The future of HSCT. John Barrett, MD, NHBLI, NIH Bethesda MD

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1 The future of HSCT John Barrett, MD, NHBLI, NIH Bethesda MD

2 Transplants today

3 Current approaches to improve SCT outcome Optimize stem cell dose and source BMT? PBSCT? Adjusting post transplant I/S to minimize GVHD and preserve GVL Select god risk patients! TXPT TXPT survival survival relapse relapse New conditioning regimens Controlled regimen intensity YEARS Better Supportive care

4 Roadblocks to sucessful outcome transplant T cell recovery B cell recovery Viral reactivation clinical endpoints Marrow recovery APC recovery NK recovery infection GVL Biological endpoints cgvhd agvhd Relapse statistical endpoints TRM OS DFS

5 Preventing GVHD Host APC Donor T cells Host target virus engagement Activation/ex pansion damage repair leuk mha MHC1 MHC2 CD8 CD4 CD8 CD4 Stop activation Remove APC Block APC Conditioning Bortezomib Remove T cells ATG Campath T cell depletion Selective T cell depletion CSA/Tacro Sirolimus Tregs block proliferation MTX Cy MMF Steroids MSC Prevent T cell target contact Steroids homing Tissue repair MSC

6 NO CHANGE IN RELAPSE AFTER HLA-IDENTICAL SIBLING MYELOABLATIVE TRANSPLANTS FOR EARLY LEUKEMIA* IBMTR, (1,071) 23% PROBABILITY, % (8,989) 22% 1995 (9,747) 20% YEARS *1 st CR for AML or ALL Chronic phase for CML

7 NO CHANGE IN RELAPSE AFTER HLA-IDENTICAL SIBLING MYELOABLATIVE TRANSPLANTS FOR ADVANCED LEUKEMIA* IBMTR, PROBABILITY, % (451) 62% (2299) 60% 1995 (2261) 62% YEARS * Not in remission for AML or ALL Blast phase for CML

8 Is BMT at an evolutionary dead end?

9 The challenges ahead A transplant for every patient that needs one? Expanding the donor pool Safer transplants for older patients

10 Related and unrelated SCT meet less than half the need for donors Percentage of patients needing donors 100% 42% 30% 21,000 no unrelated donor 4,000 unrelated 11,000 related CBT Haplo transplant

11 5,000 4,500 4,000 Allogeneic Transplants for Age 20yrs, Registered with the CIBMTR by Donor Type and Graft Source - Related BM/PB Unrelated BM/PB Unrelated CB Number of Transplants 3,500 3,000 2,500 2,000 1,500 1, * * Data incomplete Slide 10 SUM-WW11_10.ppt

12 Number of Transplants 13,000 12,000 11,000 10,000 9,000 8,000 7,000 6,000 5,000 4,000 3,000 2,000 1,000 0 Allogeneic Transplants for Age 20yrs, Registered with the CIBMTR by Donor Type and Graft Source - Related BM/PB Unrelated BM Unrelated PB Unrelated CB * * Data incomplete Slide 11 SUM-WW11_11.ppt

13 Trend: More patients will have a donor Umbilical cord and URD - Increase in banks Haploidentical donors more as the procedure gets safer Health care delivery - NMDP approach to increasing SCT availability - availability increases with socio-economic status SUM-WW11_11.ppt

14 100 Trend: Older patients will be increasingly transplanted (data shown ) 80 < 50 years >= 50 years <60 years >=60 years Transplants, % Allogeneic Transplants Autologous Transplants * Transplants for AML, ALL, NHL, Hodgkin Disease, Multiple Myeloma Slide 7 SUM11_5.ppt

15 The limits of success today RELAPSE GVHD INFECTION REGIMEN RELATED MORTALITY SUM-WW11_17.ppt

16 Cell therapy for HSCT the Vision Transplant CD34 cells unmanipulated / T cell depleted engraftment >90% survival Relapse Infection Organ toxicity GVHD DLI, T cells and NK cells Virus specific T cells MSC MSC / Tregs Sources: Allogeneic Marrow Blood Cord blood

17 Cell therapy complexity minimal manipulation minimal cell sorting culture expansion adjuvants selection feeder cells gene insertion Level 1 Level 2 Level 3 Level 4 Marrow UCB G-PBSCT G-Marrow DLI CD34 selection CD4 selection Memory cell selection T reg selection NK selection Selective immunodepletion Virus specific T cells Tumor-specific T cells TIL, MIL NK cells MSC DC CAR cells TCR insertion Suicide genes TGFb resistance Cytokine secreting Frequency of use

18 The long winding road to successful T cell therapy multi-virus specific T cells in the clinic Improved growth conditions tumor specific T cells in the clinic Low volume GMP grade culture systems 2000 Improved APC generation Target antigen identification Incorporating CD4 and CD8 responder Proof of principle Virus specific T cells Leukemia-specific T cells 1995

19 Multivirus -specific CTL Protect against EBV after HSCT 9/40 patients had EBV reactivation 9/9 patients had decrease in EBV viral load with corresponding elevation in EBV-specific CTL detected in PB No antiviral therapy required Diagnosis of PTLD 2 months later Nat Med. 2006;12(10):

20 Making LMP1 and LMP2 T cells to treat Hodgkins disease Adherent PBMC GM-CSF IL-4 IL-1b IL-6 TNF-a PGE-2 LCL LCL LCL PBMC mdc More recently Substituting ad vector for pepmixes IL-15 IL-2 IL-2 LMP-specific Cytotoxic T Lymphocytes (CTL) Bollard et al, JIT 2004, Straathof et al, JI 2005

21 Clinical Responses post LMP-CTL PRE PRE POST POST

22 Clinical Responses post LMP-CTL Relapsed Disease Arm (n=23) No toxicity 12 CR (1 also given Rituximab) (includes 1PR CR) 2 very good partial responses (up to 36 mths) 9 progressive disease (2-8 wks) Median clinical response: 1.5y (range: >6 to >40 mths) Patients with disease at CTL infusion PR CR n =23

23 New York Times, December 9, 2012

24 Progress in the general application of cell therapy Technology Clinical trials CD34 cell products Dendritic cells MSC Gene modified T cells Virus specific T cells NK cells CAR trans T cells Tumor specific T cells T regs Acceptance Commercialization

25 Black Box The future for commercial cell therapy? Apheresis Apheresis lymphocytes monocytes Add growth factors Add peptides Cell product: Virus specific T cells Controlled sterile environment

26 2043 Transplants in 30 years time? Some predictions

27 PREDICTION: APPROX 5% IMPROVED SURVIVAL / DECADE (URD myeloablative SCT shown) PROBABILITY, % (7,251) 41% (2,095) 37% 0 P = YEARS

28 PREDICTION : IMPROVED CURE RATES (HLA-ID Sib SCT for advanced disease shown) 100 PROBABILITY, % (451) 62% (2299) 60% 1995 (2261) 62% %?? YEARS * Not in remission for AML or ALL Blast phase for CML

29 Unpredictables 14,000 12,000 Autologous Related Donor Unrelated Donor 10,000 Transplants 8,000 6,000 4,000 2,000 0 '80 '81 '82 '83 '84 '85 '86 '87 '88 '89 '90 '91 '92 '93 '94 '95 '96 '97 '98 '99 '00 '01 '02 '03'04''05 '06 '07 '08 '09 '10 Slide 3 SUM11_41.ppt

30 Unpredictables: combinations that work Surgery radiation chemotherapy Cell therapy, SCT New agents cytokines Monoclonals Small molecules

31 Unpredictables: new treatments change indications for HSCT another imatinib cell therapy without HSCT gene therapy

32 The future evolution of allogeneic SCT Combination chemotherapy Small molecules Early chemotherapy High dose chemotherapy and marrow rescue Cell therapy Cytokines New drugs

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