BOSTON – The Cancer Center at Beth Israel Deaconess Medical Center (BIDMC) has launched a new research institute to harness the potential of RNA to revolutionize the way cancer and ultimately other diseases are treated and diagnosed.
The Institute for RNA Medicine (iRM), established under the leadership of renowned cancer geneticist and BIDMC Cancer Center Director Pier Paolo Pandolfi, MD, PhD, will bring together leading investigators in the field to pursue new lines of inquiry into non-coding RNA, a highly promising area of research that significantly expands the known boundaries of the human genome.
“Although the sequencing of the human genome marked a critical era in molecular biology, it has become clear that this was only the beginning of the story,” said Pandolfi. “Scientific research over the past decade has concentrated almost exclusively on the 2 percent of the genome’s protein coding regions, ignoring the other 98 percent, a vast universe of non-coding genetic material previously dismissed as nothing more than ‘junk.’ It is now apparent that these non-coding RNAs contain a wealth of crucial clues to both health and disease that will guide us in our quest to understand the mysteries of cancer and to develop therapies to cure this deadly disease.”
The iRM will capitalize on BIDMC’s large, growing and varied patient population, as well as its unique mouse modeling facility and streamlined co-clinical trial platform, to bring non-coding RNA laboratory discoveries into the clinical setting.
“Beth Israel Deaconess is proud to host one of the first dedicated research institutes within an academic medical center to focus on non-coding RNA,” said Kevin Tabb, MD, BIDMC President and Chief Executive Officer. “BIDMC’s depth of scientific talent and discovery in this field is unparalleled. Now, with the creation of the iRM, we can rapidly move this research from the lab bench to the patient bedside.”
Distinguished Scientist Named Director
Molecular biologist Frank Slack, PhD, an international leader in the study and understanding of microRNAs, a subset of non-coding RNA, will become director of the iRM on July 1. Slack will join co-founders Pandolfi and trailblazing molecular biologist John Rinn, PhD, in launching the iRM, thus bringing together three visionary leaders whose scientific breakthroughs have helped form the foundation for this dynamic field.
“Frank Slack’s research played an important role in shifting the scientific view of non-coding RNA from one of insignificance to a promising source of personalized diagnostics and therapies,” said Jeffrey Saffitz, MD, PhD, Chairman of Pathology at BIDMC and another iRM co-founder. “Dr. Slack’s discoveries into the roles played by microRNAs in regulating human disease genes have already revealed key insights into oncogenic and tumor suppression functions and have enabled us to move past the discovery stage and into the translational phase of this rapidly growing field.”
Slack will move his laboratory to the iRM’s location in the Center for Life Sciences building in the Longwood Medical Area, adjacent to BIDMC.
He has been a member of the Yale University faculty since 2000, most recently as a Professor of Molecular, Cellular and Developmental Biology and Director of the Yale Center for RNA Science and Medicine. He holds a doctorate in molecular biology from Tufts University School of Medicine and completed post-doctoral training at Stanford University School of Medicine and Harvard University.
“Beth Israel Deaconess Medical Center is recognized around the world for the novel discoveries in long non-coding RNAs (lincRNAs) and ceRNAs made by Drs. Pandolfi and Rinn,” said Slack. “Our non-coding RNA investigations complement one another and create the opportunity for the emergence of exciting synergies. Cancer takes numerous forms in many different tissues, but the disease is ultimately driven by cells that grow out of control. Non-coding RNAs are playing an integral role in this process and have revealed themselves to be great novel targets of anti-cancer therapies.”
A Rapidly Evolving Field
As part of BIDMC’s Cancer Center, the iRM will benefit from a range of established resources, most notably the Cancer Center’s “Mouse Hospital,” a unique facility in which mice are genetically altered to replicate human cancers. This enables BIDMC investigators to conduct human clinical trials in parallel with genetically relevant mouse models to determine the efficacy of new treatments in later stage cancers.
“The iRM will provide an interdisciplinary approach to expedite our scientific discoveries,” said Pandolfi. “By bringing together systems biologists, mouse modelers, bioinformaticians and molecular biologists within a clinically based platform, we will provide a unique opportunity to not only identify new non-coding RNA pathways and determine if they are targets for therapeutic intervention, but also to test candidate drugs in our Mouse Hospital.”
The iRM will initially focus on the role of non-coding RNA in blood cancers, prostate cancer, lung cancer and breast cancer. Additional investigations will explore the role of this genetic matter in other diseases, including Alzheimer’s disease and diabetes. Already cardiovascular researchers at BIDMC are looking at the impact of non-coding RNA on heart disease.
The iRM is planning an inaugural symposium on the state of non-coding RNA research that will take place in the spring of 2015. Participants are expected to include experts in disease genetics from the United States and beyond.
An advisory board chaired by Michael F. Cronin, a member of the BIDMC Board of Directors, and Tige Stading, a member of the BIDMC Board of Overseers, is helping to guide development of the iRM. Cronin is Co-Founder and Managing Partner of Weston Presidio investment company. Stading is the Founder and President of The Stading-Younger Cancer Research Foundation.
“Non-coding RNA has ushered in another revolutionary chapter in genomic medicine,” said Walter Gilbert, PhD, another advisory board member and a molecular biologist whose work led to the development of rapid DNA sequencing, for which he was awarded the Nobel Prize in 1980. “This avenue of investigation holds tremendous potential for the development of targeted cancer therapies.”
Collaboration Among Non-Coding RNA Pioneers
Through independent, parallel lines of study, Rinn, Slack and Pandolfi were among the very first in the scientific community to recognize the vast potential of non-coding RNAs.
Known for his capacity to boldly challenge traditional scientific thinking, Rinn, Associate Professor of Pathology at BIDMC and Harvard Medical School and Associate Professor of Stem Cell and Regenerative Biology at Harvard University, has carried out seminal research on a class of large non-coding RNAs called lincRNAs. His work, which has led to the discovery of more than 10,000 new RNA genes, has verified the ability of lincRNAs to regulate the immune response, cancer growth and fat and stem cell production.
Slack’s research focuses on microRNAs. His work led to the discovery of let-7, the second of more than 1,500 microRNAs ever discovered, and of the first known human microRNA. Later found to be a critical determinant of lung cancer and key to the regulation of oncogenes, let-7 provided the first mechanistic role for a microRNA in cancer.
Pandolfi, a prominent cancer geneticist whose research led to the cure of a once-deadly form of leukemia, has demonstrated that pseudogenes – 20,000 or so genetic units once thought to represent evolutionary relics – are indeed very relevant to tumorigenesis. His team has also recently discovered that coding genes and non-coding genes can communicate through a distinctive RNA language that, when regulated, can lead to the development of cancer in mouse models.
“This is the time to invest in the promise of non-coding RNAs,” said Rinn. “Over the past decade, we have developed an integrative approach combining human disease risk genetics, experimental and computational biology tools to identify key RNA candidates. Thus we have honed in on the non-coding regions that have the greatest change to impact human health and disease.”
About the Cancer Center at Beth Israel Deaconess Medical Center
The Cancer Center at BIDMC is a clinical, research and educational organization within Beth Israel Deaconess Medical Center, a teaching hospital of Harvard Medical School. Multidisciplinary teams of physicians collaborate to design and deliver an individual care plan for each patient. US News & World Report recognizes BIDMC as one of the best cancer hospitals in the United States. The Cancer Center is committed to advancing the frontiers of cancer science and compassionate care.
Beth Israel Deaconess Medical Center is a patient care, teaching and research affiliate of Harvard Medical School, and currently ranks third in National Institutes of Health funding among independent hospitals nationwide.
The BIDMC health care team includes Beth Israel Deaconess Hospital–Milton, Beth Israel Deaconess Hospital–Needham, Beth Israel Deaconess Hospital–Plymouth, Anna Jaques Hospital, Cambridge Health Alliance, Lawrence General Hospital, Signature Health Care, Commonwealth Hematology-Oncology, Beth Israel Deaconess HealthCare, Community Care Alliance, and Atrius Health. BIDMC is also clinically affiliated with the Joslin Diabetes Center and Hebrew SeniorLife and is a research partner of Dana-Farber/Harvard Cancer Center. BIDMC is the official hospital of the Boston Red Sox. For more information, visit www.bidmc.org.