FROM CORD BANK TO CORDPHARM

Transcription

1 FROM CORD BANK TO CORDPHARM ANTHONY NOLAN CORD BLOOD PROGRAMME Expanding access Improving outcomes Opening ways

2 QuickTime and a TIFF (Uncompressed) decompressor

3 THE ANTHONY NOLAN TRUST The Anthony Nolan Research Institute University College London, England

4 THE ANTHONY NOLAN TRUST Development of the Anthony Nolan Register Anthony Nolan born suffering from Wiskott-Aldrich Syndrome Donor recruitment commenced to establish a panel of donors Anthony died without receiving a transplant Number of donor on Reg = 4000, Number of donors provided for transplant = 8020 Jan/2009

5 QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor >12,000,000 donors QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor

6 ADULT MODEL: STAIR Allomandatory Jump Sibling MUD Perfect Match Adult Mismatch Quién habla de victoria.? Allotherapy Sobre ponerse es todo Rainer Maria Rilke.??

7 time a l t e r n a t i v e DRUG / AUTO THERAPY alternatives >30 W 33% 33% Diagnostic 5 W 14 W Allo Sib Allo Sib Tx 19 W 25% unfit 25% no donor UD KING S COLLEGE HOSPITAL NHS TRUST Exploratory No Donor AN Donor Overseas No Tx 21 W Survey Alloprocedures 17% 25% 34% 24% 11 W 60% 10 W Tx UD Tx 26 W 33% 14 W 42% 10 W Tx UD Tx 29 W

8 Diagnostic Allo Scoring system Urgency HLA yes CB NOD2 MUD MUD Search no CB Search CB CB Search CB Tx 11 weeks time HLA-DP mm MUD Tx >20 weeks CB Tx Objectives: DECREASING TIME INCREASING EFFECTIVENESS

9 The Large Bone Marrow Registers Name / Total donor / % ABDR typed NMDP 5,060,482 (86%) Germany 3,355,359 (63%) China Anthony Nolan 395,135 >769,372 (81%) Brazil >554,371 Israel E.M. 399,342 (70%) Italy 326,638 (67%) Japan 320,416 (100%) BBMR 310,541 (94%) Taiwan 277,936 (87%) From BMDW 23/12/08

10 Total number of HPC donations WMDA report 2007

11 Unrelated Donor Registers in the UK ANT (1974) - Anthony Nolan Trust 395,135 BBMR (1987) - British Bone Marrow Registry 310, ,974 UK donors WBMDR (1989)- Welsh Bone Marrow Donor Registry 54,298 BMDW 23/12/08

12 UK Activity new patients referred for searches 704 had their search extended internationally by end of transplants from ANT donors to patients referred from UK centres 75 transplants from BBMR donors and 22 from WBMDR donors to patients referred from UK centres 318 transplants from an international donor/cord unit to UK patients (241 adult 77 CB) 32 UK transplant centres performed at least one unrelated donor transplant (range 1-40)

13 UK Activity new patients referred for searches 857 had their search extended internationally by end of transplants from ANT donors to patients referred from UK centres 171 transplants from ANT donors to patients overseas 359 transplants from an international donor/cord unit to UK patients (265 adult 91 CB) 33 UK transplant centres performed at least one unrelated donor transplant (range 1-19)

14 Source of imported haematopoietic stem cells for UK patients in Total GERMANY USA ITALY AUSTRALIA CANADA CYPRUS FRANCE ISRAEL TAIWAN FINLAND NORWAY AUSTRIA SWITZERLAND SLOVENIA SWEDEN EIRE POLAND PORTUGAL SPAIN CHINA CORD BLOOD

15 The plan To increase the number of donations provided to UK and international patients such that 1000 patients will be treated with potential life-saving haematopoietic progenitor cells by 2013 and to increase the size of our Register to 450,000 donors

16 Donor Recruitment Predicted growth of The Anthony Nolan Trust Register Size of register No of donors deleted No of donors to be recruited Overall size of register year % increase p.a.

17 Number of donations facilitated the future No. of donations facilitated by The Anthony Nolan Trust - Future Estimates % 6% 7% 7% 7% 7% 7% est 2008 est 2009 est 2010 est 2011 est 2012 est 2013 Calendar Year

18 HLA antigens and alleles

19 Number of HLA alleles, January 2009 HLA-A HLA-B HLA-C 733(24) 1115(49) 392(9) HLA-E HLA-F HLA-G HLA-DRA HLA-DRB HLA-DQA1 HLA-DQB1 HLA-DPA1 HLA-DPB (20) (7) HLA-DMA HLA-DMB HLA-DOA HLA-DOB MICA MICB TAP1 TAP

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21 QuickTime and a TIFF (LZW) decompressor

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23 Low resolution HLA-A*02

24 Medium resolution HLA-A*0201/09/43N/66/78/88/89/90/91+

25 High resolution HLA-A*0201/0209

26 Allele level resolution HLA-A*

27 B Clinic Research aapc NK N Ste m Cells Co rdpharm Operations Department GIAS Cord Blood Services Adult Blood and Marrow Registry Cord Blood Bank Immunogenetics and Informatics Group ANRI Cellular Immunotherapy Group Immunogenetics ioinformatics aïve T Cells

28 Allo-option non Drug therapy yes HLA typing non Allomandatory non Drug therapy yes yes Sibling 5/6 Inclusion at AN MUD³ CBT GIAS score CBT>MUD MUD 9/10 CBT 4/6 Note: If a patient need a transplant in a defined time (urgencies), the donor available at this moment will be used

29 Scoring System: Proposal 1) Expected outcome of search: Individual frequencies Pair frequencies: DR-DQ, B-C Haplotype frequencies 2) Selection between 2-donors: Donor MUD CBT MAJOR HLA compatibility HLA compatibility CMV donor NC/CD34 dose Age donor Need for DLI MINOR Gender CMV recipient Previous pregnancies Fact accredited bank P. Blood vs BM Duration storage

30 The Anthony Nolan Cord Blood Programme Register Cell Pharmacy Cord Blood Bank Cord Blood Pharm

31 COST-BENEFIT QuickTime and a TIFF (Uncompressed) decompressor

32 BONE MARROW DONOR REGISTERS: EFFICIENCY QuickTime and a TIFF (Uncompressed) decompressor EFFICIENCY/YEAR ~ 1/1000 QuickTime and a TIFF (LZW) decompressor DONOR RATIO % QuickTime and a TIFF (LZW) decompressor RECIPIENT Cost: Inventory (150): 57,000,000, Transplant (4000): 14,250

33 QuickTime and a TIFF (Uncompressed) decompressor EFFICIENCY/YEAR ~ 1/1666 QuickTime and a TIFF (Uncompressed) decompressor Cost: Inventory(150): 679,000,000, Transplant (25,000): 27,180

34 EFFICIENCY: ~1/100 QuickTime and a TIFF (Uncompressed) decompressor Cost: Inventory (1,500): 206,000,000, Transplant: 37,784

35 Maintenance Cost UNRELATED DONOR AVAILABILITY Building phase (charitable) Cord= 50,000 ~ 75,000,000 MUD= 500,000 ~ 75,000,000 PROBABILITY e4 8&8 6&6 5&6 4&6 0 1,0E+07 1,0E+06 1,0E+05 1,0E+04 1,0E+03 1,0E+02 1,0E+01 1,0E+00 DONORS REGISTERED CB OPERON 75% 4/6 45% 5/ Maintenance phase Blood: needs 5000 ~ donors 5000/100,000 (500,000 )= cost 100 MUD: needs 3 but supply 1 ~ donors 100/100,000 (15,000 ) = cost 15,000 Cord: needs 3 ~ donors 3/100,000 (4,500 ) = cost 1, Blood Cord MUD Phase

36 Impact of cell dose and HLA Match on survival. Data presented by Dr Pablo Rubinstein at the 5 th International Umbilical Cord Blood Symposium held in Los Angeles in May Survival 5/6 Match 4/6 Match 5/6 match 4/6 match Cell Dose ( 10 7 /kg) 70% >10 50% % < <2.5 time after transplant

37 % Probability Of Patient Match Sample 2000 Patients Match 4 of Match 5 of Match 6 of Donor Sample Size

38 ANTHONY NOLAN CORD BLOOD PROGRAMME CordBank Cord Blood Bio-resource Donor Registry Cord Blood Transplantation Research Bank Stem Cell Biology Transplantation Immunology CordPharm Medicinal By-Products Cellular Stem and Immune Cells Non-Cellular RBC, Plasma, Platelets TheraCord New Therapies Haematology Adoptive Cell Therapy Non-Haematology Regenerative Medicine Tolerance

39 QuickTime and a TIFF (Uncompressed) decompressor QMP QuickTime and a TIFF (Uncompressed) decompressor Collection Storage Intelligent labeling SOP Register ID (confidentiality) RELEVANT DATA (critical steps) seal ELECTRONIC RECORDS transport ANCB FACTORY QC clinical data QuickTime and a TIFF (Uncompressed) decompressor Recep Proces Cryopres LT Storage freezer ship log Transport Directive 2004/23/EC and its technical requirements HTA FACT-NETCORD Grade C CD34/CFU IDM/ MICROBIOLOGY BLOOD TESTS QuickTime and a TIFF (Uncompressed) decompressor Clinical analysis

40 NTU Service Provider contract Building Security Maintenance Generall cleaning IT support Clean Modules Ltd Service Provider contract Maintenance COURIER Service provider Distribution KINGSCORD TPA Procurement contract SLA CB Human applications Testing Processing Storage Distribution ANCTC-NTU Cord Blood Research Research cord blood bank London LABS ANT Central Support IT H&S HRs Fundraising Marketting Operations & Registry Distribution Import/Export Financial contract contract contract Waste Company Service Provider BIOSAFE Service Provider CRYOSERVICE Service Provider Testing contract contract BD Flow Service Provider Testing BD BacTec Service Provider Other suppliers: Coulter, General Equipment, GMP-cleaning, Micronclean, others

41 Anthony Nolan Cord Blood Programme BANKING Automation Graft QualificationAN QuickTime and a TIFF (Uncompressed) decompressor COLLECTION Ethical Logistics Hospital CBB FACTORY CB RECRUITMENT AN REGISTER CB TRANSPLANT. DONOR SELECTION GIAS Search algorithm TRANSPLANTATION Gold standard: conditioning, follow-up, engraftment, chimerism Immune reconstitution KingsCord QuickTime and a TIFF (Uncompressed) decompressor CORD BLOOD BIOBANK bedside Multiple Donors: engraftment and immunotherapy Multi-donor Tx (pooling) Selection/Expansion New products: CD34/CD133, Naïve T regs, USSC/MSC, CELL THERAPY Neural ANCTC bench Associated groups Automation Data Management BIOINFORMATICS Cardiovascular Liver Basic Biology Orthopaedics

42 Cord Blood Bank 10,000 clinical units 10,000 Research cord units Cellular Immunotherapy Group CordPharm Stem cells for Tolerance induction T reg Stem cells for Cardiovascular intervention aapc for cord blood cell manipulation NK, Naive Investigating Engraftment of stem cells

43 ANCBB GIAS Wharton Club Tolerance Cord Blood Research Programme NIHR Adaptive Innate aapc Stem BMT Immuno Immunogenetics Genetics Programme BioInformatics Oxford Cardiovascular UCL Engraftment CIH ALLOSTEM 1 AND 2

44 As a SERVICE Biopharmaceutical perspective Stem cell therapy Cell lines Regenerative Medicine Health Investment Affordable commodity for a new stem cell biomedical industry

45 THE ANTHONY NOLAN TRUST Organizational Chart Mr SM Dyson Chairman Mr T Farnfield Finance & Admin. Director Mr D Knights Marketing & Comm. Director Mr G. Parkes Fundraising Director BOD Trustees Mrs H. Braund Chief Executive P. Makoni Operations Director Dr S Querol Cord Blood Bank Director Prof JA Madrigal Research & Scientific Director Research Institute Anthony Nolan Joint Scientific and Medical Advisory Committee Prof J Goldman Medical Director

46 ANTHONY NOLAN CORD BLOOD BANK (ANCBB) ANT BOD Corporate License Holder Advisory Committees (AC) ANCBB-AC Chair: Prof Madrigal ANCBB-MANAGEMENT Dr Steve McEwan, Rep License Holder Prof Goldman, Prof Madrigal, Dr Little ANT Central Services CU-AC Chair: Prof Mufti KingsCord Third Party Agreement Head-Cord Blood Services Dr Sergio Querol, Designated Individual Quality Supervisor IT Supervisor H&S Supervisor CB Provision and Transplantation Distribution KINGSCORD-MANAGEMENT KCH representative: Prof Mufti Medical Officer: Dr Dennes Operations: Katie, Winsome, Frank CB Bank & Lab Manager Anthony Nolan Cell Therapy Centre, NTU Collection Unit (CU) Supervisor Ms Terie Duffy, Person Designated Trained KCH Staff Bank Supervisor Lab Supervisor CB Bank & Lab Technicians ANT Round Table Labs Procurement Testing, Processing, Storing Organisational Chart (MSP-R-5, v3, 10/6/08)

47 Liver ANTU-NCH ccbbb: core Cord Blood bio Bank as a translational research model for cell therapy Creating a new scientific environment Heart / Neural Lung Bench ccbbb bedside Kidney Basic Science KingsCord GIAS

48 ANTHONY NOLAN CORD BLOOD BANK QuickTime and a TIFF (Uncompressed) decompressor Robert Davy, Roger Horton East Midlands Development Agency Laura Fry, Daniel Gibson, Salmah Mahmood Susana Garcia Gomez Hazel Forde, Kamal Khan, Peter Chenery Richard Duggleby, James Devitt Daniel Figueroa, Lia Zambetti, Mehri Daryouzeh Prof Tony Dodi Dr Steve Marsh, Dr Bronwen Shaw Dr Ann Margaret Little, Dr Steve McEwan Prof Robert Rees Terie Duffy, Maria Tarrio Katie Yannouzis Dr William Dennes Winsome Okeke Dr Antonio Pagliuca Prof Ghulam Mufti QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor Prof John Goldman Dr Sergio Querol Prof Alejandro Madrigal Prof Nigel Russell Dr Joan Garcia QuickTime and a TIFF (Uncompressed) decompressor QuickTime and a TIFF (Uncompressed) decompressor