"Cloning & Stem Cell Strategies in Transplantation" 3/6/2006

1. Cloning & stem cell strategies in transplantation
Robert Lanza, MD
VP Medical & Scientific Development
Advanced Cell Technology
Adjunct Professor
Institute for Regenerative Medicine
Wake Forest University School of Medicine

2. Alzheimer’s
Dwarfism
Parkinson ’s
Strokes
Epilepsy
Hemophilia
Kidney failure
Chronic pain
Cancer
Infertility
Burns
AIDS
Muscular dystrophy
ALS
Affective disorders
Macular degeneration
Hypoparathyroidism
Heart disease
Liver failure
Enzymatic defects
Diabetes
Osteoarthritis
Multiple sclerosis
Huntington ’s
Hypocholesterolemia
Rheumatoid
arthritis
Atherosclerosis
Ulcers
Spinal cord
injuries

3. 10
20
30
40
50
60
70
80
90
100
1988
1989
1990
1991
1992
1993
1994
1995
1996
1997
1998
1999
2000
2001
Year
NumberofPatients(inthousands)
Waiting List
Organs Transplanted

6. 2001

7. 2003 [First stem-cell stage SCNT embryos]

9. Lanza et al Science 2000Lanza et al Science 2000

11. 0.0
20.0
40.0
60.0
80.0
0.0 1.0 2.0 3.0 4.0 5.0 6.0
A
.
Telomere fluorescence (in kMESF)
0.0 10.0 20.0 30.0 40.0 50.0 60.0 70.0 80.0
Dupl. 1
Dupl. 2
Exp. 1 Exp. 2
Young cattle
5-10 mos old
Old cattle
> 10 yrs old
Newborn calves
< 2 wks old
Cloned cattle
5-10 mos old
10 20 30 40 50 60 70 10 20 30 40 50 60 700 80
C
y= - 3.62 x + 61.8
R2
= 0.54
B
Years
kMESF
23.1
9.4
1 2 3 4 5 6 7 8 9 10
Mean TRF 18.3 15.2 20.1 18.3 15.2 20.1 14.4 16.4 12.1 16.1
kb

12. 0
20
40
60
80
100
POPULATIONDOUBLINGS
NORMAL FETUS CLONED FETUS

13. •Parkinson’s ($6 billion/yr)
•Stroke ($45 billion/yr)
•Spinal cord injury ($10 billion/yr)
•Epilepsy ($3 billion/yr)
•Alzheimer’s ($100 billion/yr)
•Multiple sclerosis ($10 billion/yr)

16. Production of new hES lines that will be safe for
clinical applications
• Feeder-free system
• Serum-free system
• Elimination of all
animal products,
including sialic acids
• Generated using good
manufacturing
practice (GMP)
A B
C D
E
F
A B
C D
E
F

19. Generation of ES cells using parthenogenesis

21. Retinal Degenerative Diseases
• Age-related macular degeneration (ARMD) alone affects over 30
million people worldwide
• Leading cause of blindness in patients over 60 yrs old in the US
• Degeneration of retinal pigment epithelium (RPE) plays a critica l
role in the pathogenesis of AMRD as well as retinitis pigmentosa
and other retinal degenerative diseases
• Transplanted RPE cells have been shown to rescue host
photoreceptors and attenuate loss of visual function in animals
• Stem cell-derived RPE could be a potentially important source of
tissue for subretinal transplantation in patients

24. WBC colony from cloned stem cells

25. •Cardiovascular disease costs the US $329 billion annually

30. End-stage renal disease will cost US $1 trillion during the coming
decade