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In Vivo Differentiation of Stem Cell and Their Progeny Robert Lanza, MD VP Medical & Scientific Development Advanced Cell Technology Adjunct Professor Institute of Regenerative Medicine Wake Forest University School of Medicine

IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

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11/8/2005

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Page 1: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

In Vivo Differentiation of Stem Cell and Their Progeny

Robert Lanza, MDVP Medical & Scientific Development

Advanced Cell Technology

Adjunct Professor

Institute of Regenerative Medicine

Wake Forest University School of Medicine

Page 2: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

CAUTIONARY STATEMENT

CONCERNING FORWARD-LOOKING STATEMENTS

This presentation contains “forward-looking statements” as defined under the

federal securities laws. Actual results could vary materially. Factors that

could cause actual results to vary materially are described in our filings with

the Securities and Exchange Commission. You should pay particular attention

to the “risk factors” contained in documents we file from time to time with the

Securities and Exchange Commission. The risks identified therein, as well as

others not identified by the Company, could cause the Company’s actual

results to differ materially from those expressed in any forward-looking

statements.

Page 3: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

Alzheimer’s

Dwarfism

Parkinson’s

Strokes

Epilepsy

Hemophilia

Kidney failure

Chronic pain

CancerInfertility

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

Page 4: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

10

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1988

1989

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1991

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1998

Year

Nu

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ati

ents

(in

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san

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Waiting List

Organs Transplanted

Page 5: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

Stem cells are smart

• In vitro: Without the right environmental clues they don’t know whatwe want them to do. Left to their own devices in vitro, theydifferentiate into a hodgepodge of cell types – neurons, heating heartcells, ciliated epithelium, and even miniature “eyeballs”

• In vivo: Stem cells home towards damaged tissue where they caninitiate tissue regeneration. We need a better grasp of the roles playedby such chemical signals as insulinlike growth factor (IGF-1), whichmay also take part in causing local cells to revert to a multipotent stateand begin differentiating into the required tissue types. Thisphenomena (epimorphic regeneration) underlies the ability of newts &zebrafish to regrow limbs and organs.

Page 6: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

•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)

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Generation of ES cells using parthenogenesis

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WBC colony from cloned stem cells

Page 17: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

•Cardiovascular disease costs the US $329 billion annually

Page 18: IBC Life Sciences "Stem Cell Research Challenges" Conference Presentation

~ Biodegradable scaffolds

~ Self-assembly

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

decade

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A few last thoughts

• Moving into the clinic – perhaps the damaged tissue area can do part

of the work for us

• More basic research – we need to better understand the in vivo factors

that control stem cell fate and repair

• Regenerative medicine’s ideal – to find a means to cause controlled

de- and trans- differentiation of cells in adult tissue. Will it ever be

possible to generate an environment that replicates the ability of newts

and zebra fish to regenerate tissue, to regrow entire limbs and organs?