hi I'm Amy Adams welcome to the
California stem cell agencies ask the
expert series we asked you to submit
questions through our blog Facebook and
Twitter about stem cell research and
Alzheimer's disease many of you
submitted questions and today dr.
Lawrence Goldstein of the University of
California San Diego is going to answer
some of them for us let's go in why do
you study this disease you know
originally I started studying it because
it seemed interesting we we work on
brain cells and the behavior of brain
cells and as we started to develop new
ideas about what was going on with
Alzheimer's disease my mother developed
the disease and I you know I went
through it personally I don't want to
make a big deal out of it but it was bad
I mean it was just a terrible thing to
go through it's hard on my brothers it
was hard on me it was very hard on her
and to be honest I just got angry about
it you know personally and so I've sort
of resolved to do the best I can to
hopefully develop something that will be
meaningful in the lives of people who
developed this terrible disorder we've
had a lot of people ask why don't we
just inject stem cells directly into the
brain to treat Alzheimer's or stroke or
ALS or autism or a number of other
diseases well the several answers to
that question one is if we're going to
inject stem cells into somebody's brain
first of all we better be sure that
those stem cells are not going to do
something bad that they're going to be
safe you know so we to do that first
we'd have to have a safe prep of stem
cells that we knew would not do
something terrible and then second we'd
have to have some evidence that they
could repopulate the brain in some
normal way and that's harder than it
sounds because ordinarily when the brain
is built it's when you're a fetus and
there are all sorts of signals that say
how to build the brain that aren't there
when you're an adult so let's take an
example of your eye
so when your iPhone is being built there
are all sorts of manufacturing
instructions for you know wiring up the
pieces of the circuit boards in the
screen and everything else but then once
you've bought an iPhone those signals
those manufacturing instructions don't
come along with the phone that's the
adult phone and so if part of it breaks
just injecting metal into the phone is
not necessarily going to repopulate the
circuitry of the phone in a proper way
even though metal is the raw material
that built the phone so we have to do
something much more sophisticated than
just putting the raw materials it so
tell me about the work that you're doing
now yeah so about four or five years ago
I decided that we'd really reached
almost the end of what we could do with
so-called animal models of disease mouse
models and you know just to just remind
everybody the mouse models of disease
are engineered so that they develop some
of the hallmark defects that you find in
the brains of people with disease but
ultimately they don't develop true
Alzheimer's disease as near as we can
tell there's something different about
mice and humans perhaps that's not so
surprising we are not just big mice we
don't look like Mickey Mouse our brains
are different we're big animals all the
rest of that stuff and so I decided that
if we were going to really make a
significant impact we needed to start
working on human brain cells and that's
harder than it sounds i can't just take
a sample of your brain out and study it
and you haven't developed aziz yet
anyway and once somebody's died of the
disease it's like studying a plane crash
after the planes hit the ground and and
the analogy I like to use is you can
learn a lot about a plane crash from
studying the pattern of wreckage on the
ground but what you really want is you
want the black box you want a record of
what was happening in the cockpit when
the plane was getting ready to go down
just before it did you know where the
pilots fooling around with their laptops
was one of them locked in the bathroom
they make a mistake or to the plane ran
out of gas or whatever you got to know
that so the idea
is that if we were to take a sample of
your skin in your skin cells we would
have your genome your unique pattern of
genetic variation that makes you
susceptible to some diseases and
resistant to others we can reprogram
your skin cells to a stem cell-like
state so that they can make all your
adult tissues as far as we can tell and
they would have your unique DNA so that
if we reprogram your skin cells to a
stem cell state and then induce them to
become brain cells we would de facto
have your Amy Adams unique brain cells
in a dish that we could study and if I
had Alzheimer's disease then we could
ask questions about those of those cells
about what biochemical changes are there
that are typical of Alzheimer's disease
and start to dissect how your pattern of
DNA variation leads to unusual behavior
of a brain cells and so we're in the
process in collaboration with the
sanford-burnham screening Center setting
up a project to look directly for drug
like molecules that reverse the abnormal
behavior of those brain cells that have
Alzheimer's disease in a dish Larry
thank you so much for your time I really
appreciate it that's coming to visit
good luck with your work