La Médecine de Précision, Aujourd'hui et Demain Michel Goldman - Co-directeur, Institut I3h, Université libre de Bruxelles Editeur-en-chef ...

La Médecine de Précision,
  Aujourd’hui et Demain

            Michel Goldman
Co-directeur, Institut I3h, Université libre de Bruxelles
        Editeur-en-chef, Frontiers in Medicine

Precision medicine:
« The right drug for the right patient »

                                          BIOMARKERS
                                         (omics, imaging..)
                                          ALGORITHMS

   EFFECT     NO     ADVERSE    EFFECT
            EFFECT   REACTION

Precision Medicine in 2019

          PRECISION
          MEDICINE

Immune Checkpoint Inhibitors
       - Toxicities -

                               4

NATURE 2018 24: 255
RESPONDER   NON-RESPONDER

CAR T-Cells: Combining
Cell and Gene Therapy

CAR T Cells: The next revolution in
      cancer immunotherapy

                Acute Lymphoblastic
                     Leukemia         Current Price:
FDA-approved
                                      > 300.000 €
               B Cell Lymphoma

           Multiple Myeloma
               Solid Tumors

Nature 2018, 555

Journal of Clinical Oncology, 1 June 2018

                           June 2018

American Economic Review 2013 103: 406-411

   Can Financial Engineering Cure Cancer?

       By DAVID E. FAGNAN, JOSE MARIA FERNANDEZ,
             ANDREW W. LO, & ROGER STEIN

Detection and localization of surgically resectable cancers
                     with a multi-analyte blood test

                         Science, eaar 3247 January 18, 2018
Published by AAAS

NATURE 2018: 555:447

Watson for Oncology

La médecine de précision au-delà
          du cancer

  Diabète
  Maladies inflammatoires du tube
   digestif (maladie de Crohn)
  Arthrite rhumatoïde
  Asthme bronchique
  …………
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Stratification of diabetic patients
                   Big Data from 8980 patients

   Risk of renal                                Group 1
   insuffisiency

                                                  Group 2

                                           Group 3
                                      Group 4
                                          Group 5

                          Duration of diabetes (years)

   Ahlquist et al., Lancet Diabetes & Endocrinology 2018, 6: 361-369
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Towards a new taxonomy
      of diseases

                *Patient Reported Outcomes

Precision therapeutics
             beyond chemical drugs

Produits                           Indications
Biopharmaceuticals        Cancer, inflammatory diseases
(monoclonal antibodies)
Targeted radiotherapy     Solid tumors
(proton therapy..)
Cell therapies
       aaa                Leukemias, lymphomas….....
Gene therapies            Genetic diseases, retinal disease

Phages                    Multiresistant bacterial infections
Degradable biomaterials   Cardiac prostheses
Robotics                  Surgery, artificlal limbs
Connected pills           Mental disorders

The First Connected Drug Approved by FDA
  Abilify MyCite (aripiprazole tablets with sensor)

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Feb 1st 2018

A revolution in healthcare is
          coming
    Welcome to doctor YOU

Sensing a Shift in Healthcare

                        Steven R. Steinhubl et al.,
                        Sci Transl Med 2015;
                        7:283rv3

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The Changing Face of Clinical Trials
  Randomized registry trials

  Pragmatic trials:
  • Provide real-world evidence based on
     patient-reported outcomes

  Adaptive trials
  • Modifications in the course of the trial according
    "real-time learning"
  • Facilitates assessment of combination therapies

  Basket trials
  •   Different diseases with same biomarker/target      23

Les déterminants des maladies
chroniques non-transmissibles:
   Vers la prévention ciblée
 Génome               Microbiome

                      Tabac
                      Alcool
                      Régime alimentaire
           Exposome   Sommeil
                      Médicaments
                      Toxiques environnementaux
                      …..                    25

Deployment of Preventive Interventions
     - Time for a Paradigm Shift -
     Katherine Pryor, M.D., and Kevin Volpp, M.D., Ph.D   May 10, 2018

“For health care’s transformation from a volume- to a value-
based framework to be successful, putting coverage of
preventive services and treatments on more even footing
will deliver great value”

e.g. behavorial intervention to prevent diabetes
                                                                 26

Science 2018, 361: 1017

“…cancer death rates could be reduced by
70% around the world even without the
development of any new therapies”

“Deciphering the complexity of human
diseases and finding safe, cost-effective
solutions that help people live healthier
lives requires collaboration across
scientific and medical communities
throughout the health care ecosystem.

Open Science is Critical
to Move Health Research Forward

   Transparency and dissemination
         of data and methods

       Results Reproducibility

    Translation in Health Outcomes
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Supporting precision medicine:
      education is key!

Obermeyer Z, Lee TH. N Engl J Med 2017;377:1209-1211

Redefining the roles of the doctor

                         The Economist June 2012

A New Role for Leading Hospitals
           in Europe
  J Ribeira, G Antoja, M Rosenmöller & P Borrás

RESEARCH AND EDUCATION AS KEY MISSIONS
   Leading hospitals should develop
   multi-stakeholder collaboration for
  translational research and education

                                    http://bit.ly/2y1NUiB
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The I3h Interuniversity Program
   in Healthcare Innovation
           17 January 2019

       http://i3health.eu/

“Medicine is a science of uncertainty
and an art of probability.”

                    Sir William Osler
                            1849-1919

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Thank you

   @MichelGoldman
   @I3h_Institute

 www.i3health.eu

mgoldman@i3health.eu

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