OCT 2004 Nietfeld & Verter 1

Statistics of AutologousCord Blood Storage and Use

J.J. Nietfeld, Ph.D.University Medical CenterUtrecht, The Netherlands

Frances Verter, Ph.D.www.ParentsGuideCordBlood.com

OCT 2004 Nietfeld & Verter 2

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 2004 Nietfeld & Verter 3

J.J. Nietfeld, Ph.D., Biography

• Biochemist & Pathobiologist• Associate Professor, UMC Utrecht, The Netherlands• P.I. 2003 ZonMw report: “Desirability and feasability of a Personal Cell Bank”

Netherlands Report proposes 3-way split of storedcord blood: 1- private portion 2- public portion 3- research portion

OCT 2004 Nietfeld & Verter 4

OCT 2004 Nietfeld & Verter 5

ParentsGuideCordBlood.com• Mission Statement: Impartial review of cord blood storage options

How To use website Public Bank ListMedical motives for storage Private Bank ListDiseases Treated by SCT Types of banksResearch Regulations by nationalityNews reports Lab processingEthical & Legal Issues Suggested QuestionnaireCost & Reimbursement Summaries & Prices

OCT 2004 Nietfeld & Verter 6

Website Readership

– Hits from over 70 foreign countries in the past month

– Average monthly hits over past year: 158,933

67,494 = 36.03 % .net 38,656 = 23.51 % .com 38,590 = 21.20 % (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 2004 Nietfeld & Verter 7

Types of Cord Blood Banks

Private bank

Transplantpublic bank

Researchpublic bank

Parent cost $900-$1800 Free Free

Blood owner

Parents Bank Bank

Bank income

Parents Med. Ins. & Sales

Sales &Patents

Blood usage

Parentaldiscretion

Transplants& Research

Research

OCT 2004 Nietfeld & Verter 8

USA Cord Blood Inventory

NUMBER 9/2003 9/2004 + %

Private CB Banks 15 21 40%

Public CB Banks 18 20 11%

# private customers 179,350 > 270,991 55%# public donationsNMDP + NYBC alone

58,65049,000

> 76,14559,325

21%

# private CBT's 55 to date 71 to date 29%

OCT 2004 Nietfeld & Verter 9

Investments in CB Banking

$ 8 M NMDP 2004 infusion to cord blood banking

$ 9 M National Cord Blood Program year 1 funding

$ 75 M CBR investment in new building over 5 years

OCT 2004 Nietfeld & Verter 10

Previous Opinions Against Autologous Cord Blood Banking

• American College of Ob and Gyn (ACOG) 1997; Int J Gyn Ob, 58(2):257-259• American Academy of Pediatrics July 1999; Pediatrics, 104:116-118• 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 2004 Nietfeld & Verter 11

Previously Published Chancesfor Autologous Transplant Use

• 74 : 200,000 (< 20 yrs) F.L. Johnson, 1997; J Ped Hem Onc 19(3): 183-186. (based on statistics of diseases and treatments)

• 1 : 20,000 ( < 20 yrs) G.J. Annas, 1999; NEJM 340: 1521-1524.

(not substantiated)• 1 : 10,000 – 1 : 200,000 (lifetime) R.M.

Kline, April 2001; Sci Amer 4: 30-37. (not substantiated)

OCT 2004 Nietfeld & Verter 12

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 2004 Nietfeld & Verter 13

Probability of an autologous transplant during one’s lifetime

 

P is a function of n and pWhere: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 2004 Nietfeld & Verter 14

Data for North America 2002Transplants (Ref: CIBMTR) Number

Autologous Blood & Marrow 10,500

Allogeneic Blood & Marrow 7,200

Population Number

USA 288,000,000

Canada 31,000,000

Total 319,000,000

Population minus those over 70 290,000,000

OCT 2004 Nietfeld & Verter 15

Autologous Transplant Lifetime Odds: North America 2002

Probability P = 0.25%Usingn = maximum age for treatment = 70p = probability / year of autologous transplant = (# autol. transplants per year) / (population < 70) = (10,500)/(290 million)

OCT 2004 Nietfeld & Verter 16

Autologous Transplant Lifetime Chances up to age 70

Cha

nce

in %

0

0.05

0.1

0.15

0.2

0.25

0.3

NorthAmerica

9 EUcountries

Netherlands

Geographic Area

Chance / life

OCT 2004 Nietfeld & Verter 17

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 2004 Nietfeld & Verter 18

Example: autologous stem cells for Cellular Cardiomyoplasty

ie: stem cell transplantation for cardiac repair.

Indications: Myocardial Infarction,CongestiveHeart Failure

OCT 2004 Nietfeld & Verter 19

OCT 2004 Nietfeld & Verter 20

Cellular Cardiomyoplasty Lifetime Odds: USA 2001

Probability P = 14% Example: USA inpatient PCI up to age n=75Yearly rate p = (571,000/ 264,818,014) = 0.002(Ref: American Heart Association Statistics)

OCT 2004 Nietfeld & Verter 21

Probability per LifetimeAutologous transplant or Diagnosis

OCT 2004 Nietfeld & Verter 22

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 2004 Nietfeld & Verter 23

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)