Susan Solomon: The promise of research with stem cells

Summary

El guion del video destaca la importancia de las células madre embrionarias como herramientas de reparación del cuerpo y su potencial para transformarse en cualquier tipo celular. La investigación con estas células está cambiando nuestra comprensión de las enfermedades y el desarrollo de medicamentos. La tecnología desarrollada por la New York Stem Cell Foundation Laboratory permite crear una gran variedad de líneas de células madre, lo que abre la puerta a ensayos clínicos en un plato y a un enfoque más personalizado en la medicina. Esta innovación tiene el potencial de acelerar el descubrimiento de tratamientos más efectivos y seguros, y podría cambiar drásticamente la forma en que se abordan enfermedades como el Alzheimer y la diabetes.

Takeaways

• 🌟 Las células madre embrionarias son increíblemente versátiles y pueden transformarse en cualquier tipo de célula del cuerpo humano.
• 🛠️ La investigación con células madre tiene el potencial de cambiar la forma en que entendemos y modelamos las enfermedades, así como de desarrollar medicamentos.
• 💡 La creación de la New York Stem Cell Foundation Laboratory en 2005 proporcionó un espacio seguro para avanzar en la investigación sin interferencias.
• 🔬 La tecnología desarrollada por la fundación combina software y hardware para generar una gran cantidad de líneas de células madre genéticamente diversas, lo que representa un gran avance en la realización del potencial del genoma humano.
• 🧪 La utilización de células madre para realizar ensayos clínicos en un plato, en lugar de en células animales, puede conducir a medicamentos y tratamientos más efectivos, seguros y económicos.
• 📈 La identificación de células madre embrionarias humanas en 1998 y la posterior creación de células madre inducidas pluripotentes (IPS) en 2007 representan avances significativos en la ciencia.
• 🧬 La investigación con células madre IPS ha permitido modelar enfermedades y descubrir mecanismos de muerte celular que antes no se conocían.
• 💊 La actual metodología para la prueba de medicamentos es problemática y costosa, con un porcentaje de éxito muy bajo.
• 👫 La importancia de la genética en el desarrollo de medicamentos personalizados, ya que las respuestas a los medicamentos varían según la composición genética de cada individuo.
• 🤖 La tecnología automatizada y robótica para la producción de líneas de células madre es esencial para superar los desafíos de escala y consistencia en la producción de medicamentos.
• 🌈 La perspectiva de una medicina personalizada se acerca, donde los medicamentos y tratamientos serán probados en células relevantes para cada paciente, lo que puede mejorar la efectividad y seguridad de los tratamientos.

Q & A

¿Qué son las células madre embrionarias y por qué son importantes?

Las células madre embrionarias son células pluripotentes que pueden transformarse en cualquier tipo de célula del cuerpo humano. Son consideradas como el 'kit de reparación' natural del cuerpo y tienen un potencial enorme para la investigación médica y el tratamiento de enfermedades.

¿Cómo se relacionan las células madre con la capacidad de entender y tratar enfermedades?

Las células madre permiten modelar enfermedades y ayudar a comprender por qué las personas enferman. Además, se utilizan en la investigación para desarrollar medicamentos y terapias, lo que puede cambiar la forma en que abordamos enfermedades crónicas y degenerativas.

¿Por qué es crucial el avance en investigación con células madre?

El avance en investigación con células madre puede llevar a la prevención y curación de enfermedades como el Alzheimer y la diabetes, que actualmente son incurables. La investigación con células madre es fundamental para mejorar la salud humana y extender la vida útil de las personas.

¿Qué desafíos políticos y financieros enfrenta la investigación con células madre?

La investigación con células madre ha sido objeto de controversia y desafíos políticos y financieros, similares a los que enfrentó la fertilización in vitro hace más de 35 años. La investigación crítica está siendo desafiada en lugar de ser apoyada, lo que ha llevado a la necesidad de laboratorios seguros privados para avanzar en el trabajo sin interferencia.

¿Qué es la Fundación de Células Madre de Nueva York y su propósito?

La Fundación de Células Madre de Nueva York es una organización creada en 2005 con el objetivo de avanzar en la investigación con células madre y apoyar este trabajo. Se enfoca en cerrar la brecha entre la investigación académica y las compañías farmacéuticas y biotecnológicas que desarrollan medicamentos y tratamientos.

¿Qué es una célula madre inducida pluripotente (IPS) y cómo se relaciona con la investigación de enfermedades?

Una célula madre inducida pluripotente (IPS) es una célula que se ha reprogramado a partir de células somáticas adultas para tener propiedades pluripotentes similares a las de las células madre embrionarias. Estas células son útiles para modelar enfermedades y potencialmente para la descubrimiento de medicamentos.

¿Cómo se utilizan las células madre para modelar enfermedades y descubrir medicamentos?

Las células madre se pueden transformar en células específicas del cuerpo, como neuronas motoras, y se pueden utilizar para modelar enfermedades como la enfermedad de ALS. Esto permite observar cómo se desarrolla la enfermedad en un entorno controlado y descubrir nuevas rutas de investigación y posibles medicamentos.

¿Qué significa el término 'clínica en un plato' y por qué es importante?

El término 'clínica en un plato' se refiere a la capacidad de realizar pruebas y ensayos clínicos utilizando células humanas en lugar de animales o ensayos en seres humanos. Esto es importante porque permite realizar pruebas más seguras y precisas, reduciendo los riesgos y tiempos de desarrollo de medicamentos.

¿Qué es la nueva tecnología de software y hardware que se menciona en el guion y cómo cambiará la investigación?

La nueva tecnología de software y hardware mencionada en el guion puede generar miles de líneas de células madre genéticamente diversificadas. Esto permitirá realizar ensayos clínicos en un plato con células humanas, lo que puede acelerar el desarrollo de medicamentos y tratamientos más efectivos, seguros y económicos.

¿Por qué es necesario considerar la genética en el desarrollo de medicamentos y por qué las células madre pueden ayudar en esto?

La genética es crucial en el desarrollo de medicamentos porque las respuestas individuales a los medicamentos varían según las características genéticas de cada persona. Las células madre genéticamente diversificadas pueden ayudar a modelar estas diferencias y a desarrollar medicamentos personalizados que sean más efectivos y seguros para diferentes grupos genéticos.

Outlines

00:00

🧬 La importancia de las células madre embrionarias

El primer párrafo aborda la fascinante realidad de las células madre embrionarias, descritas como 'kits de reparación' del cuerpo humano con la capacidad de transformarse en cualquier tipo de célula. Se enfatiza su potencial para reemplazar células dañadas o enfermas y su impacto en la comprensión de las enfermedades y el desarrollo de medicamentos. La narrativa destaca la promesa de la investigación en células madre y cómo ha sido desafiada política y financieramente, similar a la fertilización in vitro hace más de 35 años. Se menciona la creación del New York Stem Cell Foundation Laboratory en 2005 para apoyar y avanzar en esta investigación crítica. Además, se discute la necesidad de cerrar la brecha entre la investigación académica y las empresas farmacéuticas y biotéicas, proponiendo nuevas tecnologías y un nuevo modelo de investigación para acelerar el desarrollo de curas y terapias.

05:02

🔬 Modelos de enfermedades con células madre

El segundo párrafo profundiza en cómo las células madre están transformando la forma en que entendemos y modelamos las enfermedades. Se destaca la capacidad de estas células para recrear enfermedades en un plato de cultivo, proporcionando una ventana sin precedentes para observar los errores celulares antes de que aparezcan síntomas en un paciente. Se critica el actual modelo de prueba de medicamentos, que es costoso y poco efectivo, y se propone el uso de células humanas para probar medicamentos, lo que podría reducir significativamente los tiempos y costos de desarrollo. Se argumenta que la genética juega un papel crucial en la reacción a medicamentos y que es necesario considerar la diversidad genética al desarrollar nuevos tratamientos. Se menciona el caso de Vioxx, un medicamento que ayudó a algunos pero causó daños cardíacos graves en otros, ilustrando la necesidad de un enfoque más personalizado en la farmacología.

10:03

🤖 Tecnología robótica para la producción de líneas de células madre

El tercer párrafo presenta la tecnología robótica automatizada que se ha desarrollado para producir una gran cantidad de líneas de células madre genéticamente diversas. Se destaca la importancia de esta tecnología para la investigación farmacológica y el desarrollo de medicamentos personalizados. Se argumenta que la producción artesanal de líneas de células madre no es escalable ni consistente, y que la nueva tecnología puede ayudar a superar estos desafíos. Se menciona la necesidad de tener una representación genética amplia y diversa para poder probar medicamentos en un rango completo de células humanas, lo que podría llevar a una medicina personalizada más efectiva. Se concluye con un enfoque emocional, destacando la relevancia personal de la investigación en células madre para el hablante y la esperanza de que avance en esta área pueda traer soluciones para enfermedades incurables como el diabetes tipo 1, la enfermedad cardiovascular y el cáncer.

Keywords

💡Células madre embrionarias

Células madre embrionarias son células pluripotentes que pueden desarrollarse en cualquier tipo de célula del cuerpo humano. En el video, se menciona que son increíblemente importantes para la reparación y el estudio de enfermedades, y que su investigación tiene un gran potencial para la humanidad, comparando su importancia con el desarrollo de la fertilización in vitro hace más de 35 años.

💡Pluripotencia

La pluripotencia es la capacidad de una célula madre embrionaria para transformarse en cualquier tipo de célula del cuerpo. Es un concepto clave en el video, ya que esta propiedad es fundamental para el potencial terapéutico de las células madre y para el modelado de enfermedades.

💡Modelado de enfermedades

El modelado de enfermedades se refiere a la creación de sistemas en los que se pueden estudiar los procesos patológicos. En el video, se destaca cómo las células madre permiten modelar enfermedades de manera más precisa y personalizada, lo que es crucial para entender mejor las causas de las enfermedades y para el desarrollo de tratamientos.

💡Células madre inducidas pluripotentes (iPS)

Las células madre inducidas pluripotentes son células somáticas que han sido reprogramadas para tener propiedades similares a las de las células madre embrionarias. En el video, se describe cómo estas células fueron creadas a partir de células de piel y cómo han revolucionado el campo de la investigación médica.

💡Investigación de medicamentos

La investigación de medicamentos es el proceso de descubrimiento y desarrollo de nuevos fármacos. El video resalta cómo el uso de células madre puede mejorar este proceso, haciendo que sea más efectivo, seguro y económico al permitir pruebas con células humanas en lugar de animales.

💡Células motoras neuronales

Las células motoras neuronales son las células afectadas en la enfermedad de ALS. En el video, se describe cómo un científico transformó biopsias de piel de pacientes con ALS en células madre inducidas pluripotentes y luego en células motoras neuronales, lo que permitió modelar la enfermedad y descubrir nuevas perspectivas sobre su causa.

💡Toxicidad

La toxicidad se refiere a la capacidad de una sustancia para causar daño en el cuerpo. En el contexto del video, se menciona cómo la toxicidad de ciertos fármacos puede variar según el perfil genético de las personas, lo que destaca la importancia de la personalización en la medicina.

💡Medicamentos genéticamente diversos

El video habla sobre la necesidad de probar medicamentos en una variedad genética amplia de células para entender mejor cómo diferentes perfiles genéticos pueden responder de manera diferente a un mismo fármaco, lo que puede llevar a una medicina más personalizada y segura.

💡Tecnología de automatización y robótica

La tecnología de automatización y robótica se utiliza para producir grandes cantidades de líneas de células madre de manera más eficiente y precisa. En el video, se describe cómo esta tecnología puede cambiar el proceso de descubrimiento de medicamentos y la realización de ensayos clínicos.

💡Medicamentos personalizados

Los medicamentos personalizados son fármacos diseñados para satisfacer las necesidades individuales de los pacientes basándose en su perfil genético. El video enfatiza cómo el uso de células madre y tecnología avanzada puede llevar a un enfoque más personalizado en el tratamiento de enfermedades.

💡Medicamentos existentes

El video sugiere la posibilidad de volver a probar los medicamentos existentes en nuevas matrices de células madre genéticamente diversas para evaluar mejor sus efectos y side effects, lo que podría mejorar la seguridad y eficacia de los tratamientos.

Highlights

Embryonic stem cells are pluripotent and can morph into all cells in our bodies, acting as our body's repair kit.

Stem cells have the potential to replace damaged or diseased cells in the future.

Stem cells are being used to model diseases, understand why we get sick, and develop drugs.

Stem cell research could change the perception of major diseases like Alzheimer's and diabetes, viewing them as preventable.

The New York Stem Cell Foundation Laboratory was established in 2005 to advance stem cell research without interference.

Large organizations sometimes struggle to innovate in new fields, leading to a gap between academic research and drug development.

New technologies and a new research model are needed to bridge the gap between research and drug development.

A new technology has been developed to generate genetically diverse stem cell lines, creating a global array of human 'avatars'.

This technology allows for clinical trials with human cells instead of animal cells, leading to more effective and safer drugs.

The first human embryonic stem cells were identified in 1998, and nine years later, induced pluripotent stem cells (IPS cells) were created.

IPS cells, derived from skin cells, are useful for disease modeling and drug discovery, despite not being embryonic stem cells.

Researchers were able to reprogram skin biopsies from ALS patients into motor neurons, creating a model of the disease in a dish.

Stem cell models have revealed new insights into disease mechanisms, such as the role of certain cells in ALS.

Stem cells provide an unprecedented window into disease, allowing researchers to see cellular conversations before symptoms appear.

Drug development currently takes 13 years on average, with a cost of 4 billion dollars and a 1% success rate.

Traditional drug testing on animal cells does not accurately represent human diseases.

Human stem cells can create 'avatars' for drug testing, providing a more accurate and faster method.

The need for a genetically diverse array of cells to test drugs, accounting for individual genetic differences in disease response.

The pharmaceutical industry has not yet adopted a personalized approach to drug development due to lack of tools.

An automated robotic technology has been developed to produce a scalable and genetically diverse array of stem cell lines.

This technology could lead to personalized medicine, testing drugs for side effects on relevant human cells.

The potential for re-screening existing drugs on genetically diverse cell arrays to improve safety and efficacy.

Stem cell research is crucial for advancing treatments for diseases like type 1 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)