Stem Cells:Stem Cells:Scientific and Commercial ChallengesScientific and Commercial Challenges

Scott SternScott SternNorthwestern University and NBERNorthwestern University and NBER

Chicagoland Stem Cell SymposiumChicagoland Stem Cell SymposiumFebruary 2008February 2008

The Challenge of Stem Cell ResearchThe Challenge of Stem Cell Research Stem cell research is likely to be among the most important Stem cell research is likely to be among the most important

areas of scientific research over the next 50 years – we just areas of scientific research over the next 50 years – we just don’t know exactly howdon’t know exactly how

Stem cell research takes place in Pasteur’s Quadrant – where Stem cell research takes place in Pasteur’s Quadrant – where research is simultaneously of scientific and commercial research is simultaneously of scientific and commercial interestinterest

Stem cell research is global – local (or even national) efforts Stem cell research is global – local (or even national) efforts at regulation have had little impact on whether research is at regulation have had little impact on whether research is actually conducted but has had a significant impact on actually conducted but has had a significant impact on wherewhere the research is conducted and whether the research takes the research is conducted and whether the research takes place in an effective institutional environmentplace in an effective institutional environment

Heart Muscle

Vessels

Blood

KidneyBone Skin

Nervous System

Liver

Pancreas

neuronsastrocytesoligodendrocytes

blood cells

cardiomyocytes

liver cells

insulin producing cells

endothelial cells

osteoblasts

While the isolation of stem cells was originally developed in relatively narrow applications, the potential applications are enormous. Despite a decade of work, however, the research is still a highly exploratory phase

based on Martin F. Pera (2005)

The traditional distinction between science and technology The traditional distinction between science and technology fails when knowledge has fails when knowledge has both both basic and applied value, basic and applied value,

with implications for discovery, development and with implications for discovery, development and commercialization.commercialization.

Consideration of Use?Consideration of Use?

Quest forQuest forFundamentalFundamental

Understanding?Understanding?

NONO YESYES

NONOPure Applied Pure Applied

ResearchResearch(Edison)(Edison)

YESYESPure Basic Pure Basic ResearchResearch

(Bohr)(Bohr)

Use-Inspired Use-Inspired Basic Basic

ResearchResearch(Pasteur)(Pasteur)

Similar to many other cutting-edge areas of life sciences Similar to many other cutting-edge areas of life sciences research, stem cell research is conducted in Pasteur’s research, stem cell research is conducted in Pasteur’s

Quadrant, where research is simultaneously of scientific Quadrant, where research is simultaneously of scientific and commercial interestand commercial interest

Over the past several years, despite policy debates over Federal Over the past several years, despite policy debates over Federal funding of basic research for publication purposes, an explosion funding of basic research for publication purposes, an explosion

in in patent applicationspatent applications over various stem cell technologies over various stem cell technologies

Bergman and Graff, NBiotech, 2007

In Pasteur’s Quadrant, we will see fundamental scientific research in both In Pasteur’s Quadrant, we will see fundamental scientific research in both the public and private sector, and both universities and firms will the public and private sector, and both universities and firms will

aggressively protect intellectual property through patents (or even aggressively protect intellectual property through patents (or even secrecy!). More than 40% of all stem cell patent applications are by secrecy!). More than 40% of all stem cell patent applications are by

university researchers university researchers

Bergman and Graff, NBiotech, 2007

The Geography of Stem Cell Research:The Geography of Stem Cell Research:While some countries support embryonic stem cell research, While some countries support embryonic stem cell research,

others – including the United States – have imposed others – including the United States – have imposed complex restrictions on their use (or ability to get funding) complex restrictions on their use (or ability to get funding)

Allowing derivation & Allowing derivation & NTNT

Allowing cell lines Allowing cell lines derivationderivation

BelgiumBelgium China China IsraelIsrael Japan Japan South KoreaSouth Korea SingaporeSingapore (Sweden)(Sweden) United KingdomUnited Kingdom

AustraliaAustralia CanadaCanada Czech RepublicCzech Republic DenmarkDenmark EstoniaEstonia FinlandFinland FranceFrance GreeceGreece

HungaryHungary IranIran LatviaLatvia NetherlandsNetherlands RussiaRussia SloveniaSlovenia SpainSpain TaiwanTaiwan

Research allowed, but Research allowed, but no new derivation, no no new derivation, no NTNT

No explicit permission for No explicit permission for research with hESCresearch with hESC

GermanyGermany AustriaAustria Ireland Ireland Italy Italy Slovak RepublicSlovak Republic

LithuaniaLithuania NorwayNorway PolandPoland Based on

Martin F. Pera (2005)

NT = nuclear transfer

Perhaps in part because of these policy differences Perhaps in part because of these policy differences human embryonic stem cell research publications has human embryonic stem cell research publications has

become increasingly globalizedbecome increasingly globalized

Owen-Smith and McCormick, NBiotech, 2006

Indeed, the emerging international stem cell research “network” seems Indeed, the emerging international stem cell research “network” seems to reflect strategies by researchers to avoid locations or research to reflect strategies by researchers to avoid locations or research

materials hindered by IP restrictions or onerous regulationsmaterials hindered by IP restrictions or onerous regulations

Some ImplicationsSome Implications Stem cells are a “general purpose” research tool of Stem cells are a “general purpose” research tool of

fundamental scientific, commercial and therapeutic interestfundamental scientific, commercial and therapeutic interest• We are unlikely to be able to forecast the precise ways in which We are unlikely to be able to forecast the precise ways in which

these exciting new discoveries will impact clinical practice or the these exciting new discoveries will impact clinical practice or the human experiencehuman experience

• A proactive debate about the ethics of certain practices (and A proactive debate about the ethics of certain practices (and providing effective global institutions and policies) ensures that providing effective global institutions and policies) ensures that the ethical issues are debated in advance of having to make hard the ethical issues are debated in advance of having to make hard choices about alternative research directionschoices about alternative research directions

So far, most policy has been national and narrow – providing So far, most policy has been national and narrow – providing narrowly tailored exceptions for Federal funding that narrowly tailored exceptions for Federal funding that simultaneously undermine the effectiveness of the scientific simultaneously undermine the effectiveness of the scientific research process while having little impact on the actual research process while having little impact on the actual direction and conduct of researchdirection and conduct of research• The United States has begun to cede its early lead in stem cell The United States has begun to cede its early lead in stem cell

research to other countriesresearch to other countries• A great deal of research is now funded by a patchwork of private A great deal of research is now funded by a patchwork of private

or non-profit funders who may actually impose or non-profit funders who may actually impose fewerfewer ethical ethical obligations or requirements on researchersobligations or requirements on researchers