Precision Medicine: A Columbia University Initiative

Precision medicine encompasses all the right reasons for a new approach to health cares

Columbia University and our hospital partner, NewYork-Presbyterian, are at the forefront of genomics, data science, and the core science, policy, and engineering disciplines essential to this emerging field of humanistic medicine known as precision medicine, which the White House announced on Jan. 30 is the mission of a new $215 million federal initiative.

The potential for progress in this broad field goes beyond new cures for disease and the practice of medicine. It encompasses virtually every part of the university, including areas that explore fundamental issues of human self-knowledge and the legal, policy, and economic implications of revolutionary changes in our understanding of human biology.

Tom Maniatis, PhD, was appointed by Columbia University President Lee C. Bollinger as director of Columbia’s university-wide precision medicine initiative. Dr. Maniatis, one of the pioneers of modern molecular biology, and Wendy Chung, MD, PhD, associate professor of pediatrics and of genetics & development, were invited to the White House to participate in the Jan. 30 presidential announcement.

This compilation of materials about, and articles on, precision medicine illustrates the interdisciplinary approach Columbia University and CUMC are taking to define and advance the field. By harnessing genomic data to better predict the future course of disease and the efficacy of treatment for individual patients, precision medicine has the potential to improve and protect health and dismantle one-size-fits-all therapies.

Precision medicine in practice and research at CUMC, in particular, is realized via collaborations across all of our clinical disciplines and many basic sciences. Our diverse scientific expertise readily contributes to enhancing precision medicine: genomics, proteomics, bioinformatics, systems biology, data and computational science, as well as core science, engineering, and other disciplines. The results should improve patient outcomes, reduce adverse treatment effects, and yield greater patient satisfaction.

CUMC’s efforts play a vital role in Columbia University’s institution-wide priority to realize the potential of precision medicine. Through the efforts of the Precision Medicine Task Force, the University’s internal expertise is coordinated and growing. The synergies of CUMC specialists’ biomedical expertise with that of other University faculty and leaders will define the medical, legal, policy, and economic implications anticipated from the applications of precision medicine.

Many of the more than 40 state-of-the-art shared research facilities within CUMC participate in precision medicine initiatives, including the JP Sulzberger Columbia Genome Center. Already our discoveries are making a difference. For example, using genomic analysis, scientists sequence the DNA of individual tumors to find FDA-approved drugs likely to target crucial areas of each tumor’s genetics. In addition, scientists developed a way to recreate an individual’s immune system in a mouse, an unprecedented tool for customized analysis of autoimmune diseases such as type 1 diabetes. The tool also may be useful to analyze a patient’s response to existing treatments or to develop new therapies.

Among CUMC’s plans for precision medicine is a comprehensive biological repository that will store and allow analysis of 100,000 patient specimens to enable translational researchers to develop new therapies that, in turn, will transform the way clinicians diagnose and treat patients.

With our clinical partner, NewYork-Presbyterian Hospital, we participate in the New York Genome Center, an international consortium of academic, medical, and industry leaders dedicated to translating genomic research into clinical solutions for disease.

Read more and follow the links below to learn about precision medicine, from recruitment of faculty with related expertise, to new diagnostic tests, to research that will enable individualized treatment of intractable cancers.

Precision Medicine Shaping the Future of Cancer Research
Immunotherapy: New Hope for Patients with Advanced Lung Cancer
Tom Maniatis Named Director of Precision Medicine Initiative
David Goldstein to Direct Columbia’s Institute for Genomic Medicine
Biogen Idec and CUMC to Conduct Collaborative Genetics Research
Profiles of Faculty Leaders in Precision Medicine
Columbia Precision Medicine Links

Precision Medicine Shaping the Future of Cancer Research

March 26, 2015

A conversation on the future of cancer research

Panelists discussing the future of cancer research (from left): Stephen Emerson, MD, PhD; Sid Mukherjee, MD, DPhil; Andrew Kung, MD, Phd; Cory Abate-Shen, PhD; William Nelson, MD, PhD; Gary Schwartz, MD; Dennis Slamon, MD. (Photo: Eileen Barroso)

Nearly 50 years after the “war on cancer” was declared in the United States, precision medicine presages an era of increased understanding of the molecular basis of cancer and of the ability to design treatments tailored to a patient’s own genetic profile, a panel of experts said Tuesday at a briefing sponsored by Columbia University.

The event, a media conversation on the future of cancer, was held to mark the upcoming PBS broadcast of “Ken Burns Presents Cancer: The Emperor of All Maladies,” based on the Pulitzer Prize-winning book by Columbia oncologist Siddhartha Mukherjee, MD, DPhil.

“In recent years we’ve learned cancer is not one disease, but hundreds of diseases,” said Andrew Kung, MD, PhD, professor of pediatrics and chief of pediatric oncology, NewYork-Presbyterian/Morgan Stanley Children’s Hospital at Columbia. “Eighty percent of children with cancer are now cured. By sequencing every gene in the cancers of pediatric patients, we can start chipping away at the remaining 20 percent.”

With precision, or targeted, cancer therapies, oncologists determine the genetic profile of a tumor and match that profile to a specific drug or, increasingly, novel drug combination. The genetic signature reveals how a tumor is hijacking a patient’s cellular machinery to wreak havoc. Once the tumor’s behavior is unmasked, scientists can target the pathways and signaling proteins that are allowing the cancer to grow.

Researchers are looking not only at the genetics, but also at the cell biology of cancer. Dr. Mukherjee described his most recent work investigating the roles played by stem cells in various forms of cancer. The most exciting discovery that’s come out of his lab recently, he said, is that through the lens of cancer, we’re able to find normal stem cells, including a stem cell that seems to build the entire vertebrate skeleton—a skeleton stem cell.

Executive producer Ken Burns, who participated in the event, said he has been motivated to learn about cancer since he was 11, when his mother died of the disease, and that he hoped the conversation spurred by the film will increase research and improve treatments. Director Barak Goodman said, during the opening remarks, he hesitated at the idea of spending a couple of years immersed in cancer, but it turned out to be one of his most enjoyable film experiences. “Science is fun,” he said, after showing a clip from the film about the discovery of oncogenes in the 1970s, considered the first major milestone in modern cancer science.

Though targeted therapies offer the best opportunities for patients, most adults diagnosed with cancer are unable to receive personalized treatment based on precision medicine outside of major academic centers. Community oncologists have limited access to sequencing and informatics technologies and still largely treat patients with standard chemotherapy and radiation, which Katie Couric, global anchor for Yahoo! News, described as “the scorched body approach.”

Couric, whose sister and first husband died of cancer, is cofounder of the research group Stand Up To Cancer (SU2C), launched in May 2008 to support innovative cancer research. “So far, we have created 13 ‘dream teams,’ involved more than 800 scientists from 115 institutions, and launched more than 120 clinical trials with 5,200 patients,” said Couric. “We are pressing the gas pedal on cancer research and won’t stop until our scientists develop new therapies.”

Dennis Slamon, MD, PhD, director of the UCLA Jonsson Comprehensive Cancer Center’s Clinical/Translational Research, and a SU2C Dream Team leader, said “We are no longer focused on the organ in which a cancer originates. Identifying which genes are broken allows us to determine which pathways are broken. This sounds very logical now, but it represents a major change in thinking from just a few decades ago. ”

With many of the targeted therapies, said Dr. Slamon—whose research led to development of the antibody conjugate Herceptin® (trastuzumab)—there are now minimal side effects compared with what oncologists are used to seeing. Physically linking Herceptin, for example, to the chemotherapy rather than administering it alongside the chemotherapy has had a big impact on safety. Women taking it no longer have the hair loss, nausea, and vomiting they had when they took the antibody alongside the chemotherapy. Dr. Slamon said there is a lot of excitement that not only are we going to be able to dial up efficacy but also to dial down the safety concerns and toxicities.

The front line of the war on cancer, launched in 1971, has been fought primarily by pediatric oncologists. “In our pediatric studies, we fully sequence every gene in the body and every gene in the cancer,” said Dr. Kung. “Precision medicine will not only allow us to do better for the 20 percent we don’t yet cure but also decrease toxicities in the 80 percent we do cure. With the evolution and revolution of the precision medicine era, we can do better for everyone.”

Cory Abate-Shen, PhD, professor of urology and pathology at Columbia, said now that we’re at the point of being able to identify many cancers at an early stage, we have to determine whom to treat. “For example, we’ve brought a systems approach to decide which prostate cancers need to be treated. We now can test biopsy samples to differentiate between aggressive and harmless tumors.”

Gary Schwartz, MD, professor of medicine and chief of the division of hematology-oncology at Columbia, said that the day will come when the physical exam of every cancer patient will include DNA sequencing. “We will have personalized medicine for every patient with cancer in the United States. Without this type of information, we are not giving patients the full benefit of treatment. ”

One highly promising path in cancer treatment is immunotherapy. Dr. Schwartz told how researchers had noticed that patients with autoimmune diseases don’t get cancer. Through immunotherapy, he said, we’ve seen some formerly untreatable cancers go away. Just this month, he noted, the drug Opdivo® (nivolumab) was approved by the FDA for treatment of advanced squamous non-small cell lung cancer.

William Nelson, MD, PhD, vice chair, SU2C Scientific Advisory Committee, and director of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, said that eventually, a smart missile will deliver drugs to where the biomarkers are. “You can see the beginning of precision medicine with the discovery of that first gene. One of the remarkable things about the documentary is that you can’t watch it without thinking about where we are going.”

The film aired over three consecutive nights, beginning on March 30.

Watch the entire panel discussion here.
Read more about cancer research and treatment at Columbia here.

Immunotherapy: New Hope for Patients with Advanced Lung Cancer

March 12, 2015

In the late 1800s, a New York surgeon named William Coley noticed that some patients with cancer seemed to fare better if they developed an infection after undergoing surgery. Suspecting that the immune system played a role in this mysterious response, he tried treating cancer patients with bacteria in an effort to turn on the immune system. Coley’s bold experiments largely failed, however, and faded into obscurity as other cancer treatments, such as radiation and chemotherapy, were put into practice.

Coley wasn’t wrong—just far ahead of his time. More than a century later, advances in how the immune system can be harnessed to fight cancer have offered new hope to patients with lung cancer. One of the latest immunotherapies to reach the market is a drug called nivolumab (Opdivo), which the FDA recently approved for the treatment of patients with advanced squamous non-small cell lung cancer (NSCLC).

“‘Groundbreaking’ and ‘revolutionary’” often overstate the case, but they truly apply to the impact of the new immunotherapy agents that target the PD-1 pathway for NSCLC,” says Naiyer A. Rizvi, MD, of the Herbert Irving Comprehensive Cancer Center and director of thoracic oncology and immunotherapeutics in the Department of Medicine at NewYork-Presbyterian Hospital/Columbia University Medical Center.

Nivolumab restores the immune system’s delicate balance by disabling the PD-1 protein on T cells. PD-1 works by suppressing T cell activity, so when this “checkpoint” protein is inhibited, T cells can go about their business.

Dr. Rizvi led a lung cancer trial recently published in Lancet Oncology that was key to approval of nivolumab for squamous lung cancer. “When I first started treating patients with nivolumab in 2008, it was hard to imagine how dramatically this could help patients who were resistant to all of our standard treatments,” says Dr. Rizvi. “We have some patients who are still alive many years after taking this drug, with no evidence of cancer. This has never been seen with standard lung cancer treatment.”

While some patients with NSCLC respond well to PD-1 inhibitors, others do not. Dr. Rizvi and his colleagues (at Memorial Sloan Kettering Cancer Center) thought that the cancers that had accumulated the most DNA damage were more likely to have worn out the immune system and would likely be helped the most by PD-1 inhibitors.

They tested this by sequencing tumor DNA from both responders and non-responders to treatment with pembrolizumab (Keytruda), a PD-1 inhibitor. Among their findings, published in March in Science, was that patients with a great deal of DNA damage were far more responsive to treatment than those with less DNA damage. “We were able to use advances in sequencing technology to study the entire exome—the protein-coding genes of the genome—of tumors from patients with NSCLC who were treated with pembrolizumab. We found that the more genetically damaged the tumor was, the more likely the patient was to respond to PD-1 inhibitors.”

“This is an important first step toward being able to predict who will respond to PD-1 inhibitors and could be a new way to think about precision medicine based on the sequencing of tumor DNA,” says Dr. Rizvi. “This collaboration among clinical researchers, geneticists, and immunologists shows how a team of scientists can work together to help patients fight cancer.”

How does immunotherapy work?

Cancer cells can bind to T-cells and “turn-off” their ability to detect and kill tumor cells. Immunotherapy drugs bind to T-cells and keep them “on” by blocking a tumor cell’s access to the T-cell. (Credit: Columbia University Medical Center)

Papers discussed above:


“Activity and safety of nivolumab, an anti-PD-1 immune checkpoint inhibitor, for patients with advanced, refractory squamous non-small-cell lung cancer (CheckMate 063): a phase 2, single-arm trial,” published February 20, 2015, in the online edition of Lancet Oncology.


“Mutational Landscape Determines Sensitivity to Programmed Cell Death-1 Blockade in Non-Small Cell Lung Cancer,” published March 12, 2015, in the online edition of Science. Dr. Rizvi is a lead author of the paper.


Tom Maniatis Named Director of Precision Medicine Initiative

Click here to download hi-res version for media use.

Tom Maniatis, PhD, has been appointed director of Columbia’s university-wide precision medicine initiative. Dr. Maniatis is one of the pioneers of modern molecular biology, having led the development of recombinant DNA methods and their application to both basic biomedical research and biotechnology. He is the Isidore S. Edelman Professor of Biochemistry and chair of the Department of Biochemistry and Molecular Biophysics at Columbia University Medical Center and a cofounder of the New York Genome Center, of which Columbia is a partner.

His appointment is a crucial step in the university’s initiative, intended to position the university at the forefront of genomics, data science, and the core science and engineering disciplines essential to this emerging field of humanistic medicine. The potential for progress in this broad field goes beyond new cures for disease and the practice of medicine. It encompasses virtually every part of the university, including areas that explore fundamental issues of human self-knowledge and the legal, policy, and economic implications of revolutionary changes in our understanding of human biology.

Dr. Maniatis coauthored the definitive laboratory manual on genetic engineering, published by the Cold Spring Harbor Laboratory, which set a standard for international dissemination of recombinant DNA methods. His past research has led to major advances in our understanding of the mechanisms of gene expression at the level of RNA transcription and splicing. His current research focuses on the role of single-cell diversity in neural connectivity, as well as on the molecular mechanisms that underlie the neurodegenerative disease ALS (Lou Gehrig’s disease).

After receiving his BA and MS from the University of Colorado at Boulder and his PhD in molecular biology from Vanderbilt University, Dr. Maniatis did postdoctoral work at Harvard and at the Medical Research Council Laboratory of Molecular Biology in Cambridge, England. He has held faculty appointments at Harvard, the Cold Spring Harbor Laboratory, and Caltech. He is a member of the U.S. National Academy of Sciences and the Institute of Medicine and is the recipient of numerous prestigious awards, including the 2012 Lasker~Koshland Special Achievement Award in Medical Science.

David Goldstein to Direct Columbia’s Institute for Genomic Medicine


David Goldstein (Photo: Duke Photography)

Dr. Goldstein’s role includes serving as an adviser to Columbia University President Lee C. Bollinger and Executive Vice President for Health and Biomedical Sciences Lee Goldman, MD, on the genetic and genomic components of Columbia’s university-wide initiative in precision or “personalized” medicine, which was announced in February.

“Having a pioneering researcher like David Goldstein join us marks a crucial next step in our initiative to be at the forefront of genomics, data science, and the core science and engineering disciplines essential to this emerging field of truly humanistic medicine,” said President Bollinger. “The potential for progress in this broad subject encompasses not only new cures for disease, but also virtually every part of the University, including areas that explore fundamental issues of human self-understanding, as well as the legal, policy, and economic implications of revolutionary changes in knowledge and practice.”

Dr. Goldstein’s research has focused on identifying the relationship between human genetic variations and diseases such as epilepsy, hepatitis C, and schizophrenia, as well as the response of these diseases to pharmacologic treatments. In addition to his leadership of the Institute for Genomic Medicine at CUMC, he will have a faculty appointment at the New York Genome Center, as well as one in neurology at P&S.

“David Goldstein has shown himself to be both an innovative scientist and a visionary leader in genetic, genomic, and personalized medicine,” said Dr. Goldman, who is also the Harold and Margaret Hatch Professor of the University and dean of the Faculties of Health Sciences and Medicine at CUMC. “Working with our partners across Columbia and at New York-Presbyterian, Dr. Goldstein will help us establish a fully integrated genetics and genomics research environment to maximize the scientific possibilities and apply them to the frontiers of patient care and public health.”

“Personalized medicine and targeted therapies represent the future of patient-centered health care,” said Steven J. Corwin, MD, CEO, NewYork-Presbyterian. “Dr. Goldstein’s expertise in genetics will help us not only to tailor individualized treatments for patients, but  also to identify diseases before they develop. His work will have a transformative impact on patient care at NewYork-Presbyterian.”

Dr. Goldstein comes to Columbia from Duke University, where he has been director of the Center for Human Genome Variation and the Richard and Pat Johnson Distinguished University Professor, with appointments in the departments of molecular genetics & microbiology and biology. He joined Duke in 2005 after six years at University College London, which named him Honorary Professor in 2007. He received his PhD in biological sciences from Stanford University in 1994.

“The vision of Columbia University and NYP to create a truly integrated environment for research, clinical application, and student instruction is exactly the right vision,” said Dr. Goldstein. “Human genomics is creating breathtaking new opportunities to better understand the biology of disease and to provide more effective and more accurately targeted therapies. Capitalizing on these opportunities and ensuring that clinical applications adhere to the highest-possible scientific standards requires close collaborations among researchers, the clinical community, and patients and their families. I am thrilled to be joining Columbia University at this pivotal time in my field, and I am honored to participate in Columbia’s university-wide initiative in precision medicine.”

Dr. Goldstein was elected a fellow of the American Association for the Advancement of Science in 2013 and received one of the first seven nationally awarded Royal Society/Wolfson research merit awards in the United Kingdom for his work in human population genetics. Also in 2013, Dr. Goldstein chaired the Gordon Research Conference in Human Genetics. He serves on the Advisory Council at NIH’s National Institute of Neurological Disorders and Stroke.

Read about an ALS study led by Dr. Goldstein.

Biogen Idec and CUMC to Conduct Collaborative Genetics Research

Sequencing facility and shared postdoctoral program to support genetic discovery research to advance development of new treatments

Biogen Idec and Columbia University Medical Center have formed a $30 million strategic alliance to conduct genetics discovery research on the underlying causes of disease and to identify new treatment approaches. As part of this agreement, a sequencing and analysis facility and shared postdoctoral program will be established at Columbia to support collaborative genetics research. The agreement will integrate genomics research conducted at Columbia with Biogen Idec’s understanding of disease mechanisms and pathways, and expertise in discovering new medicines.

“Our understanding of human genetics is rapidly expanding, and there is growing recognition that the elucidation of the genetic causes of disease will have a transformative effect on both patient care and drug development in many different diseases,” said David Goldstein, PhD, founding director of Columbia University’s Institute for Genomic Medicine. “This collaboration marries the exceptional drug development expertise of Biogen with cutting-edge genomics expertise at Columbia University Medical Center. It will not only focus on target identification and validation at the early stages of drug development, but also facilitate genetically informed evaluation of treatments.”

“Human genetic technologies and analytics have advanced to the point where they are becoming central to the discovery and development of new medicines,” said Tim Harris, PhD, DSc, senior vice president, Technology and Translational Sciences at Biogen Idec. “We are committed to working with leading institutions such as Columbia to advance basic genetic research and, by combining our unique strengths, accelerating the discovery of potential new treatments.”

The collaboration will enable Biogen Idec and Columbia to investigate the genomes of patients showing unusual treatment responses or unique disease presentations and to explore the connections among genes, pathways, and disease processes. The ultimate goal will be to provide multiple qualified targets for new therapeutic approaches, increasing the potential for the development of new treatments.

“This collaboration with Biogen, with its focus on the genetic causes of diseases, fits in perfectly with Columbia’s commitment to precision medicine,” said Lee Goldman, MD, the Harold and Margaret Hatch Professor of the University and dean of the faculties of health sciences and medicine. “The development of new treatments based on this genetic understanding will have profound effects on clinical practice.”

The new facility will have broad genetic research capabilities and the capacity to launch and complete whole-genome sequencing projects rapidly. It will allow for rapid population-scale DNA sequencing across a broad range of disease areas, focusing on diseases with significant unmet clinical need such as amyotrophic lateral sclerosis and idiopathic pulmonary fibrosis.

Tom Maniatis, PhD, the Isidore S. Edelman Professor of Biochemistry, chair of the Department of Biochemistry & Molecular Biophysics at Columbia, and director of Columbia’s university-wide precision medicine initiative, said, “The strong clinical and basic science programs in neurodegenerative diseases at Columbia will significantly benefit from the Columbia/Biogen alliance. We expect that the alliance will dramatically advance our understanding of the genetics of these devastating diseases and ultimately lead to mechanism-based treatments, a key aspect of Columbia’s precision-medicine initiative.”

Profiles of Faculty Leaders in Precision Medicine

Andrea Califano, PhD, is chair of the Department of Systems Biology at P&S. The emerging field of systems biology uses principles of biology, genetics, mathematics, computer science, physics, chemistry, and other fields to discover and model the complex interactions within biological systems. Dr. Califano’s research focuses on a new, personalized approach to cancer, called the N-of-1 approach, whereby therapies are individually designed and tested for a single patient. In this approach, each patient’s tumor is “reverse engineered” to determine its unique genetic characteristics and to identify existing U.S. Food and Drug Administration-approved drugs that may target them. View video:

Wendy Chung, MD, PhD, directs the clinical genetics program at Columbia, which helps physicians identify the precise genetic factors that cause disease in specific cases. She also directs clinical programs in risk assessment for cardiomyopathy and arrhythmias and develops novel molecular diagnostic methods to improve genetic testing, including pre-implantation genetic diagnosis for family planning. Her research is on the genetic basis of metabolic disorders such as diabetes, cardiac disorders, neuromuscular disease, and neuropsychiatric disorders. Read a Q&A with Wendy Chung. In a TED talk, Dr. Chung discussed genetic testing in newborns.

Hematologist/oncologist Stephen G. Emerson, MD, PhD, is director of the Herbert Irving Comprehensive Cancer Center, a collaboration of Columbia and NewYork-Presbyterian Hospital/Columbia. His research on bone marrow stem cell biology, particularly as applied to bone marrow stem cell transplantation, has led to new medical therapies used worldwide. His work in stem cell biology has broader implications for many types of cancers. Read a Q&A with Dr. Emerson.


Wayne Frankel, PhD, is an epilepsy expert at Columbia’s Institute for Genomic Medicine. After 24 years at the Jackson Laboratory, Dr. Frankel joined Columbia in November 2015, where he is the Director of Preclinical Models at the IGM and a professor in the Department of Genetics and Development. Dr. Frankel’s lab investigates the molecular mechanisms of complex disease, using gene editing to create mouse models that more precisely reproduce human disease. The research is particularly focused on neurological disorders involving recurrent seizures. Read a Q&A with Dr. Frankel.

Ali Gharavi, MD, is chief of the nephrology division in the Department of Medicine at P&S. His research focuses on the molecular genetics of kidney diseases, particularly glomerular and developmental disorders, as well as congenital abnormalities of the kidney and urinary tract. He recently found that 10 percent of children born with kidney defects have large alterations in their genomes known to be linked with neurodevelopmental delay and mental illness, which should lead to more personalized treatment for such patients. The long-term objective of his lab is to identify specific genes and dysregulated pathways underlying kidney disorders, to enable the development of new diagnostic tools and therapies.

Richard Mayeux, MD, is chair of the Department of Neurology, director of the Gertrude H. Sergievsky Center, and co-director of the Taub Institute for Research on Alzheimer’s Disease and the Aging Brain. Dr. Mayeux helps lead the Alzheimer’s Disease Genetics Consortium, which has in recent years uncovered many of the genes that increase the risk of developing Alzheimer’s. Those findings may lead to prognostic testing that can better identify those at risk of developing the disease and guide treatment for those with the disease. Since 1989, he has led a multidisciplinary, population-based, epidemiological investigation of Alzheimer’s disease and related conditions known as the Washington Heights-Inwood Community Aging Project. He also directs a genetic linkage study of Alzheimer’s disease in Caribbean Hispanic families.

Gary Schwartz, MD, chief of the hematology/oncology division, is an expert in the treatment of melanoma, sarcoma, and cancers of the gastrointestinal tract and in new drug development. His studies encompass the molecular and cellular mechanisms of cancer, its unique behavior in different tissues, and statistical aspects of its occurrence and treatment in large populations. His research goal is to understand the mechanisms underlying cell cycle and cell death, in order to improve the effectiveness of currently available treatments.

Megan Sykes, MD, studies ways to coax the immune system to better tolerate transplanted organs, which could allow transplant patients to forgo lifelong immunosuppressant drugs. She has worked on ways to re-create an individual’s immune system in a mouse, which would offer an unprecedented tool for individualized analysis of abnormalities that contribute to autoimmune diseases such as type 1 diabetes, starting at the onset of disease. Her research has shown that combining bone marrow transplant and organ transplantation can induce tolerance and allow acceptance of the donor organ without long-term immunosuppressant therapy.

Ronald J. Wapner, MD, is vice chair for research in the Department of Obstetrics & Gynecology at P&S and director of reproductive genetics at NewYork-Presbyterian Hospital/Columbia. Dr. Wapner’s research on first-trimester screening is helping to bring personalized genomics technology to the prenatal diagnosis of developmental delays, structural abnormalities, and treatable or life-threatening diseases. As a result of his 2012 study showing that microarray technology in prenatal testing provides more clinically relevant information than current standard techniques, genomics techniques are becoming more commonplace in the prenatal setting. Dr. Wapner also was instrumental in developing chorionic villi sampling and multi-fetal reduction procedures. His clinical practice focuses on high-risk obstetrics and prenatal diagnosis.


Research Facilities
Facilities that Support Patient Care
Collaborations with Other Organizations
Columbia Technology Ventures
Precision Medicine and Cancer
Precision Medicine and Stem Cells
Precision Medicine and Alzheimer’s Disease
Precision Medicine and Psychiatry
Precision Medicine and Kidney Disease
Precision Medicine and Patient Care
Precision Medicine and Dentistry
Precision Medicine and Nutrition
Precision Medicine and Infectious Diseases
Precision Medicine and Heart Disease
Precision Medicine and Systems Biology
Precision Medicine and Genetics
Precision Medicine and Children’s Health
Precision Medicine and ALS
Precision Medicine and Organ Transplantation
Precision Medicine and Autoimmune Diseases
Overview of Precision Medicine

Precision Medicine Links

Research Facilities
Among the more than 40 state-of-the-art shared research facilities within CUMC, many participate in precision medicine initiatives, including:
Center for Genome Technology and Biomolecular Engineering
Center for Human Genetics
Columbia Center for Translational Immunology
Columbia Stem Cell Initiative
Department of Biomedical Informatics
Department of Biostatistics
Department of Genetics & Development
Department of Pathology & Cell Biology
Laboratory of Personalized Genomic Medicine
Department of Pediatrics/ Division of Molecular Genetics
Kavli Institute for Brain Science
General Thoracic Surgery
Hypertrophic Cardiomyopathy Research
Department of Systems Biology
Center for Computational Biology and Bioinformatics (C2B2)
J.P. Sulzberger Columbia Genome Center
Herbert Irving Comprehensive Cancer Center
Program in Cancer Regulatory Networks
Human Genetics Resources Core
Irving Institute for Clinical and Translational Research/Biomarkers Core Laboratory
Institute for Cancer Genetics

Facilities that Support Patient Care
Laboratory of Personalized Genomic Medicine 

Collaborations with Other Organizations
New York Genome Center
Genome Center Opens in New York

Columbia Technology Ventures
Profile of Dr. Jingyue Ju

Precision Medicine and Cancer
Cancer’s New Vocabulary: Life-saving, Turnarounds, Cures
Precision Medicine Puts Lung Cancer in Its Sights
Drug Combo Blocks Rare Cancer’s Escape Route
State-of-the-Art Proteomics Lab Opens at CUMC
Columbia Launches Clinical Cancer Exome and Transcriptome Test
Drug Shows Promise for the First Time Against Metastatic Melanoma of the Eye
Two Genes Together Drive Aggressive Prostate Cancer
Common Blood Cancer May Be Initiated by Single Mutation in Bone Cells
NewYork-Presbyterian/Columbia Opens New Radiation Oncology Center
Study Reveals Genes That Drive Brain Cancer
Columbia Awarded One of First NCI “Provocative Questions” Grants
Research May Lead to New Treatment for Type of Brain Cancer
Vast Hidden Network Regulates Gene Expression in Cancer
Family History of Breast Cancer May Raise Risk for Ovarian Cancer
Herbert Irving Comprehensive Cancer Center Program in Cancer Regulatory Networks
Herbert Irving Comprehensive Cancer Center Breast Cancer Management 2013 [PDF]
Institute for Cancer Genetics
New Cancer Treatment: Personalized Radiation Therapy During Cancer Surgery

Precision Medicine and Stem Cells
Patient-Specific Stem Cells and Personalized Gene Therapy
Tom Jessell Wins Prestigious Neuroscience Award
Dan Doctoroff, David Rubenstein, and Bloomberg Philanthropies Unveil Target ALS
New Technique to Transform an ALS Patient’s Skin Cells Into Motor Neurons
Columbia Receives $2.5 Million To Support Stem Cell Initiatives
Scientists at New York Stem Cell Foundation, Columbia U. Make Advance in Development of Patient-Specific Stem Cells
First Patient-Specific Human Embryonic Stem Cells Created
Scientists Develop Technique to Help Prevent Inherited Disorders in Humans

Precision Medicine and Alzheimer’s Disease
Researchers Receive $12.6M NIH Grant to Study Alzheimer’s Genetics
Alzheimer’s Disease Consortium Identifies Four New Genes for Alzheimer’s Disease Risk
Global Study Discovers Flurry of New Alzheimer’s Genes
Key Molecular Pathways Leading to Alzheimer’s Identified

Precision Medicine and Psychiatry
DocTalks: Psychiatry and Precision Medicine
Profile of Dr. Patrick J. McGrath
Cognitive Behavioral Therapy Tailored for Adolescent Anxiety Disorders
New Strategy for Stimulating Neurogenesis May Lead to Drugs to Improve Cognition and Mood
New de novo Genetic Mutations in Schizophrenia Identified

Precision Medicine and Kidney Disease
New Nephrology Chief Named at NewYork-Presbyterian/Columbia University Medical Center

Precision Medicine and Patient Care
CUMC Project Adds Patient DNA Data to Electronic Health Records
Acute Palliative Care Units Provide Compassion and Medical Care for Patients with End-Stage Disease
Irving Institute for Clinical and Translational Research Precision Medicine Resource
NewYork-Presbyterian/Columbia Opens New Radiation Oncology Center
Herb Chase, Biomedical Informatics: Will a Machine Be My New Doctor?

Precision Medicine and Dentistry
Gene Expression Signature Reveals New Way to Classify Gum Disease

Precision Medicine and Nutrition
Four Tips on Thinking about Fat in Your Diet

Precision Medicine and Infectious Diseases
Combination Approach Reduces Spread of Drug-Related HIV
Computational Analysis Helps Researchers Understand Emerging Pathogenic Foe

Precision Medicine and Heart Disease
New Genetic Cause of Pulmonary Hypertension Identified
NewYork-Presbyterian/Columbia Surgeons Are First in NYC Area to Implant Total Artificial Heart

Precision Medicine and Systems Biology
Clinical Trials for Cancer, One Patient at a Time

Precision Medicine and Genetics
CRISPR Used to Repair Blindness-causing Genetic Defect in Patient-derived Stem Cells 
Wendy Chung, Pediatrics, Is GATTACA Soon to Become a Reality?
Many Birth Defects in Heart Caused by Spontaneous Mutations
Tom Maniatis: A Deep Sense that Science Must Be Shared
New York Genome Center Launches Unprecedented Collaboration of 11 Leading Medical/Research Institutions
New Columbia Center Aims To Advance Next Generation Of Genomics, Proteomics Research
Study of Ashkenazi Origins Will Improve Personalized Medicine

Precision Medicine and Children’s Health
Genome Study Finds Link Between Congenital Heart and Brain Disorders
Common Childhood Asthma Not Rooted in Allergens, Inflammation
Looking for the Telltale Gene
Study Shows Why Leukemia Returns in Some Children
New Prenatal Gene Test Proposed as Standard of Care

Precision Medicine and ALS
New Technique To Transform An ALS Patient’s Skin Cells Into Motor Neurons

Precision Medicine and Organ Transplantation
An Inkjet for Living Tissue

Precision Medicine and Autoimmune Diseases
“Personalized Immune” Mouse Offers New Tool for Studying Autoimmune Diseases

Overview of Precision Medicine (previously known as personalized medicine)
Columbia Medicine Spring 2014 cover story