OCT 2004Nietfeld & Verter1 Statistics of Autologous Cord Blood Storage and Use J.J. Nietfeld, Ph.D. University Medical Center Utrecht, The Netherlands Frances Verter, Ph.D.

OCT 2004Nietfeld & Verter2 Contents Introduction to authors Review of cord blood industry Inventory of stored cord blood in USA Previous opinions and estimates re usage Calculate lifetime probability of autologous transplant Forecast lifetime probability of autologous use for regenerative medicine Implications

OCT 2004Nietfeld & Verter3 J.J. Nietfeld, Ph.D., Biography Biochemist & Pathobiologist Associate Professor, UMC Utrecht, The Netherlands P.I ZonMw report: “Desirability and feasability of a Personal Cell Bank” Netherlands Report proposes 3-way split of stored cord blood: 1- private portion 2- public portion 3- research portion

OCT 2004Nietfeld & Verter4

OCT 2004Nietfeld & Verter5 ParentsGuideCordBlood.com Mission Statement: Impartial review of cord blood storage options How To use websitePublic Bank List Medical motives for storagePrivate Bank List Diseases Treated by SCTTypes of banks ResearchRegulations by nationality News reportsLab processing Ethical & Legal IssuesSuggested Questionnaire Cost & ReimbursementSummaries & Prices

OCT 2004Nietfeld & Verter6 Website Readership –Hits from over 70 foreign countries in the past month –Average monthly hits over past year: 158,933 67,494 = %.net 38,656 = %.com 38,590 = % (numerical IP) 3,837 = 2.85 %.br (Brazil) 4,338 = 2.81 %.edu (USA Edu) 1,930 = 1.24 %.org (Non-Profits) 2,140 = 1.19 %.de (Germany) 1,938 = 1.06 %.ca (Canada) 1,942 = 1.05 %.au (Australia) 2,468 = 1.03 %.mx (Mexico) 14,716 = 8.03 % ALL OTHERS

OCT 2004Nietfeld & Verter7 Types of Cord Blood Banks Private bank Transplant public bank Research public bank Parent cost$900-$1800Free Blood owner ParentsBank Bank income ParentsMed. Ins. & Sales Sales & Patents Blood usage Parental discretion Transplants & Research Research

OCT 2004Nietfeld & Verter8 USA Cord Blood Inventory NUMBER9/20039/2004+ % Private CB Banks152140% Public CB Banks182011% # private customers179,350> 270,99155% # public donations NMDP + NYBC alone 58,650 49,000 > 76,145 59,325 21% # private CBT's55 to date71 to date29%

OCT 2004Nietfeld & Verter9 Investments in CB Banking $ 8 MNMDP 2004 infusion to cord blood banking $ 9 MNational Cord Blood Program year 1 funding $ 75 MCBR investment in new building over 5 years

OCT 2004Nietfeld & Verter10 Previous Opinions Against Autologous Cord Blood Banking American College of Ob and Gyn (ACOG) 1997; Int J Gyn Ob, 58(2): American Academy of Pediatrics July 1999; Pediatrics, 104: Royal College of Ob and Gyn Oct. 2001; Opinion Paper 2 European Group on Ethics in Science and New Technologies March 2004; Opinion No.19

OCT 2004Nietfeld & Verter11 Previously Published Chances for Autologous Transplant Use 74 : 200,000 (< 20 yrs) F.L. Johnson, 1997; J Ped Hem Onc 19(3): (based on statistics of diseases and treatments) 1 : 20,000 ( < 20 yrs) G.J. Annas, 1999; NEJM 340: (not substantiated) 1 : 10,000 – 1 : 200,000 (lifetime) R.M. Kline, April 2001; Sci Amer 4: (not substantiated)

OCT 2004Nietfeld & Verter12 Lifetime storage of Cord Blood: source of autologous stem cells At – 196 °C it takes thousands of years for cosmic radiation to damage cells Cord Blood Transplants on adults have been successful in clinical trials Numerous companies are “expanding” cord blood cell counts Current research focus is speeding cord blood engraftment

OCT 2004Nietfeld & Verter13 Probability of an autologous transplant during one’s lifetime P is a function of n and p Where: n = maximum age for treatment p = probability / year of autologous transplant The probability P depends on a formula which the authors wish to reserve until it is published.

OCT 2004Nietfeld & Verter14 Data for North America 2002 Transplants (Ref: CIBMTR)Number Autologous Blood & Marrow10,500 Allogeneic Blood & Marrow7,200 PopulationNumber USA288,000,000 Canada31,000,000 Total319,000,000 Population minus those over 70290,000,000

OCT 2004Nietfeld & Verter15 Autologous Transplant Lifetime Odds: North America 2002 Probability P = 0.25% Using n = maximum age for treatment = 70 p = probability / year of autologous transplant = (# autol. transplants per year) / (population < 70) = (10,500)/(290 million)

OCT 2004Nietfeld & Verter16 Autologous Transplant Lifetime Chances up to age 70 Chance in %

OCT 2004Nietfeld & Verter17 Forecast of cord blood use for regenerative medicine Given long “shelf life” of cord blood, today’s babies can use autologous cord blood for future regenerative medicine Auto CB carries advantages over Auto BM: –Stem cells have more “plasticity” (under study) –No surgical harvest –Lower cost (depending on storage time) Lifetime probability of diagnosis gives an UPPER LIMIT for treatment

OCT 2004Nietfeld & Verter18 Example: autologous stem cells for Cellular Cardiomyoplasty ie: stem cell transplantation for cardiac repair. Indications: Myocardial Infarction, Congestive Heart Failure

OCT 2004Nietfeld & Verter19

OCT 2004Nietfeld & Verter20 Cellular Cardiomyoplasty Lifetime Odds: USA 2001 Probability P = 14% Example: USA inpatient PCI up to age n=75 Yearly rate p = (571,000/ 264,818,014) = (Ref: American Heart Association Statistics)

OCT 2004Nietfeld & Verter21 Probability per Lifetime Autologous transplantorDiagnosis

OCT 2004Nietfeld & Verter22 Implications Autologous cord blood storage outnumbers public donations and is growing faster. USA National Cord Blood Program will invest less $$ towards growth than private companies. Correct probability of autologous stem cell use is much higher than standard assumptions. Regenerative medicine dramatically increases opportunities for autologous use of stem cells. Private cord blood banking is here to stay.

OCT 2004Nietfeld & Verter23 Future: harness private inventory for public good? Need 150,000 CB for all USA transplants (Ref: Pablo Rubinstein, M.D., NYBC) Private banks will add that much to inventory in less than 2 years. Instead of the USA government funding a program of public banks --- Why not offer parents in private banks a REGISTRY OPTION? (with quality standards)