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


 

Dear Colleague:


Recognizing that almost 40 percent of those who responded to the strategic planning survey underpinning the plan did not agree that “it is inevitable that all doctors will someday practice personalized medicine [because] the science and technology will demand it,” the Coalition has outlined robust efforts to encourage and nurture medical progress through education, advocacy, and evidence development.
On behalf of the Personalized Medicine Coalition (PMC), I am pleased to share PMC’s Strategic Plan for 2019.

Unless payers, providers, patients, and policymakers are aware of personalized medicine, it will be harder to increase research funding and propose smart public policies that encourage integrating diagnostics into therapeutic decisions.

And unless we develop the evidence base necessary to demonstrate the clinical and economic value of personalized medicine, those decision-makers are not going to embrace it, and, in fact, may propose policies, like “step therapy” — paying for what costs least first — that fly in the face of the principles of personalized medicine.

In short, in keeping with the scientific and technological progress of recent years, we need to redouble our efforts to deliver the promise of personalized medicine — better clinical outcomes and more efficient health systems. The stakes are very high.

We welcome your participation in PMC this year. Without it, progress will be much slower than any of us would like.

Please contact PMC Membership & Development Director Kayla Smith at ksmith@personalizedmedicinecoalition.org with questions about how to get involved with any of the projects listed in the plan.

Sincerely yours,

Edward Abrahams

President

SOURCE

rom: Personalized Medicine Coalition <messages@app.production.membersuite.com>

Reply-To: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>

Date: Wednesday, January 9, 2019 at 9:36 AM

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

Subject: PMC Strategic Plan for 2019

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Role of Informatics in Precision Medicine: Notes from Boston Healthcare Webinar: Can It Drive the Next Cost Efficiencies in Oncology Care?

Reporter: Stephen J. Williams, Ph.D.

 

Boston Healthcare sponsored a Webinar recently entitled ” Role of Informatics in Precision Medicine: Implications for Innovators”.  The webinar focused on the different informatic needs along the Oncology Care value chain from drug discovery through clinicians, C-suite executives and payers. The presentation, by Joseph Ferrara and Mark Girardi, discussed the specific informatics needs and deficiencies experienced by all players in oncology care and how innovators in this space could create value. The final part of the webinar discussed artificial intelligence and the role in cancer informatics.

 

Below is the mp4 video and audio for this webinar.  Notes on each of the slides with a few representative slides are also given below:

Please click below for the mp4 of the webinar:

 

 


  • worldwide oncology related care to increase by 40% in 2020
  • big movement to participatory care: moving decision making to the patient. Need for information
  • cost components focused on clinical action
  • use informatics before clinical stage might add value to cost chain

 

 

 

 

Key unmet needs from perspectives of different players in oncology care where informatics may help in decision making

 

 

 

  1.   Needs of Clinicians

– informatic needs for clinical enrollment

– informatic needs for obtaining drug access/newer therapies

2.  Needs of C-suite/health system executives

– informatic needs to help focus of quality of care

– informatic needs to determine health outcomes/metrics

3.  Needs of Payers

– informatic needs to determine quality metrics and managing costs

– informatics needs to form guidelines

– informatics needs to determine if biomarkers are used consistently and properly

– population level data analytics

 

 

 

 

 

 

 

 

 

 

 

 

What are the kind of value innovations that tech entrepreneurs need to create in this space? Two areas/problems need to be solved.

  • innovations in data depth and breadth
  • need to aggregate information to inform intervention

Different players in value chains have different data needs

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Data Depth: Cumulative Understanding of disease

Data Depth: Cumulative number of oncology transactions

  • technology innovators rely on LEGACY businesses (those that already have technology) and these LEGACY businesses either have data breath or data depth BUT NOT BOTH; (IS THIS WHERE THE GREATEST VALUE CAN BE INNOVATED?)
  • NEED to provide ACTIONABLE as well as PHENOTYPIC/GENOTYPIC DATA
  • data depth more important in clinical setting as it drives solutions and cost effective interventions.  For example Foundation Medicine, who supplies genotypic/phenotypic data for patient samples supplies high data depth
  • technologies are moving to data support
  • evidence will need to be tied to umbrella value propositions
  • Informatic solutions will have to prove outcome benefit

 

 

 

 

 

How will Machine Learning be involved in the healthcare value chain?

  • increased emphasis on real time datasets – CONSTANT UPDATES NEED TO OCCUR. THIS IS NOT HAPPENING BUT VALUED BY MANY PLAYERS IN THIS SPACE
  • Interoperability of DATABASES Important!  Many Players in this space don’t understand the complexities integrating these datasets

Other Articles on this topic of healthcare informatics, value based oncology, and healthcare IT on this OPEN ACCESS JOURNAL include:

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

Broad Institute launches Merkin Institute for Transformative Technologies in Healthcare

HealthCare focused AI Startups from the 100 Companies Leading the Way in A.I. Globally

Paradoxical Findings in HealthCare Delivery and Outcomes: Economics in MEDICINE – Original Research by Anupam “Bapu” Jena, the Ruth L. Newhouse Associate Professor of Health Care Policy at HMS

Google & Digital Healthcare Technology

Can Blockchain Technology and Artificial Intelligence Cure What Ails Biomedical Research and Healthcare

The Future of Precision Cancer Medicine, Inaugural Symposium, MIT Center for Precision Cancer Medicine, December 13, 2018, 8AM-6PM, 50 Memorial Drive, Cambridge, MA

Live Conference Coverage @Medcity Converge 2018 Philadelphia: Oncology Value Based Care and Patient Management

2016 BioIT World: Track 5 – April 5 – 7, 2016 Bioinformatics Computational Resources and Tools to Turn Big Data into Smart Data

The Need for an Informatics Solution in Translational Medicine

 

 

 

 

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Gene-editing Second International Summit in Hong Kong: George Church, “Let’s be quantitative before we start being accusatory”

 

Reporter: Aviva Lev-Ari, PhD, RN

UPDATED on 11/30/2018

Gene editing takes a foreboding leap forward

He Jiankui. Photo: Zhang Wei/Chinese News Service/VCG via Getty Images

 

China is temporarily suspending the work of scientists who claimed twins were born after being genetically edited as embryos.

Why it matters: The scientific consensus is that gene editing embryos at this stage of science is “irresponsible.” But, while this particular experiment has not been verified, the fact is the technology is available to researchers, so there’s a growing call for international limitations on its use.

ICYMI: Chinese scientist He Jiankui announced earlier this week that twins were born after he used the gene-editing tool CRISPR-Cas9 to cut the CCR5 gene that’s known to play a role in HIV infection.

  • He stirred even more dismay when he mentioned the possibility of a second pregnancy.
  • China currently bans human implantation of gene-edited embryos. Its Ministry of Science and Technology is investigating the claims, per Xinhua.

There are concerns about the safety, efficacy and possible mosaicism, where a person can contain genes in both its edited and unedited forms, from cutting genes.

  • Editing embryos raises an even bigger concern: The genetic changes and all the unknowns around them can be passed down to future generations.

Between the lines: Not everyone viewed it as a complete disaster. For instance, Harvard Medical School’s George Daley suggested that it may be time to reconsider the massive amounts of research done over the past several years and look for plausible methods of moving forward.

What to watch: Scientists are cautious about predicting what the impact will be, in part because the details of this claim are thin. However, the debate is heating up and one concern is it will dampen important research.

  • Medical ethicist Jonathan Moreno from the University of Pennsylvania says the situation reminds him of other times in history where there were tremors in the science world, like the death of 18-year-old Jesse Gelsinger in 1999 from a gene therapy trial that led to years of diminished research.

The bottom line: The alarm over what could be next is real. But scientists hope the current debate will promote consensus on firm limits and promote transparency.

Go deeper:

SOURCE

From: Andrew Freedman <andrew.freedman@axios.com>

Date: Thursday, November 29, 2018 at 5:33 PM

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

Subject: Axios Science: About that climate report — Gene editing takes a foreboding step — Building in harms’ way

 

 

He Jiankui spoke at the second international summit on human genome editing in Hong Kong. (Alex Hofford/EPA-EFE/Shutterstock)

CRISPR-baby scientist faces the music

The scientist who claims to have helped produce the first people born with edited genomes faced a tough crowd yesterday at a gene-editing summit in Hong Kong. He Jiankui gave a 20-minute talk about his unpublished work in animals and humans before opening a 40-minute Q&A session (watch it here). He faced difficult questions about the ethics of his work and his choice to keep it mostly under wraps until after the babies were born, and left many unanswered.

Meanwhile, prominent geneticist George Church is one of the few scientists who seem to be looking on the bright side of He’s controversial claim. “Let’s be quantitative before we start being accusatory,” Church told Science. “As long as these are normal, healthy kids it’s going to be fine for the field and the family.”

Nature | 9 min read & Science | 6 min read

Read more: Genome-edited baby claim provokes international outcry

 

SOURCE

From: Nature Briefing <briefing@nature.com>

Reply-To: Nature Briefing <briefing@nature.com>

Date: Thursday, November 29, 2018 at 12:18 PM

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

Subject: CRISPR-baby scientist faces the music at gene-editing summit

 

See

SAVE

The ethical red flags of genetically edited babies

Driving the news: Chinese scientist He Jiankui announced Sunday night that a pair of twin girls had been born from embryos he modified using the gene-editing tool known as CRISPR.

  • He hasn’t provided solid proof, but if it‘s true, it would be the first time the technology has been used to engineer a human.

What they’re saying: The inventors of CRISPR technology did not seem pleased with the development — one called for a moratorium on implantation edited embryos into potential mothers.

  • “I hope we will be more cautious in the next thing we try to do, and think more carefully about when you should use technology versus when you could use technology,” said Jessica Berg, a bioethicist at Case Western Reserve University.

Between the lines: Several specific factors in He’s work sent up ethical red flags.

  • Many scientists had assumed that, when this technology was first used in humans, it would edit out mutations tied to a single gene that were certain to cause a child pain and suffering once it was born — essentially, as a last resort.
  • But He used CRISPR to, as he put it, “close a door” that HIV could have one day traveled through. That has prompted some speculation that this project was more about testing the technology than serving an acute medical need.
  • “That should make us very uneasy about the whole situation,” Berg said. “Of all the things to have started with, it does make you a little suspicious about this particular choice.”

The intrigue: There’s a lot we still don’t know about He’s work, and that’s also contributing to an attitude of skepticism.

  • How many embryos did he edit and implant before these live births?
  • How will he know it worked? As the children age, they’ll likely have their blood drawn and those samples will be exposed to HIV in a lab, but researchers aren’t going to tell them to go out and have unprotected sex or use intravenous drugs — another reason HIV seems like an odd starting place for human gene editing.
  • How did this even happen? The university where He worked said he was on leave, and Chinese officials have said he’s under investigation. But gene editing is a pretty hard thing to freelance.

The other side: He defended his work in a video message, saying, “I understand my work will be controversial but I believe families need this technology and I’m willing to take the criticism for them.”

  • “Their parents don’t want a designer baby, just a child who won’t suffer from a disease which medicine can now prevent,” He said.

Yes, but: Now that this threshold may have been crossed, attempts to create “designer babies” — within the limitations of what CRISPR can do — probably aren’t far off, some experts fear.

  • There are “likely to be places that are less regulated than others, where people are going to attempt to see what they can do,” Berg said. “I wouldn’t say everything in the world has changed now, but it’s certainly the next step.”
SOURCE

https://www.axios.com/genetic-editing-baby-china-ethics-controversy-b33f8414-8b83-445c-bad5-d8407f8841f4.html

https://pharmaceuticalintelligence.com/2018/11/26/jennifer-doudna-and-npr-science-correspondent-joe-palca-several-interviews/

Read Full Post »


The Future of Precision Cancer Medicine, Inaugural Symposium, MIT Center for Precision Cancer Medicine, December 13, 2018, 8AM-6PM, 50 Memorial Drive, Cambridge, MA

Reporter: Aviva Lev-Ari, PhD, RN

#CPCM2018 @AVIVA1950 @pharma_BI

 

 

Aviva Lev-Ari, PhD, RN, Editor-in-Chief, will attend and cover this event in REAL TIME for

 http://pharmaceuticalintelligence.com 


Over the past decade, there have been major advancements in the field of precision medicine, leading to exciting new treatments for some cancer patients. Much attention has been focused on genomic profiling of tumors to identify genomic alterations that might guide selection of specific therapies for individual patients. Beyond genomics, however, there is a variety of other precision approaches that can identify and exploit cancer-specific biological mechanisms including proteomics, metabolomics, and computational modeling, resulting in the more effective use of existing cancer medicines. On Thursday, December 13, 2018, the MIT Center for Precision Cancer Medicine will hold its inaugural annual symposium in the Samberg Conference Center at MIT. This full-day event will feature leading researchers and clinicians, who will highlight recent advances in precision cancer medicine and share perspectives on the future. An industry panel will also discuss the barriers to instituting precision medicine into current and future clinical trials.

 


Keynote Address

Charles Sawyers

Charles Sawyers, MD

Chair, Human Oncology and Pathogenesis Program
Memorial Sloan Kettering Cancer Center

Speakers

Andrea Califano

Andrea Califano, PhD

Clyde and Helen Wu Professor of Chemical Systems Biology, Columbia University
Chair, Department of Systems Biology, Columbia University
Director, JP Sulzberger Columbia Genome Center
Associate Director, Herbert Irving Comprehensive Cancer Center

Chris Love

J. Christopher Love, PhD

Professor of Chemical Engineering, MIT
Associate Member, Ragon Institute of MGH, MIT and Harvard
Member, Koch Institute, MIT

Richard Marais

Richard Marais, PhD

Professor of Molecular Oncology
Director, CRUK Manchester Institute
The University of Manchester

Kenna Mills Shaw

Kenna Mills Shaw, PhD

Executive Director
Sheikh Khalifa Bin Zayed al Nahyan Institute for Personalized Cancer Therapy
MD Anderson Cancer Center

Alice Shaw

Alice Shaw, MD, PhD

Professor, Harvard Medical School
Director, Thoracic Cancer Program, Massachusetts General Hospital

Matt Vander Heiden

Matthew Vander Heiden, MD, PhD

Associate Professor of Biology, MIT
Associate Director, Koch Institute
Member, MIT Center for Precision Cancer Medicine

Mike Yaffe

Michael B. Yaffe, MD, PhD

David H. Koch Professor of Science, MIT
Professor of Biology and Biological Engineering, MIT
Director, MIT Center for Precision Cancer Medicine
Director, Koch Institute Clinical Investigator Program

Jean Zhao

Jean Zhao, PhD

Professor of Biological Chemistry and Molecular Pharmacology
Harvard Medical School and Dana-Farber Cancer Institute


Panelists: Barriers to Instituting Precision Medicine in Clinical Trials

Hammerman

Peter Hammerman, MD, PhD

Global Head, Translational Research
Oncology Disease Area
Novartis Institutes for BioMedical Research

Ho

Steffan N. Ho, MD, PhD

Vice President, Head of Translational Oncology
Pfizer Global Product Development

Shiva Malek

Shiva Malek, PhD

Director and Principal Scientist
Department of Discovery Oncology
Genentech Inc

Marks

Kevin Marks, PhD

VP of Biology
Agios Pharmaceuticals

Michael Rothenberg

S. Michael Rothenberg, MD, PhD

Vice-President, Research and Development
Loxo Oncology, Inc.

Angela Koehler

Moderator:

Angela Koehler, PhD

Goldblith Career Development Professor in Applied Biology, MIT
Member, Koch Institute for Integrative Cancer Research
Member, MIT Center for Precision Cancer Medicine

 

Speakers:

Panelists:

  • Peter Hammerman, Novartis Institutes for BioMedical Research
  • Steffan Ho, Pfizer
  • Shiva Malek, Genentech, Inc
  • Kevin Marks, Agios Pharmaceuticals
  • S. Michael Rothenberg, Loxo Oncology, Inc

Moderated by Angela Koehler, MIT’s Koch Institute

Agenda:

8:00 am Registration and continental breakfast

8:45 am Opening remarks by Michael Yaffe (MIT’s Koch Institute)

  • Season of great expectation, tumor genetics is just the beginning, beyond: science, engineering, medicine: beyond genomics: immunology, cell biology, early detection, new drug development for the undrugable, system biology, RNAi
  • Jack Tyler was the initiator to find a donor for CPCM

9:00 am Keynote Address by Charles L. Sawyers (Memorial Sloan Kettering Cancer Center)

  • developed a drug for prostate cancer
  • Clinical trained oncologist/genomics
  • Lineage Plasticity:
  1. luminal cells in histology of origin and basal cells and require androgen receptor AR) function
  2. deprive lunimal cells fro growth factor
  3. Hormonal therapy Leuprolite, degarelix [castration methastatic]
  4. after relapse 2nd generation anti-androgens abirateron
  5. PING MU ENZALUTAMIDE RESISTANCE P53/RB! DELETION CONFER
  6. TRANSCRIPTION CHANGE: ANTIADROGEN RESISTANCE
  7. Lineage shift Sox2 level goes up – prevent drug resistance, in vivo and in vitro
  8. SOX2 promotes lineage placticity and antiadrogen resistance in TP53 and RBI-deficient prostate cancer
  9. Evolution of Lineage plasticity over time
  10. AR Pathway inhibition accelerates lineage plasticity: synaptophysin-positive disease in-vivo
  11. scRNA-seq time course – modeled by diffusion map displayed in luminal and basal cells
  12. Emergence of EMT phenotype, with retention of epithelial features
  13. Use CRISPR to perturb luminal plasticity by phyeno type
  14. Genomic landscape of Primary Prostate Cancer: ERG gain drives luminal layer
  15. Different classes of FOXA1 mutations in Prostate organoid Cancer – Missense, inframe, truncated
  16. FOXA1 key in hormone receptor signaling
  17. Hypermorphic peaks – ATAC-seq neomorphic FOXA1 pioneering activity
  18. Common Prostate Cancer Genes:differentiation phenotypes: TP53 Loss, RB1 – Loss,
  19. work of Matan Hofree – four subtypes of luminal cells
  20. involution and regeneration of single cell RNAseq
  21. Transcriptional shifts in response to castration/androgen addback
  22. androgen addback: 50% of luminal cells are proliferation in 48 hours
  23. cell responsible for organ regeneration

 

9:45 am Alice T. Shaw (Massachusetts General Hospital)

  • evolution of drug resistance in Lung Cancer
  • oncogenic drivers in lung adenocarcenoma –
  1. EGFR – sensitizing 19.4% of all patients
  2. KRAS
  3. ALK
  4. ROS1
  5. CMET
  6. BRAF
  7. NTRK1
  8. RET

Delay and prevention of drug resistance: liquid biopsy of pleural fluids and serial blood collections

  • Crizotinib patient with ROS1 + nsclc
  • acquired mutation in ROS1 G2032R – resistance to Crizotinib – Michael Lawrence, MGH – analysis of mutation and resistance
  • Repotrectinib – for ROS1 – Resistance mediated by this mutation
  • If patient fails three antiinhibitor drugs: secondary ALK mutations mediate Crizotinib Resistance
  • 2nd generation of  ALK inhibitors are structurally Distinct molecules
  • Lorlatinib – 3rd generation –>> back to 1st generation Crizotinib
  • Clonal evolution of resistance in ALK in NSCLC
  • compound mutations in ALK mutations – Lorlatinib Resistance
  • Sequential TKI therapy foster the development of compound mutation refractory to all generations og ALK TKIs – compound mutation can’t be overcome
  • Intratumoral Heterogeneity revealed by multiregion sequencing of renal cell carcinoma and resected NSCLC
  • somatic mutations: Pre-treatment to Lorlatinib resistance
  • Clonal Analysis: Multiple Drivers of resistance underlie clinical relapse
  • genomic instability – eradicate residual disease to eliminate drug resistance and tolerance persistance

 

10:25 am Networking Break

10:45 am Richard Marais (Cancer Research UK, Manchester Institute)

  • Melanoma – Precision Medicin
  • Request – NOT TO PUBLISH on the INTERNET, some of the work presented is not PUBLISHED.
  • Request is honored

11:25 am Matthew Vander Heiden (MIT’s Koch Institute)

  • Targeting Metabolism is altered in cancer
  • Metabolism is glucose carbohydrates, lipids – conversion of nutrients into biomass: ATP, Protein, Nucleic acid,
  • Not -proliferating cells vs proliferating cells
  • genetic mutations, tissue of origin, lineage of cells — metabolism takes place: combination of these three facto
  • environment consists the metabolic network definers.d by cell intrinsic network
  • Assessment of nutrient levels in tumor microenvironment
  • Metabolite analysis: ion suppression vs nutrients
  • nutrients are available to cells in tumors
  • depletion of glucose vs enrichment
  • metabolite most different: Gluthamine, needed for cancer to grow
  • Lineage can contribute – tryptophane and argenine
  • gluthamine – Cyctine affect gluthamine sensitivity to gluthamine inhibitors
  • what you eat, where is the tumor locate, tissue environment — more important
  • therapeutic window: metabolism processes – cell proliferation
  • ability to make aspartate – given to mice pancreatic  — tumor grow faster
  • cellular oxidation state correlate with pyruvate oxidation — PDH Activator suppress oxidation
  • Aspartate vs NAD+/NADH – lactate TCA – form more carbon
  • PDH activation reduces Redux
  • Serine availability can limit proliferation even in cells with increase
  • Serine vs NAD regeneration
  • which cancer falls into which group : Serine pathway – increase serine synthesis: Melanoma vs Breast cancer
  • growth of breast cancer: Serine availability dependent – accelerate of inhibit growth by level of serine
  • Model for how nutrient limitation affect tumor growth, tumor size depends of serine levels

 

12:05 pm Box lunch

12:30 pm Industry panel: Barriers to instituting precision medicine into clinical trials

  • Long term benefits of Precision Medicine
  • What phynotype are now looked for?

Michael Rothenberg

  1. short term, identify mutations
  2. more testing is needed
  3. sequencing the therapies
  4. challenge getting tissue, doing experiments in house
  5. Industry needs Academia collaboration for accelerated innovations
  6. AI may lower the cost of drug discovery

KEVIN MARX:

  1. MECHANISM OF RESISTANCE – COMBINATORIAL DRUG DISCOVERY
  2. phynotyping, tissue acquisition immune phenotype, what drive therapeutic response?
  3. genetic drivers
  4. HR seeks Scientistist that worked in TEAMS, collaborative science

STEPHAN HO

  1. long term benefits are very important
  2. Stage III disease – technology advances
  3. advanced in the regulatory space
  4. smaller cohort size to approve a drug
  5. biologic complexity, driver oncogenes, precision to imprecision
  6. cost of risk in investment in innovations
  7. check point inhibitor – known biology and immuno-modulation, data hypothesis and moving forward
  8. Organizational culture, interaction in teams, functional behavior
  9. commit to deliverable, perfect timing contingent on work of others.

Peter Hammerman

  1. single cell tumor immunity in combination drug therapy
  2. Tumor monitoring over time
  3. Novartis is interested to collaborate with innovators in Academia and in other institutions
  4. critical thinking on DATA and on negative data
  5. Combination drug therapy: orthogonal mechanism of actions and drug classed – toxicity is an issue

Shiva Malek

  1. How to drug mutations on DATA
  2. Acquired and intrinsic mutations
  3. exposure and patient safety
  4. UCSF’s Ashkenazi’s Team and Genetech – basic biology area selection
  5. Failure are not talked about
  6. Round table for problem solvers, how you approach a problem
  7. translational work require skills beyond technical expertise
  8. learning the navigation inside an organization
  9. leadership in R&D, expected to demonstrate leadership, the Scientist needs to have command of the field and of desirable directions of research

 

2:00 pm J. Christopher Love (MIT’s Koch Institute)

Acceleration of the PROCESS to develop Precision Medicine products

  • design, build, test – PROCESS
  • New drugs and vaccines – the process is iterative
  • measurements, with use of smallest number of samples
  • deliver precision medical: small f patients or large population or
  • clinical samples provide rich source of information: Blood or tissue sample
  • Tissue – extract RNA, component cells, single-cell RNA sequencing,
  • Challenges of enabling scRNA-seq in clinical labs
  • Probability, scale, capture efficiencies, temporal uniformity
  • single-cell sequencing
  • Seq-Well: method for scRNA-Seq
  • New Chemistries for T-cell
  • Blood: cell, cfDNA, Exosomes
  • map cancer genome from blood
  • Tissue:
  • Single circulating Tumor cells:
  • yield genomic landscape of cancer
  • cell free DNA, vells, proteins, metabolite, Tumor is existence, draw blood
  • cfDNA Tumor Fraction is prognostic of survival in mTNBC
  • automate to 13 cancer types
  • Rs is now possible
  • reduce sample requirement
  • cost is low digital information from clinical samples
  • Keytruda – is a molecular Signature
  • low volume product, advanced preparation (mo-years) __>>> agile solutions (days to years)
  • bentchtop, on-demand manufacturing system: Production, Purification, Formulation
  • hand-free production of formulated G-CSF: comparable to licensed products.
  • Plug and play manufacturing using  InSeq
  • Novel MAbs from patients
  • Many molecules to many products

 

2:40 pm Andrea Califano (Columbia University, System Biology)

Mechanistic Framework for the systematic pharmacological targeting of Non-Oncogene Dependencies – Precise Precision Oncology

  • systematic elucidation od critical cancer cell dependencies
  • drug MOA
  • Tumor dependencies to Drug MOA
  • Tumor heterogeneity
  • ARACNe – regulatory targets of regulatory proteins
  • Combinational Therapy: HER@ inhibitor and JAK1/JAK2 inhibitor
  • Driver Mutations
  • ARACNe; MINDy DIGGIT; Expression VIPER: MetaVIPER
  • Aberrantly activated protein for Prioritizing treatment in patients
  • Checkpoint activity reversal – prioritize drugs based on
  • Tumor model selection: GIST
  • 260 patients, 14 untreatable cancers — N of 1 Study
  • Single cell Studies – active proteins in stem-like progenitor cells
  • Ivermectin Treatment vs Control (7d vs 14d)

 

3:20 pm Networking Break

3:40 pm Jean Zhao (Dana Farber Cancer Institute)

Immunotherapy and Targeted Therapy in Cancer Therapy

  • Targeting cancer with CDK4/6 inhibitors
  • CDK4/6 inhibitors causes tumor regression in breast cancer and regression of CT-26 colorectal cancer
  • CDK4/6DNMT1 inducing viral mimicry
  • PARP inhibitors  changing treatment in ovarian cancer
  • FDA approved three drugs for ovarian cancer
  • p53-null; BRCA-null; myc high – model testing

 

4:20 pm Kenna Mills Shaw (MD Anderson Cancer Center)

  • PM nor a Silver bullet nor a Dream Illusion
  • 2013: not all mutations are equally actionable
  • Context of Biomarkers
  • co-mutations in lung cancer identity – therapeutic vulnerability
  • NGS cost decrease leads to increases in Data generation
  • there are only 125 genes ACTIONABLE IN THE CLINIC
  • finding biomarkers beyond direct targets
  • clinical actionability:80K mutation – 32%
  • patients: No standard treatment available
  • Enrollment inGenotype Matched TRIALS
  • MUTATIONS SCREENED: LACK OF ENROLLMENT NOT DUE TO LACK OF MATCHING PROCESS
  • 69% GOT NEW REGIMEN, 17% did not come back — no one called them
  • 58% enrolled on genotrype-matched trials
  • Beyond NGS:

www.personalizedcancertherapy.org

  • DECISION SUPPORT IN REAL TIME IMPROVES “MATCHING” TO RIGHT DRUG.
  • MULTIFACTORS: CO-MOEBIDITIES, MICROBIOME, IMMUNE PHYNOTYPING, GENOMICS, MICROBIOME, ZIP CODE, INFECTION

5:00 pm Michael Yaffe (MIT)

  • AUGMENTED SYNTHETIC LETHALITY
  • CANCER CELLS ARE UNDER CONSTANT STRESS
  • inflammation
  • Therpeutics-targeted Synthetic Lethality
  • BRCA mutation seen in 10%-20% of patients
  • p53 mutations DNA demage – leads to apoptosis p38 MK2 as a pathway is taking over repair DNA and no apotosis occurs.
  • doxorubicin
  • Nanoparticle targeting of siRNAs to established tumors
  • The Concept of augmented Synthetic Lethality   —- enhance a prevosly known synthetic interaction by targeting additional pathways
  • combination of repair pathway  and checkpoint activation – lead to better therapeutic results
  • MK2 – targets hnRNP A0 (an RNA binding protein)  – Cleaved Caspase 3 – is synthetic lethal with p53 mutuant tumors, not just p53 null alleles
  • MK2 links Inflammation and Cancer – IBD –>> polyps and Colon Cancer
  • myeloid cell recruitment to inflammatory tumors in
  • MK2 KO mice: IL-4 –M2 magrophage – tumor progression; regulate the tumor microenvironment
  • IFNgamma –>M1 macrophages – tumor suppression

 

 

 

SOURCE

https://ki.mit.edu/news/events/cpcmsymposium-2018

https://www.eventbrite.com/e/mit-center-for-precision-cancer-medicine-inaugural-symposium-tickets-50424019600?utm_campaign=event_reminder&utm_medium=email&utm_source=eb_email&utm_term=eventname

https://www.eventbrite.com/e/mit-center-for-precision-cancer-medicine-inaugural-symposium-tickets-50424019600

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Precision Medicine Market to surpass USD 96.6 Bn by 2024, study by Global Market Insights, Inc.

Reporter: Aviva Lev-Ari, PhD, RN

 

Precision Medicine Market to surpass USD 96.6 Bn by 2024

The Precision Medicine Market is set to grow from its current market value of more than $47.8 billion to over $96.6 billion by 2024; as reported in the latest study by Global Market Insights, Inc.

Advancements in cancer biology will ensure development in gene sequencing technique and other platforms available for cancer treatment. Furthermore, rising development in field of bioinformatics that support gene sequencing to initiate formulations of precision medicines will enhance the precision medicine market growth in foreseeable future.

Increase in adoption rate of gene therapy in developed economies with relatively high-income level will further enhance the precision medicine market growth. Rising incidences of genetic disorders has positively influenced the business growth. According to WHO, sickle cell anemia affects around millions of people globally every year. Along with sickle cell anemia, there are many other genetic diseases such as thalassemia, hemophilia and others that are prevalent globally. Moreover, increase in research and development activities that boosts the production and manufacturing of precision medicine in emerging countries such as Africa and Brazil will augment the industry growth in future. However, high cost of precision medicines may restrain the market growth.

Precision Medicine Market

 

 

Bioinformatics segment was valued at USD 7.4 billion in 2017 and is expected to have considerable growth during the forecast timeframe. Bioinformatics is composed of information that includes data for gene sequencing, amino acid sequences of proteins and structural classification of protein enabling efficient drug development process for treating diseases such as cancer at molecular level. Additionally, bioinformatics is also used to determine the treatment for hereditary metabolic disorders that will drive the segmental growth in forthcoming years.

Oncology segment will experience 11.1% CAGR during the forecast timeframe. High segmental growth can be attributed to the increasing prevalence of cancer globally. According to Globocan 2018, the number of new cancer cases are estimated to be 18.1 million cases. Development of target novel therapies and precision medicines has drastically reduced the mortality rates and hence, high demand for precision medicine in cancer treatment will drive the segmental growth in foreseeable future.

Pharmaceutical companies segment of precision medicine market accounted for 37.1% in 2017 owing to the increasing R&D efforts for drug development activities. Pharmaceutical companies are currently trying to develop precision medicines for rare genetic diseases. According to Global genes, there are around 7,000 rare genetic diseases discovered.

U.S. precision market will experience 9.7% CAGR during the forecast timeframe. Enormous market growth will be due to increasing cases of rare genetic diseases. Furthermore, certain initiatives undertaken by the regulatory bodies have also positively impacted availability of precision medicines.

U.S. Precision Medicine Market Size, By Technology, 2017 & 2024 (USD Million)

Germany precision medicine market was valued at USD 2.6 billion in 2017 and high segmental growth will be due to the growing pediatric as well as adult population susceptible to metabolic diseases such as diabetes. According to NCBI, prevalence of Type 2 diabetes is high in the German population. Researchers and scientists have geared up efforts to develop precision medicines that are altered according to patient’s genome ensuring efficient treatment of genetic disorders.

Prominent industry players operational in the precision medicines market include Biocrates Life Sciences AG, Eagle Genomics Ltd, Ferrer inCode, Intomics, Laboratory Corporation of America Holdings, NanoString Technologies, Novartis, Pfizer, Qiagen, Quest Diagnostics, F. Hoffmann-La Roche, Silicon Biosystems, Tepnel Pharma Services, Teva Pharmaceuticals.

Source: https://www.gminsights.com/industry-analysis/precision-medicine-market

 

SOURCE for Request to Publish in pharmaceuticalintelligence.com

From: Shankar Khatkale <shankar.k@gminsights.com>

Date: Tuesday, November 27, 2018 at 6:48 AM

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

Subject: Article Submission Request

Dear Editor,

An industry news titled ‘Precision Medicine Market Size worth $96.6bn by 2024’ by Global Market Insights is relevant to your esteemed website https://pharmaceuticalintelligence.com. This email is a suggestion to publish this news (content attached in word format or can be picked from link mentioned below) on your website with an objective to share the information with your audiences.

News Link: https://www.gminsights.com/pressrelease/precision-medicine-market

Looking forward to hear from you.

On behalf of and as instructed by Global Market Insights, Inc.

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Tweeter and Real Time Conference Press Coverage: Aviva Lev-Ari, PhD, RN
  1. LIVE eProceeding Day Two – The 14th Annual Personalized Medicine Conference: The Business of Personalization, November 15, 2018, HMS, Boston via

  2. Cary Pfeffer, M.D., Partner, Third Rock Ventures IP can’t be reduced by other country unfair trade

  3. Pellini – CMS will take the lead partnerships early and often in clinical trials,

  4. Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition Patients and Consumers will force in five years figuring out – every diagnosis of cancer will be sequenced to interpret results and paid for

  5. Salveen Richter, Goldman Sachs Sequencing cost plunged, public investors placing funding in start ups even without return in the horizon, companies with multiple modalities spurring innovation – Europe vs US, China has no FDA ,talent from US China

  6. Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition Diagnostics component inside 4.8 Trillion in the therapeutics selection in the system as a whole Foundation Medicine saw Roche for International reach

  7. William A. Sahlman, Ph.D., Baker Foundation Professor, Harvard Business School Biotech IPO, VC, windows slam shut, drug failure – drivers and non Increasing return to scale: AI, NGS, screening, – foreign money, Tsinghua went back to China from CA

  8. William A. Sahlman, Ph.D., Baker Foundation Professor, Harvard Business School market – can it sustain the opportunity – winners and losers innovative financial models Biotech IPO, VC, windows slam shut, drug failure – drivers and non

  9. Salveen Richter, C.F.A., Vice President, Research Division, Goldman Sachs Europe successful in financing Health care in the US system must change – investment will flee, to fund pricing drug is key in changing the system CART Pricing is key

  10. Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition Impasse or Inflection Point? it s Inflection Point NOT an Impasse Diagnostics component inside 4.8 Trillion in the therapeutics selection in system

  11. Cary Pfeffer, M.D., Partner, Third Rock Ventures MS drug efficacy was in 50% non respondents 25% Genomic sequencing to identify patient populations – target therapy Mayocardia – drug in CVD for patients identified by Genomics Genomics develop drugs

  12. Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition Impasse or Inflection Point? it s Inflection Point NOT an Impasse

  13. Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana CMS Guideline: every test ordered must guide treatment otherwise not covered

  14. Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana Guidelines on ordering genomic testing, AI can assist providers, MDs need to catch up on a weekly basis companion diagnostics and pharmaceutical paid together SOCare is paid

  15. Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana show us any value as good value – avoiding patient going to MDs Office, Hospital, ER – cost increase due to Pharmacogenomics testing $5K per test

  16. Scott Ramsey, M.D., Ph.D., Full Member, Fred Hutchinson Cancer Research Center; Director, Hutchinson Institute for Cancer Outcomes Research Pricing of Testing NGS and Targeted therapy represent a threat to adoption of Genomics in Medicine

  17. Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana What test needed to be ordered? Patient stay healthy NGS $650 – $2000 in 2018, in 2016 it was $25,000 cost of testing, cost of drugs

  18. Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana cost of doing the test vs not doing this test – assess value CMS – provide data on what is covered and what is not Humana: any missed opportunities, MD order tests of no impact MR

  19. Dr. Ramsey – Survival in this cohort NGS vs EGFR – improved survival 6 month longer, Increased survivals, why? cost of sequencing – #14 most influential – cost does not drive value #1 drug cost, out of pocket expense was the factor #2 survival

  20. Scott Ramsey, M.D., Ph.D., Full Member, Fred Hutchinson Cancer Research Center; Director, Hutchinson Institute for Cancer Outcomes Research Value and utility are interconnected cost effectiveness of NGS in melanoma: single gene testing, EGFR vs NGS

  21. Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humanalabs, payers, providers, pharma — the GAP to be bridged opportunities to prevent and treat disease Payers, MDs, cost and impact, markers, Humana has a research division Use Tests

  22. MODERATOR | Daryl Pritchard, Ph.D., Senior Vice President, Science Policy, Personalized Medicine Coalition genetic profiling, adopt policy for mass deployment of NGS demonstrate value, payers needs little more that evidence exist for payer to cover

  23. Luba Greenwood, J.D., Strategic Business Development and Corporate Ventures, Verily (an Alphabet company) Patient need to own the genome data not a Databank

  24. Birgit Funke, Ph.D., F.A.C.M.G., Vice President, Clinical Affairs, Veritas Genetics; Associate Professor of Pathology (Part-Time), Harvard Medical School Risk prevention, driving DOWN operating cost curation of the Genome

  25. Luba Greenwood, J.D., Strategic Business Development and Corporate Ventures, Verily (an Alphabet company) Diagnostics in use to keep patients OUT of hospitals – management of chronic diseases

  26. Luba Greenwood, J.D., Strategic Business Development and Corporate Ventures, Verily (an Alphabet company) treatment solution therapeutics except og Oncology threatment is a strugle in the genomics field and pharmacogenomics General Medicine: CVD DM

  27. Keith Stewart, M.B., CH.B., Carlson and Nelson Endowed Director, Center for Individualized Medicine, Mayo Clinic genomics for detection of predisposition, inherited Barriers to deploy genomics: Knowledge, readiness of providers, cost of uninsured,

  28. Ellen Sigal Recipient of 14th PMC Award

  29. Sigal: Patient deserve right answers right choices, calls doctors on behave of patients treatments done out of the community in Academic hospitals – patients are scared to death. Patients are asking for options: Right testing, access to testing

  30. The 14th Annual Leadership in Personalized Medicine Award to Ellen V. Sigal, Ph.D., Chairperson, Founder, Friends of Cancer Research Patient Challenges: 90% are treated Community and they need a second opinion, insurance, access, clinical trials

  31.  Retweeted

    Frederick Banting, awarded the Nobel Prize for the discovery of insulin, was born on this day in 1891. The discovery of insulin is one of the biggest breakthroughs in medicine and has saved millions of lives.

  32. Amy Abernethy, M.D., Ph.D., Chief Medical Officer, Chief Scientific Officer, Senior Vice President, Oncology, Flatiron Health Technologies: AI, Countries with platforms Regulatory framework, reproducibility of results Taking care of people,

  33.   Retweeted

    I am covering this Conference at Harvard Medical School in Real Time, 11/14-11/15/2018 ⁦

  34. Edward Tepporn, Executive Vice President, Asian & Pacific Islander American Health Forum 1985 minorities education all surveys conducted in English, Asian American access to affordable health care services to accommodate services for communities

  35. Adolph P. Falcón, Executive Vice President, National Alliance for Hispanic Health community based organization 50 million improving healthcare access improve inclusion in science, no advancement in 45 years Hard to reach through – academic language

  36. Alex J. Carlisle, Ph.D., Chairman, CEO, National Alliance Against Disparities in Patient Health PM with focus on disparities, racialbiologic, socioecological Patient centered – raise health education Translation for interpretation Physicians&Patients

  37. Vence L. Bonham, Jr., J.D., Senior Advisor, Director on Genomics and Health Disparities, U.S. NHGRI Genomics data is of European dissents no diversity minority populations not represente sland populations not represented hispanics not

  38. Kimberly Popovits, proprietary test vs. test offered by all labs — different markets Utility agreed upon like “” demonstrate a pathway of product development that was already followed

  39. Kimberly Popovits, Chairman of the Board, CEO, President, Genomic Health – Oncology, Breast cancer molecular diagnostics Genomic testing saved the Health care System billions of dollars Genomic testing will not be placebo, 12 years study controlled

  40. Julie Khani, President, American Clinical Laboratory Association FDA will establish a center for Diagnostics, proposal for pre-certification like in Medical devices congress is involved in the decision making

  41. Michael Doherty, Senior Vice President, Head of Product Development, Head of Research & Development, Foundation Medicine – ex Genetech/Roche Operate in regulated environment, how to establish a company for long term companion diagnostics

  42. Joseph V. Ferrara, CEO, Boston Healthcare Associates Regulatory action for reimbursement of test new categories of tests: new payment if Innovation, PLA codes, 45 months approval, CPT codes

  43. Best Talk like last year by David King , keynote at

  44. David King, J.D., Chairman, CEO, LabCorp Non respondent – further researched MDs understanding, confidence of results PM Promise: close education GAP, convene on VALUE for individual cases not populations assess value among initiatives PM

  45. Tom Miller, Managing Partner, GreyBird Ventures LLC algorithms are behind the firewall of the Hospital, for privacy. the patient’s identity is not of central point, privacy is the key

  46. Radiology: SW used in detection of disease Personalized Medicine and AI Data mining of Text using AI Vital signs monitoring – providers can spot Arrhythmias earlier tagging images is a matual process

  47. Darrell M. West, Ph.D., Vice President of Governance Studies and Director of Center for Technology Innovation, Douglas Dillon Chair in Governance Studies, Interest in AI and in particular: Health care large part of the economy

  48. Gregg Talbert, Ph.D., Global Head of Digital and Personalized Health Care Partnering, Roche – Data on mutations Data falls short on Patient follow up (longitudinal data on Patients) curation of EMR IS NOT AN EASY OR AUTOMATED PROCESS i.e., IMAGES

  49. Tom Miller, Managing Partner, GreyBird Ventures LLC AI applied to capture unusual movement allowing to detect a forthcoming neurological event. Technologies used now

  50. Colin Hill, Chairman, CEO, Co-Founder, GNS Healthcare is moderator for and Medicine, panelists from , and The ⁦⁩ ⁦⁩ ⁦

  51. Bryce Olson, Global Marketing Director, Health and Life Sciences Group, Intel Corporation; stage IV prostate cancer patient Patient engage in their care, involvement in interpretation og ONES OWN Genome sequence is ate most engaging

  52. Emily Kramer-Golinkoff, Co-Founder, Emily’s Entourage, cystic fibrosis patient “Better for a is the Patient him/herself”

  53. Great Panel moderation by Toni Andreu scientific director of

  54.   Retweeted

    can help cancer patients live longer, healthier lives. Learn more about how we’re working to advance the field ‘s 11/15 when Ellen Sigal receives the 14th Annual Leadership in Personalized Medicine Award:

  55. Enjoyed most learning about the leading evolution in Genomics in Israel, UK, Canada and Finland

  56.   Retweeted

    In 14th Annual Elizabeth Nabel- made an excellent overview exploring the Promise of Personalized Medicine: Healthcare state-of-the-art; Digital health; Professionals-Payers-Providers relationships and next future challenges

  57. Amazing International Panel: UE, Israel, UK, Canada, Finland

  58. Great talk of a visionary in Management of Academic

  59. Great Confernec, 14th in one row

  60. Antonio L. Andreu, M.D., Ph.D., Scientific Director, EATRIS European Infrastructure for Translational Medicine General Medicine: Metabolomics, biological systems vs GENOMICS Medical care w/genomic testing, MDs will call to tell patients future news

    Translate Tweet

  61. Ora Dar, Ph.D., Senior Expert, Medical Sciences, consultant to the Israel Innovation Authority $300Miliion R&D sponsored clinical data on genomics, MDs are trained to place genomics data on EMR, epidemiology, sequencing of genetic diseases

  62. Liisa-Maria Voipio-Pulkki, M.D., Ph.D., Director General, Chief Medical Officer, Ministry of Social Affairs and Health, Finland Public sector is the majority of Health care systems, Expertise is as high as can be, entrepreneurship is on the rise

  63. Marc LePage, President, CEO, Genome Canada Social impact, adoption systems for focusing on rare diseases, following UK and US trends, 10 sites in Canada, aggregate the datethe National level, extract clinical data securely implementation expertise

  64. Tom Fowler, Ph.D., Deputy Chief Scientist, Building infrastructure, education, future, National approach to genomic testing, built in a National lab, scaling research

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LIVE eProceedings Day Two – The 14th Annual Personalized Medicine Conference: The Business of Personalization, November 15, 2018, HMS, Boston

Real Time Coverage: Aviva Lev-Ari, PhD, RN

 

PART II

 

The Business of Personalization

The successful implementation of [personalized medicine] will depend on the embrace of [its] principles in the business community.

 RAJU KUCHERLAPATI, PH.D.
Paul C. Cabot Professor of Genetics, Harvard Medical School

*** Speakers will be added to the schedule on a rolling basis as they are confirmed. ***

7:00 a.m.
Registration and Continental Breakfast

Joseph B. Martin Conference Center at Harvard Medical School
77 Avenue Louis Pasteur, Boston, MA 02115

8:00 a.m.
Opening Remarks

SPEAKER | Stephen L. Eck, M.D., Ph.D., Chief Medical Officer, Immatics U.S. Inc; Board Chair, Personalized Medicine Coalition

8:10 a.m.
Pioneering Precision: Inside the Pharmaceutical Industry’s Push Toward Personalized Medicine — A Fireside Chat

MODERATOR | Meg Tirrell, Reporter, CNBC

Daniel O’Day, CEO, Roche Pharmaceuticals

8:55 a.m.
Considering Costs: Evaluating Emerging Pharmaceutical and Insurance Industry Business Models in Personalized Medicine

The pharmaceutical industry is deeply invested in commercializing personalized therapies that must recoup fixed development costs from smaller patient populations covered by health insurance companies that are increasingly concerned about rising health care costs. In that context, this diverse panel will explore the viability of the business model for developing and paying for personalized medicines, tackling issues related to costs, prices, and access.

MODERATOR | Meg Tirrell, Reporter, CNBC

Peter Juhn, M.D., M.P.H., Global Head of Value-Based Partnerships, Amgen

Nick Leschly, CEO, Bluebird Bio

Michael Sherman, M.D., Chief Medical Officer, Senior Vice President, Harvard Pilgrim Health Care

Sean Tunis, M.D., Founder, CEO, Center for Medical Technology Policy

9:55 a.m.
Networking Break

Light refreshments provided.

Sponsored By

10:25 a.m.
Reinventing Research: Are Adaptive Platform Trials the Model of the Future? (A Harvard Business School Case Study)

Recognizing that traditional randomized controlled clinical trials can only study the safety and efficacy of a single therapy in one large population of patients, researchers in personalized medicine increasingly hope that “adaptive platform trials,” which employ advanced statistical techniques to simultaneously test the effectiveness of several personalized treatments in multiple sub-populations of patients, may be the key to new drug approvals in the future. Adaptive platform trials may make drug development more efficient by revealing which of several drug candidates are most promising for which patients, but maximizing the potential of these trials requires unprecedented collaboration among the institutions conducting and sponsoring research on various personalized treatments — and no obvious business models have emerged.

During this interactive case study discussion, professors from Harvard Business School will help us examine how researchers at the Dana-Farber Cancer Institute considered and addressed myriad challenges in their effort to design and operationalize an adaptive platform trial for glioblastoma patients, a deadly disease state for which there are few existing treatment options.

PRESENTED BY

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

Ariel D. Stern, Ph.D., Assistant Professor, Technology and Operations Management Unit, Harvard Business School

11:40 a.m.
The 14th Annual Leadership in Personalized Medicine Award

INTRODUCTION | Steven D. Averbuch, M.D., Vice President, Head of Precision Medicine, Bristol-Myers Squibb

  • Ellen V. Sigal head of Friends of Cancer Research
  • Advanced science by Diagnostics Tests
  • Cancer Moonshot Program
  • Revolution therapies brought to market by Sigel’s sponsorship

AWARDEE | Ellen V. Sigal, Ph.D., Chairperson, Founder, Friends of Cancer Research

Friendly conversation:

  • Thanks to PMC
  • sister die on breast cancer at 40 with child of 4 1/2.
  • appointed to celebrate 20th year of American Cancer Association – Funding for Research, money spend in Washington is for Patients.
  • After ten years, interested in measurement of achieving evaluation, FDA structure was of interest.
  • Precision Medicine: biomarkers and targets for patients to define success for each patient, WHat is the right population for any drug, responders to drug therapy, if no response, change the drug.
  • Patient perspective: Challenges: 90% are treated in the Community and they need a second opinion, insurance, access, clinical trials done out of the community in Academic hospitals – patients are scared to death. Patients are asking for options: Right testing, access to testing involve insurance
  • combination therapy  – 6-8 months in advance,
12:10 p.m.
Bag Lunch
1:10 p.m.
Predicting and Preventing: Evaluating Progress Toward Personalized Medicine

The original architects of the personalized medicine paradigm envisioned an era in which clinicians could predict, prevent and treat disease based on an improved understanding of how human biology interacts with external environments. During this session, a panel of experts will examine our progress on each of these fronts during a wide-ranging conversation about personalized medicine’s past, present and future.

MODERATOR | Cynthia Casson Morton, Ph.D., William Lambert Richardson Professor of Obstetrics, Gynecology and Reproductive Biology, Harvard Medical School

  • 1 Million volunteer participants for genome sequencing and microbiome data
  • PM Past, Present and Future
  • Issues that are rapidly evolving: Physician, Patients

Birgit Funke, Ph.D., F.A.C.M.G., Vice President, Clinical Affairs, Veritas Genetics; Associate Professor of Pathology (Part-Time), Harvard Medical School

  • Risk prevention,
  • driving DOWN operating cost curation of the Genome

Luba Greenwood, J.D., Strategic Business Development and Corporate Ventures, Verily (an Alphabet company)

  • view on diagnostics from Roche, CHemist and lawyer, Venture capital, beyond Genomics, from diagnosis to prognosis,at Verily projects tapping into the entire life span aspect of health
  • treatment solution therapeutics except og Oncology threatment is a strugle in the genomics field and pharmaco-genomics
  • Power the patients vs Genomics in Diabetes
  • Diagnostics in use to keep patients OUT of hospitals – management of chronic diseases
  • Patient need to own the genome data not a Databank

Keith Stewart, M.B., CH.B., Carlson and Nelson Endowed Director, Center for Individualized Medicine, Mayo Clinic

  • Hematologist, genomics apply genomics for detection of predisposition, inherited , Health genome sequencing,
  • Barriers to deploy genomics: Knowledge, readiness of providers, cost of uninsured,
  • Diagnostics high value low cost
  • drug adherence, pharmacists to be involved in drug adherence before refill
2:10 p.m.
Assessing the Assays: Determining the Clinical and Economic Utility of Genomic Sequencing

Advocates for personalized medicine have contended that genomic sequencing can deliver clinical and economic value to patients and the health system by allowing providers to more efficiently diagnose disease and develop treatment plans. Following increased use of genomic sequencing in clinical settings, many stakeholders, including payers, have begun to examine that value proposition more closely. During this session, a pharmaceutical industry representative, a payer, and a health economist will discuss the status and future of the emerging evidence regarding the clinical and economic utility of genomic sequencing, including studies recently commissioned by the Personalized Medicine Coalition.

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

  • genetic profiling, adopt policy and procedures for mass deployment of NGS
  • show that it works – demonstrate value, payers and providers
  • a little more that evidence exist for payer to cover
  • rare diagnosed disease

Kristine Bordenave, M.D., F.A.C.P., Corporate Medical Director, Humana

  • labs, payers, providers, pharma — the GAP to be bridged
  • opportunities to prevent and treat disease
  • Payer, MDs, cost and impact, markers,
  • Humana has a research division Use Testing to find value, pharmacogenomics  – on Medicare, Medicaid patients
  • cost of doing the test vs not doing this test – assess value
  • pharmacisit, economist, statisticians – CMS – provide data on what is covered and what is not Humana: any missed opportunities, MD order tests of no impact per medical record
  • What test needed to be ordered? Patient stay healthy
  • NGS $650 – $2000 in 2018, in 2016 it was $25,000 cost of testing, cost of drugs
  • show us any value as good value – avoiding patient going to MDs Office, Hospital, ER – cost increase due to Pharmacogenomics testing $5K per test
  • Guidelines on ordering genomic testing, AI can assist providers, MDs need to catch up on a weekly basis
  • CMS Guideline: every test ordered must guide treatment otherwise not covered

Scott Ramsey, M.D., Ph.D., Full Member, Fred Hutchinson Cancer Research Center; Director, Hutchinson Institute for Cancer Outcomes Research

  • Value and utility are interconnected
  • cost effectiveness of NGS in melanoma: single gene testing – EGFR vs NGS – help clinicians to evaluate Lung Cancer
  • Flariton Database, 300 centers  – 140,000 – Patients got NGS – 7% ADDITIONAL patients founded mutations beyond EGFR
  • Survival in this cohort NGS vs EGFR – improved survival 6 month longer, mean survival 3 weeks long, not significant.
  • Increased survivals, why? cost of sequencing  – #14 most influential – cost does not drive value
  • #1 drug cost was the factor
  • #2 survival
  • marginal cost in platform comparison
  • Pricing of Testing NGS and Targeted therapy represent a threat to adoption of Genomics in Medicine
  • disparities and access – cost and patients: Partners and Mayo clinic patients are lucky

 

3:10 p.m.
PhRMA Foundation Challenge Awards: Developing Value Assessment Strategies That Align With Personalized Medicine

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

PRESENTER | Shreeram Aradhye, M.D., Head of Global Medical Affairs, Chief Medical Officer, Pharmaceuticals, Novartis; Board Member, PhRMA Foundation

#1 Prize $50,000 – Dr. Garrison, UK

#2 Prize $25,000 – Dr. Robim Hayeems, Hospital for Sick Children Institute, Toronto, Canada

#3 Prize @ $10,000  – Dr. A Le, PharmD., PhD, Western University of Health Sciences

3:20 p.m.
Networking Break

Light refreshments provided.

3:50 p.m.
Impasse or Inflection Point? — An Investment Analysis

Sustaining the pace of innovation in personalized medicine will require continued investment in new initiatives, but the financial outlook for the field remains unclear. In that context, this panel of investors will examine whether personalized medicine is at an impasse, an inflection point or somewhere in between.

MODERATOR | William A. Sahlman, Ph.D., Baker Foundation Professor, Harvard Business School

  • market – can it sustain the opportunity – winners and losers
  • innovative financial models
  • Biotech IPO, VC, windows slam shut, drug failure – drivers and non
  • Increasing return to scale: AI, NGS, screening, – foreign money, China
  • Tsinghua University went back to China from Silicon Valley

Cary Pfeffer, M.D., Partner, Third Rock Ventures

  • was a decade at Biogen, MS indication drug, no biomarkers for patients – efficacy was in 50% non respondents 25%
  • Genomic sequencing to identify patient populations – no good effective medicine without target therapy
  • Mayocardia – drug in CVD for patients identified by Genomics
  • Genomics information needed to develop drugs

Michael Pellini, M.D., Managing Partner, Section 32; Board Member, Personalized Medicine Coalition

  • Impasse or Inflection Point? it s Inflection Point NOT an Impasse
  • Diagnostics component inside 4.8 Trillion in the therapeutics selection in the system as a whole
  • Foundation Medicine saw Roche as Big brother with International reach
  • Patients and Consumers will force in five years figuring out – every diagnosis of cancer will be sequenced and the infrastructure to interpret results and paid for

Salveen Richter, C.F.A., Vice President, Research Division, Goldman Sachs

  • innovative and disruptive, orphan drugs, Health IT, US Market 3 trillion – size of the opportunity 80% genetically driven
  • Cancer, CART therapy, easier to pay by performance, cost of the drug itself. profit in the 1st generation od Pharma manufacturers
  • One time pricing vs further indications, annuity type system, Hemophilia – $19Million market,
  • Europe successful in financing Health care — in the US — system must change – investment will flee, to fund pricing drug is key in changing the system CART Pricing is still difficult to pay for
  • Sequencing cost plunged, public investors placing funding in start ups even without return in the horizon, companies with multiple modalities spurring innovation – confusing in the investment side, technologies become obsolete very fast
  • Europe vs US, China is different no regulation like FDA,talent from US Pharma went back to China

 

4:50 p.m.
Closing Remarks

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

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