Report on The Empire State Stem Cell Program

(NYSTEM)
April 2012
AMSNY MEMBER INSTITUTIONS
Albany Medical College
Albert Einstein College of Medicine of Yeshiva University
Columbia University College of Physicians & Surgeons
Hofstra North Shore-LIJ School of Medicine at Hofstra University
Mount Sinai School of Medicine
New York College of Osteopathic Medicine
New York Medical College
New York University School of Medicine
Sophie Davis School of Biomedical Education at City College of New York
State University of New York Downstate Medical Center
State University of New York Upstate Medical University
Stony Brook School of Medicine
Touro College of Osteopathic Medicine
University at Buffalo State University of New York School of Medicine and
Biomedical Sciences
University of Rochester School of Medicine and Dentistry
Weill Cornell Medical College
Associated Medical Schools of New York
1270 Avenue of the Americas, Suite 606
New York, NY 10020
Phone: 212-218-4610 Fax: 212-218-4278
E-mail: info@amsny.org Web: www.amsny.org
The Associated Medical Schools of New York (AMSNY) is a consortium of the 16 public and private
medical schools in New York State. AMSNY works in partnership with its members to improve
health care through education, advocacy and collaboration. AMSNYs focus areas include, but are not
limited to: faculty development, diversity/inclusion of medical students and medical school faculty, and
development of best practices around medical education, educational informatics, and global health. In
addition, AMSNY works with its members to promote research initiatives that aim to improve health
care outcomes.
Front cover photos courtesy of University of Rochester Medical Center (Nancy Ann Oberheim, Takahiro
Takano) and Columbia University College of Physicians and Surgeons (Gordana Vunjak-Novakovic)
2012 Report on the Empire State Stem Cell Program

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Acknowl edgement s

The 2012 Report on t he Empire St at e St em Cell Program is an updat e of t he 2010 report t hat described t he
development and impact of New York St at es st em cell program. This report provides current dat a on t he
number of j obs creat ed and maint ained by NYSTEM f unding at New York St at es medical schools and out lines
recent advances in research t hat are a direct result of t he NYSTEM grant s.

The report was developed wit h t he help of many people. The publicat ion was produced under t he direct ion of
Jo Wiederhorn, Cryst al Mainiero and Samuel Mot t . We owe special t hanks t o t he eff ort s of t he AMSNY
Government Relat ions Commit t ee who helped facilit at e discussions and inf ormat ion gat hering from various
depart ment s wit hin t heir respect ive inst it ut ions. I n addit ion, we are grat eful for t he careful edit ing of Janet
Silver and Jonat han Teyan.

We welcome comment s and suggest ions f or fut ure edit ions of t his report .

Please direct your correspondence t o:
Cryst al Mainiero, Chief Operat ing Off icer
Associat ed Medical Schools of New York ( AMSNY)
cryst almainiero@amsny.org





2012 Report on the Empire State Stem Cell Program


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Tabl e of Cont ent s


I nt roduct ion...Page 3

Measure of NYSTEMs Success....Page 4

Economic I mpact ....Page 5

Job Creat ion...Page 5

Leveraging of NYSTEM Funding.....Page 7

Cost Savings...Page 8

Scient ific I nnovat ion and Medical Advances.......Page 9

St em Cell Research Wor kf orce.....Page 12

Cross-inst it ut ional Collaborat ion...Page 14

Conclusion....Page 17

Key Definit ions...Page 19

Commonly Used Acronyms..Page 21

Addit ional ResourcesPage 22






















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I nt roduct i on

Over t he past several years, New York St at e has experienced a maj or economic downt urn as it st ruggles t o
weat her t he local, nat ional and global economic crises. This t ype of economic sit uat ion can lead t o a pot ent ial
loss of federal aid in t he f oreseeable fut ure. As a result , all st at e-funded programs are at risk. Given New Yorks
current economic climat e, it is import ant t o cont inue t o fund st at e programs t hat provide signif icant and
demonst rable economic and social ret urns.

A prime example is t he Empire St at e St em Cell Program ( NYSTEM) . I n 2007, New York St at e commit t ed $600
million over eleven years t o NYSTEM, making it t he largest government -financed st em cell program in t he
count ry, out side of Californias. The st at es obj ect ive was t o sponsor a st rong research communit y t hat could
invest igat e t he pot ent ial of st em cell science t o alleviat e disease and improve human healt h. Of nearly equal
import ance was t he goal of economic development t he invest ment in research and facilit ies t hat would creat e
j obs, bot h direct ly and indirect ly, and fuel t he local economy. The Empire St at e St em Cell Board was creat ed
and charged wit h making grant s f or basic, applied, t ranslat ional and ot her research and development act ivit ies
t o advance st em cell research t hroughout t he st at e. Aft er t he first year, which j ump-st art ed t he program wit h
$100 million, $50 million was t o be allocat ed t o t he program in subsequent years. This plan was fully
implement ed unt il FY 2010-11 when t he NYSTEM budget was decreased t o $44.8 million.

New Yorks funding commit ment is crit ical t o t he st at es st em cell research and pat ient communit ies given it s
unique nat ure. NYSTEM funds early st age proj ect s t hat have had diff icult y accessing ot her f unding sources like
t he Nat ional I nst it ut es of Healt h ( NI H). NYSTEM also is dist inct ive among ot her research grant s in t hat it
provides funding f or capit al proj ect s and equipment , allowing inst it ut ions t o develop or expand t heir st em cell
research infrast ruct ure. I n 2001, t he federal government severely limit ed federal financing f or embryonic st em
cell research, a move t hat set off int ense compet it ion among st at es t o creat e publicly f unded st em cell
programs. Alt hough t he Obama administ rat ion has expanded t he number of st em cell lines available f or use in
NI H funded research from 21 t o 136 lines
1
, cert ain funding limit at ions st ill remain. The NYSTEM program has
made it possible f or st em cell research t o t hrive in New York St at e.



1
u.S. naLlonal lnsLlLuLes of PealLh. nlP Puman Lmbryonlc SLem Cell 8eglsLry. Avallable aL hLLp://granLs.nlh.gov/sLem_cells/reglsLry/
current.htm. Accessed November 22, 2011.
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Measur e of NYSTEMs Success

Since awarding it s first grant in early 2008, NYSTEM has allocat ed nearly $223 million t hrough a compet it ive,
peer-reviewed grant process. To dat e, import ant progress has been made as scient ist s use NYSTEM funding t o
furt her unlock t he myst eries of human biology and make progress in t reat ment s and develop pot ent ial cures t o
many of t he diseases t hat affect so many Americans. The program has also st imulat ed New York St at e research
inst it ut ions t o make maj or invest ment s in support of st em cell research, which in t urn has improved t heir abilit y
t o win NI H grant s and at t ract privat e sect or and philant hropic funding.

NYSTEMs success can be out lined in t he f ollowing areas:
Economic impact
o Job creat ion
o Leveraging of funds t o obt ain grant s from federal and philant hropic sources
o Cost -savings
Scient ific innovat ion and medical advances
Educat ion and t raining of a fut ure biomedical research workforce
Enhanced cross-inst it ut ional collaborat ion and public/ privat e part nerships
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Economi c I mpact

New Yorks invest ment in st em cell research creat es and sust ains j obs while seeking t o improve t he populat ions
healt h. New Yorks medical schools have been awarded over 62 percent , or $137.5 million, of NYSTEM f unding
t o dat e
2
. The remainder has been awarded t o ot her healt h care and research inst it ut ions including t he
Regenerat ive Research Foundat ion and Cold Spring Harbor Laborat ory. Because of New Yorks funding
commit ment , t he st at es biomedical research and commercial sect ors have grown and New York has achieved a
scient if ic and economic advant age over st at es t hat do not have dedicat ed st em cell programs. Even in st at es
t hat do have programs, NYSTEM has proven t o be a great example of a simple, well run and ef fect ive program.
I n addit ion, t he st at e grant s have result ed in a mult iplier effect , as grant recipient s have been able t o raise
addit ional monies eit her t hrough federal grant s or privat e donat ions t o f urt her enhance t he impact of t he init ial
invest ment .

New Yorks cont inued invest ment in st em cell research will generat e significant benefit s t o t he st at es economy.
NYSTEMs economic impact can be broken down int o t hree different segment s: j ob creat ion, leveraging of funds
and cost -savings.

Job Creat ion
New York St at e is home t o one of t he st rongest biomedical research communit ies in t he world, wit h sixt een
medical schools, and approximat ely 100 t eaching hospit als and ot her t op qualit y research inst it ut ions. Leading
scient ist s and medical professionals from across t he count ry are coming t o New York because t hey are able t o
conduct cut t ing-edge research in t he st at e. I n doing so, t hese scient ist s are oft en bringing wit h t hem t heir
Nat ional I nst it ut es of Healt h ( NI H) grant s and post -doct oral st udent s. Furt hermore, medical schools and
research laborat ories are hiring new researchers t o complement t heir st em cell programs. The growing
research infrast ruct ure brings increased revenue f or research facilit ies and st aff t o t rain new graduat e st udent s,
develop new drug t herapies, and spin of f clinical businesses t hroughout t he region.

As a result of NYSTEM funding received by t he medical schools, approximat ely 404 j obs in st em cell research
labs have been ident if ied as being creat ed and/ or maint ained since 2008.
At t he Albert Einst ein College of Medicine, 12 new researchers have been hired.

2
Although NYS medical schools have been awarded a total of $137.5m in NYSTEM grants, only partial funding has been
received by the medical schools to date.
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At Columbia Universit y Medical Cent er, an est imat ed 100 posit ions have been filled and/ or maint ained
wit h NYSTEM funding.

The Mount Sinai School of Medicine support s up t o 73 research and relat ed j obs t hrough NYSTEM
funding. Wit h cont inued support t hrough NYSTEM and ot her philant hropic sources, Mount Sinai
ant icipat es t hey will double t he number of new j obs creat ed over t he next f ive years.

At New York Medical College, t hree post -doct orat e fellows and t wo new facult y members were hired.

NYSTEM funding t o New York Universit y School of Medicine support s approximat ely 86 FTEs wit hin t he
Kimmel Cent er for St em Cell Biology. I f NYSTEM funding were t o be eliminat ed, seven laborat ories at
NYU would no longer conduct st em cell research and a t ot al of 27 proj ect s would cease.

The Universit y at Buffalo SUNY School of Medicine and Biomedical Sciences est imat es 30 FTEs are
covered t hrough NYSTEM funding. This includes facult y, graduat e st udent s, post -doct orat es, and
t echnicians.

The Universit y of Rochest er est imat es 50 FTEs have been creat ed or maint ained wit h st at e funding
received t o dat e.

Weill Cornell Medical College est imat es 48 FTEs are support ed t hrough NYSTEM grant s.

The dollars t hat New Yorks st em cell program awards f or research provide real and direct economic benef it s t o
t he communit y, including increased employment , growt h opport unit ies for universit ies, medical cent ers, and
local businesses. Moreover, t hese j obs and t he const ruct ion or renovat ion of st em cell lab space has led t o a
mult iplier ef fect creat ing hundreds of addit ional const ruct ion j obs as a result of t he init ial NYSTEM invest ment .
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Leveraging of NYSTEM Grant Funds
Researchers at t he inst it ut ions t hat have received NYSTEM funding have been able t o leverage t heir awards t o
receive addit ional funding from federal and philant hropic sources. This leveraged funding includes bot h direct
follow-up awards and awards f or ot her st em cell research proj ect s by NYSTEM f unded scient ist s.

Some examples include:

I n March of 2010, NYSTEM funding was inst rument al in assist ing Albert Einst ein College of Medicine in
obt aining $10 million from t he Nat ional I nst it ut es of Healt h (NI H) , issued under t he American Recovery
and Reinvest ment Act (ARRA), t o expand it s st em cell research capabilit ies. These f unds are being
used t o creat e new laborat ories in order t o expand it s already subst ant ial cohort of st em cell
invest igat ors.

Columbia Universit y College of Physicians & Surgeons est imat es t hat funding f or st em cell research from
federal sources has increased roughly four t imes since NYSTEM began awarding grant s in 2008, and
wit hin t he same t ime period by about six t imes from ot her privat e/ corporat e sources..

The Mount Sinai School of Medicine was also able t o secure ARRA federal st imulus funding aft er
receiving st at e funding f or a shared facilit ies grant . I n April 2010, t he Nat ional Cent er for Research
Resources, part of t he Nat ional I nst it ut es of Healt h ( NI H), awarded nearly $10 million t o Mount Sinai
Medical Cent er t o support t he expansion, improvement , and consolidat ion of space for laborat ories at
Mount Sinai School of Medicine.

The st at es st em cell invest ment also leverages inst it ut ional support . At St ony Brook Univer sit y Medical
Cent er, NYSTEM funding spurred inst it ut ional support f or renovat ions, facult y cost s and new hires.

At t he Universit y of Rochest er , dat a t hat result ed from NYSTEM funded research at t he Cardiovascular
Research I nst it ut e was crit ical in obt aining a healt h-relat ed research and development grant from NI H
for $1,543,500 in order t o st udy t he role of immune response in vascular dysfunct ion in hypert ension.
Addit ionally, dat a developed from t wo NYSTEM grant s at t he Depart ment of Ort hopedics were
inst rument al in a grant award of $537,320 from t he NI H for st udies on skelet al repair and regenerat ion.
Overall, t he Universit ys st em cell labs have direct cost commit ment s of more t han $80 million.


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At Weill Cornell Medical College, t he Ansar y I nst it ut e t akes a collaborat ive approach t o st em cell
research by bringing t oget her scient ist s fr om acr oss a wide spect rum of biomedical research. The
I nst it ut e was est ablished in 2004 wit h a generous gift f rom Mr. Hushang Ansary. Since t hen, t he
I nst it ut e has garnered approximat ely $26 million in ext ernal funding, including support fr om t he St arr
Foundat ion Tr i- I nst it ut ional St em Cell I nit iat ive and NYSTEM.

I f NYSTEM funding were t o cease, t he risk of losing st em cell researcher s t o ot her inst it ut ions out side of New
York St at e would increase.

Cost -savings
As research advances t o t he st age of successful t herapies, New Yor k is posit ioned t o realize billions of dollar s in
reduced healt h care cost s. I n New Yor k, more t han $94 billion is spent every year on healt h care wit h
t axpayer s assuming $35 billion of t he t ot al cost .
3
A significant port ion is spent t reat ing individuals wit h chr onic
condit ions, many of whom could benefit fr om st em cell research. I n t ot al, t hese diseases account f or 73 percent
of deat hs in New York and 70 percent of t ot al medical cost s. Given t he pot ent ial of st em cell research, New
York could realize significant cost savings in f ut ure year s.


3
See A Scient if ic, Policy and Economic Analysis: New York and St em Cell Research. Univer sit y of Rochest er .
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Sci ent i f i c I nnovat i on and Medi cal Advances

St em cells are self-renewing and have t he abilit y t o develop or generat e int o ot her t ypes of cells. Given t heir
unique capacit y, st em cells are at t he core of development al biology and clinical applicat ions. Scient ist s
speculat e t hat in t he near f ut ure, st em cells may be used t o replace or repair damaged cells and have t he
pot ent ial t o drast ically change t he t reat ment of condit ions like Alzheimers disease, amyot rophic lat eral sclerosis
( i.e. ALS or Lou Gehrigs disease) , burns, cancers, spinal cord inj ury, Parkinsons disease, j uvenile diabet es and
ot her condit ions, t hus bringing hope t o millions of people suffering from a range of debilit at ing diseases. St em
cells are also a powerful research t ool t hat will allow scient ist s t o recreat e and st udy diseases in a way t hat was
never bef ore possible, t hus leading t o bet t er prevent ion st rat egies, t reat ment s, and even cures.

NYSTEM funded st em cell research proj ect s being conduct ed at New York St at es medical schools and research
inst it ut ions include:

At t he Albert Einst ein College of Medicine, researchers are working t o t reat and cure diseases ranging
from cancer and anemia t o heart and liver diseases, obesit y, and brain disorders. Einst ein scient ist s are
doing some highly advanced work on blood st em cell funct ion and different iat ion. Researchers are also
replicat ing liver cells t hat could reduce t he need f or liver t ransplant s using live donors and cadavers,
resources t hat are in chronically short supply. Recognizing t he import ance of st em cell research f or t he
fut ure, Einst ein has opened a new $25 million inst it ut e for st em cell and regenerat ive medicine research
funded by philant hropy.

At Columbia Universit y Medical Cent er, facult y members are developing human mot or neurons from
skin f ibroblast s using a t echnique t hat changes f ibroblast s t o pluripot ent st em-like cells. Pluripot ent cells
have t he abilit y t o become nearly any t ype of cell in t he body. I n t he past year, st em cell work
conduct ed by researchers at t he Columbia St em Cell I nit iat ive has result ed in several import ant
discoveries. Among t hese breakt hroughs is t he ident if icat ion of t ranscript ional net works t hat underlie
t he mesenchymal t ransf ormat ion of brain t umors. I nvest igat ors also demonst rat ed t he abilit y of a novel
st em cell t ype, called XEN, t o cont ribut e t o cells wit hin t he mouse embryo, suggest ing t heir pot ent ial
ut ilit y for new approaches in regenerat ive medicine t hat do not require t he use of embryonic st em cells.


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Through NYSTEM funding, researcher s at t he Mount Sinai School of Medicine discovered a met hod t o
t ransf or m human skin cells int o st em cells and t urned different iat ed human st em cells int o heart cells.
These discoveries will enable deeper under st anding of how heart disease develops and allows f or init ial
t est ing of new t reat ment s on st em cells bef ore being used on human subj ect s. Ot her research
programs f ocus on t ransf or ming skin cells fr om pat ient s wit h schizophrenia, aut ism, and Alzheimers
disease int o brain cells in order t o st udy, in t he laborat ory, t he abnor malit ies in t hese cells t hat lead t o
t hese crippling diseases.

At t he New York Universit y College of Dent ist ry, a dent al research t eam is st udying t he pot ent ial use of
st em cells in regenerat ing facial muscles t o enable people wit h t raumat ic inj ur ies t o speak, eat and
smile nor mally again. The t eam plans t o ext ract a small number of st em cells fr om t he facial muscles of
a pig, gr ow t he cells int o facial muscle on a t issue scaff old t o develop a prost hesis, and t ransplant t he
prost hesis ont o t he pigs face t o repair damaged muscle. Pigs are used for t he st udy because t heir
ext racellular mat r ix is similar t o t hat of humans, and because successfully t ransplant ing t he st em cells in
a large animal model would pave t he way f or clinical t rials on humans.

At New York Universit y School of Medicine, researchers are act ively creat ing t herapies f or brain
degenerat ion. I n addit ion, NYU is ut ilizing NYSTEM f unding on new t arget s f or t reat ing melanoma,
neurological disorder s, and pot ent ial pat hs t hat could lead t o breakt hr oughs in t he t reat ment of cancer ,
diabet es, and spinal cord inj ur ies.

At t he Universit y at Buf falo SUNY School of Medicine and Biomedical Sciences resear ch falls under t he
development al genomics t heme of t he UB2020 St rat egic St rengt h in Molecular Recognit ion in Biological
Syst ems and Bioinfor mat ics ( MRBS/ BI O) . This st rat egic st rengt h is based on four t hemat ic component s:
Molecular Signaling, DNA Replicat ion and Repair, Development al Genomics, and Chemical/ Molecular
Diversit y. Development al Genomics represent s a group of t hirt een invest igat ors wit h int erest s in a
number of biological syst ems including morphogenesis, cell different iat ion and t he cont r ol of cell
division. These int erest s range fr om t he explorat ion of f undament al biological pr ocesses such as early
embr yological development , t hr ough t he bioengineering of specific organs and t issues, int o t he
t herapeut ic applicat ion of st em cells for t he t reat ment of neur odegenerat ive, cardiovascular and ot her
diseases.


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The Universit y of Rochest er, home t o several groundbreaking st em cell research programs in f ields such
as neurological diseases, cancer, bone repair, and musculoskelet al diseases, has been awarded more
t han $18 million in compet it ive grant s t hrough NYSTEM. I n one import ant proj ect , scient ist s at t he
Universit y of Rochest er Medical Cent er have developed a novel met hod t o t arget and eradicat e
leukemia st em cells. This research has t he pot ent ial t o have significant impact on t he t reat ment of
pat ient s wit h specif ic t ypes of leukemia and will be usef ul in t reat ing lymphoma and mult iple myeloma.

Weill Cornell Medical College scient ist s and physicians have recent ly ident ified new cancer st em cells.
This discovery could signal a change in t he direct ion of research on cancer st em cell biology and
st imulat e t he search for new aut hent ic cancer st em cell markers. I n addit ion, Weill Cornell has ut ilized
it s NYSTEM funding t o observe how st em cells replace cells t hat are damaged by disease and t o
underst and t he abilit y of st em cells in bone marrow t o help heal wounds. One of t he most recent
awards went t o Dr. Bet sy Ross who will compare human and mouse embryonic st em cells at various
st ages of brain development t o ident if y t he point at which cert ain cells could be direct ed t o regenerat e
brain t issue t hat has been damaged by diseases such as Alzheimers.

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Educat i on and Tr ai ni ng of t he Fut ure St em Cel l Resear ch Wor kf or ce

Educat ion and t raining are crit ically import ant t o t he fut ure of st em cell research, as it represent s a relat ively
new and expanding f ield t hat requires a highly specif ic knowledge base. NI H has not hist orically direct ed it s
resources t o support st em cell t raining programs, making st at e funding essent ial t o support t he development of
t he fut ure st em cell workf orce. As a result of NYSTEM f unding, several of t he medical schools have developed
t raining programs geared t owards educat ing and t raining undergraduat es, post -doct oral st udent s and
physicians in st em cell research:

Columbia Universit y College of Physicians & Surgeons offers a fellowship t hat aims t o t each t he next
generat ion of scient ist s t he t ools of t he t rade for performing st em cell research. This program involves
hands-on experience in st em cell labs, developing curricula wit h secondary school t eachers and
exposure t o research carried out in t he st em cell field.

New Yor k Universit y School of Medicine received t raining grant s from t he Nat ional Cancer I nst it ut e and
NYSTEM. Six slot s are current ly funded, including t hree post doct oral and t hree graduat e posit ions.

Wit h a grant from NYSTEM, St ony Br ook Universit y Medical Cent er developed a summer undergraduat e
program on st em cell educat ion wit h a cohort of t en undergraduat e st udent s from across t he nat ion in
it s f irst year. St udent s learn about st em cell research, work in labs on a specif ic st em cell proj ect , and
present t heir findings once t he semest er is over.

The Univer sit y of Rochest er School of Medicine and Dent ist r y has developed a series of programs
int ended t o help t rain t he next generat ion of st em cell scient ist s. Using a mult idisciplinary t eam of
facult y, t he Universit y of Rochest er has developed st em cell courses for undergraduat e, non-biology
maj ors. The courses, which are offered at t he Universit y of Rochest er and at Monroe Communit y
College, cover scient ific concept s, as well as et hical, legal and social implicat ions of st em cell science. I n
addit ion, t hey have used a NYSTEM t raining grant t o provide t raining fellowships t o four PhD st udent s
and t wo post doct oral fellows working in six dif ferent st em cell laborat ories.


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Any furt her reductions in funding for the states stem cell program will worsen t he brain drain and diminish
the work t hat has been done t o expand t he stem cell workforce pipeline and make the recruit ment of talented
j unior scientists more difficult as the appearance of research and career advancement opportunities becomes
uncertain or unstable in the state.

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Enhanced Cr oss- i nst i t ut i onal Col l abor at ion

NYSTEM funding support s shared-use facilit ies t o enhance st em cell research by maint aining qualit y cont rol and
laborat ory supplies needed f or st em cell experiment s. NYSTEMs shared facilit y grant s allow inst it ut ions t o
collaborat e wit h one anot her and expand t heir research capabilit ies. Bot h t echnological resources and dat a can
be shared, creat ing a st rong and efficient research communit y. These shared-use facilit ies enable researchers t o
isolat e, derive, and charact erize st em cell lines. Cross-inst it ut ional collaborat ions pave t he way for
advancement s in such indust ries as biot echnology and pharmaceut icals.

Albert Einst ein College of Medicines part nership wit h Mont efiore Medical Cent er offers t he real prospect
of bringing t he product s of Einst ein st em cell research t o pat ient s in need of novel t reat ment
approaches. The Mont efiore-Einst ein Transplant Cent er provides an int erdisciplinary approach t o
pat ient s, adult and pediat ric, in need of organ t ransplant . High-t hroughput screening of human
embryonic st em cells and induced pluripot ent st em cells will enable discovery of fact ors required f or
regenerat ion of new heart , blood, lung, liver, pancreat ic, kidney and brain cells. Not only cell
replacement t reat ment , but also t reat ment of regenerat ive fact ors, will likely represent t he f ut ure in
t reat ing current ly incurable diseases.

For Mount Sinai Medical Cent er, $5 million went t o a shared research facilit y t hat was init ially f unded by
NI H. Mt . Sinai lost federal support f or t he facilit ys operat ions and was t hreat ened wit h closure when
t he NYSTEM funding was awarded. The shared facilit y provides t he opport unit y for hands-on t raining
and development of reagent mat erials. Approximat ely t wo dozen laborat ories operat e wit hin t he facilit y.
Mt . Sinai works in collaborat ion wit h Memorial Sloan Ket t ering and ot her inst it ut ions (t he NY St ruct ural
Biology Cent er at CUNY, for example) . A maj orit y of t heir proj ect s focus on individual, early-st age
research.

New York Universit y School of Medicines Ribonucleic Acid I nt erference Core (RNAi) provides an
int egrat ed, st at e-of-t he-art , high-t hroughput screening facilit y and is open t o bot h int ernal and ext ernal
users. I t has facilit at ed numerous proj ect s t hat would ot herwise not be feasible under t he purview of
individual invest igat ors. RNA int erference is a process wit hin living cells t hat moderat es t he act ivit y of



2012 Report on the Empire State Stem Cell Program


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t heir genes. The equipment cont ained wit hin t he RNAi Core facilit y is not f ound in a t ypical laborat ory,
and t he st aff has t he expert ise and specialized knowledge t o t rain users on operat ion. I n addit ion, t he
Core has developed t ools and acquired soft ware for dat a analysis, pat hway analysis, and dat a
visualizat ion.

Five pr oj ect s are t aking place at t he facilit y as a result of NYSTEM funding, and it is est imat ed t hat at
least seven more will begin wit hin t he next six mont hs. Drugs based on RNA int erference are expect ed
t o be t he next maj or class of human t herapeut ics.

St ony Brook Universit y Medical Cent er is poised t o creat e a mult i-user facilit y t hat will support 25
funded invest igat ors and eight invest igat or s seeking funding, as a single-point - of-ent ry resource t o
broadly pr ovide scient ific and educat ional support in st em cell pr ocessing, gene t ransfer and analysis.

Wit h a NYSTEM planning grant , SUNY Downst at e Medical Cent er helped develop t he SUNY EYE I nst it ut e
t hat int egrat es t he complement ary st rengt hs of t he f our SUNY medical cent ers and SUNY opt omet r y.
The SUNY EYE inst it ut e is a prime example of advancing t he SUNY mission t o increase opport unit ies f or
and support of int er-campus collaborat ion. As a result , t he I nst it ut e received a U54 grant ( an award f or
I nt erdisciplinary Research Consort ia) from t he NI H.

The Universit y at Buffalo St at e Universit y of New York ( SUNY) School of Medicine and Biomedical
Sciences received $3.5 million fr om NYSTEM t o est ablish a West ern New York St em Cell Cult ure and
Analysis Cent er . These funds will be used t o pr omot e and facilit at e research in t he use of mouse and
human embryonic, adult , induced pluripot ent and cancer st em cells; not only at t he Universit y at Buf falo
but at t he part ner inst it ut ions Roswell Par k Cancer I nst it ut e and t he Haupt man-Woodward I nst it ut e.

Wit h funding fr om NYSTEM, t he Universit y of Rochest er School of Medicine and Dent ist ry is const ruct ing
a facilit y t hat meet s current Good Manufact uring Pr ocesses ( cGMP) . cGMP are t he pr ocedures t hat
biot ech companies must follow t o ensure t hat t he pr oduct s t hey pr oduce meet Food and Drug
Administ rat ion ( FDA) st andards. An FDA compliant facilit y is necessary t o pr oduce st em cells t hat are
suit able f or t est ing in humans. Meet ing t hese st andards requires highly specialized facilit ies. The Federal
gover nment does not provide any funding f or t hese facilit ies t herefore New York medical schools have
been using NYSTEM shared facilit y grant s for t his purpose. Current ly, t here are no cGMP manufact uring
facilit ies available f or human st em cell pr oduct s in upst at e New Yor k. This facilit y will be a regional

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resource available t o scient ist s t hroughout upst at e New York and will accelerat e t he clinical applicat ion
of st em cell research. A number of biot echnology companies have also formally expressed an int erest in
using t he facilit y.

Weill Cornell Medical College is part of t he Ansary St em Cell I nst it ut e, which t akes a collaborat ive
approach t o st em cell research by t apping t he expert ise of scient ist s across a range of areas in
biomedical research. The I nst it ut e has garnered approximat ely $26 million in ext ernal funding, including
st em cell support from NYSTEM and t he Tri-I nst it ut ional St em Cell I nit iat ive, which also includes t he
Rockefeller Universit y and Memorial Sloan Ket t ering Cancer Cent er. One NYSTEM grant , for example, is
being used t o est ablish new, and maint ain exist ing, core facilit ies at t he Rockefeller Universit y and Weill
Cornell Medical College.


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Concl usi on

Cut s t o NYSTEM will creat e a negat ive ripple effect on t he economy
Last years budget included $44.8 million for NYSTEM, down $5.2 million from t he originally planned $50 million
per year. Alt hough New York is facing an upcoming def icit , any furt her reduct ion t o New Yorks st em cell
program will creat e a negat ive ripple ef fect on t he st at es economy and halt t he advancement s being made in
medical research. NYSTEM, and t he work t hat is funded t hrough t he program, are crucial component s of New
Yorks economic development st rat egy.

The dollars t hat New Yorks st em cell program sends out int o communit ies provide real and direct economic
benefit s at t he local level, including increased employment ; growt h opport unit ies f or universit ies, medical
cent ers, and local companies; and addit ional economic st imulus f or t he communit y. When st at e f unding is cut ,
communit ies across t he st at e suffer.

There is likewise signif icant risk t hat researchers in New York will be recruit ed away t o inst it ut ions in ot her
st at es such as California, Connect icut , New Jersey, I llinois or Maryland - where t hey would have great er
access t o resources t o support t heir research. The loss of t hese scient ist s would have a negat ive effect on t he
st at e's ent ire research communit y as research grant s, j unior scient ist s, biot ech companies, and vent ure capit al
will similarly migrat e t o st at es t hat are perceived t o be on t he cut t ing edge of biomedical research. Even now,
inst it ut ions in ot her st at es and even ot her count ries are at t empt ing t o lure away New Yorks t op scient ist s
wit h t he promise of public funding and a more welcoming regulat ory climat e f or st em cell research. Any f urt her
reduct ion or eliminat ion t o t he st at es st em cell program will only worsen t he brain drain. New York would not
only lose some of it s t op scient ist s t o inst it ut ions in ot her st at es, but t he fut ure recruit ment of t alent ed j unior
scient ist s would become more difficult as t he appearance of research and career advancement opport unit ies
becomes uncert ain or unst able. A loss of j ust a few of t he st at es st em cell scient ist s could lead t o t he loss of
millions in federal funding.

I f NYSTEM funding is lost , New Yorks medical schools and research inst it ut ions would be f orced t o end or
signif icant ly reduce exist ing research proj ect s. As such, t his would slow t he progress in developing t herapies
t hat have t he pot ent ial t o drast ically change t he t reat ment of condit ions like Alzheimers disease, ALS, burns,
cancers, heart disease, spinal cord inj ury, Parkinsons disease, j uvenile diabet es and ot her condit ions. Moreover,
t he new research facilit ies recent ly const ruct ed would merely become empt y shells. At a t ime when grant

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submissions t o NYSTEM are at a hist orical high due t o declining NI H funding, a loss of t his funding st ream could
be devast at ing, part icularly t o j unior invest igat ors who lack subst ant ial t rack records in obt aining NI H grant s.

As illust rat ed, it is essent ial f or New York St at e t o preserve it s commit ment t o st em cell research in order t o
sust ain one of t he st rongest research communit ies in t he world.


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Key Def i ni t i ons

Appl i ed r esear ch: refers t o scient if ic st udy and research t hat seeks t o solve pract ical problems.

Basi c r esear ch: refers t o st udy and research on pure science t hat is meant t o increase our scient ific
knowledge base.

Bi omedi cal r esear ch: also known as medical research and encompasses basic, applied and t ranslat ional
research.

Br ai n dr ai n: refers t o the migrat ion of highly educat ed people due t o lack of opport unit y, hazards or ot her
ext ernal fact ors.

Cel l s: the basic st ructural and functional unit of all known living organisms.

Cel l di vi si on: t he process by which a parent cell divides int o t wo or more daught er cells.

Devel opment al bi ol ogy: t he st udy of t he process by which organisms grow and develop.

Di f f er ent i at i on: A process that occurs during development by which cells take on their specialized funct ions,
such as t he abilit y of a red blood cell t o carry oxygen or a nerve cell t o send an elect rical signal.

Ext r acel l ul ar : in cell biology, means out side t he cell

Fi br obl ast s: a t ype of cell t hat synt hesizes t he ext racellular mat rix and collagen, t he st ruct ural framework for
animal t issues, and plays a crit ical role in wound healing.

Hi gh- t hr oughput scr eeni ng: a method for scientific experimentat ion especially used in drug discovery and
relevant f ields of biology and chemist ry.

Mot or neur ons: classically applies t o neurons locat ed in t he cent ral nervous syst em and direct ly or indirect ly
cont rols muscles.

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I nduced pl ur i pot ent st em cel l s: a type of pluripotent stem cell artificially derived from a non-pluripotent cell
typically an adult somatic cell

Mesenchymal st em cel l s: cells that can differentiate into a variet y of cell t ypes.

Mor phogenesi s: the biological process that causes an organism to develop its shape. I t is one of three
fundamental aspects of developmental biology, along wit h the control of cell growth and cellular differentiat ion.

Mul t i pl i er ef f ect : in economics, t his refers t o the subsequent impact that direct expendit ures have on the
economy, as money is re-spent or circulated.

Pl ur i pot ent st em cel l s: Also known as embryonic stem cells they have the ability t o become any type of cell
in the body (http:/ / nyscf.org/ about-stem-cells/ stem-cells-101)

Reagent mat er i al s: a substance or compound that is added to a system in order to bring about a chemical
reaction, or added to see if a react ion occurs.

Regener at i on: t he process of renewal, rest oration, and growth.

Ski n f i br obl ast s: cells which are responsible for generating connect ive t issue and allowing t he skin to recover
from inj ury.

St em cel l : Early stage cells that can become any t ype of cell in the body.

Tr ansl at i onal r esear ch: a way of conduct ing scientific research t o make the results of research applicable t o
the population under study.

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Commonl y Used Acr onyms

AMSNY Associat ed Medical Schools of New Yor k
ARRA American Recover y and Reinvest ment Act of 2009
cGMP Good Manufact ur ing Processes
FDA Food and Dr ug Administ rat ion
hESC Human Embr yonic St em Cell
NI H Nat ional I nst it ut es of Healt h
NYSTEM New Yor k St at e St em Cell Science ( refer s t o st at e grant program)







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Addi t i onal Resour ces

New York St at e St em Cell Science Program (ht t p: / / st emcell.ny.gov/ )

St em Cells 101 New York St em Cell Foundat ion ( ht t p: / / nyscf .org/ about - st em-cells/ st em-cells-101)





















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I ncorporat ed in 1967, t he Associat ed Medical
Schools of New York (AMSNY) is a consort ium of
t he sixt een public and privat e medical schools in
New York St at e. Working in part nership wit h it s
members, AMSNYs mission is t o ensure t he st at es
preeminence in t he f ields of medical educat ion,
research and pat ient care.

AMSNY facilit at es collaborat ion and knowledge-
sharing bet ween t he medical schools and
coordinat es an int ensive st at ewide eff ort t o develop
a more diverse medical st udent populat ion in order
t o address physician workf orce issues wit hin t he
st at e. I t s f ocus areas include, but are not limit ed
t o: advocacy, facult y development ,
diversit y/ inclusion of t he medical st udent and
facult y populat ions, and promot ion of research
init iat ives t hat aim t o improve healt h care
out comes.