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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

  • Seeking new therapeutic approaches for the treatment of metastatic breast cancer.
  • Laboratory studies are ongoing to validate a promising RNA-based therapy.
  • If successful, this strategy could move quickly to clinical trials to benefit breast cancer patients.

Stage IV or metastatic breast cancer—breast cancer that have spread to different organs—is an incurable disease and the leading cause of breast cancer deaths. While new therapies have been approved and are improving the lives of patients, new treatments are urgently needed. Dr. Pandolfi is conducting studies to test a novel treatment approach that is showing promising results in laboratory studies and could quickly advance to clinical trials for patients with metastatic breast cancer.

Full Research Summary

RNA molecules that do not encode proteins (called non-coding RNAs) are critical in the development of human cancer, and as such offer new opportunities for cancer treatment and prevention. Dr. Pandolfi identified a non-coding RNA designated as miR-22 as a potent tumorigenic molecule that promotes the development of invasive and metastatic breast cancer. In laboratory studies, they have shown that high amounts of miR-22 lead to tumor growth and spread, while inhibition of miR-22 blocks tumor growth.
The team previously developed an anti-miR-22 molecule that can reverse the effects of miR-22.  They have successfully tested this molecule in cultured cancer cell lines, further demonstrating that targeting miR-22 could be an effective therapy for metastatic breast cancer. 
They have developed a laboratory model of liver metastasis from human breast cancer cell lines that should be a powerful tool for testing specific therapeutic approaches for metastatic breast cancer. This year, they will test the anti-miR-22 molecule in this model using a variety of approaches and delivery systems. This project represents the first RNA-based therapy to be tested for breast cancer and metastatic breast cancer.
Critically, the translation of these findings in human clinical trials will be immediate, as this approach can be tested in humans utilizing the very same RNA small molecule that the Pandolfi group is utilizing in their preclinical studies.


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


Donor Recognition

The Amy Robach Award

Area(s) of Focus