable disease and the leading cause of breast cancer deaths. Dr. Pandolfi and his team have discovered that MicroRNA-22 (miR-22) is a major player in the development of breast cancer, specifically, in the mechanism that turns normal cells into metastatic cells. They have developed a series of new anti-miR-22 molecules that can reverse the effects of miR-22, thus preventing the metastatic spread to liver and lungs.

Current research: He and his colleagues are using the anti-miR-22 molecules previously developed to test specific therapeutic approaches for metastatic breast cancer.

What he’s learned so far: Dr. Pandolfi and his team have shown that miR-22 is a major player in both the initial development of breast cancer and the mechanisms that throw the “switch” to metastasis. They have developed a series of novel molecules that can target and reverse the effects of miR-22, which have been tested in breast cancer cells. They have developed a laboratory model for liver and lung metastasis for testing therapeutic approaches for metastatic breast cancer.

What’s next: The team will continue to conduct tests on the most promising anti-miR-22 molecule. They will translate the findings of these studies to clinical trials and begin the first RNA-based therapy for the treatment of metastatic breast cancer.

Biography

Pier Paolo Pandolfi received his MD and PhD from the University of Perugia, Italy. He received post-graduate training at the National Institute for Medical Research and the University of London in the UK. Dr. Pandolfi presently holds the Reisman Endowed Chair of Medicine, and is Professor of Medicine and Pathology at Harvard Medical School (HMS). He serves as Scientific Director of the Cancer Center at Beth Israel Deaconess Medical Center (BIDMC), the Director of the Cancer Genetics Program, and the Chief of the Division of Genetics in the Department of Medicine and is also a Member of the Department of Pathology at BIDMC. He was recently appointed to serve as the Cancer Center Director and the Director of the Cancer Research Institute at BIDMC and HMS.

Dr. Pandolfi has been the recipient of numerous awards throughout his career including the Pezcoller Foundation–AACR International Award for Cancer Research in 2011, the Scanno International Award for Medicine in 2012, the Pomilio Ethic International Award in Biomedicine, the European Foundation Guido Venosta Award for Cancer Research in 2013, and the America International Award in 2014. On June 2, 2015, Dr. Pandolfi was Knighted by the president of the Italian Republic and received the Medal of Honor as “Officer of the Order of the Star of Italy”.

The research carried out in Dr. Pandolfi’s laboratory has been seminal to elucidating the molecular mechanisms and the genetics underlying the pathogenesis of leukemias, lymphomas and solid tumors as well as in modeling these cancers in the laboratory. Dr. Pandolfi and colleagues have characterized the function of the fusion oncoproteins and the genes involved in the chromosomal translocations of acute promyelocytic leukemia (APL), as well as of major tumor suppressors such as PTEN and p53, and novel proto-oncogenes such as POKEMON. The elucidation of the molecular basis underlying APL pathogenesis has led to the development of novel and effective therapeutic strategies. As a result of these efforts, APL is now considered a curable disease. Additional novel therapeutic concepts have emerged from this work and are currently being tested in clinical trials. More recently, Dr. Pandolfi and colleagues have presented a new theory describing how mRNA, both coding and non-coding, exerts their biological functions with profound implications for human genetics, cell biology and cancer biology.

Grid Researcher Headshot - Pandolfi

BCRF Investigator Since

2015

Area(s) of Focus