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Archive for the ‘Personalized and Precision Medicine & Genomic Research’ Category


Medical Scientific Discoveries for the 21st Century & Interviews with Scientific Leaders at https://www.amazon.com/dp/B078313281 – electronic Table of Contents 

Author, Curator and Editor: Larry H Bernstein, MD, FCAP

Available on Kindle Store @ Amazon.com since 12/9/2017

List of Contributors & Contributors’ Biographies

Volume Author, Curator and Editor

Larry H Bernstein, MD, FCAP

Preface, all Introductions, all Summaries and Epilogue

Part One:

1.4, 1.5, 1.6, 2.1.1, 2.1.2, 2.1.3, 2.1.4, 2.2.1, 2.2.2, 2.2.3, 2.3, 2.4, 2.4.1, 2.4.2, 2.5, 2.6.1, 2.6.2, 2.6.3, 2.6.4, 2.7, 2.8, 2.9, 2.10, 3.1, 3.2, 3.3, 3.4, 4.1, 4.2, 4.3

Part Two:

5.2, 5.3, 5.6, 6.1.2, 6.1.4, 6.2.1, 6.2.2, 6.3.2, 6.3.4, 6.3.5, 6.3.6, 6.3.8, 6.3.10, 6.4.1, 6.4.2, 6.5.1.2, 6.5.1.3, 6.5.2.2, 7.1, 7.2, 7.3, 7.4, 7.5, 8.1, 8.2, 8.3, 8.4, 8.5, 8.6, 8.7, 8.8, 8.9, 8.9.1, 8.9.3, 8.9.4, 8.9.5, 8.9.6, 8.10.1, 8.10.2, 8.10.3, 8.10.4, 9.2, 9.3, 9.5, 9.6, 9.7, 9.8, 9.9, 9.10, 9.11, 9.12, 9.13, 9.14, 9.15, 9.16, 10.2, 10.5, 10.6, 10.7, 10.8, 10.10, 10.11, 11.1, 11.2, 11.3, 11.5, 11.6, 11.7, 12.1, 12.2, 12.3, 12.4, 12.5, 12.7, 12.8, 12.9, 12.10, 12.11, 12.12, 13.1, 13.2, 13.3, 13.6, 13.12, 13.13, 14.1, 14.2

Guest Authors:

Pnina Abir-Am, PhD Part Two: 6.1.1

Stephen J Williams, PhDPart Two: 6.2.6, 6.5.2.2, 10.4, 10.9, 13.4

Aviva Lev-Ari, PhD, RN:

Part One:

1.1, 1.2, 1.3, 1.4, 1.5, 1.7, 2.2.1, 2.3

Part Two:

5.1, 5.4, 5.5, 5.7, 5.8, 5.9, 5.10, 5.11, 6.1.3, 6.2.3, 6.2.4, 6.2.5, 6.3.1, 6.3.3, 6.3.7, 6.3.9, 6.4.3, 6.5.1.1, 6.5.2.1, 6.5.2.2, 6.5.3.1, 6.5.4, 6.5.5, 6,5,6, 8.9.2, 8.10.2, 9.1, 9.4, 10.1, 10.3, 11.4, 12.6, 13.5, 13.7, 13.8, 13.9, 13.10, 13.11

Adam Sonnenberg, BSC, MSc(c)Part Two: 13.9

 

electronic Table of Contents

PART ONE:

Physician as Authors, Writers in Medicine and Educator in Public Health

 

Chapter 1: Physicians as Authors

Introduction

1.1  The Young Surgeon and The Retired Pathologist: On Science, Medicine and HealthCare Policy – Best writers Among the WRITERS

1.2 Atul Gawande: Physician and Writer

1.3 Editorial & Publication of Articles in e-Books by  Leaders in Pharmaceutical Business Intelligence:  Contributions of Larry H Bernstein, MD, FCAP

1.4 Abraham Verghese, MD, Physician and Notable Author

1.5 Eric Topol, M.D.

1.6 Gregory House, MD

1.7 Peter Mueller, MD  Professor of Radiology @MGH & HMS – 2015 Synergy’s Honorary Award Recipient

Summary

Chapter 2: Professional Recognition

Introduction

2.1 Proceedings

2.1.1 Research Presentations

2.1.2 Proceedings of the NYAS

2.1.3 Cold Spring Harbor Conference Meetings

2.1.4 Young Scientist Seminars

2.2 Meet Great Minds

2.2.1 Meet the Laureates

2.2.2 Richard Feynman, Genius and Laureate

2.2.3 Fractals and Heat Energy

2.3 MacArthur Foundation Awards

2.4 Women’s Contributions went beyond Rosie the Riveter

2.4.1 Secret Maoist Chinese Operation Conquered Malaria

2.4.2 Antiparasite Drug Developers Win Nobel

2.5 Impact Factors and Achievement

2.6   RAPsodisiac Medicine

2.6.1 Outstanding-achievements-in-radiology-or-radiotherapy

2.6.2 Outstanding-achievement-in-anesthesiology

2.6.3 Outstanding-achievement-in-pathology

2.6.4 Topics in Pathology – Special Issues from Medscape Pathology

2.7 How to win the Nobel Prize

2.8 Conversations about Medicine

2.9 Current Advances in Medical Technology

2.10 Atul Butte, MD, PhD

Summary

Chapter 3:  Medical and Allied Health Sciences Education

Introduction

3.1 National Outstanding Medical Student Award Winners

3.2 Outstanding Awards in Medical Education

3.3 Promoting Excellence in Physicians and Nurses

3.4 Excellence in mentoring

Summary

Chapter 4: Science Teaching in Math and Technology (STEM)

Introduction

4.1 Science Teaching in Math and Technology

4.2 Television as a Medium for Science Education

4.2.1 Science Discovery TV

4.3 From Turing to Watson

Summary

PART TWO:

Medical Scientific Discoveries Interviews with Scientific Leaders

Chapter 5: Cardiovascular System

Introduction

5.1 Physiologist, Professor Lichtstein, Chair in Heart Studies at The Hebrew University elected Dean of the Faculty of Medicine at The Hebrew University of Jerusalem

5.2 Mitochondrial Dysfunction and Cardiac Disorders

5.3 Notable Contributions to Regenerative Cardiology

5.4 For Accomplishments in Cardiology and Cardiovascular Diseases: The Arrigo Recordati International Prize for Scientific Research

5.5 Becoming a Cardiothoracic Surgeon: An Emerging Profile in the Surgery Theater and through Scientific Publications

5.6 Diagnostics and Biomarkers: Novel Genomics Industry Trends vs Present Market Conditions and Historical Scientific Leaders Memoirs

5.7 CVD Prevention and Evaluation of Cardiovascular Imaging Modalities: Coronary Calcium Score by CT Scan Screening to justify or not the Use of Statin

5.8 2013 as A Year of Revolutionizing Medicine and Top 11 Cardiology Stories

5.9 Bridging the Gap in Medical Innovations – Elazer Edelman @ TEDMED 2013

5.10 Development of a Pancreatobiliary Chemotherapy Eluting Stent for Pancreatic Ductal Adenocarcinoma PIs: Jeffrey Clark (MGH), Robert Langer (Koch), Elazer Edelman (Harvard:MIT HST Program)

5.11 Publications on Heart Failure by Prof. William Gregory Stevenson, M.D., BWH

Summary

Chapter 6: Genomics

Introduction
6.1 Genetics before the Human Genome Project

6.1.1 Why did Pauling Lose the “Race” to James Watson and Francis Crick? How Crick Describes his Discovery in a Letter to his Son

6.1.2 John Randall’s MRC Research Unit and Rosalind Franklin’s role at Kings College

6.1.3 Interview with the co-discoverer of the structure of DNA: Watson on The Double Helix and his changing view of Rosalind Franklin

6.1.4 The Initiation and Growth of Molecular Biology and Genomics, Part I

6.2 The Human Genome Project: Articles of Note  @ pharmaceuticalintelligence.com by multiple authors

6.2.1 CRACKING THE CODE OF HUMAN LIFE: The Birth of BioInformatics & Computational Genomics

6.2.2 What comes after finishing the Euchromatic Sequence of the Human Genome?

6.2.3 Human Genome Project – 10th Anniversary: Interview with Kevin Davies, PhD – The $1000 Genome

6.2.4 University of California Santa Cruz’s Genomics Institute will create a Map of Human Genetic Variations

6.2.5 Exceptional Genomes: The Process to find them

6.2.6 Multiple Lung Cancer Genomic Projects Suggest New Targets, Research Directions for Non-Small Cell Lung Cancer

6.3 The Impact of Genome Sequencing on Biology and Medicine

6.3.1 Genomics in Medicine – Establishing a Patient-Centric View of Genomic Data

6.3.2 Modification of genes by homologous recombination – Mario Capecchi, Martin Evans, Oliver Smithies

6.3.3 AAAS February 14-18, 2013, Boston: Symposia – The Science of Uncertainty in Genomic Medicine

6.3.4 The Metabolic View of Epigenetic Expression

6.3.5  Pharmacogenomics

6.3.6 Neonatal Pathophysiology

6.3.7 Genetics of Conduction Disease: Atrioventricular (AV) Conduction Disease (block): Gene Mutations – Transcription, Excitability, and Energy Homeostasis

6.3.8 3D mapping of genome in combine FISH and RNAi

6.3.9 Human Variome Project: encyclopedic catalog of sequence variants indexed to the human genome sequence

6.3.10 DNA mutagenesis and DNA repair

6.4 Scientific Leadership Recognition for Contributions to Genomics

6.4.1 Interview with Elizabeth H. Blackburn, Carol W. Greider and Jack W. Szostak (44 minutes)

6.4.2 DNA Repair Pioneers Win Nobel – Tomas Lindahl, Paul Modrich, and Aziz Sancar 2015 Nobel Prize in Chemistry for the mechanisms of DNA repair

6.4.3  Richard Lifton, MD, PhD of Yale University and Howard Hughes Medical Institute: Recipient of 2014 Breakthrough Prizes Awarded in Life Sciences for the Discovery of Genes and Biochemical Mechanisms that cause Hypertension

6.5 Contemporary Field Leaders in Genomics

6.5.1 ROBERT LANGER

6.5.1.1 2014 Breakthrough Prizes Awarded in Fundamental Physics and Life Sciences for a Total of $21 Million – MIT’s Robert Langer gets $3 Million

6.5.1.2 National Medal of Science – 2006 Robert S. Langer

6.5.1.3  Confluence of Chemistry, Physics, and Biology

6.5.2 JENNIFER DOUDNA

6.5.2.1 Jennifer Doudna, cosmology teams named 2015 Breakthrough Prize winners

6.5.2.2 UPDATED – Medical Interpretation of the Genomics Frontier – CRISPR – Cas9: Gene Editing Technology for New Therapeutics

6.5.3 ERIC LANDER

6.5.3.1  2012 Harvey Prize in April 30: at the Technion-Israel Institute of Technology to Eric S. Lander @MIT & Eli Yablonovitch @UC, Berkeley

6.5.4 2013 Genomics: The Era Beyond the Sequencing of the Human Genome: Francis Collins, Craig Venter, Eric Lander, et al.

6.5.5 Recognitions for Contributions in Genomics by Dan David Prize Awards

6.5.6   65 Nobel Laureates meet 650 young scientists covering the fields of physiology and medicine, physics, and chemistry, 28 June – 3 July, 2015, Lindau & Mainau Island, Germany

Summary

Chapter 7: The RNAs

Introduction

7.1 RNA polymerase – molecular basis for DNA transcription – Roger Kornberg, MD

7.2  One gene, one protein – Charles Yanofsky

7.3 Turning genetic information into working proteins – James E. Darnell Jr.

7.4 Small but mighty RNAs – Victor Ambros, David Baulcombe, and Gary Ruvkun, Phillip A. Sharp

7.5 Stress-response gene networks – Nina V. Fedoroff

Summary

Chapter 8: Proteomics, Protein-folding, and Cell Regulation
Introduction.

8.1 The Life and Work of Allan Wilson

8.2 Proteomics

8.3 More Complexity in Protein Evolution

8.4 Proteins: An evolutionary record of diversity and adaptation

8.5 Heroes in Basic Medical Research – Leroy Hood

8.6 Ubiquitin researchers win Nobel – Ciechanover, Hershko, and Rose awarded for discovery of ubiquitin-mediated proteolysis

8.7 Buffering of genetic modules involved in tricarboxylic acid cycle metabolism provides homeostatic regulation

8.8 Dynamic Protein Profiling

8.9 Protein folding

8.9.1 Protein misfolding and prions – Susan L. Lindquist, Stanley B. Prusiner

8.9.2 A Curated Census of Autophagy-Modulating Proteins and Small Molecules Candidate Targets for Cancer Therapy

8.9.3 Voluntary and Involuntary S-Insufficiency

8.9.4 Transthyretin and Lean Body Mass in Stable and Stressed State

8.9.5 The matter of stunting in the Ganges Plains

8.9.6 Proteins, Imaging and Therapeutics

8.10 Protein Folding and Vesicle Cargo

8.10.1 Heat Shock Proteins (HSP) and Molecular Chaperones

8.10.2 Collagen-binding Molecular Chaperone HSP47: Role in Intestinal Fibrosis – colonic epithelial cells and sub epithelial myofibroblasts

8.10.3 Biology, Physiology and Pathophysiology of Heat Shock Proteins

8.10.4 The Role of Exosomes in Metabolic Regulation 


Summary

Chapter 9:  Neuroscience

Introduction

9.1 Nobel Prize in Physiology or Medicine 2013 for Cell Transport: James E. Rothman of Yale University; Randy W. Schekman of the University of California, Berkeley; and Dr. Thomas C. Südhof of Stanford University

9.2 Proteins that control neurotransmitter release – Richard H. Scheller

9.3 Heroes in Basic Medical Research – Robert J. Lefkowitz

9.4 MIND AND MEMORY: BIOLOGICAL AND DIGITAL – 2014 Dan David Prize Symposium

9.5 A new way of moving – Michael Sheetz, James Spudich, Ronald Vale

9.6 Role the basal ganglia

9.7 The Neurogenetics of Language – Patricia Kuhl – 2015 George A. Miller Award

9.8 The structure of our visual system

9.9 Outstanding Achievement in Schizophrenia Research

9.10 George A. Miller, a Pioneer in Cognitive Psychology, Is Dead at 92

9.11 – To understand what happens in the brain to cause mental illness

9.12 Brain and Cognition

9.13 – To reduce symptoms of mental illness and retrain the brain

9.14 Behavior

9.15 Notable Papers in Neurosciences

9.16 Pyrroloquinoline quinone (PQQ) – an unproved supplement

Summary

Chapter 10: Microbiology & Immunology

Introduction

10.1 Reference Genes in the Human Gut Microbiome: The BGI Catalogue

10.2 Malnutrition in India, high newborn death rate and stunting of children age under five years

10.3 In His Own Words: Leonard Herzenberg, The Immunologist Who Revolutionized Research, Dies at 81

10.4 Heroes in Medical Research: Dr. Robert Ting, Ph.D. and Retrovirus in AIDS and Cancer

10.5 Tang Prize for 2014: Immunity and Cancer

10.6 Halstedian model of cancer progression

10.7 The History of Hematology and Related Sciences

10.8 Pathology Emergence in the 21st Century

10.9 Heroes in Medical Research: Barnett Rosenberg and the Discovery of Cisplatin

10.10  T cell-mediated immune responses & signaling pathways activated by TLRs – Bruce A. Beutler, Jules A. Hoffmann, Ralph M. Steinman

10.11 Roeder – the coactivator OCA-B, the first cell-specific coactivator, discovered by Roeder in 1992, is unique to immune system B cells

Summary

Chapter 11: Endocrine Hormones

Introduction

11.1 Obesity – 2010 Douglas L. ColemanJeffrey M. Friedman

11.2 Lonely Receptors: RXR – Jensen, Chambon, and Evans – Nuclear receptors provoke RNA production in response to steroid hormones

11.3 The Fred Conrad Koch Lifetime Achievement Award—the Society’s highest honor—recognizes the lifetime achievements and exceptional contributions of an individual to the field of endocrinology

11.4 Gerald D Aurbach Award for Outstanding Translational Research

11.5 Roy O. Greep Award for Outstanding Research in Endocrinology – Martin M. Matzuk

11.6 American Physiology Society Awards

11.7 Solomon Berson and Rosalyn Yalow

Summary

Chapter 12. Stem Cells

Introduction

12.1 Mature cells can be reprogrammed to become pluripotent – John Gurdon and Shinya Yamanaka

12.2 Observing the spleen colonies in mice and proving the existence of stem cells – Till and McCulloch

12.3 McEwen Award for Innovation: Irving Weissman, M.D., Stanford School of Medicine, and Hans Clevers, M.D., Ph.D., Hubrecht Institute

12.4 Developmental biology

12.5  CRISPR/Cas-mediated genome engineering – Rudolf Jaenisch

12.6 Ribozymes and RNA Machines –  Work of Jennifer A. Doudna

12.7 Ralph Brinster, ‘Father of Transgenesis’

12.8 Targeted gene modification

12.9 Stem Cells and Cancer

12.10 ALPSP Awards

12.11 Eppendorf Award for Young European Investigators

12.12 Breaking news about genomic engineering, T2DM and cancer treatments

Summary
Chapter 13: 3D Printing and Medical Application

Introduction

13.1 3D Printing

13.2 What is 3D printing?

13.3 The Scientist Who Is Making 3D Printing More Human

13.4 Join These Medical 3D Printing Groups on Twitter and LinkedIn for great up to date news

13.5 Neri Oxman and her Mediated Matter group @MIT Media Lab have developed a technique for 3D-printing Molten Glass

13.6 The ‘chemputer’ that could print out any drug

13.7 3-D-Bioprinting in use to Create Cardiac Living Tissue: Print your Heart out

13.8 LPBI’s Perspective on Medical and Life Sciences Applications – 3D Printing: BioInks, BioMaterials-BioPolymer

13.9 Medical MEMS, Sensors and 3D Printing: Frontier in Process Control of BioMaterials

13.10 NIH and FDA on 3D Printing in Medical Applications: Views for On-demand Drug Printing, in-Situ direct Tissue Repair and Printed Organs for Live Implants

13.11 ‘Pop-up’ fabrication technique trumps 3D printing

13.12 Augmentation of the ONTOLOGY of the 3D Printing Research

13.13 Superresolution Microscopy

Summary

Chapter 14: Synthetic Medicinal Chemistry

Introduction

14.1 Insights in Biological and Synthetic Medicinal Chemistry

14.2 Breakthrough work in cancer

Summary to Part Two

Volume Summary and Conclusions

EPILOGUE

 

 

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PMC Comment Letter on Centers for Medicare & Medicaid Services: Innovation Center New Direction

Guest Author: Cynthia A. Bens, Vice President, Public Policy, PMC

cbens@personalizedmedicinecoalition.org

NOVEMBER 27, 2017

In response to a Wall Street Journal op-ed and request for information about innovative ways to pay for and deliver health care in the U.S., the Personalized Medicine Coalition has encouraged the Centers for Medicare & Medicaid Services (CMS) to spearhead models that empower physicians to move away from the current standard of care when patient outcomes can be improved by tailoring care to a patient’s genetics and other factors:

In keeping with PMC’s mission to underline the significance of personalized medicine to patients and the health system, the Coalition’s comment letter contends that personalized medicine products and services can increase the overall value of dollars spent by improving health outcomes.

CMS’ previous efforts to advance new payment models, the letter notes, were met with resistance largely because they focused on reducing overall health care costs without adequately considering whether those reductions would result in a disproportionate decrease in the outcomes that matter to patients.

PMC indicates in the letter that the guiding principles put forth in CMS’ request for information provide “reasonable assurance” that the agency plans to proceed at “a more measured pace” going forward.

“We believe that personalized medicine has the potential to help CMS deliver on its goal of [affordable, accessible health care] if [the agency] focuses on maximizing individual patient outcomes, if new models are fully evaluated before large-scale implementation, if payment is not rooted in current standard of care, and if physicians have the flexibility to tailor care based on a patient’s genetics and other factors,” the letter reads.

Please contact Cynthia A. Bens, Vice President, Public Policy, at cbens@personalizedmedicinecoalition.org with questions about PMC’s comment letter.

###

Personalized Medicine Coalition
1710 Rhode Island Ave. NW; Suite 700
Washington, D.C. 20036
Blog: Education & Advocacy
Twitter: @PerMedCoalition
www.PersonalizedMedicineCoalition.org

SOURCE

From: Personalized Medicine Coalition <messages@app.production.membersuite.com>
Reply-To: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>
Date: Monday, November 27, 2017 at 2:58 PM
To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>
Subject: PMC to CMS: To Increase Value, Empower Physicians to Tailor Care, Optimize Outcomes

 and

From: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>

Date: Tuesday, November 28, 2017 at 7:34 AM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: Re: PMC to CMS: To Increase Value, Empower Physicians to Tailor Care, Optimize Outcomes

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Reporter and Curator: Dr. Sudipta Saha, Ph.D.

 

A mutated gene called RAS gives rise to a signalling protein Ral which is involved in tumour growth in the bladder. Many researchers tried and failed to target and stop this wayward gene. Signalling proteins such as Ral usually shift between active and inactive states.

 

So, researchers next tried to stop Ral to get into active state. In inacvtive state Ral exposes a pocket which gets closed when active. After five years, the researchers found a small molecule dubbed BQU57 that can wedge itself into the pocket to prevent Ral from closing and becoming active. Now, BQU57 has been licensed for further development.

 

Researchers have a growing genetic data on bladder cancer, some of which threaten to overturn the supposed causes of bladder cancer. Genetics has also allowed bladder cancer to be reclassified from two categories into five distinct subtypes, each with different characteristics and weak spots. All these advances bode well for drug development and for improved diagnosis and prognosis.

 

Among the groups studying the genetics of bladder cancer are two large international teams: Uromol (named for urology and molecular biology), which is based at Aarhus University Hospital in Denmark, and The Cancer Genome Atlas (TCGA), based at institutions in Texas and Boston. Each team tackled a different type of cancer, based on the traditional classification of whether or not a tumour has grown into the muscle wall of the bladder. Uromol worked on the more common, earlier form, non-muscle-invasive bladder cancer, whereas TCGA is looking at muscle-invasive bladder cancer, which has a lower survival rate.

 

The Uromol team sought to identify people whose non-invasive tumours might return after treatment, becoming invasive or even metastatic. Bladder cancer has a high risk of recurrence, so people whose non-invasive cancer has been treated need to be monitored for many years, undergoing cystoscopy every few months. They looked for predictive genetic footprints in the transcriptome of the cancer, which contains all of a cell’s RNA and can tell researchers which genes are turned on or off.

 

They found three subgroups with distinct basal and luminal features, as proposed by other groups, each with different clinical outcomes in early-stage bladder cancer. These features sort bladder cancer into genetic categories that can help predict whether the cancer will return. The researchers also identified mutations that are linked to tumour progression. Mutations in the so-called APOBEC genes, which code for enzymes that modify RNA or DNA molecules. This effect could lead to cancer and cause it to be aggressive.

 

The second major research group, TCGA, led by the National Cancer Institute and the National Human Genome Research Institute, that involves thousands of researchers across USA. The project has already mapped genomic changes in 33 cancer types, including breast, skin and lung cancers. The TCGA researchers, who study muscle-invasive bladder cancer, have looked at tumours that were already identified as fast-growing and invasive.

 

The work by Uromol, TCGA and other labs has provided a clearer view of the genetic landscape of early- and late-stage bladder cancer. There are five subtypes for the muscle-invasive form: luminal, luminal–papillary, luminal–infiltrated, basal–squamous, and neuronal, each of which is genetically distinct and might require different therapeutic approaches.

 

Bladder cancer has the third-highest mutation rate of any cancer, behind only lung cancer and melanoma. The TCGA team has confirmed Uromol research showing that most bladder-cancer mutations occur in the APOBEC genes. It is not yet clear why APOBEC mutations are so common in bladder cancer, but studies of the mutations have yielded one startling implication. The APOBEC enzyme causes mutations early during the development of bladder cancer, and independent of cigarette smoke or other known exposures.

 

The TCGA researchers found a subset of bladder-cancer patients, those with the greatest number of APOBEC mutations, had an extremely high five-year survival rate of about 75%. Other patients with fewer APOBEC mutations fared less well which is pretty surprising.

 

This detailed knowledge of bladder-cancer genetics may help to pinpoint the specific vulnerabilities of cancer cells in different people. Over the past decade, Broad Institute researchers have identified more than 760 genes that cancer needs to grow and survive. Their genetic map might take another ten years to finish, but it will list every genetic vulnerability that can be exploited. The goal of cancer precision medicine is to take the patient’s tumour and decode the genetics, so the clinician can make a decision based on that information.

 

References:

 

https://www.ncbi.nlm.nih.gov/pubmed/29117162

 

https://www.ncbi.nlm.nih.gov/pubmed/27321955

 

https://www.ncbi.nlm.nih.gov/pubmed/28583312

 

https://www.ncbi.nlm.nih.gov/pubmed/24476821

 

https://www.ncbi.nlm.nih.gov/pubmed/28988769

 

https://www.ncbi.nlm.nih.gov/pubmed/28753430

 

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Tweets by @pharma_BI and @AVIVA1950 for #PMConf  at The 13th Annual Personalized Medicine Conference, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

Curator: Aviva Lev-Ari, PhD, RN

 

@pharma_BI

@AVIVA1950

 

All TWEETS from LPBI’s Twitter.com handles at #PMConf 

@pharma_BI

@AVIVA1950

  1. Aviva Lev-Ari Retweeted Gary An

    nice comment

    Aviva Lev-Ari added,

  2. Narrative plan unsupported by facts

  3. Robert C. Green, M.D., M.P.H., Director, Genomes2People Research Program, Professor of Medicine (Genetics), Brigham and Women’s Hospital, Broad Institute and Harvard Medical School pharmacogenomics can harm if odds are so low adherence will be lower

  4. Michael Snyder, Ph.D., Stanford W. Ascherman Professor, Chair, Department of Genetics, Director, Center of GenomiPersonalized Medicine, Stanford University School of Medicine Personal sequencing for multiple etiologies rich people are sequenced

  5. Tom Miller, M.S., Founder, Managing Partner, GreyBird Ventures LLC duty to call up therapies that will not work, balance addressed by PM – diagnostics in PM clinical utility from patient selection for the therapy the patient will respond to

  6. Sandro Galea, M.D., School of Public Health, Boston University US expense on Health care the highest in the World comes on the expense of housings, mental health, education – curative vs preventive care MDs are insentiviced to keep patients sick

  7. Robert C. Green, M.D., Broad Institute and HMS Platinum vs gold standard 59 genes will identify 80,000 will get the disease and 47,000 will never get the disease, is the technology the reason for investment vs Family history?

  8. Bryce Olson, Global Marketing Director, Health and Life Sciences Group, Intel Corporation Genome sequencing found his Pi3K Pathway – PIK3CA p.E54 – Anti Inhibitor for Pi3K = Precision Medicine

  9. Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA 21st Century – metastatic solid tumors – 900 patients: accommodated plan Lab developed Tests: new approach Efficiency, transparency

  10. innovation INFORMS at NIH Center of Excellence – data collection and analysis of multiple data types Biometric sensors collecting data on cancer patients collaboration with Academia, single arm vs randomized decentralized devices are collecting data

  11. FDA considers N of One, small samples, EGFR drug was approved in 2 1/2 years since Phase 1 of NDA New trial designs: reduce bias and alternative end points narrow criteria for participation, more personalized and more patient-centered innovation

  12. Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA Advances of technology of biomarkers, disease indication Accelerated approval by FDA a collaborative of speeding the process companion diagnosis assays

  13. Unmet need, commitment is there, innovation and connectivity drive access, collaboration not competition – helps Precision medicine in emerging nations. Access to PM anywhere in the world suggested Kristin Pothier, MS, Global Head, Life Sciences EY

  14. Stephen L. Eck, M.D., Ph.D., President, CEO, Aravive Biologics; Board Chairman, PMC Laxo – A molecular target to be found by diagnostics TEST — as a basis to develop a drug Pricing and value – dimensions of Value to society How PM is done today?

  15. Marc S. Williams, MD Geisinger Clinical Genomics vs Physician specialist (i.e.,hypercholestoralemia), both in same place – paper and EMR Outcomes – tracking patients over decades – systems in place to capture the data Virtual Cycle Clinical data

  16. Timothy Cannon, M.D., Inova Molecular Tumor Board, 5 hospital in VA, Precision Genomics Cancer Therapy Poor understanding of molecular results by MDs, Refractory Patients no Forum to discuss other options 220 patients presented beyond InovaOncologi

  17. Scott A. Beck, Mayo Clinic, MN, AZ PM, Genomics sequencing, BioEthics, IT, Translational Perspective in Epi-genomics, Discovery to Translation Applicattions Pharmacy- Formulary – EMR – Champions from Disease areas to practice environment Testing

  18. payment dominates delivery of care, future PM from Genomics cost to patients Transform acceptability of PM suggested Ronald A. Paulus, M.D., M.B.A., President, CEO, Mission Health, NC, ex-Geisinger, CIO

  19. Genomics based PM to be turned into Wellness Strategy – the path not yet knows said Jeffrey R. Balser, M.D., Ph.D., Dean, Vanderbilt University School of Medicine; President, CEO, Vanderbilt University Medical Center, Nashville, TN

  20. Millianlian Diabetics NOT on Medicare, Analytics: iPhone telling patient dishes to order since SYSTEM KNOWS BLOOD SUGER 24×7 – target care by Analytics Genomics paid by NIH PM Analytics is built at Vanderbilt University MC, Jeffrey R. Balser, CEO

  21. Survival of patient with mutation and targeted drug LIVE LONGER David B. Roth, M.D., Ph.D., Simon Flexner Professor Chair, Pathology and Laboratory Medicine, Perelman School of Medicine at University of Pennsylvania

  22. Lotte Steuten, Ph.D., School of Pharmacy at University of Washington, Seattle aggregate big data , models as evidence, has value to clinical, the model under development NGS Profile of Patient vs current standard of care.

  23. David B. Roth, M.D., Ph.D., UPenn Director, Penn Center for Precision Medicine 5000 patients underwent genome sequencing Interpretation is the issue that is hard Health IT are still in silos: Pharmacy data, financial data, EMR

  24. Michael Pellini, M.D., M.B.A., Chairman, Board of Directors, Foundation Medicine; Board Member, Personalized Medicine Coalition, we know there is value in PM we need to work together on the challenges — to prove the value in PM

  25. Andrea Stern Ferris, M.B.A., President, Chairman of the Board, LUNGevity Foundation – PATIENT to be included in the conversation patient after successful treatment have hope work pay taxes pay to health plans continue family life

  26. Molecular Era, NEJM, 2017, 377, 1813-1823, BRAF in Melanoma – 80% do not need additional therapy vs 20% benefitted in the Non-Molecular Era, data by Dane J. Dickson, CureOne (formerly MED-C); Oregon Health and Science University

  27. CURES – CAR-T are they cures??? A teen-ager’s Value-based Price: $475,000 x years lived suggests  Steven D. Pearson, M.D., M.Sc., Founder, President, Institute for Clinical and Economic Review (ICER)

  28. Of 134 drugs in development – 42 have the potential to become Personalized medicine therapies, said Stephen J. Ubl, President, CEO, PhRMA

  29. Transplantation vs enhancement – resistance to senescence and pathogens to be achieved by gene editing suggests George M. Church, Ph.D., Professor of Genetics

  30. Regulatory oversight on engineering embrios is coming, metric of success in recruitment of patients said Arthur L. Caplan, Ph.D., Drs. William F. and Virginia Connolly Mitty Chair, Director, Division of Medical Ethics, New York Univ

  31. CRISPR does not handle all mutation many require a different editing tool said George M. Church, Ph.D., Professor of Genetics, Health Sciences and Technology, Harvard-MIT Division of Health Sciences and Technology

  32. understand well enough  the gentic application where CRISPR will assist medicine: Retinal degeneration, two aspects one worked in Japan said Katrine Bosley, CEO, Editas Medicine

  33. Aviva Lev-Ari Retweeted Aviva Lev-Ari

    Amazing Power in hands of informed patients

    Aviva Lev-Ari added,

  34. Patients input and sophistication increased – IRB is not aware of the engagement of Patients and their challenging feedback say Deborah Schrag, M.D., M.P.H, Dana Farber

  35. Physicians needs interfaces, dashboard information delivered to MDs, data sits unused, new tools are needed for the data display by relevance to the MDs – clinicians needs decision support in their office

  36. Standards: Toxicity criteria – library of 882 symptoms, Patient reported outcomes by Patients, Resist criteria applied to imaging data criteria for brain tumors said Deborah Schrag, M.D., M.P.H., Chief Medical Oncology, Dana-Farber

  37. drafting document on Verify data integrity in clinical trials, detect discrepancies compromise the integrity of the data – audits by FDA said Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA

  38. pre-existing autoimmune disease – not indicated for them Immunotherapy even though patients wish to try said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute

  39. Drug approved for one indication, provide new data for supplemental indications said Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA

  40. Eric G. Klein, Pharm.D, Eli Lilly Aggregate burden of disease, existence of co-morbidities Genomics: WHY is explained – precise tools data vs intelligence – interoperability Past clinical trial, replicate studies retrospective data

  41. linkages vs computational techniques we do not have consistent data, data structured Vital sign or WBC count – we have data standardization is evolving said Deborah Schrag, M.D., M.P.H., Chief, Dana-Farber Cancer Institute

  42. use data sets prospective vs retrospective studies asked Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Flatiron Health; Board Member, Personalized Medicine Coalition

  43. Clinical sense vs research context, FDA is more comfortable with other than oncology products beyond drugs, namely diagnostics, diagnostics company seeking partnership with many drug areas Thermo FIscher and Novartis partnership

  44. Cost of CT Scan vs an NGS Test – Genomic testing is much cheaper yet volume is still low said Jacob S. Van Naarden, Chief Business Officer, Loxo Oncology

  45. NGS – time results come back what the mutation mean? NOW results come in few days, data analysis assist the said Joydeep Goswami, Ph.D., M.B.A., M.S., President, Clinical Next-Generation Sequencing, Oncology, Thermo Fisher Scientifi

  46. 3D BioPrinting of Drugs and the innovation storm of agents — are both benefits, value based pricing, elasticities, is that price sufficient to support R&D, dynamic environment said Joshua Ofman, SVP, Global Value, Access, Amgen

  47. Awardee of Leadership in PM, Illumina, HC system not yet ready for Precision Medicine

  48. Amgen and Harvard Pilgrims interpretation of Values related to partnerships: Novartis

  49. at Illimina – Consumer Advocacy added to Technology breakthroughs in genome sequencing said Jay T. Flatley, M.S., Executive Chairman, Illumina

  50. National Genomic Service – Sequencing becoming STANDARD of Care, phynotypes, $10 million to be spent NIH said Jay T. Flatley, M.S., Executive Chairman, Illumina

  51. 13th Annual Leadership in Personalized Medicine Award AWARDEE | Jay T. Flatley, M.S., Executive Chairman, Illumina

  52. 13th Annual Leadership in PM Award to Jay T Flatlet, Illumina

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LIVE Day Two – 13th Annual Personalized Medicine Conference, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

 

JOSEPH B. MARTIN CONFERENCE CENTER

HARVARD MEDICAL SCHOOL, BOSTON, MA 02115

http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/13th_Annual_PM_Conference37.pdf

Leaders in Pharmaceutical Business Intelligence (LPBI) Group, Boston

pharma_bi-background0238

will cover in REAL TIME the 13th Annual Personalized Medicine, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

In attendance, covering LIVE using Social Media

Aviva Lev-Ari, PhD, RN

Editor-in-Chief

http://pharmaceuticalintelligence.com

@pharma_BI

@AVIVA1950

#PMConf

Agenda · Part II  NOVEMBER 16, 2017 · CONFERENCE PROGRAM | AGENDA

7:00 am Registration and Breakfast

8:00 am Opening Remarks

SPEAKER | Stephen L. Eck, M.D., Ph.D., President, CEO, Aravive Biologics; Board Chairman, Personalized Medicine Coalition

  • 2005 at Pfizer new initiative on Personalized Medicine
  • Left to go to Lilly – not to give a drug to  Patients with KRAS mutation – beginning of PM
  • Laxo – A molecular target to be found by diagnostics TEST  — as a basis to develop a drug
  • Pricing and value – dimensions of Value to society
  • How PM is done today

8:10 am Clinical Adoption of Personalized Medicine: A Two-Part Discussion Pioneering health care providers have begun to explore the business models, operational processes, IT infrastructure and educational programs that are needed to catalyze the paradigm shift toward personalized medicine. This two-part session on clinical adoption will examine the strategic and day-to-day challenges clinical organizations face as they seek to integrate personalized medicine in clinical settings — and the solutions they employ to address those challenges.

SESSION CHAIR | Marcia A. Kean, M.B.A., Chairman, Strategic Initiatives, Feinstein Kean Healthcare

Discussion Part 1

8:15 am The Case for Personalized Medicine in the Clinic: The View From the Corner Office Inspiring an organizational commitment to a new way of practicing medicine requires visionary leadership. This fireside chat will highlight the viewpoints and approaches of leaders who are spearheading efforts to adopt personalized medicine at clinical institutions, with an eye on the value proposition for changing existing norms and practices.

MODERATOR | Howard L. McLeod, Pharm.D., Medical Director, The DeBartolo Family Personalized Medicine Institute, Chair, Department of Individualized Cancer Management, Senior Member, Division of Population Sciences, Moffitt Cancer Center; Board Member, Personalized Medicine Coalition

Jeffrey R. Balser, M.D., Ph.D., Dean, Vanderbilt University School of Medicine; President, CEO, Vanderbilt University Medical Center, Nashville, TN

  • Vanderbilt University Medical Center [$500 Million in Grants] is a HealthCare System, split from Vanderbilt University School of Medicine [1200 Medical Residents], now they are like Partners
  • BioMedical Informatics very strong at Vanderbilt University School of Medicine
  • Jeffrey R. Balser, M.D., Ph.D. was first student of Dan, A pioneer in Personalized Medicine
  • PM and Non-Oncology: PLAVIX – DSS when MD order drugs DSS trigger No PLAVIX to this patient, made second decision in real time by MDs
  • Nashville – four flagship hospitals, largest HMO not for profit in the country
  • PM at Vanderbilt University Medical Center – 25 drugs – given ONLY after Genomic sequencing
  • Millianlian Diabetics NOT on Medicare, Analytics: iPhone telling patient what dishes to order since SYSTEM KNOWS BLOOD SUGER 24×7 – target care by Analytics
  • Genomics paid by NIH
  • PM Analytics is built at Vanderbilt University Medical Center said Jeffrey R. Balser, M.D., Ph.D., Dean, Vanderbilt University School of Medicine; President, CEO, Vanderbilt University Medical Center
  • what is the economics benefit of Genomic sequencing: DSS on Drugs – Peterson is documenting drug class by economic benefit – bundle to show value
  • Genomics based PM is on drugs — polimorthism – this drug will not work
  • Disease counseling is harder than drug = wellness strategy is the Fututre
  • Genomics based PM to be turned into Wellness Strategy – the path not yet knows said Jeffrey R. Balser, M.D., Ph.D., Dean, Vanderbilt University School of Medicine; President, CEO, Vanderbilt University Medical Center, Nashville, TN
  • Research Investment is R&D, decisions made to guide all research and investment in PM initiative – not generating money. Pilot studies leads to grants. One study is on Economic benefit of PM

Ronald A. Paulus, M.D., M.B.A., President, CEO, Mission Health, NC, ex-Geisinger, CIO

  • 8 most western counties in NC
  • small group of Genetists:
  1. PM and Oncology and – Cancer therapy from a Genomics stand point
  2. PM and Non-Oncology – Drug-Drug interactions
  3. Clinicians needs actionable information
  4. Primary care practices to adopt PM – HOW, where, why? what will be out of pocket expense to the individual
  5. subsidization is a must
  6. payment dominates delivery of care, future PM from Genomics cost to patients
  7. Transform acceptability of PM suggested Ronald A. Paulus, M.D., M.B.A., President, CEO, Mission Health, NC, ex-Geisinger, CIO

Discussion Part 2

9:00 am Practicing Personalized Medicine: Lessons From the Front Lines To successfully integrate personalized medicine into a health system, administrators and clinicians must also design and implement new processes related to program infrastructure and informatics; help educate physicians and patients about the field; and inspire cultural change within the institution. During this panel discussion, a group of early adopters will share lessons learned from implementing pilot programs across the United States.

MODERATOR | Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, Personalized Medicine Coalition

Bonnie J. Addario, Founder, Chair, Bonnie J. Addario Lung Cancer Foundation; Board Member, Personalized Medicine Coalition; Lung Cancer Survivor

Scott A. Beck, M.B.A., Administrator, Center for Individualized Medicine, Mayo Clinic, MN, AZ

  • PM, Genomics sequencing, BioEthics, IT, Translational Perspective in Epi-genomics,
  • Discovery to Translation Applicattions
  • Pharmacy- Formulary – EMR – Champions from Disease areas to practice environment
  • Testing offering, approve value
  • 25 Projects: PharmacoGenomics Testing to patients, change drug before repeat endoscopy

Timothy Cannon, M.D., Clinical Director, Inova Schar Cancer Institute Molecular Tumor Board

  • 5 hospital in VA,
  • Precision Genomics Cancer Therapy
  • Poor understanding of molecular results by MDs, Refractory Patients – no Forum to discuss other options
  • Molecular Tumor Board for Inova Health System: 220 patients presented beyond Inova
  • Oncologists had concerned that patients are aware of drug that MD can’t deliver to client

Peter Hulick, M.D., M.M.Sc., Medical Director, Center for Personalized Medicine, NorthShore University HealthSystem

  • 4 hospitals – 950 MDs
  • PCP to get engaged
  • Neurology
  • Genetic Assessment tool DSS offers PCP option for electronic ordering of the Genetic Testing, Results appear in Patients’ charts
  • Proactive testing
  • 20,000 patients in the system to be tested for pharmaco-genomics testing

Marc S. Williams, M.D., F.A.A.P., F.A.C.M.G., F.A.C.M.I., Director, Genomic Medicine Institute, Geisinger – Central PA

  • 30% of providers are Geisinger the rest are not
  • Genomics: PM — Microbiome bank – broad user consent: recontact and return results
  • Genomics Medicine Institute – 2014 Partnership with Regeneron – genomics sequencing and profiling — all result to be used by Geisinger
  • 170,000 patient consented – 90% responded, 8,000 sequences available
  • 80 gene with potential actionability – interpretation by Scientists
  • Pathologic calls return results –
  • Clinical Genomics vs Physician specialist (i.e.,hypercholestoralemia), both in same place – paper and EMR
  • Outcomes – tracking patients over decades – systems in place to capture the data
  • Virtual Cycle Clinical data – dashboards were created to deliver results per week.
  • 1/2 people do not need criteria
  • Geisinger will disseminate internationally

10:15 am Networking Break (sponsored by Moffitt Cancer Center)

10:45 am Harvard Business School Case Study — Intermountain Healthcare: Pursuing Precision Medicine Intermountain has a long history of being at the forefront of health care quality improvement and the development of treatment protocols. In 2013, Intermountain Precision Genomics (IPG) was started with Dr. Lincoln Nadauld as its Executive Director. IPG focused on stage 4 cancer patients and performed three distinct functions: genomic sequencing, interpretation of sequencing results with recommendations for precision therapies, and drug acquisition and reimbursement. A paper published in February 2017 reported that in addition to having a higher quality of life, patients who received the targeted therapies had progression-free survival rates of almost twice as long as other patients. The purpose of our case discussion will be to assess these efforts, to consider their broader applicability and to review IPG’s plans for the future.

PRESENTER | Richard Hamermesh, D.B.A., Co-Faculty Chair, Harvard Business School Kraft Precision Medicine Accelerator

12:00 pm Overview of the International Landscape for Personalized Medicine

PRESENTER | Kristin Pothier, M.S., Global Head, Life Sciences, Parthenon-EY

  • Precision Medicine in the Globe – stackholder ecosystem
  • India
  • Latin America
  • USA
  • Africa
  • EU
  • APAC
  • Middle East

Regional Spotlight:

China – strength emerging 1.4 Billion people, 4.2 million annual incidence of Cancer – systemic challenges will limit access

Brazil – 440,000 cancer incidents a year, 207 Million Corporate, Hospital, Government, Academic research, collaboration vs competition, collaboration will win

Dubai – 28.5 Million population

Qatar

UAE – Initiative to sequence the entire population, 6,000 done

Saudi Arabia

Middle East – 0.4 oncologist for 100,000

Summary – Unmet need, commitment is there, innovation and connectivity drive access, collaboration not competition – helps Precision medicine in emerging nations. Access to PM anywhere in the world suggested Kristin Pothier, M.S., Global Head, Life Sciences, Parthenon-EY

12:30 pm Bag Lunch

1:30 pm Personalized Medicine at FDA: An Inside Look at the Agency’s Priorities for the Field

INTRODUCTION | Cynthia A. Bens, Vice President, Public Policy, Personalized Medicine Coalition

KEYNOTE | Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA

  • Advances of technology of biomarkers, disease indication
  • Accelerated approval by FDA a collaborative of speeding the process
  • companion diagnosis assays and drug or biologics
  • FDA considers N of One, small samples, EGFR drug was approved in 2 1/2 years since Phase 1 of NDA
  • New trial designs: reduce bias and alternative end points
  • narrow criteria for participation, more personalized and more patient-centered
  • innovation INFORMS at NIH Center of Excellence – data collection and analysis of multiple data types
  • Biometric sensors collecting data on cancer patients
  • collaboration with Academia, single arm vs randomized, decentralized
  • devices are collecting data in clinical trials
  • 21st Century – metastatic solid tumors – 900 patients: accommodated plan
  • Lab developed Tests: new approach
  • Efficiency, transparency

2:00 pm The Patient Perspective on Personalized Medicine

INTRODUCTION | Susan McClure, Founder, Publisher, Genome magazine; Board Member, Personalized Medicine Coalition

  • Precision medicine and relevant information for Patients
  • Genomic sequencing is the Opening Gate to PM

KEYNOTE | Bryce Olson, Global Marketing Director, Health and Life Sciences Group, Intel Corporation

  • genome sequencing found his Pi3K Pathway – PIK3CA p.E54 – Anti Inhibitor for Pi3K = Precision Medicine

2:30 pm Patient 2.0: Exploring the Future of Personalized Medicine Many observers speculate that the coming wave of gene editing, gene therapy, direct-to-consumer genetic tests and the personalized use of wearables will change the psychology, sociology, economy and efficacy of health care. Informed by the previous panel discussions, this conversation will examine the future of personalized medicine and the merits of these emerging trends.

MODERATOR | Robert C. Green, M.D., M.P.H., Director, Genomes2People Research Program, Professor of Medicine (Genetics), Brigham and Women’s Hospital, Broad Institute and Harvard Medical School

  • Neurologist first , Geneticist thereafter
  • Platinum vs gold standard
  • 59 genes will identify 80,000 will get the disease and 47,000 will never get the disease
  • is the technology the reason for investment vs Family history
  • pharmacogenomics can harm, if odds are so low than adherence will be lower

Sandro Galea, M.D., M.P.H., Dr.P.H., Dean, Robert A. Knox Professor, School of Public Health, Boston University

  • Skeptical of societal aspects: race, 50% of the country health getting better vs 50% getting worse but enthusiast on technology
  • US expense on Health care the highest in the World comes on the expense of housings, mental health, education – curative vs preventive care
  • MDs are insentiviced to keep patients sick
  • Folic Acid
  • Nudge behavior
  • invest in long livivng

Tom Miller, M.S., Founder, Managing Partner, GreyBird Ventures LLC

  • duty to call up therapies that will not work, balance addressed by PM – diagnostics in PM
  • clinical utility from patient selection for the therapy the patient will respond to
  • fantasy: Medical decision making to be made to avoid un neccesary care

Michael Snyder, Ph.D., Stanford W. Ascherman Professor, Chair, Department of Genetics, Director, Center of Genomics and Personalized Medicine, Stanford University School of Medicine

  • Personal sequencing for multiple etiologies
  • rich people come to get sequenced
  • libraries
  • Providers to be incentivized if patients are health

3:30 pm Closing Remarks

SPEAKER | Edward Abrahams, Ph.D., President, Personalized Medicine Coalition

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LIVE Day One – 13th Annual Personalized Medicine Conference, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

 

JOSEPH B. MARTIN CONFERENCE CENTER

HARVARD MEDICAL SCHOOL, BOSTON, MA 02115

http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/13th_Annual_PM_Conference37.pdf

Leaders in Pharmaceutical Business Intelligence (LPBI) Group, Boston

pharma_bi-background0238

will cover in REAL TIME the 13th Annual Personalized Medicine, From Concept to the Clinic, November 14–16, 2017, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur Boston, MA 02115

In attendance, covering LIVE using Social Media

Aviva Lev-Ari, PhD, RN

Editor-in-Chief

http://pharmaceuticalintelligence.com

@pharma_BI

@AVIVA1950

#PMConf

Agenda · Part I  NOVEMBER 15, 2017 · CONFERENCE PROGRAM | AGENDA

7:00 am Registration and Breakfast

8:00 am Opening Remarks SPEAKER | Edward Abrahams, Ph.D., President, Personalized Medicine Coalition

  • 13th Annual PM Conference at HMS
  • Paradigm shift from One medicine FITS ALL – 13 years ago was a promise in 2017 it is a REALITY
  • Liquid biopsies, read and write gene therapy
  • Value, Pricing, Access
  • Evidence for reimbursement and FDA directions
  • Introduction od PM to the Clinic
  • Case study on Value and Healthcare System
  • Future of PM Stanford, BU, Investor
  • Translation to Chinese, 50 guests from 20 countries and 21 from CA

 

8:10 am The State of Personalized Medicine

INTRODUCTION |

Steven D. Averbuch, M.D., Head, Precision Medicine Research & Development, Bristol-Myers Squibb; Board Member, Personalized Medicine Coalition

  • Known Tom for few decades, collecting tissue on lung cancer.
  • EGFR – discovered at HMS

KEYNOTE |

Thomas J. Lynch, Jr., M.D., Executive Vice President, Chief Scientific Officer, Research & Development, Bristol-Myers Squibb

  • Apply right drug to right patient
  • Gefitinig (AstraZeneca) vs Carboplatin/Paclitaxel – lung cancer – advanced NSCLC
  • Gene mutation – EGFR negative vs Positive – cost of sequencing inverse to Moore’s Law
  • Genome Sequenced at <$1,000
  • Lung Adenocarcinoma: 2016: PIK3CA, KRAS, BRAF
  • 2015NEJM – Design drugs: Resistance mutation vs. negative – no mutation EGFR
  • Immune -Biology of Cancer is Complex – Tumor, Effector Cells, Immune regulatory & APGs
  • NEXT opportunities: Novel I-O mechanisms, patient selection is key
  • Biomarkers: PDL-1, MSI – H Tumor mutation Burden (TMB) LAG-3
  • Future: Gene signature
  • Pembrolizumab Free survivsls – 50%
  • BMS – FoundationOne – calibration used by BMS: Mutational Burden: FoundationOne assey: Exploratory analysis: High TMB (Nivoluman vs Chemo, Nivo id bettervs LowTMB
  • Combination Drug Therapy: Nivo +Ipi
  • Assessment of TMB: coding regions of 21K genes: WES vs FoundationOne (F1)
  • TMB in Blood (bTMB) in 2L + NSCLC (POPLAR and OAK)
  • bTMB <16 vs bTMB >16
  • PM – molecular profiling of Hundreds of Cancers and PM of drug treatment driven by molecular profiles
  • F1 is promising

8:40 am 13th Annual Leadership in Personalized Medicine Award

AWARDEE | Jay T. Flatley, M.S., Executive Chairman, Illumina

  • Precision Medicine – still inpact is limited
  • Regulatory and Reimbursement are BARRIERS in the HealthCare system,
  • Kidney Cancer treated off-label – great
  • less 10% of tumors have been sequenced, sufficient tissue needed, physician voted not to sequence deeming it un-actionable
  • Cystic Fibrosis – only sequencing lead to FDA approval, POST marketing required significant resources
  • Translational researchers validated 200 genes
  • 400 genes at FoundationOne
  • DIagnostics companies – reimbursement process is TOO LONG
  • Regulatory – emerging diagnostics: Product in use in clinical trials
  • Risk models to be shared with Diagnostics companies – reimbursement at end of period when results are available
  • Blockchain technology is promising for handling data
  • Rare diseases in Cancer  – One milion genomes sequenced
  • National Genomic Service – Sequencing becoming STANDARD of Care, phynotypes, $10 million to be spent NIH said Jay T. Flatley, M.S., Executive Chairman, Illumina
  • at Illimina – Consumer Advocacy added to Technology breakthroughs in genome sequencing said Jay T. Flatley, M.S., Executive Chairman, Illumina

9:10 am Networking Break

9:35 am Progress in Partnerships: A Two-Part Discussion Aligning the constructs of the health system with the principles of personalized medicine will require stakeholders to scale the most promising cross-sector partnership models. This series of conversations will examine the potential of several of the most promising models that have emerged thus far.

Discussion

Part 1 9:35 am A Model for Risk-Sharing Agreements Between Payers and the Pharmaceutical Industry Many payers are reluctant to assume that covering personalized medicines will help mitigate costs associated with major medical events that require hospitalization. During this fireside chat, however, representatives from Amgen and Harvard Pilgrim Health Care will discuss the logic and implications of their groundbreaking agreement to share the financial risks of covering a targeted medicine based on that premise. Under the terms of the agreement, Amgen agreed to cover treatment costs for patients who have a heart attack or stroke while taking its personalized therapy for familial hypercholesterolemia.

MODERATOR | Meg Tirrell, M.S.J., Reporter, CNBC

Joshua Ofman, M.D., M.S.H.S., Senior Vice President, Global Value, Access and Policy, Amgen

  • Hyperlipidemia – Partnering, Amgen with Harvard Pilgrim Health Care
  • Arrangements: Patients who need the medication will get access to the medication
  • effective stuards: Co-Pay not too high, replacement of medication by generics
  • Medicare has requirements
  • Migraine medications are coming out from Amgen – novel payment
  • Combination drug therapy – Payment system not ready for it yet
  • 3D BioPrinting of Drugs and the innovation storm of agents — are both benefits, value based pricing, elasticities, is that price sufficient to support R&D, dynamic not linear environment said Joshua Ofman, M.D., M.S.H.S., Senior Vice President, Global Value, Access and Policy, Amgen

Michael Sherman, M.D., M.B.A., M.S., Chief Medical Officer, Senior Vice President, Harvard Pilgrim Health Care; Board Member, Personalized Medicine Coalition

  • complications,  cost, outcome work vs does not work
  • Gene therapies coming from Novartis and Partnership with Harvard Pilgrims
  • Value proposition for one drug and one cancer – to assure access Pharma and Payers Partnership
  • Drug and Outcome, high cost drug coming, very expensive, life saver for who needs them

Discussion

  • Pharma’s revenue stream is international, it is in the US that payers require Value

 

Part 2 10:05 am Models for the Development of Personalized Medicine Diagnostics Pharmaceutical and diagnostics companies have responded to a host of complex scientific, regulatory and reimbursement challenges partly by developing innovative partnership models around companion diagnostics. This panel discussion will feature representatives from the pharmaceutical and diagnostics industries, who will discuss the challenges partnerships have helped industry overcome as well as the obstacles that continue to inhibit the development of the diagnostic tools upon which personalized medicine depends. Agenda ·

 

MODERATOR | Alexander Vadas, Ph.D., Managing Director, L.E.K. Consulting

Joydeep Goswami, Ph.D., M.B.A., M.S., President, Clinical Next-Generation Sequencing, Oncology, Thermo Fisher Scientific

  • monitor disease , biopsy changes in 6 month and repeat is needed
  • NGS – results come back in a month what the mutation mean? NOW results come in few days, data analysis  assist the MDs for action in treatment said Joydeep Goswami, Ph.D., M.B.A., M.S., President, Clinical Next-Generation Sequencing, Oncology, Thermo Fisher Scientific
  • How to accelerate the need for safety and the avalangue of innovations
  • Clinical sense vs research context, FDA is more comfortable with other than oncology products beyond drugs, namely diagnostics

Jacob S. Van Naarden, Chief Business Officer, Loxo Oncology

  • Increase in need of drugs in NGS World, Tissue agnostics, ALL the drugs and all the tumors
  • reduction in cost with technology, can’t be too expensive,
  • LOXO drug development  – each testing addresses a focused medical issue  – cancer alterations  for ALL extremely aggressive cancer
  • DNA and RNA based events detected by tools
  • cost of test need to be low, Reference Labs need to collaborate to standarize technology and explain to the Payers
  • Cost of CT Scan vs an NGS Test – Genomic testing is much cheaper said Jacob S. Van Naarden, Chief Business Officer, Loxo Oncology
  • Educational

10:35 am Real-World Personalized Medicine: Examining the Role of Real-World Evidence in Personalizing Health Care FDA has offered a definition of real-world evidence, but the community continues to debate what is needed to fully integrate it into decision-making. This panel will explore what real-world evidence is, how it is being used and what regulatory requirements are needed to realize its potential.

MODERATOR | Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Flatiron Health; Board Member, Personalized Medicine Coalition

  • Collection of evidence to accelerate from lab to the Clinic
  • use data sets prospective vs retrospective studies asked Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Flatiron Health; Board Member, Personalized Medicine Coalition

Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA

  • 3/2017 established to have an integrative approach by FDA – real world dat ais important to FDA
  • Drug approved for one indication, provide new data for supplemental indications said Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA
  • co-morbidities cause EXCLUSION from clinical trials, i.e., HIV patients, experience of patients excluded to learn how differently they can be treated
  • drafting a document on Verify data integrity in clinical trials, detect discrepancies compromise the integrity of the data – audits by FDA said Sean Khozin, M.D., M.P.H., Associate Director (Acting), Oncology Center of Excellence, FDA
  • Validation of devices- FDA Innovation Initiative,

Eric G. Klein, Pharm.D., Senior Director, Oncology, Global Patient Outcomes and Real-World Evidence, Eli Lilly and Company

  • Aggregate burden of disease, existence of co-morbidities
  • Why it was occurring – Genomics: WHY is explained – precise tools
  • data vs intelligence – interoperability
  • Past clinical trial – replicate studies with retrospective data
  • finding the responding patients – pragmatic trial, not randomized, collect end point – very expensive and requires statisticians
  • end pint definition changed

Eleanor M. Perfetto, Ph.D., M.S., Senior Vice President, Strategic Initiatives, National Health Council

  • How patients  wish to see usage of their experience – how side effect information can be used.
  • New indication
  • reimbursement
  • improved used of existing drug higher rating vs new indications
  • clinical trial design gets input from patients, Patient can announce, dropping participation if a change is not made

Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute

  • Major gene mutation and Drugs
  • Drug exposure correlate with evidence, Worldwide,
  • linkages vs computational techniques we do not have consistent data, data structured or not, respond to medication: symptoms, prospects vs Vital sign or WBC count – we have data standardization is evolving said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute
  • clinical decision support what is structured is data upon admission, monitoring the drugs given in this period, turning to patients willing to offer feedback and cooperate
  • pre-existing autoimmune disease – not indicated for them Immunotherapy even though patients wish to try said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute
  • Standards: Toxicity criteria – library of 882 symptoms, Patient reported outcomes by Patients, Resist  criteria applied to imaging data criteria for brain tumors said Deborah Schrag, M.D., M.P.H., Chief, Division of Population Sciences, Medical Oncology, Dana-Farber Cancer Institute
  • Physicians needs interfaces, dashboard information delivered to MDs, data sits unused, new tools are needed for the data display by relevance to the MDs
  • Patients input and sophistication increased – IRB is not aware of the engagement of Patients and their challenging feedback say Deborah Schrag, M.D., M.P.H, Dana Farber

11:50 am Luncheon

1:00 pm The Designer Genome: Exploring the Implications of Gene Editing and Gene Therapy for the Future of Medicine and Humanity Many scientists believe the clustered regularly interspaced short palindromic repeats (CRISPR) genetic engineering tool and recent developments in gene therapy will dramatically alter the trajectory of medicine, but the specific implications of these developments for health systems around the world remain unclear. During this session, a panel of experts will discuss the status of these new technologies and how the medical community and regulatory agencies may have to adapt to keep up with forthcoming developments.

MODERATOR | Kevin Davies, Ph.D., Co-Author, DNA: The Story of the Genetic Revolution (with Jim Watson and Andrew Berry); Executive Editor, The CRISPR Journal

Katrine Bosley, CEO, Editas Medicine

  • spectrum of ease to correct a mutation, some mutation are easier than others for editing,
  • understand well enough  the gentic application where CRISPR will assist medicine: Retinal degeneration, two aspects one worked in Japan said Katrine Bosley, CEO, Editas Medicine

Arthur L. Caplan, Ph.D., Drs. William F. and Virginia Connolly Mitty Chair, Director, Division of Medical Ethics, New York University Langone Medical Center

  • Bioethics, super babies, engineering embrios,
  • Regulatory oversight on engineering embrios is coming, metric of success in recruitment of patients said Arthur L. Caplan, Ph.D., Drs. William F. and Virginia Connolly Mitty Chair, Director, Division of Medical Ethics, New York University Langone Medical Center
  • cell repair is cheaper that transplantation,
  • clone of super person next door
  • Bioterrorism accomplished by gene engineering !!

George M. Church, Ph.D., Professor of Genetics, Health Sciences and Technology, Harvard-MIT Division of Health Sciences and Technology; Director, Harvard Medical School NHGRI-Center of Excellence in Genomic Science; Director, Harvard Medical School Personal Genome Project; Founding Member, Wyss Institute for Biologically Inspired Engineering at Harvard University

  • Delivery, more precise,
  • Longevity and aging – one blockbuster is needed
  • Engineered mutation, machine learning
  • CRISPR does not handle all mutation many require a different editing tool said George M. Church, Ph.D., Professor of Genetics, Health Sciences and Technology, Harvard-MIT Division of Health Sciences and Technology; Director, Harvard Medical School NHGRI-Center of Excellence in Genomic Science; Director, Harvard Medical School Personal Genome Project; Founding Member, Wyss Institute for Biologically Inspired Engineering at Harvard University
  • over regulation – Do not touch germ line – is not desired
  • Transplantation vs enhancement – resistance to senescence and pathogens to be achieved by gene editing suggests George M. Church, Ph.D., Professor of Genetics
  • Bringing back genes – elephants with fur

Jeffrey D. Marrazzo, M.B.A., M.P.A., CEO, Spark Therapeutics

  • Retina degeneration causes blindness, deliver drug to back of the retine, inject genetic material and achieved remarkable results, drug approval of genetic therapy in the US for a genetic disorder in Retina causing blindness
  • 21st Century schema of Payment and benefits

 

2:15 pm Pricing Personalized Medicines The increasing pressure on industry stakeholders to alter their drug pricing practices has particular significance for personalized medicines, which must recoup research and development costs from smaller patient populations. This conversation will explore the pharmaceutical industry’s strategies for facilitating sustainable access to these innovative therapies.

MODERATOR | Meg Tirrell, M.S.J., Reporter, CNBC

Stephen J. Ubl, President, CEO, PhRMA

  • minimum 10% invested in R&D at each Pharma
  • Of 134 drugs in development – 42 have the potential to become Personalized medicine therapies, said Stephen J. Ubl, President, CEO, PhRMA
  • Icer methodology – average patient aggregate data, value pricing is a better model: RA targeted to subset of patients
  • Price gauging is a problem – bring solutions to the table, Patients asks for incentives
  • amortizing costs like mortgage
  • Outcome-based arrangements: If money-based guaranteed it negate Medicaid negotiation power. If transportation is covered – it leads to locking product in use

2:45 pm Networking Break (sponsored by GreyBird Ventures)

3:15 pm Precision Valuation: A Discussion of How Value Assessment Frameworks Can Account for Personalized Medicine Payers control access to personalized medicine, and some have begun to take an interest in findings from value assessment frameworks that are challenged to account for developments in the field. In addition to exploring their potential impact on individualized care, this session will examine how value assessment frameworks can and should consider personalized medicine as part of their processes for evaluating therapeutic options.

MODERATOR | Jennifer Snow, M.P.H., Director, Health Policy, Xcenda, AmerisourceBergen

  • Quality Era moved to Value Era

 

Dane J. Dickson, M.D., CEO, Founder, CureOne (formerly MED-C); Director, Precision Medicine Policy and Registries, Knight Cancer Institute at Oregon Health and Science University

Molecular Era

  • NEJM, 2017, 377, 1813-1823
  • BRAF in Melanoma – 80% do not need additional therapy vs 20% benefitted in the Non-Molecular Era

data by Dane J. Dickson, M.D., CEO, Founder, CureOne (formerly MED-C); Director, Precision Medicine Policy and Registries, Knight Cancer Institute at Oregon Health and Science University

 

Robert Dubois, M.D., Ph.D., Executive Vice President, Chief Science Officer, National Pharmaceutical Council

  • Value Assessment and PM: ACC, ASCO, ICER< Memorial Sloan,
  • The patient: survival, QOL, Adverse events, Out of pocket cost, extra survival by disease, treatment burden,
  • PAYERS: One size does not fit all – AVERAGE is meaningless
  • MDs vs Patients – are different in preference

Andrea Stern Ferris, M.B.A., President, Chairman of the Board, LUNGevity Foundation

  • PATIENT to be included in the conversation

Steven D. Pearson, M.D., M.Sc., Founder, President, Institute for Clinical and Economic Review (ICER)

  • Precision Medicine vs Value Assessment
  • Novartis CAR-T Kymriah:  relapsed B-cell precursor ALL under 25 – 5 yr survival – 10%
  • Changes associated with Gene therapies: single arm trials, surrogate outcomes, less certainty safety and benefits
  • Gene therapy – >> innovations Kymriah – $475,000 price
  • Long term value for money vs Short term affordability

PRICE-based on Value – discount from prices after rebate to meet ICER value-based Price range

  • More targeted = higher value more favorable cost-effectiveness
  • Rare/ultra-rare populations: broader value range:
  1. Use EARLIER
  2. will it work for patients without genetic marker?
  • Years of Life weighted by an INDEX of quality of life (1=perfect health;  0=dead)
  • willingness to pay: WHO and ACC: 1-3x
  • individual x2 salary
  • Opportunity cost x1 per capita GDP in UK
  • Future of value assessment and precision medicine
  • CURES – CAR-T are they cures???
  • A teen-ager’s Value-based Price: $475,000 x years lived suggestsSteven D. Pearson, M.D., M.Sc., Founder, President, Institute for Clinical and Economic Review (ICER)

4:30 pm The Utility Proposition: An Analysis of Case Studies in the Economic Value of Personalized Medicine Although personalized medicine’s proponents contend that the field can deliver economic value by helping doctors avoid prescribing costly but ineffective therapies, the field lacks literature testing that hypothesis. This session will highlight recent studies on the clinical and economic value of personalized medicine, shedding light on what we know about personalized medicine’s clinical and economic utility — and what we don’t.

MODERATOR | Michael Pellini, M.D., M.B.A., Chairman, Board of Directors, Foundation Medicine; Board Member, Personalized Medicine Coalition

  • we know there is value in Personalized Medicine
  • we need to work together to acknowledge the challenges — to prove the value in PM

Lincoln Nadauld, M.D., Ph.D., Executive Director, Precision Medicine, Precision Genomics, Intermountain Healthcare

  • Interpretation by Medical Oncologists beyond: KRAS, BRAF
  • Measuring the value and presenting that to the payers and inside the organizations
  • 2013 –

David B. Roth, M.D., Ph.D., Simon Flexner Professor Chair, Pathology and Laboratory Medicine, Perelman School of Medicine at University of Pennsylvania; Director, Penn Center for Precision Medicine

  • 5000 patients underwent genome sequencing
  • Interpretation is the issue that is hard
  • Health IT are still in silos: Pharmacy data, financial data, EMR are not integrated yet
  • Survival of patient with mutation and targeted drug LIVE LONGER

Lotte Steuten, Ph.D., M.Sc., Associate Faculty Member, Hutchinson Institute for Cancer Outcomes Research (HICOR), Fred Hutchinson Cancer Research Center; Affiliate Associate Professor, Pharmaceutical Outcomes Research and Policy Program, School of Pharmacy at University of Washington, Seattle

  • aggregate big data , models as evidence, has value to clinical, the model under development NGS Profile of Patient vs current standard of care. Model Payer advisor committee, Oncologist Advisory Committee great benefit to PM on the cost side data is for targeting treatment using the promise of PM

5:45 pm Elements Café Cocktail Reception

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Novartis’ Kymriah (tisagenlecleucel), FDA approved genetically engineered immune cells, would charge $475,000 per patient, will use Programs that Payers will pay only for Responding Patients

Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED on 9/1/2017:

This Pioneering $475,000 Cancer Drug Comes With A Money-Back Guarantee

Novartis defends the eye-popping price of its pioneering gene therapy with arguments about its $1 billion expenditure—and novel “value-based” pricing.

https://www.fastcompany.com/40461214/how-novartis-is-defending-the-record-475000-price-of-its-pioneering-gene-therapy-cancer-drug-car-t-kymriah

 

On 8/30/2017 we wrote:

FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of KitePharma, no approved drug and Canakinumab for Lung Cancer (may be?)

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2017/08/30/fda-has-approved-the-worlds-first-car-t-therapy-novartis-for-kymriah-tisagenlecleucel-and-gileads-12-billion-buy-of-kite-pharma-no-approved-drug-and-canakinumab-for-lung-cancer-may-be/

 

The Price for the Treatment was published on 8/31/2017, a Value-based Pricing Payment Model of a $475,000 per patient charge for the responding patients after ONE month of treatment. Novartis says it takes an average of 22 days to create the therapy, from the time a patient’s cells are removed to when they are infused back into the patient. Kymriah will initially be available at 20 U.S. hospitals within a month, Novartis says. Eventually, 32 total sites will offer the therapy. 

CAR-T gained national attention three years ago when Carl June, a researcher at the University of Pennsylvania, used to put a young girl’s acute lymphoblastic leukemia. Genetically altering the girl’s immune cells had made her deathly ill, but June had used a Roche drug, Actemra, to treat the side effects. She lived, and the results were published in The New England Journal of Medicine. Novartis bought the rights to the Penn treatment for just $20 million up front.

Pharma Buying the right to use from an Academic Institution is a known route to leap frog the R&D lengthy process of Drug discovery.

“I’ve told the team that resources are not an issue. Speed is the issue,” says Novartis’ Chief Executive Joseph Jimenez, told Forbes in a cover story about the work then.

The FDA calls this CAR-T therapy treatment, made by Novartis, the “first gene therapy” in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. The FDA defines gene therapy as a medicine that “introduces genetic material into a person’s DNA to replace faulty or missing genetic material” to treat a disease or medical condition. This is the first such therapy to be available in the U.S., according to the FDA.

Two gene therapies for rare, inherited diseases have already been approved in Europe.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA’s Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

FDA commissioner Scott Gottlieb in a statement.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” 

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm

The Protocol

A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.

https://www.technologyreview.com/s/608771/the-fda-has-approved-the-first-gene-therapy-for-cancer/?utm_campaign=add_this&utm_source=email&utm_medium=post

CAR-T Therapies: Product/Molecules/MOA under Development:

  • Similar CAR-T treatments were being developed at other institutions including
  • Memorial Sloan-Kettering Cancer Center,
  • Seattle Children’s Hospital, and
  • The National Cancer Institute.
  • The Memorial and Seattle work was spun off into a startup called Juno Therapeutics, which has fallen behind. Juno Therapeutics ended a CAR-T study earlier this year after patients died from cerebral edema, or swelling in the brain.
  • The NCI work became the basis for the product being developed by Kite Pharma. Kite Pharma, which is awaiting FDA approval for its CAR-T therapy to treat a form of blood cancer in adults, was this week bought out by Gilead in a deal worth $11.9 billion.

On Cambridge Healthtech Institute’s 4th Annual Adoptive T Cell Therapy, Delivering CAR, TCR, and TIL from Research to Reality, August 29 – 30, 2017 | Sheraton Boston | Boston, MA

TUESDAY, AUGUST 29 – I covered in Real Time the talk on Juno Therapeutics: Building Better T Cell Therapies: The Power of Molecular Profiling by Mark Bonyhadi, Ph.D., Head, Research and Academic Affairs, Juno Therapeutics

https://pharmaceuticalintelligence.com/2017/08/29/live-829-chis-oncolytic-virus-immunotherapy-and-adoptive-cell-therapy-august-28-29-2017-sheraton-boston-hotel-boston-ma/

 

Precision Medicine is Costly and not a Rapid manufacturing process

All of the CAR-T products are expensive to make, and must be manufactured on an individual basis for each new patient from the patient’s own T-cells, a type of white blood cells, a process that takes weeks.

  • How quickly companies can speed up manufacturing.
  • Kymriah will be manufactured at a facility in Morris Plains, N.J.
  • CAR-T technology, which has so far been used only in patients with blood cancers that have not been cured by other treatments, can be used earlier in the disease or for solid tumors: Breast, Prostate, Melanomas.

https://www.forbes.com/sites/matthewherper/2017/08/30/fda-approves-novartis-treatment-that-alters-patients-cells-to-fight-cancer/#2aecb25b4400

Prediction How Patients will Far Well – Researchers use a big-data approach to find links between different genes and patient survival.

https://www.technologyreview.com/s/608666/a-cancer-atlas-to-predict-how-patients-will-fare/?set=

A pathology atlas of the human cancer transcriptome

+ See all authors and affiliations

Science  18 Aug 2017:
Vol. 357, Issue 6352, eaan2507
DOI: 10.1126/science.aan2507

Modeling the cancer transcriptome

Recent initiatives such as The Cancer Genome Atlas have mapped the genome-wide effect of individual genes on tumor growth. By unraveling genomic alterations in tumors, molecular subtypes of cancers have been identified, which is improving patient diagnostics and treatment. Uhlen et al. developed a computer-based modeling approach to examine different cancer types in nearly 8000 patients. They provide an open-access resource for exploring how the expression of specific genes influences patient survival in 17 different types of cancer. More than 900,000 patient survival profiles are available, including for tumors of colon, prostate, lung, and breast origin. This interactive data set can also be used to generate personalized patient models to predict how metabolic changes can influence tumor growth.

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