Case study II - Stem cells
Part One
JIIP
JOANNEUM RESEARCH POLICIES
Michael Ploder
Angelika Sauer
17th November 2014
17 November 2014
JIIP Symposium 2014
1
STEM CELLS
• Stem cells are literally the mother cells of every tissue.
• The development of stem cell research over the last centuries
opens four major paths of applications
1)
Clinical Stem Cell Treatment:
Reproduction and Transplantation
(Bone marrow/Blood, Skin, Cornea, Cartilage, Fat)
2)
Drug Development and Disease Modelling
(Differentiated disease models and test systems
both for block buster and personalized medicine)
3)
(Re) Programming of Tissue Cells
(Organ Recreation: Pancreas, Neuronal Syst.)
4)
Identification /Treatment of Cancer Stem Cells
(Diagnosis, Therapy)
2
Research Landscape
critical contributions
• The last two decades showed an exponential increase of stem cell research
• The research landscape is (increasingly) global
USA
1988: Hematopoietic
(blood) stem cells from
adult mice are purified and
characterized
USA
1998: The
first human
1992:Stem cells are embryonic
identified in the adult stem cells
human brain
are isolated
CN
1950
1955
1960
1955: First successful
bone marrow
transplant
USA
1965
1970
1975
1980
1985
1981: Embryonic
Stem Cells are
isolated from
mouse
blastocysts
UK, USA
1990
1995
2000
2004: The type of
nerve cell lost in
2014: Retina sheet
Parkinson’s disease made of induced
is produced from
pluripotent stem cells
human embryonic
stem cells
USA
2005
2010
UK JP USA
2015
2006/7: Induced
pluripotent stem cells are
discovered
1996:Dolly the sheep,
the first mammal
to be cloned from
an adult stem cell
UK
JP
2002: Pancreatic cells
derived from mouse
embryonic stem cells cure
diabetes in mice
CN
2013: neural
organoid
generated
from hPSC
AT
57
196
477
Total costs:
Avera
ge dura tion (months ):
55
649,4
34
1105,4
44
OF FP-ACTIVITIES:
Average
(m€):
0,9
SUMMARY
OF FP-ACTIVITIES:
Pa
rtici paproject
tionsSUMMARY
: size
303
SUMMARY
OF FP-ACTIVITIES:
2,2
1277
1,9
1627
EC contribution
companies:
Countri
es i nvolto
ved:
9,1%
38
16,1%
41
NUMBERS
Stem
Stem
cellscells
Stem cells
Projects :
30,7%
24
38
303303
53,7
24
24 425,8
BUDGET
EC contribution
(m€):
EC contribution
(m€):
BUDGET
39,3%
53,7
55
2,3%
0,9
0,9
27,7%
--
Average
project
(m€):
Average
size size
(m€):
Uni vers i ti esproject
:
39,3%
INSTRUMENTS
Companies:
PARTICIPANTS
PARTICIPANTS
RTDvers
a ctions
84,5%
Uni
i ties::
39,3%
EC contribution
to companies:
Rescontribution
ea rch org:
30,7%
EC
to companies:
Compa ni es :
2,3%
RTDea
knowl
edge traUni
nsvefer:
14,0%
Universities:
Res
rch org:
30,7%
Others
: rs i ti e s :
27,7%
Research
Adoption a nd i nnova
0,0%
Re
s etion:
a rch
org: org:
RTD a cti ons :
84,5%
Compa ni es :
2,3%
Companies:
RTD knowlni
edge
tra ns fer:
14,0%
Compa
e
s
:
Actions to s upportAdopti
poloni cy:
1,5%
a nd i nnova ti on:
0,0%
Others:
Othe
Others
27,7%
Acti onsrsto: s upport pol i cy:
1,5%
N/A :
0,0%
N/A
0,0%
2,3%
COUNTRIES
INSTRUMENTS
RTD a ctiRTD
onsactions:
:
RTD knowledge transfer:FR
RTD knowl e dge tra ns fe84,5%
r:FR
RTD a ctions :
UK
Top-5 participating
countries
Adoption
and innovation:
Adopti
on a nd i nnova ti on:IT
(participation activity)
UK
to support policy:
RTD knowl edge traActi
nsons
fer:Actions
14,0%
to s upport pol i cy:DE
Top-5 participating countries
ES
N/A
N/A tion:
IT
Adoption a nd i nnova
0,0%
INSTRUMENTS
477
Pa
rti ciespa
ti ons
Countri
iParticipations:
nvol
ved: :
Countri
eCountries
s (m€):
i nvol ve
d:
involved:
EC contribution
54Mio
196
Ave
ge
dura
tiduration
on (months
):
Average
(months):
Pa rtira
ci pa
ti ons
:
303
Average project size (m€):
Others
:
EC
contribution
to companies:
Total
costs:
Total
EC contribution
to costs:
companies:
BUDGET
57
49,0%
425,8
426Mio
Avera ge dura ti on (months ):
Compa niproject
es : sizeTotal
Average
(m€):
costs:
PARTICIPANTS
24
57 57
34
55 55
1277
Proje cts :Projects:
NUMBERS
Res eacosts:
rch org:
Total
NUMBERS
Unicontribution
vers i ties : (m€):
EC
EC
Contribution
in €:
(participation activity)
DE
Actions to s upport pol i cy:
1,5%
Top-5 participating countries
Top-5 participating
countries
ES
(participation activity)
N/A
0,0%
(participation activity)
COUNTRIES
PARTICIPANTS
INSTRUMENTS
INSTRUMENTS
COUNTRIES
•
EU-FP has funded Stem Cell
Research since the 90‘
Significant increase in
FP 6 and FP7
COUNTRIES
•
PARTICIPANTS
BUDGET
FP Stem Cell Research
COUNTRIES
NUMBER
BUDGET
Projects :
Top-5 participating countries
(participation activity)
FR
UK
IT
DE
ES
19644
196
477
341627
34
44
1277
1627
38
41
425,8
890,2
33,0%
649,4
127741
38890,2
12,5%
2,2
1105,4
649,4
53,7
53,7
425,8
2,2
- 9,1%
1,9
5,3%
9,1%
649,4
16,1%
0,90,9
49,0%
2,2
48,5%
- 33,0%
12,5%
39,3%
39,3%
5,3%
30,7%
30,7%
77,2%
2,3%
21,2%
2,3%
0,4%
27,7%
27,7%
1,2%
0,0%
84,5%
84,5%
14,0%DE
14,0%
UK
0,0%
FR
0,0%
1,5% IT
1,5%
0,0% ES
0,0%
FR
FR
UK
UK
IT
IT
DE
DE
ES
ES
649,4 1105,4
2,2
77,2%
49,0%
9,1%
28,0% 9,1%
20,5%
21,2%
49,0% 49,0%
33,0%
3,0%
33,0% 33,0%
0,4%
83,0%
12,5%
12,5%
12,5% 12,5%
1,2%
3,0%
5,3%
5,3%
1,4% 5,3%
0,0%
0,0%
12,5%
77,2%
UK
21,2%
DE
0,4%
FR
1,2%IT
0,0%ES
77,2%
DE
UK
21,2%
FR
0,4%
DE
IT
1,2%
UK
FR
ES
0,0%
IT
ES
DE
UK
FR
IT
ES
48,5%
890,2
890Mio
477
44
28,0%
1105,4
1627
41
20,5%
1,9
890,2
3,0%
16,1%
1105,4
1,9
16,1%
48,5%
28,0%
20,5%
3,0%
77,2% 83,0%
12,5%
21,2%
3,0%
0,4%
1,4%
1,2%
0,0%
0,0%
UK
DE
DE
UK
FR
FR
IT
IT
ES
ES
1,9
83,0%
48,5%
16,1%
12,5%
48,5%
28,0%
3,0%
28,0%
20,5%
20,5%
1,4%
3,0%
3,0%
0,0%
20,5%
83,0%
12,5%
83,0%
UK
3,0%
DE
12,5%
1,4%
0,0%
FR
3,0%
ITUK
1,4%
DE
ESFR
0,0%
IT
UKES
DE
FR
IT
ES
(Currently)
4
Research Landscape actors
Hospitals
Clinical
Laboratories
Development
of laboratory
equipment
Equipment,
Materials
Suppliers
Pharma
Expansion,
differentiation
of celltypes,
disease
modells
Clinical Stem
Cell Treatment
Biobanks,
Storage of
Patient
Samples
Medical
Faculties/
Universities
Life
Sciences
PROs
(Re) Programming
of Tissue Cells
(Regenerative Medicine)
Equipment
Suppliers
Developing
Testing
Disease
Modelling
(Drug development)
Biobanks,
Storage of
Patient
Samples
Hospitals
Clinical
Laboratories
Tissue
collection
Equipment,
Materials
Suppliers
Tissue
collection
Spin offs
Biotech
Start ups
Development of
Diagnostics and
Therapeutics (VC)
Cancer
Cancer
Treatment
Treatment
(Personalised
(Personalized
medicine)
medicine)
Pharma
Hospitals
Clinical
Laboratories
Clinical tests
5
Market development:
•
Many stem cell treatments are still far from market
• Stem cell treatments can save expenses of the clinical health care system
and create jobs mainly in academic institutions
MARKET
READINESS:
since Mid 90‘
Clinical Stem Cell Treatment:
Reproduction and Transplantation
(Skin, Bone marrow, Cornea, Fat Tissue)
Drug Development and Disease Modelling
(Test Systems)
Identification / Treatment of Cancer Stem Cells
(Diagnosis, Therapy)
(Re) Programming of Tissue Cells
(Organ Recreation: Pancreas, Neuronal Syst.)
MARKET
PLAYERS:
CLINICAL
LABORATORIES
LAB-EQUIPMENT
SUPPLIERS
0 to 7 years
7 to 15 years
15 to 40 years
PHARMACEUTICAL
FIRMS
6
Regulation as competing elements
• Development of stem cell research and innovations has been accompanied by an
increasing number of different national legislative regulative measures
concerning embryonic stem cells
– In Germany 2002 the “Stem Cell Act” defined the derivation of human
embryonic stem cell (hESC) lines as a criminal act
– In UK the Parliament amended the “Human and Embryology Act 1990” to allow
research on human embryos for specific purposes.
– In Italy clear restrictions of research using embryos are set by the “Law 40” in 2004
– In 2011 the European Court of Justice rules that technologies or inventions based on
hESC cannot be patented in the EU (challenge for IPRs and private equity)
• 2007 stem cell therapies are classified as “Advanced Therapy Medicinal Products”
by the European Commission Regulation 1394/2007 (ATMP)
– definition (gene-therapy, somatic-cell therapy, tissue engineering, combined advancedtherapy), authorization
• 2008 Establishment of the “Committee for Advanced Therapies” (CAT)
located at the European Medical Agency (EMA)
– No medicines based on pluripotent stem cells have received marketing authorization in
the EU
7
Lessons which might be learned
from the stem cell case
•
Stem cell research and innovation is and will remain a global effort,
Biological systems are significantly more complex than engineering based technologies
•
However local conditions and regulatory and legal frame highly relevant
– Institutional Thickness
– Regulatory coordination (hESC, preclinical models etc.)
– Public awareness
•
•
Stem cell innovation vastly in its infancy
Stem cells value chain in public domain to a large extent, (even in the long term)
with certain potentials for private economic growth.
– high societal and economical impact via clinical treatment, health insurance systems
– willingness to pay (private) vs. challenges for reimbursement (public)
•
FPs could contribute to a significant extent:
–
–
–
–
Building large consortia (open innovation) including third countries (INCO)
Attracting private firms
Filling the funding gap between basic research and applied research/application
Building ground-work for regulatory development, good practice and co-ordination
8