Millie Ray Harvard Medical School, Mass General Hospital Breakthrough Stem Cell Research Have you ever wondered just what a stem cell really is? Or why your tax dollars fund stem cell research? Can stem cells cure a disease? Do we have the technology to clone ourselves? The rapidly growing field of stem cell biology holds the promise of enormous benefits for medicine and science, and is a platform for capital gains as well as many legal and ethical issues. This talk will address these questions and offer a viewpoint on how recent advents in stem cell biology will affect the future of stem cell research and medicine. Millie (Mridula) is currently a 3rd year graduate student in the Biological and Biomedical Sciences program at Harvard Medical School. She is also an associate producer at an online media company, BioBusiness.tv. Recently (Summer 2009) she helped produce a 10-part series on stem cells, interviewing key opinion leaders in academia, finance, biotech and Pharma/medicine about the basics of stem cell biology and the future of the field. In her undergraduate research, she worked for two years in a leading stem cell lab at MIT. Her current research involves characterizing the interactions of a group of proteins which are essential to development.

1. iPS
cells:
A
BREAKTHROUGH
IN
STEM
CELL
RESEARCH
Presented
by
Mridula
Ray
H+
2010

2. Outline
• Embryonic
Stem
Cells
• iPS
cells
–
turning
Adult
Cells
into
Embryonic-­‐
like
cells
• ImplicaCons
in
research
&
drug
design
• ImplicaCons
in
medicine

3. Embryonic
Stem
Cells
• Self-­‐renewal
Can
divide
indefinitely
into
two
iden/cal
“daughter
cells”
that
have
the
same
proper/es
as
the
original
cell
• Pluripotency
Can
turn
into
ANY
cell
in
the
body

4. Where
do
ES
cells
come
from?
Modified
from
h@p://www.biology-­‐online.com

5. differenCaCon What
can
ES
cells
do?
reprogramming
Courtesy
Biobusiness.tv

6. Induced
Pluripotent
Stem
(iPS)
Cells
• RevoluConary
technique
to
turn
cells
from
adult
Cssue
into
an
ES
cell-­‐like
state
Shinya
Yamanaka
2006
• RelaCvely
easy
to
do,
phenomenon
reproduced
by
many
labs
around
the
world

7. How
are
iPS
cells
made?
THE
TECHNIQUE
DNA
human
DNA
Replace
most
of
a
virus’s
DNA
with
human
DNA. Infect
a
human
cell The
the
human
DNA
(and
the
virus’s
DNA)
The
part
of
the
virus
DNA
that
makes
it
get
integrated
into
the
cell’s
DNA
dangerous
is
removed
THE
EXPERIMENT
• Put
in
4*
genes
involved
in
development
and
cancer
• Conferred
ES
cell
like
properCes

8. Embryonic
Stem
Cells lls!!
S
Ce
And
iP
• Self-­‐renewal
Can
divide
indefinitely
into
two
iden/cal
“daughter
cells”
that
have
the
same
proper/es
as
the
original
cell
• Pluripotency
Can
turn
into
ANY
cell
in
the
body

9. WHAT
CAN
IPS
CELLS
DO?

10. The
near-­‐future
of
scienPfic
discovery
THE
PROBLEM
• Cannot
always
obtain,
idenCfy
or
purify
stem
cells
from
a
diseased
paCent
• Cannot
always
study
diseased
cells
in
a
lab
(limited
lifespan
even
if
you
can)
• May
take
years
to
see
effect
iPS
SOLUTION
• Disease
model
is
improved
• Drug
targets
found
 discovery
improved
• Toxicity
studies
are
improved

11. iPS
cells
are
a
LONG
way
off
from
• Mice
grown
from
iPS
cells
are
more
likely
to
get
cancer
than
natural
babies
• Is
an
iPS
cell
completely
reprogrammed
on
the
molecular
level?
• Quality
standards?
• Making
iPS
cells
takes
a
long
Cme!
• Stem
cells
 increases
risk
of
cancer
• Delivery
of
a
stem
cell
to
the
right
place
may
be
a
challenge!
• Ge^ngthe
right
signals
to
the
stem
cells
is
hard
to
control

12. Autologous
versus
Allogenic
• Autologous
–
from
your
own
body
• Allogenic
–
from
a
donor
Your
immune
system
can
recognize
“self”
from
“non-­‐self”
If
the
donor
does
not
have
similar
enough
geneCc
properCes
as
you,
your
immune
system
will
reject
the
transplanted
organ,
blood,
cells
etc
PaCents
who
have
had
organ
transplants
oden
have
to
take
immuno-­‐suppressive
medicines
for
the
rest
of
their
lives

13. Current
and
PotenPal
Uses
of
Stem
Cells
Current
Use PotenCal
use
with
iPS
• IVF • N/A
• Bone
marrow
transplants • Autologous
• Cord
blood
banking • Not
necessary
• Drug
Screening
&
toxicity
• Personalized
medicine
• Disease
models
• Diabetes • From
actual
diseased
cells
• Cornea
repair
(Australia,
India) • Autologous
• Skin
grads • Autologous
(no
difference)
• Spinal
Cord
injury
(1st
ES
C
FDA
• Autologous
approved
clinical
trial
in
the
US)
• Cardiac
and
bone/carClage
regen. • Autologous
• Full
organ
regeneraCon
challenges

14. THANK
YOU!