What is Precision Medicine?
Precision medicine is targeted, individualized care that is tailored to each patient based on his or her specific genetic profile and medical history. Unlike in traditional one-size-fits-all medicine, practitioners of precision medicine use genomic sequencing tools to interrogate a patient’s entire genome to locate the specific genetic alterations that have given rise to and are driving his or her tumor. With this information they can identify small molecule drugs, monoclonal antibodies, vaccines, and other therapies that are most precisely targeted and are therefore most effective and have the fewest side effects.
Precision medicine can be helpful to patients at different stages of disease. In patients with advanced disease who no longer respond to available therapies and lack treatment options, genomic analyses of tumor tissue may isolate the causes of drug resistance and highlight therapies with a better likelihood of success. Precision medicine can also allow physician-scientists to identify a patient’s risk before diseases develop, and enable them to take steps toward prevention through medical treatment, lifestyle modification or both. Precision medicine is an exciting, emerging field that is transforming the existing paradigm for diagnosing and treating patients with many types of disease.
About the Institute
The Caryl and Israel Englander Institute for Precision Medicine at Weill Cornell Medical College and NewYork-Presbyterian Hospital is a translational research hub, which opened under the direction of renowned pathologist and prostate cancer expert Dr. Mark Rubin in 2013. The IPM team includes clinicians, basic scientists, pathologists, molecular biologists, and computational biologists. These physician-scientists are directing their collective expertise and wealth of knowledge to pinpoint the molecular underpinnings of disease and spur the discovery of novel therapies.
The Institute’s three main resources, next-generation genomics, biobanking, and computational biology, will enable the IPM team to most efficiently match new, biologically-targeted therapies to the cancer patients who will benefit most. Our physician-scientists use advanced gene-sequencing technologies to identify the genetic alterations that give rise to and drive each patient’s illness.
The IPM has already sequenced the genomes of more than 100 patients, and is building a comprehensive biobank of tissue samples. The biobank also includes more than 2,000 prostate samples collected from patients treated at Weill Cornell Medical Center. Over the coming months and years as IPM scientists sequence additional patients’ genomes and add samples to the biobank, IPM researchers will gain insight into specific genetic alterations that recur from patient to patient, and that may be targetable with existing or new drugs.
Members of the institute work collaboratively with physician-scientists nationally and internationally; this team science approach is critical to advancing treatment and speeding the handoff from discovery to clinical implementation. By linking cutting-edge research and next-generation sequencing in the laboratory to the patient’s bedside the IPM hopes to revolutionize the way we treat disease.
About Our Team
Mark A. Rubin, MD, a genitourinary pathologist and physician scientist, is the founding Director of The Caryl and Israel Englander Institute for Precision Medicine, the Homer T. Hirst Professor of Oncology in Pathology, and the Vice Chair for Molecular and Genomic Pathology at Weill Cornell Medical College and NewYork-Presbyterian Hospital. Dr. Rubin is among the country’s foremost anatomic pathologists and has received wide recognition for advancing the field of biomarkers in prostate cancer. When used in combination with clinical and pathology parameters in prostate cancer, these biomarkers may increase the accuracy of risk assessment in patients.
His laboratory has pursued the development of molecular markers in prostate cancer that may be able to distinguish indolent from aggressive disease using techniques including real-time polymerase chain reaction, laser-capture microdissection, and high-density tissue microarrays, as well as single-nucleotide polymorphism, tiling, and cDNA expression arrays. For their seminal work on TMPRSS2-ETS fusion in prostate cancer, Dr. Rubin was the co-team leader for the First Annual American Association of Cancer Research Team Science Award (2007).
Dr. Rubin is a member of the World Health Organization Prostate Cancer Tumor Classification and the Cancer Genome Atlas advisory group. He has been associated with Prostate Specialized Programs of Research Excellence (SPOREs) at the University of Michigan and Harvard University, a National Cancer Institute grant that supports translational research to provide new approaches to the prevention, early detection, diagnosis, and treatment of cancers. With a focus on establishing standards in translational research across the 11 Prostate Cancer SPOREs throughout the country, he spearheaded development of the National Biospecimens Network pilot project. Dr. Rubin serves on numerous scientific advisory boards and was the co-chair of an NCI-sponsored workshop that established guidelines in biobanking.
Himisha Beltran, MD, a medical oncologist and physician scientist, is Director of Clinical Activities at The Caryl and Israel Englander Institute for Precision Medicine. Dr. Beltran’s clinical specialty is the care of patients with prostate cancer. Her particular focus is next-generation genomic sequencing and integrative molecular analysis of advanced metastatic prostate tumors to develop precision cancer care and biomarker driven clinical trials. Her research has brought increased attention to identifying and targeting androgen receptor (AR) negative neuroendocrine prostate cancer, an aggressive subtype of prostate cancer. Dr. Beltran has developed novel biomarkers and a multi-institutional Phase 2 trial of a targeted therapy for patients with this form of prostate cancer.
She is developing a research program to study the RNA and DNA of neuroendocrine prostate cancer to learn how these cancers evolve, to identify molecular alterations to target with drugs, and develop clinical trials to bring new effective therapy to patients. Dr. Beltran is the recipient of numerous awards including a Prostate Cancer Foundation Young Investigator Award, AACR Women in Cancer Research Scholar Award, and Damon Runyon Cancer Foundation Clinical Investigator Award.
Olivier Elemento, PhD leads The Caryl and Israel Englander Institute for Precision Medicine’s Computational Biology Group, which focuses on cancer systems biology. Dr. Elemento and members of his group use high-throughput sequencing to study cancer cells to uncover the patterns of activities in aberrant pathways, to learn how regulatory networks are rewired, and how tumors evolve at the genomic and epigenomic level. His lab has developed several computational approaches to analyze deep sequencing data, and additional computational approaches including a pathway analysis tool (iPAGE), tools for regulatory element detection (FIRE and FastCompare), and RRBseeqer for ERRBS analysis (including detection of differentially methylated regions).
His group is applying known drugs and compounds to new indications (drug repositioning) to identify small molecules that can target mutated signaling pathways and classically undruggable proteins such as transcription factors. The researchers model complex signaling pathways to identify drug combinations that can most efficiently shut down aberrantly active pathways. The computational group has established a data analysis pipeline based on many years of experience in the area of research genomics.
Juan Miguel Mosquera, MD, MSc, a surgical pathologist with subspecialty expertise in Genitourinary Pathology, leads the The Caryl and Israel Englander Institute for Precision Medicine's Pathology Team. Dr. Mosquera is an Associate Professor of Pathology and Laboratory Medicine at Weill Cornell Medicine. His background includes training in Molecular Pathology and Toxicology at the University of Leicester in the United Kingdom, and Fellowships in Genitourinary Pathology and Translational Research at Brigham and Women's Hospital, the latter under supervision of Dr. Mark A. Rubin.
Dr. Mosquera's research interests include genomics of prostate cancer and soft tissue tumors, and development of tissue-based biomarkers. The Pathology Team is comprised of pathologists with different subspecialties, research and clinical fellows as well as technicians, who work to optimize next-generation biobanking protocols and to develop in situ assays as potential biomarkers.
David Rickman, PhD, a molecular biologist, leads the IPM Functional Validation Team and is an Assistant Professor of Pathology and Laboratory Medicine. The Functional Validation Team has established a variety of approaches, including gene editing to recapitulate mutations and other genomic alterations in pre-clinical models to assess their impact on enhanced drug sensitivity.
Dr. Rickman's interest in cancer biology began during his post-doctoral fellowship at the University of Michigan Medical School where he used genome-wide approaches to characterize malignant gliomas. He then joined France's Ligue Nationale Contre le Cancer based in Paris as a project leader for a national cancer biomarker program that aimed to identify clinically relevant biomarkers for multiple cancer types. Dr. Rickman's research lab is focused on elucidating the role of ETS family members and N-Myc in coordinating gene regulation, chromatin 3D architecture and response to chemotherapy in the context of prostate adenocarcinoma and neuroendocrine prostate cancer. To this end, Dr. Rickman's lab has been developing in vitro and in vivo model systems that better mimic the context of the expression of these factors in vivo.
Jenny Xiang, MD manages the IPM’s clinical and research genomic resources. Dr. Xiang, who has been running the Genomics Resources Core Facility (GRCF) since 2000, has many years of experience with high throughput genomics technologies as well as with experimental design, sample preparation, instrumentation, data analysis, interpretation, and validation. Next-generation sequencing (NGS) is exceptionally powerful and versatile, and has been used for a wide range of researches related to human diseases, including whole genome sequencing; all exome sequencing; protein-DNA/RNA interactions (ChIP-Seq, CLIP, etc.); small RNA discovery/expression; RNA sequencing for SNP detection, expression analysis, and splice variant detection, and targeted resequencing. The goal of GRCF is to provide high quality services at low costs to the entire research community. The services on this technology platform have been critical to the publications and securing of external grants by many investigators in the WCMC research community.