Susan Solomon: The promise of research with stem cells
Summary
TLDRThe speaker discusses the transformative potential of stem cell research, highlighting its role in modeling diseases, understanding illness, and developing personalized medicine. They emphasize the need for new technologies and research models to bridge the gap between academic research and pharmaceutical development, ultimately aiming to revolutionize drug testing and treatment.
Takeaways
- 🌟 Embryonic stem cells are pluripotent, meaning they can develop into any cell type in the body, and are crucial for disease modeling and drug development.
- 🔬 Stem cell research is transforming our understanding of diseases and could lead to the development of more effective treatments and preventive measures.
- 💡 The New York Stem Cell Foundation Laboratory was established in 2005 to provide a safe haven for stem cell research, free from political and financial interference.
- 🤖 A new technology combining software and hardware has been developed to generate a diverse array of stem cell lines, essentially creating 'avatars' of humans for disease modeling and drug testing.
- 🧬 The potential of human genome sequencing is being realized through the use of stem cells, allowing for more personalized and effective clinical trials.
- 🧪 Induced pluripotent stem cells (iPS cells), derived from reprogrammed skin cells, offer a powerful tool for disease modeling and drug discovery, though they are not as versatile as embryonic stem cells.
- 🧬 A breakthrough in 2008 involved turning skin biopsies from ALS patients into motor neurons, providing the first model of a disease in living human cells.
- 💊 Current drug testing methods are inefficient and costly, with only 1% of tested drugs successfully making it to market, highlighting the need for better disease models and testing platforms.
- 👩🔬 The development of genetically diverse stem cell lines is essential for personalized medicine, allowing for more targeted and effective drug treatments based on individual genetic profiles.
- 🔎 The pharmaceutical industry needs to move away from a one-size-fits-all approach to drug development, considering the genetic diversity of the population to ensure safety and efficacy.
- 🏥 Automated robotic technology for stem cell production is set to revolutionize drug discovery, making the process more scalable, consistent, and personalized.
Q & A
What are embryonic stem cells and why are they significant for medical research?
-Embryonic stem cells are pluripotent cells that have the potential to develop into any cell type in the human body. They are significant for medical research because they can be used to replace damaged or diseased cells and have the potential to revolutionize the understanding and treatment of various diseases.
How can stem cells be used to model diseases and contribute to drug development?
-Stem cells can be used to create models of diseases by reprogramming them into the specific cell types affected by the disease. This allows researchers to study the disease process in a controlled environment, understand why cells become sick, and test potential drugs for effectiveness and safety.
What is the significance of induced pluripotent stem cells (iPS cells) in medical research?
-iPS cells are a type of pluripotent stem cell derived from adult cells that have been reprogrammed to an embryonic-like state. They are significant because they can be generated from a patient's own cells, avoiding ethical issues associated with embryonic stem cells, and can be used for disease modeling and drug discovery.
What is the New York Stem Cell Foundation Laboratory and what was its purpose?
-The New York Stem Cell Foundation Laboratory was established in 2005 to provide a private, safe haven for stem cell research. Its purpose was to advance stem cell research without political or financial interference and to bridge the gap between academic research and the development of drugs and treatments by the pharmaceutical industry.
What challenges did the speaker identify in the current system of drug development?
-The speaker identified several challenges, including the long time frame (13 years on average), the high cost (4 billion dollars per drug), and the low success rate (only 1% of drugs make it to market). Additionally, current drug testing often relies on animal models, which may not accurately represent human diseases.
What is the concept of 'avatars of ourselves' in the context of stem cell research?
-The concept of 'avatars of ourselves' refers to the use of genetically diverse stem cell lines that represent different individuals. These avatars can be used to conduct clinical trials in a dish with human cells, allowing for more personalized and effective drug development.
How does the speaker describe the potential impact of stem cell research on the future of disease treatment?
-The speaker believes that stem cell research will lead to a future where major diseases like Alzheimer's and diabetes are viewed as preventable, similar to how polio is viewed today. This is due to the ability of stem cells to model diseases and contribute to the development of more effective and safer treatments.
What is the role of genetics in the development of personalized medicine using stem cells?
-Genetics plays a crucial role in personalized medicine as it helps in understanding the individual differences in how diseases affect people and how they respond to treatments. By using stem cells from a genetically diverse array of individuals, researchers can develop drugs and treatments tailored to specific genetic profiles.
What was the issue with the drug Vioxx mentioned in the script, and how could stem cell technology have prevented it?
-Vioxx was a drug that helped alleviate severe arthritis pain but caused severe and sometimes fatal heart side effects in some patients. Stem cell technology could have prevented this by allowing for testing on a genetically diverse array of cardiac cells, identifying which genetic profiles would be affected by the side effects and thus preventing the drug from being prescribed to those individuals.
What is the significance of the automated robotic technology developed by the New York Stem Cell Foundation Laboratory?
-The automated robotic technology is significant because it can produce thousands of genetically diverse stem cell lines, which can be used to test drugs and treatments on a wide range of genetic profiles. This technology has the potential to revolutionize drug development by making it more efficient, cost-effective, and personalized.
How does the speaker view the current state of personalized medicine in relation to stem cell research?
-The speaker views the current state of personalized medicine as being on the threshold of a new era, thanks to stem cell research. The ability to create genetically diverse stem cell lines and test drugs on them allows for the development of treatments that are tailored to individual genetic profiles, moving away from a one-size-fits-all approach.
Outlines
🌟 The Promise of Stem Cell Research
The speaker, Joseph Geni, discusses the incredible potential of embryonic stem cells as a form of body repair. These pluripotent cells can transform into any cell type in the body, offering hope for replacing damaged or diseased cells. He emphasizes the transformative impact of stem cell research on disease modeling and drug development, comparing its potential to the eradication of polio. Despite political and financial challenges, the establishment of the New York Stem Cell Foundation Laboratory in 2005 aimed to advance this critical research. The speaker highlights the gap between academic research and pharmaceutical development, and the need for new technologies and research models to bridge this gap. The development of a new technology that generates genetically diverse stem cell lines is presented as a solution to accelerate cures and therapies, allowing for more effective and safer drug development.
🔍 The Power of Human Stem Cell Models
The speaker continues by describing the groundbreaking discovery of induced pluripotent stem cells (IPS cells), which can be reprogrammed from skin cells. These cells, though not identical to embryonic stem cells, are valuable for disease modeling and drug discovery. An example is given where skin biopsies from patients with ALS were reprogrammed into IPS cells, which were then transformed into motor neurons. This allowed researchers to observe the disease process in a controlled environment, leading to the discovery of previously unknown contributing factors. The speaker argues that without human stem cell models, understanding diseases would be akin to investigating a plane crash without a black box recorder. The potential for using human cells to test drugs is highlighted, emphasizing the current inefficiency and high cost of drug development. The speaker also discusses the need for a more personalized approach to drug development, considering genetic diversity, to avoid one-size-fits-all treatments.
🛠️ Advancing Stem Cell Technology for Personalized Medicine
The speaker addresses the limitations of current stem cell line production methods, which are manual and time-consuming, leading to inconsistencies. To overcome this, an automated robotic technology has been developed to produce thousands of genetically diverse stem cell lines. This technology is capable of massively parallel processing, which will revolutionize drug discovery by allowing for testing on a wide array of cell types. The speaker envisions a future where drugs are re-screened on these arrays, leading to more personalized and effective treatments. The potential to test drugs on relevant cells, such as brain, heart, and liver cells, is highlighted as a step towards personalized medicine. The speaker concludes by sharing personal connections to diseases like diabetes and heart disease, emphasizing the importance of stem cell research for all.
Mindmap
Keywords
💡Embryonic Stem Cells
💡Pluripotent
💡Stem Cell Research
💡Induced Pluripotent Stem Cells (iPS Cells)
💡Disease Modeling
💡Pharmaceutical Companies
💡Genetically Diverse Stem Cell Lines
💡Personalized Medicine
💡Drug Discovery
💡One-Size-Fits-All Model
💡New York Stem Cell Foundation Laboratory
Highlights
Embryonic stem cells are pluripotent and can morph into all cells in our bodies, acting as our body's repair kit.
Stem cells will enable us to replace damaged or diseased cells in the future.
Stem cell research is revolutionizing our understanding of disease and drug development.
Stem cell research could make major diseases like Alzheimer's and diabetes preventable in the future.
The New York Stem Cell Foundation Laboratory was established in 2005 to advance stem cell research without interference.
Large organizations often struggle to innovate in new fields like stem cell research.
A gap exists between academic research and pharmaceutical companies in delivering drugs and treatments.
New technologies and a new research model are needed to accelerate cures and therapies.
A new technology generates genetically diverse stem cell lines to create avatars of ourselves for disease modeling and drug testing.
This technology enables clinical trials in a dish with human cells, making drug development more effective, safer, faster, and cheaper.
Induced pluripotent stem cells (iPS cells) were created by reprogramming skin cells, allowing disease modeling and drug discovery.
Researchers used iPS cells to model ALS and discovered a previously unknown cellular mechanism contributing to the disease.
Stem cells provide an unprecedented window into disease mechanisms, acting as a 'black box' for understanding disease causes.
Current drug development takes 13 years on average, costs $4 billion, and has a 1% success rate.
Drug testing on human stem cells allows for more accurate and faster assessment of drug efficacy and safety.
Genetic diversity must be considered in drug development to avoid 'one-size-fits-all' approaches that may not work for everyone.
The example of Vioxx highlights the dangers of not accounting for genetic differences in drug responses.
Stem cell lines need to be produced in a scalable, automated way to ensure consistency and quality control.
The new technology enables the creation of a genetically diverse array of stem cell lines to advance personalized medicine.
Stem cell research is crucial for developing treatments for common diseases like diabetes, heart disease, and cancer.
Transcripts
Translator: Joseph Geni Reviewer: Morton Bast
So, embryonic stem cells
are really incredible cells.
They are our body's own repair kits,
and they're pluripotent, which means they can morph into
all of the cells in our bodies.
Soon, we actually will be able to use stem cells
to replace cells that are damaged or diseased.
But that's not what I want to talk to you about,
because right now there are some really
extraordinary things that we are doing with stem cells
that are completely changing
the way we look and model disease,
our ability to understand why we get sick,
and even develop drugs.
I truly believe that stem cell research is going to allow
our children to look at Alzheimer's and diabetes
and other major diseases the way we view polio today,
which is as a preventable disease.
So here we have this incredible field, which has
enormous hope for humanity,
but much like IVF over 35 years ago,
until the birth of a healthy baby, Louise,
this field has been under siege politically and financially.
Critical research is being challenged instead of supported,
and we saw that it was really essential to have
private safe haven laboratories where this work
could be advanced without interference.
And so, in 2005,
we started the New York Stem Cell Foundation Laboratory
so that we would have a small organization that could
do this work and support it.
What we saw very quickly is the world of both medical
research, but also developing drugs and treatments,
is dominated by, as you would expect, large organizations,
but in a new field, sometimes large organizations
really have trouble getting out of their own way,
and sometimes they can't ask the right questions,
and there is an enormous gap that's just gotten larger
between academic research on the one hand
and pharmaceutical companies and biotechs
that are responsible for delivering all of our drugs
and many of our treatments, and so we knew that
to really accelerate cures and therapies, we were going
to have to address this with two things:
new technologies and also a new research model.
Because if you don't close that gap, you really are
exactly where we are today.
And that's what I want to focus on.
We've spent the last couple of years pondering this,
making a list of the different things that we had to do,
and so we developed a new technology,
It's software and hardware,
that actually can generate thousands and thousands of
genetically diverse stem cell lines to create
a global array, essentially avatars of ourselves.
And we did this because we think that it's actually going
to allow us to realize the potential, the promise,
of all of the sequencing of the human genome,
but it's going to allow us, in doing that,
to actually do clinical trials in a dish with human cells,
not animal cells, to generate drugs and treatments
that are much more effective, much safer,
much faster, and at a much lower cost.
So let me put that in perspective for you
and give you some context.
This is an extremely new field.
In 1998, human embryonic stem cells
were first identified, and just nine years later,
a group of scientists in Japan were able to take skin cells
and reprogram them with very powerful viruses
to create a kind of pluripotent stem cell
called an induced pluripotent stem cell,
or what we refer to as an IPS cell.
This was really an extraordinary advance, because
although these cells are not human embryonic stem cells,
which still remain the gold standard,
they are terrific to use for modeling disease
and potentially for drug discovery.
So a few months later, in 2008, one of our scientists
built on that research. He took skin biopsies,
this time from people who had a disease,
ALS, or as you call it in the U.K., motor neuron disease.
He turned them into the IPS cells
that I've just told you about, and then he turned those
IPS cells into the motor neurons that actually
were dying in the disease.
So basically what he did was to take a healthy cell
and turn it into a sick cell,
and he recapitulated the disease over and over again
in the dish, and this was extraordinary,
because it was the first time that we had a model
of a disease from a living patient in living human cells.
And as he watched the disease unfold, he was able
to discover that actually the motor neurons were dying
in the disease in a different way than the field
had previously thought. There was another kind of cell
that actually was sending out a toxin
and contributing to the death of these motor neurons,
and you simply couldn't see it
until you had the human model.
So you could really say that
researchers trying to understand the cause of disease
without being able to have human stem cell models
were much like investigators trying to figure out
what had gone terribly wrong in a plane crash
without having a black box, or a flight recorder.
They could hypothesize about what had gone wrong,
but they really had no way of knowing what led
to the terrible events.
And stem cells really have given us the black box
for diseases, and it's an unprecedented window.
It really is extraordinary, because you can recapitulate
many, many diseases in a dish, you can see
what begins to go wrong in the cellular conversation
well before you would ever see
symptoms appear in a patient.
And this opens up the ability,
which hopefully will become something that
is routine in the near term,
of using human cells to test for drugs.
Right now, the way we test for drugs is pretty problematic.
To bring a successful drug to market, it takes, on average,
13 years — that's one drug —
with a sunk cost of 4 billion dollars,
and only one percent of the drugs that start down that road
are actually going to get there.
You can't imagine other businesses
that you would think of going into
that have these kind of numbers.
It's a terrible business model.
But it is really a worse social model because of
what's involved and the cost to all of us.
So the way we develop drugs now
is by testing promising compounds on --
We didn't have disease modeling with human cells,
so we'd been testing them on cells of mice
or other creatures or cells that we engineer,
but they don't have the characteristics of the diseases
that we're actually trying to cure.
You know, we're not mice, and you can't go into
a living person with an illness
and just pull out a few brain cells or cardiac cells
and then start fooling around in a lab to test
for, you know, a promising drug.
But what you can do with human stem cells, now,
is actually create avatars, and you can create the cells,
whether it's the live motor neurons
or the beating cardiac cells or liver cells
or other kinds of cells, and you can test for drugs,
promising compounds, on the actual cells
that you're trying to affect, and this is now,
and it's absolutely extraordinary,
and you're going to know at the beginning,
the very early stages of doing your assay development
and your testing, you're not going to have to wait 13 years
until you've brought a drug to market, only to find out
that actually it doesn't work, or even worse, harms people.
But it isn't really enough just to look at
the cells from a few people or a small group of people,
because we have to step back.
We've got to look at the big picture.
Look around this room. We are all different,
and a disease that I might have,
if I had Alzheimer's disease or Parkinson's disease,
it probably would affect me differently than if
one of you had that disease,
and if we both had Parkinson's disease,
and we took the same medication,
but we had different genetic makeup,
we probably would have a different result,
and it could well be that a drug that worked wonderfully
for me was actually ineffective for you,
and similarly, it could be that a drug that is harmful for you
is safe for me, and, you know, this seems totally obvious,
but unfortunately it is not the way
that the pharmaceutical industry has been developing drugs
because, until now, it hasn't had the tools.
And so we need to move away
from this one-size-fits-all model.
The way we've been developing drugs is essentially
like going into a shoe store,
no one asks you what size you are, or
if you're going dancing or hiking.
They just say, "Well, you have feet, here are your shoes."
It doesn't work with shoes, and our bodies are
many times more complicated than just our feet.
So we really have to change this.
There was a very sad example of this in the last decade.
There's a wonderful drug, and a class of drugs actually,
but the particular drug was Vioxx, and
for people who were suffering from severe arthritis pain,
the drug was an absolute lifesaver,
but unfortunately, for another subset of those people,
they suffered pretty severe heart side effects,
and for a subset of those people, the side effects were
so severe, the cardiac side effects, that they were fatal.
But imagine a different scenario,
where we could have had an array, a genetically diverse array,
of cardiac cells, and we could have actually tested
that drug, Vioxx, in petri dishes, and figured out,
well, okay, people with this genetic type are going to have
cardiac side effects, people with these genetic subgroups
or genetic shoes sizes, about 25,000 of them,
are not going to have any problems.
The people for whom it was a lifesaver
could have still taken their medicine.
The people for whom it was a disaster, or fatal,
would never have been given it, and
you can imagine a very different outcome for the company,
who had to withdraw the drug.
So that is terrific,
and we thought, all right,
as we're trying to solve this problem,
clearly we have to think about genetics,
we have to think about human testing,
but there's a fundamental problem,
because right now, stem cell lines,
as extraordinary as they are,
and lines are just groups of cells,
they are made by hand, one at a time,
and it takes a couple of months.
This is not scalable, and also when you do things by hand,
even in the best laboratories,
you have variations in techniques,
and you need to know, if you're making a drug,
that the Aspirin you're going to take out of the bottle
on Monday is the same as the Aspirin
that's going to come out of the bottle on Wednesday.
So we looked at this, and we thought, okay,
artisanal is wonderful in, you know, your clothing
and your bread and crafts, but
artisanal really isn't going to work in stem cells,
so we have to deal with this.
But even with that, there still was another big hurdle,
and that actually brings us back to
the mapping of the human genome, because
we're all different.
We know from the sequencing of the human genome
that it's shown us all of the A's, C's, G's and T's
that make up our genetic code,
but that code, by itself, our DNA,
is like looking at the ones and zeroes of the computer code
without having a computer that can read it.
It's like having an app without having a smartphone.
We needed to have a way of bringing the biology
to that incredible data,
and the way to do that was to find
a stand-in, a biological stand-in,
that could contain all of the genetic information,
but have it be arrayed in such a way
as it could be read together
and actually create this incredible avatar.
We need to have stem cells from all the genetic sub-types
that represent who we are.
So this is what we've built.
It's an automated robotic technology.
It has the capacity to produce thousands and thousands
of stem cell lines. It's genetically arrayed.
It has massively parallel processing capability,
and it's going to change the way drugs are discovered,
we hope, and I think eventually what's going to happen
is that we're going to want to re-screen drugs,
on arrays like this, that already exist,
all of the drugs that currently exist,
and in the future, you're going to be taking drugs
and treatments that have been tested for side effects
on all of the relevant cells,
on brain cells and heart cells and liver cells.
It really has brought us to the threshold
of personalized medicine.
It's here now, and in our family,
my son has type 1 diabetes,
which is still an incurable disease,
and I lost my parents to heart disease and cancer,
but I think that my story probably sounds familiar to you,
because probably a version of it is your story.
At some point in our lives, all of us,
or people we care about, become patients,
and that's why I think that stem cell research
is incredibly important for all of us.
Thank you. (Applause)
(Applause)
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