TUESDAY JUNE 26th, 2018
7:00 – 8:15am
Registration & Breakfast Buffet
8:15 – 8:20am
Welcome & Opening Remarks
8:20 – 9:10am
9:10 – 10:10am
PLENARY – A World Without Disease – Janssen Diagnostics
Coffee & Networking
10:40 – 11:30am
Curing the Precision Deficit Disorder in Precision Medicine: A Unified,
Regulatory Network-Based Model for Cancer Drug Discovery
Moderator: Mariano Alvarez, Chief Scientific Officer, DarwinHealth, Inc
Panelists: Andrew Kung, MD, Ph.D., Chair, Department of Pediatrics – MSKCC, Kevin Kalinsky, MD, MS – Assistant Professor of Medicine, New York Presbyterian/Columbia University Medical Center.
Unfortunately, the gene-centric approach to precision-based cancer treatment which, for the most part, has relied on using a single drug to attack an individual mutation, has proved effective for only a relatively small percent of patients. This is because most patients lack actionable mutations and, even among those who do have such molecular alterations, most either fail to respond to the targeted therapy or rapidly develop drug resistance. Some call this the “precision deficit disorder” in precision medicine.
To find their way out of this impasse, many clinicians, scientists and researchers now believe that by complementing its initial focus on mutated genes, precision oncology must now also address the activity of otherwise normal proteins and molecular pathways that are ultimately responsible for cancer cells’ malignant behavior. The catch is that while genes can be readily sequenced to assess the presence of mutations, a complete tally of the wayward activity of potentially relevant proteins is much harder, thus thwarting previous attempts to identify the precise set of proteins that controls the cancer cell’s “engine room.”
In this panel we will discuss the progress that has been made using algorithms that can identify these critical proteins—dubbed “master regulators”—that hold the key to the malignant behavior of cancer cells. The algorithms use mRNA levels—easily measured with conventional methods—of the proteins’ numerous targets as an indirect but highly accurate indicator of protein activity. Importantly, unlike any other available approach, these algorithms uncover the hidden networks of proteins that work together to control abnormal cell activity in an individual patient’s tumor. These master regulators represent the ultimate on-off switches in the cancer cell’s regulatory ‘malware,’ providing a new class of targets for anticancer therapy, a potentially revolutionary advance in how we put more precision into precision medicine.
11:35 – 11:55am
12:00 – 1:00pm
1:00 – 1:50pm
Treatment, Patient, Time: Are We Getting it “Right”?
The goal of precision medicine is to ensure that the right treatment is delivered to the right patient at the right time. Buried in that near-religious statement are assumptions about our ability to identify the “right” things that are proving to be, if not completely untrue, then at least less robust than hoped in the early heady days. The three “right” choices that have to be made in precision medicine are inextricably linked, but it might help to consider each in isolation.
Treatment: What do we mean by the “right” treatment, how do we select it, and how do we measure its efficacy?
Patient: Who is the “right” patient, how do we select him/her, how do we know we have chosen correctly?
Time: What is the “right” time, how do we select it, and how do we assess our choice?
Successful answers to all of these questions ultimately depend on making a sufficient number of the right measurements, identifying real signals over noise in those measurements, and interpreting those signals in a meaningful way. Early precision medicine focused primarily on genomics, which still drives most of the awareness and hope for the field (finding signal in genomic noise is difficult, but “if we only sequence enough people/genomes/exosomes/transcriptomes/epigenomes, the knowledge will fall into place”). More recently, advocates for measuring “everything” possible are questioning the genomics-alone paradigm (omics-mania), but are also be running into some of the same challenge of measuring enough of everything to get reliably meaningful insights to fall out of the data in a consistent way, without swamping out any potential “signal” with a lot of “noise.”
This panel will take a hard look at where we are in terms of being “right,” and advocate for varying approaches to get us closer.
2:20 – 3:10pm
No More Trial and Error – Developing the Right Trials for the Right Patients.
Panelists: Michele Becci, VP, Industry Strategy – Medidata Solutions
Industry has moved away from the classic three phased design to approvals based on data found as early as in Phase 1b open-label studies. There is increased use of adaptive designs and master protocols as well as discussion on the level of redundancy with control arms for studies of similar drugs in the same tumor type. Clinical teams must become more agile, adapting process more frequently with faster development paths; increasingly need to work across company boundaries; harness advanced analytics to drive effective and efficient clinical development through patient finding, improved site selection, in trial monitoring, other approaches Critical to success of oncology research is adopting a flexible, unified platform that supports the modern clinical research paradigms and streamlines study conduct across the entire process
Almost 80% of cancer patients are diagnosed in a community setting and require logistical support to reach the nearest trial center. Better patient engagement and more personalized study designs are becoming more and more desirable in clinical research. Virtual trial designs already implemented have been shown to dramatically reduce or eliminate the need for ongoing site visits, allowing for research to be done at a huge cost savings to the sponsor and in a much more inclusive and convenient way for patients. A future of hybrid trial models that would embrace the use of technology to allow patients to participate remotely with little disruption to their personal lives coupled with the reality that drug development will continue still require in-person tests and evaluation. The balance between virtual and in-person settings will vary according to the needs of the study We estimate that in the future about 25 percent of clinical trials in the U.S. could be done as [completely] virtual trials. By leveraging broad capabilities for both standard and virtual protocols, customers can use what Medidata calls the “Trial Dial” to choose the right balance of site-based visits and virtual visits best suited for achieving maximum study efficiency and data quality. This type of hybrid trial will play a central role in the future of clinical research.
How Data and Technology Can Accelerate Precision Trials:
Nearly 90% of the data ever created originated in the past two years. Enormous volumes of data are generated by many data streams of different types, billions of nucleotides in the genome, hundreds laboratory test results, wearable sensor reading of dozens of kinds, medical imaging data containing many thousands or millions of pixels per image; These data present formidable challenges in how to structure, integrate and interpret this data for relevant insights to inform clinical trials. In order to achieve the tremendous gains of oncology research, data sharing and AI can fundamentally change the way trials are executed, bringing new comparators and approaches to the process. Extracting ‘fit-for-purpose’ value from the tsunami of data while maintaining quality of the data is required to rapidly bring these novel therapies to market.
3:10 – 3:40pm
Coffee & Networking
3:40 – 4:30pm
The Illusion of ‘Art’ in Medicine – Can Artificial Intelligence Bring Us Closer to Science?
4:35 – 5:25pm
Commercialization Strategies in Precision Medicine
5:30 – 6:30pm
False Negative or False Positives – The Critical Need for Consistency in NGS Controls
Single gene to targeted sequencing to whole Exome: Value Based Healthcare – what number of genes provides the best outcome for an oncology patient?
6:45 – 9:45pm
WEDNESDAY JUNE 27th, 2018
Haematological Malignancies – Progress on patient selection and response prediction
COFFEE & NETWORKING
The Power of Prediction – New Models for Neurodegenerative Disease
Moderator: Todd Sherer, Ph.D. CEO – The Michael J. Fox Foundation for Parkinson’s Research
Panelists: Darryle D. Schoepp, Ph.D., Vice President, Neuroscience Discovery Research, Merck, Melissa Nirenberg, MD, PhD – CMO – The New York Stem Cell Foundation,
Is Data Mining of Real World Evidence from Large Populations the Fastest Route to Routine Precision Medicine?
Moderator: Michael Kolodziej, MD – VP and Chief Innovation Officer, ADVI Health
Panelists: Clifford Hudis, CEO – ASCO, Mark G Kris, MD, FACP, FACCP, Oncologist, MSKCC
Despite the profound progress in the technical aspects of tumor genotyping, the clinical utility of NGS panels for somatic mutation analysis of solid tumors has continued to be an area of considerable controversy. With the recent finalization of the CMS National Coverage Determination for NGS in solid tumors, there is a real possibility that many more patients will be tested identifying potential targets. But because of coverage policy governing use of therapeutic agents directed against these targets, we will hit a new impasse in moving precision medicine forward. Certainly clinical trials like ASCO’s Tapur and NCI Match offer solutions, but the eligible patient base is huge compared to clinical trial opportunities. One potential solution is the use of real world evidence to rapidly expand the knowledge base and identify the clinical utility of novel marker/therapeutic agent combinations. But real world evidence has its own challenges. Our panelists are leaders in the real world evidence space. They will describe the current state of affairs in real world evidence; identify challenges in data collection, curation, and application; and offer a vision of how the promise of real world evidence can be realized.
Precision Exposomics: Where Practice-Based Evidence Meets Environmental Data and Network Science
Chair: Yves A. Lussier, MD, Chief Knowledge Officer, The University of Arizona
Why should we care?
• It affects precise choices of treatment (gene x environment interactions in cancer),
• Personal disease risk (exposures and zip codes – not everyone has the same susceptibility),
• opportunities for R&D: many complex combinations of exposures under studies, yet can be approaches through network science by mining practice-based evidence together with zip codes and exposures, how would the precision medicine initiative cohort contribute ?
• opportunities for practical portal (one potential outcome would be a portal where one can find unpublished – yet statistically significant- associations of diseases for a zip code where one contemplates to buy a house or establish a business unit)
NETWORKING & EXHIBITS
Blockchain in Precision Medicine – A Path to Privacy, Traceability, and Reproduceability.
Moderator: Susan Ramonat, CEO – Spiritus
Panelists: Maria Palombini, MBA. Director Emerging Technology GBSI – IEEE Standards Association, Tony Little, ND, Senior Director Integration Strategy, Optum360
Immunotherapies in the Age of Precision Medicine
Legal Challenges in the Field of Precision Medicine
Moderator: Nephi Walton, MD, MS, Assistant Professor of Genomic Medicine, Geisinger Health
Panelists: Lucy L Thompson, Esq. CISSP, Founding Principal, Livingston PLLC, Arthur E. Peabody Jr. JD, Principal at Arthur E. Peabody Jr. PLLC, Jennifer K Wagner, JD, PhD, Associate Director, Bioethics Research Assistant Professor Center for Translational Bioethics and Health Care Policy, Geisinger Research
Free Evening – Enjoy the sights and sounds of Jersey City or visit the vibrant nightlife of the City that never sleeps.
THURSDAY JUNE 28th, 2018
Investment Strategies in Precision Medicine
Moderator: Patrick Huddie, Ph.D, Partner Westbury Group LLC
COFFEE & NETWORKING
Patient Advocate Session – Meaningful Research through Collaboration to Enable Transformative Clinical Outcomes
Moderator: Peter Kapitein – Patient Advocate, Inspire2Live
Panelists: Lara Sullivan, MD, MBA, President & Founder, SpringWorks Therapeutics, Shridar Ganesan, MD, PhD. Chief Molecular Oncology, Rutgers Cancer Institute of New Jersey, Ilona Scherr – Patient Advocate, Inspire2Live, Aled Edwards, PhD., Professor, University of Toronto, CEO of the Structural Genomics Consortium
Health Disparities in Precision Medicine
Moderator: Ysabel Duron, CEO – Latino Cancer Institute
Panelists: Nishadi Rajapakse, Ph.D. Director of the Transdisciplinary Collaborative Centers for Health Disparities Research Focused on Precision Medicine, NIH, Michael Yudell, Ph.D., MPH, Associate Professor & Chair – Community Health and Prevention – Drexel University, Jay Kaufman, Ph.D., Professor, Dpt. of Epidemiology, Biostatistics and Occupational Health, McGill University