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Providing the Cure

                                                       GIL 2012: RUSSIA
                                      The Global Community of Growth, Innovation and Leadership


                                              A Novel Therapy for the Treatment of
                                                    Leukemia and Cancer


                                       “The Stem-Cell Cocktail”
                            Steve Shor                                                   Brian Denker
                    Founder & Executive Director                                 Vice President & Global Head
                    NCLF Cancer Research Center                                Growth, Innovation and Leadership
                                                                                        Frost & Sullivan


In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te   www.leukemiafoundation.org
Providing the Cure


                                                          Why is cancer so challenging?

         Cancer is dynamic & adaptive.

         Cancer is strong & resilient.

         Cancer is complex & innovative.

         Cancer is opportunistic.

In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te   www.leukemiafoundation.org
Providing the Cure


                 The Typical Question:                                    The BIG Question…
                 In what ways does the                                        The NCLF Question
                typical cancer cell differ
                                                                       • What are the mechanisms that
                from the normal cell?
                                                                         foster growth in cancer cells?

                                                                       • Are there multiple pathways of
                                                                         growth?

                                                                       • How does the cancer cell
                                                                         harness the energy of the human
                                                                         body to facilitate growth?
In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te   www.leukemiafoundation.org
Providing the Cure


                                                      NCLF Bio Medical Research Center




In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te    www.leukemiafoundation.org
Cancer/Leukemia cells                                         Providing the Cure
                                                      The Stem Cell & Cancer
                                                         Interaction Paradigm
                                                      Molecular pathways analysis




In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te
                          Adult Stem Cells
Providing the Cure


                                                      Unleashing the Potential of Stem Cells
                                                                         in Cancer Therapy
               Cancer -Stem cell Interaction Paradigm


             Identify target molecular pathways
         regulating cancer cell growth & development


                    Design of interventions using
                  multiple target molecular pathways


                       Addressing the complexity of
                     cancer development and diversity


In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te     www.leukemiafoundation.org
Providing the Cure


                                                           Combination Therapy Approach


                                                                        Pathway
                                                                           #1


                                                          Pathway
                                                             #3


                                                                             Pathway #2




In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te   www.leukemiafoundation.org
Providing the Cure




                                                          Stem Cells
                                                                                   • Multiple Pathways
                                        Compound
                                           #2
                                                                                   • Multiple Targets
                                                                                   • Multiple Compounds
                                                      Compound
                                                         #3




In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te              www.leukemiafoundation.org
Providing the Cure


                                                                   Growth Inhibition of Leukemia Cells using
                                                                               “Stem Cell Cocktail” Therapy
                                         Growth inhibition of Leukemia cells after 72h of culture
        Viable cell number (OD)




                                   1.8
                                  1.75
                                   1.7


                                                                                                                  (in vitro results)
                                  1.65
                                   1.6
                                  1.55
                                   1.5
                                  1.45
                                   1.4
                                  1.35
                                   1.3
                                                                 A



                                                                              B



                                                                                          C




                                                                                                       )
                                          l
                                        ro




                                                                                                     +C
                                                        d



                                                                              d



                                                                                          d
                                      nt



                                                      un



                                                                            un



                                                                                        un



                                                                                                   +B
                                   Co



                                                    po



                                                                          po



                                                                                      po



                                                                                                 (A
                                                   m



                                                                         m



                                                                                     m



                                                                                                l"
                                                Co



                                                                      Co



                                                                                  Co



                                                                                              ai
                                                                                           kt
                                                                                         oc
                                                                                       "C




In S t rate gi c P art n e rs h i p wi th :


                                   F ro s t & S u lli v an In s ti tu te          www.leukemiafoundation.org
Providing the Cure


                                                                                               “Stem Cell Cocktail”
                                                                                           A Therapy to Treat Cancer
                                                                                                     (in vivo results)
               Survival of Leukemia harboring animals treated with Adult stem cells
                                                                    1.1
                                                                    1.0                                                control
                                                                    0.9                                                SC treated
                                                Fraction survival




                                                                    0.8
                                                                    0.7                                              P<0.0001
                                                                    0.6
                                                                    0.5
                                                                    0.4
                                                                    0.3
                                                                    0.2
                                                                    0.1
                                                                    0.0
                                                                          0    10    20   30    40    50   60   70
In S t rate gi c P art n e rs h i p wi th :
                                                                                            Days

              F ro s t & S u lli v an In s ti tu te                           www.leukemiafoundation.org
Providing the Cure



                                                      A Cure IS POSSIBLE in our Generation


                                              National Children’s Leukemia Foundation

                                       Use of Mesenchymal Stem Cells
                                        for the Treatment of Leukemia
                          United States Provisional Patent Application #61/564,317
                                             November 29, 2011


In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te     www.leukemiafoundation.org
Providing the Cure



                                                                                                  Trajectory of Progress


                                                                                                         Clinical Trials



                                                                                 Pre-Clinical Trials




                                              *Testing Additional Combinations
                                              *Creating additional “Cocktails”
                                              *Expanding the paradigm to
                                              other cancer types.



In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te             www.leukemiafoundation.org
Providing the Cure


                                                        The Cure IS Within Our Reach
                                                               • Explore these advancements in cancer
                                                                 research further, be apart of it.
                                                               • Determine how you too can help
                                                                 NCLF to contribute to these
                                                                 advancements.
                                                               • Make a Difference – Find your
                                                                 visionary innovation, support each
                                                                 other, members of the GIL
                                                                 Community and it’s global partners
                                                                 contributing and changing the world.

In S t rate gi c P art n e rs h i p wi th :


              F ro s t & S u lli v an In s ti tu te   www.leukemiafoundation.org
GIL 2012: Beyond the Journey to Visionary Innovation
   Next Steps




Share, Engage and Inspire 24x365        Schedule a one-on-one         Develop Your Visionary and Innovative Skills
  via the online GIL Community         Growth Strategic Dialogue             Growth Partnership Service




Leverage the GTL in your monthly   Envision Your Future and Succeed    Get Your Entire Team Innovating to Zero
GIL Community Newsletter (GCN)          with Growth Workshops          with an on-site Mega Trends Workshop

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Gil 2012 Stem Cell Cocktail

  • 1. Providing the Cure GIL 2012: RUSSIA The Global Community of Growth, Innovation and Leadership A Novel Therapy for the Treatment of Leukemia and Cancer “The Stem-Cell Cocktail” Steve Shor Brian Denker Founder & Executive Director Vice President & Global Head NCLF Cancer Research Center Growth, Innovation and Leadership Frost & Sullivan In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 2. Providing the Cure Why is cancer so challenging?  Cancer is dynamic & adaptive.  Cancer is strong & resilient.  Cancer is complex & innovative.  Cancer is opportunistic. In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 3. Providing the Cure The Typical Question: The BIG Question… In what ways does the The NCLF Question typical cancer cell differ • What are the mechanisms that from the normal cell? foster growth in cancer cells? • Are there multiple pathways of growth? • How does the cancer cell harness the energy of the human body to facilitate growth? In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 4. Providing the Cure NCLF Bio Medical Research Center In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 5. Cancer/Leukemia cells Providing the Cure The Stem Cell & Cancer Interaction Paradigm Molecular pathways analysis In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te Adult Stem Cells
  • 6. Providing the Cure Unleashing the Potential of Stem Cells in Cancer Therapy Cancer -Stem cell Interaction Paradigm Identify target molecular pathways regulating cancer cell growth & development Design of interventions using multiple target molecular pathways Addressing the complexity of cancer development and diversity In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 7. Providing the Cure Combination Therapy Approach Pathway #1 Pathway #3 Pathway #2 In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 8. Providing the Cure Stem Cells • Multiple Pathways Compound #2 • Multiple Targets • Multiple Compounds Compound #3 In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 9. Providing the Cure Growth Inhibition of Leukemia Cells using “Stem Cell Cocktail” Therapy Growth inhibition of Leukemia cells after 72h of culture Viable cell number (OD) 1.8 1.75 1.7 (in vitro results) 1.65 1.6 1.55 1.5 1.45 1.4 1.35 1.3 A B C ) l ro +C d d d nt un un un +B Co po po po (A m m m l" Co Co Co ai kt oc "C In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 10. Providing the Cure “Stem Cell Cocktail” A Therapy to Treat Cancer (in vivo results) Survival of Leukemia harboring animals treated with Adult stem cells 1.1 1.0 control 0.9 SC treated Fraction survival 0.8 0.7 P<0.0001 0.6 0.5 0.4 0.3 0.2 0.1 0.0 0 10 20 30 40 50 60 70 In S t rate gi c P art n e rs h i p wi th : Days F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 11. Providing the Cure A Cure IS POSSIBLE in our Generation National Children’s Leukemia Foundation Use of Mesenchymal Stem Cells for the Treatment of Leukemia United States Provisional Patent Application #61/564,317 November 29, 2011 In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 12. Providing the Cure Trajectory of Progress Clinical Trials Pre-Clinical Trials *Testing Additional Combinations *Creating additional “Cocktails” *Expanding the paradigm to other cancer types. In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 13. Providing the Cure The Cure IS Within Our Reach • Explore these advancements in cancer research further, be apart of it. • Determine how you too can help NCLF to contribute to these advancements. • Make a Difference – Find your visionary innovation, support each other, members of the GIL Community and it’s global partners contributing and changing the world. In S t rate gi c P art n e rs h i p wi th : F ro s t & S u lli v an In s ti tu te www.leukemiafoundation.org
  • 14. GIL 2012: Beyond the Journey to Visionary Innovation Next Steps Share, Engage and Inspire 24x365 Schedule a one-on-one Develop Your Visionary and Innovative Skills via the online GIL Community Growth Strategic Dialogue Growth Partnership Service Leverage the GTL in your monthly Envision Your Future and Succeed Get Your Entire Team Innovating to Zero GIL Community Newsletter (GCN) with Growth Workshops with an on-site Mega Trends Workshop