Cellular Therapy in Transfusion Medicine : Is the Glass Half-Full or Half-Empty? Tuan N. Le, MD 1/21/2017

Transcription

1 Cellular Therapy in Transfusion Medicine : Is the Glass Half-Full or Half-Empty? Tuan N. Le, MD 1/21/2017

2 Snapshot of Cellular Therapy (CT) & Regenerative Medicine Industry Five Lessons from the Rise & Fall of Dendreon CT Opportunities in Transfusion Medicine

3 Cellular Therapy is a part of Regenerative Medicine ARM-Alliance for Regenerative Medicine

4 Outside the setting of hematopoietic reconstitution and a few other wellestablished indications, the assertion that stem cells are intrinsically able to sense the environment into which they are introduced and address whatever functions require replacement or repair whether injured knee cartilage or a neurologic deficit is not based on scientific evidence. Marks P Witten C and Califf R. Perspective: Clarifying stem-cell therapy s benefits and risks. NEJM Epub 11/30/16 Turner L and Knoepfler P. Selling stem cells in the USA: assessing the direct to consumer industry Cell Stem Cell 2016;19:

5 Snapshot of 2016

6 ARM 2014 Annual Report,p.26

7 ARM 2014 Annual Report, p. 28

8 Therapeutic Opportunities in Regenerative Medicine ARM 2014 Annual Report, p. 31 ARM 2013 Annual Report, p. 37, 47

9 Current Cellular Therapy s Darling: CAR-T cells ARM State of the Industry, 1/11/2016

10 CAR-T-cell Designs Jackson, H. J. et al. (2016) Driving CAR T-cells forward Nat. Rev. Clin. Oncol. doi: /nrclinonc

11 CD19-specific-CAR T-cell therapy outcomes in patients with B-ALL Jackson, H. J. et al. (2016) Driving CAR T-cells forward Nat. Rev. Clin. Oncol. doi: /nrclinonc

12 Half-Full vs. Half-Empty? Snapshot of Cellular Therapy Industry Five Lessons from the Rise & Fall of Dendreon CT Opportunities in Transfusion Medicine

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14 Lesson #1: The road to FDA approval is long and arduous Dendreon Milestones: Activated Cell Therapy formed by Engelman & Strobber later changed name to Dendreon phase 1 study published 2009-phase 3 Impact study results released 2010-FDA approval

15 Dendreon Analyst Day Webcast, 9/24/2009 available at via Internet Archive Wayback Machine

16 Dendreon Analyst Day Webcast, 9/24/2009 available at via Internet Archive Wayback Machine

17 Figure 1 Mechanism of action of Sipuleucel-T Di Lorenzo, G. et al. (2011) Immunotherapy for the treatment of prostate cancer Nat. Rev. Clin. Oncol. doi: /nrclinonc

18 Dendreon Analyst Day Webcast, 9/24/2009 available at via Internet Archive Wayback Machine

19 dvaccines/cellulargenetherapy Products/ApprovedProducts/uc m htm

20 Lesson #2: Autologous vaccines can be produced efficiently in an outsourced network Dendreon Analyst Day Webcast, 9/24/2009 available at via Internet Archive Wayback Machine

21 Lesson#3: Transfusion medicine community can provide apheresis expertise and support throughout the various cellular therapy clinical trial phases Dendreon Analyst Day Webcast, 9/24/2009 available at via Internet Archive Wayback Machine

22 Dendreon Analyst Day Webcast, 9/24/2009 available at via Internet Archive Wayback Machine

23 Lesson #4: Pricing Strategy vs. Price Gouging Provenge ~$93,000/total treatment Forbes Top Ten Healthcare Charts for

24 Sermo Survey, 2011

25 Lesson #5: One who knows neither the enemy nor himself will invariably be defeated in every engagement. Sun Tzu, The Art of War, RD Sawyer translation, chapter 3, p. 179,

26 cle/325975

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28 Seattle Times, 11/10/14: _dendreonchapter11xml.html Have you ever heard of the shoeshine boy story?

29 Half-Full vs. Half-Empty? Snapshot of Cellular Therapy Industry Five Lessons from the Rise & Fall of Dendreon: The road to FDA approval is long and arduous Autologous vaccines can be produced efficiently in an outsourced network Transfusion medicine community can provide apheresis expertise and support throughout the various cellular therapy clinical trial phases Pricing Strategy vs. Price Gouging One who knows neither the enemy nor himself will invariably be defeated in every engagement. CT Opportunities in Transfusion Medicine

30 Silberstein LE and Toy P. Research opportunities in transfusion medicine. JAMA 2000;285:

31 Transfusion Medicine has Many Footholds and Opportunities in Cell Therapy

32 TM in a Horizontal CT Service Spectrum Donor Collections, Recruitment Infectious Disease Testing Emerging Technologies: Cellular Therapies, Modified Blood Products Medical Indications for Treatment Transfusion Monitoring Patient

33 Alliance for Regenerative Medicine Annual Industry Report 2014, p. 8

34 Apheresis is key in obtaining the T cell source in CAR-T cell therapy.

35 Ummm shouldn t that be apheresis?? S4E7: Dr. Adrian Shaw, 11/3/16:

36 TM is strategically positioned to collaborate with CT industry due to experience and compliance with various regulatory standards Erben RG et al White paper on how to go forward with cell based therapies in Europe. Tiss Engin 2014;20:

37 Human Platelet Lysate: another area of opportunity

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39 TM in a CT Vertical Service Spectrum Adapted from Blood, Tissues, and Cells from Human Origin-a European Blood Alliance Perspective,2013, p. 252

40 Whatever happened to stem-cell derived O-neg RBCs? DARPA, 2007 Stem-cell derived O- neg RBCs Bioreactor (size of washing machine) able to withstand parachuting into battlefield Completion within 27 months

41 Li X et al How far are stem cell-derived erythrocytes from the clinical arena? BioScience 2013;63:

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44 ARM Annual Industry Report 2014, p. 3 Blood, Tissues, and Cells from Human Origin-a European Blood Alliance Perspective,2013, p. 249

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