Raeka Aiyar, PhD, Symposium Moderator | Stem Cells and the Future of Medicine

Okay, there have been some emotional speeches. I wasn’t ready for that. I’m really happy to be back here at this excellent
meeting. I’m really grateful to Ron for inviting me
to come back. [Can you not hear? Sorry. Okay, yes, I will speak louder.] Sorry, I’m just a little bit emotional after
hearing this morning’s speeches. So I’m really grateful to Ron and to the entire
community for welcoming me back to this meeting. I’m really excited to be here at this event. It was a really inspiring event last year. And last year at this time, as he [Ron] mentioned,
I was working with him at Stanford and with this great team. And I’d like to tell you a little bit today
– before we get things kicked off with some of our very ME/CFS- focused talks – what I’ve
been up to since then. Because I wasn’t just recruited away. I was drawn to another really exciting area
in science that I think is going to have profound implications for disease research, including
for ME/CFS. And I’m looking forward to telling you about
that today. I’m working now in in the area of stem cell
research, which I believe has the potential to absolutely transform the way that we research
and treat diseases in a similar way that we’ve seen genomics transform research. And that was something I got to participate
in during my early research career, my PhD, and working in Ron’s lab of course as one
of the drivers of that. I think stem cell research is about to do
the same thing. It’s still earlier days for that, but I think
it’s really exciting. I want to share that excitement and potential
with you. So right now I’m working at the New York Stem
Cell Foundation. We are a non-profit foundation. We are a patient advocacy organization. Our CEO is is the mother of a patient, much
like the Open Medicine Foundation. And like many of the of the foundations that
support ME/CFS, and our mission is to accelerate cures for the major diseases of our time through
stem cell research. We do this in several different ways. The first is that we have a worldwide community
of innovators that we fund all over the world, this includes early career investigators in
stem cell and neuroscience research. It’s a really excellent community. We convene them every year, kind of similar
to this, and try to promote collaboration and exchange of ideas. We also have a huge focus on outreach through
our conferences and symposia. We do an annual conference as well with about
500 people. We have a lot of events that are dedicated
to educating patients about the different diseases that we study and how stem cell research
can help them. And finally, we have a Research Institute
where we do our own research in-house. So it’s a bit of a unique model. I was really drawn to that because there’s
the opportunity for both education and outreach, which I’ve come to love so much in working
with the ME/CFS community, and for being at the cutting edge of this new field of stem
cell research. We also have these collaborations all over
the world. I’m hoping to build some as well with this
community while I’m here to drive stem cell research forward and really accelerate these
cures. So what do we research at the New York Stem
Cell Foundation? We do a lot of different areas, this includes
bone regeneration. Cancer. We have diabetes, which was one of our founding
programs in autoimmune diseases. We also have a program for macular degeneration
to restore eyesight in patients with age-related macular degeneration. Our major focus is on neurological disorders,
and that’s particularly exciting for me because I’m hoping that there will be some lessons
that we can also translate from, especially diseases like multiple sclerosis, to ME/CFS. But we also work on other neurodegenerative
disorders, like Parkinson’s and Alzheimer’s disease as well as neuropsychiatric disorders. So there’s a really broad range of diseases
that you can study a lot better with stem-cell research. I realize that not everybody might know what
I mean by stem cell research, because there’s a lot of things people mean when they say
that, so I just want to show a very short video to explain why we think this is exciting. Stem cells are the superheroes of the cellular
universe. They are the only cells in the human body
with the ability to turn into more than 200 types of cells, including heart, brain, spinal
cord, kidney, liver, eyes, skin, and more. Stem cells also have the power to regenerate
indefinitely, filling up the entire room you are sitting in. These cells are uniquely powerful because
they allow us to do three things that we have never been able to do before. First, by studying the stem cells of patients,
we can see how those cells become damaged, causing disease. Second, we are able to test the safety and
effectiveness of drugs on those diseased patient cells. And, third, we are learning how to replace
those cells affected by disease with the patient’s own healthy cells. Imagine being able to use your own personal
cells to replace an organ, bone, or other damaged tissue. Not by waiting in line for a transplant match
that may never come, but through a safe, natural process for regenerating healthy tissues and
organs [Music; end of video] Just to summarize what you just saw in that
video. There are three major areas that we are pursuing
in stem-cell research. One of those is disease research, understanding
the molecular basis of disease. What we heard from Ron and from many of us
over the last three days, is that if we do want to cure a disease like ME/CFS, we need
to understand how it works . And we need to try to do that in many different ways. Another major area is drug discovery. We believe that by testing drugs on patient-derived
cells that you can get an idea of which drugs might be effective for which patients, and
that’s especially important in a disease as heterogeneous as ME/CFS. And finally there’s cell replacement therapy. As I mentioned we’re working on this for macular
degeneration and people are working on it for Parkinson’s disease to replace the cells
that are lost. Now that’s not really I don’t think a possibility
yet in ME/CFS because we don’t know what cells are being damaged or need to regenerate. But if we do figure that out, as it happened
for Parkinson’s disease when it was determined that the mid-brain dopaminergic neurons are
the prominent cells that are lost, then we can move forward with that. And I do want to point out about Parkinson’s
disease. When it was first discovered people thought
it was a psychosomatic disease driven by viral infection. A lot of the same things were being said. So we might just be where that was 50 years
ago. So the reason that all of this is possible
is because of technological revolutions. We can actually make your patients’, your
stem cells, from your skin or blood. We can induce them and that’s why they’re
called induced pluripotent stem cells. And this was a discovery made in 2006 by Shinya
Yamanaka. And just six years later he won the Nobel
Prize in medicine. That’s an incredibly fast turnaround for a
Nobel Prize, and that’s because of the promise that they’ve shown to revolutionize research. So why is this exciting technology? Because we don’t have to do invasive surgeries. We can use a skin biopsy or blood cells and
we can make your stem cells in a dish and convert them into all of these different cell
types that are affected in disease to understand what’s going on. These are what your skin cells look like. They’re actually quite pretty when you zoom
in on them in a microscope. And then we can convert them into these induced
pluripotent stem cells. So just to give you a bit more of an idea
of what I mean here. This gives you a bit of a window into disease. When you convert stem cells in the lab, you
can convert them into the specific cells that are affected in disease. So in neurodegenerative diseases this means
neurons, this means microglia, astrocytes, oligodendrocytes, all the major cell types
that are involved. And you can study those in a dish. This really gives you windows into many different
kinds of diseases. And having these in a patient-specific way
allows you then to test different drugs on those cells in the lab and see what their
effects are. And as I wanted to point out especially in
ME/CFS. This can help us to get an idea of which drugs
might work for which patients. Again, once we are able to see phenotypes
or characteristics of these cells that make them different from healthy controls, and
I think we’re able we’re going to be able to do that shortly. I want to show you an example of cardiomyocytes. These are heart muscle cells. When you grow them from stem cells in a dish,
they beat at the same rate as a human heart beats. You can test drugs on those, and you can detect
arrhythmias that drugs that are going to induce arrhythmia on certain people or not. You can make neurons and see how well their
electrical potential carries throughout a dish and how connected they are. So there are all these kinds of exciting features
of these cells that you can see that are able to distinguish patients from healthy people
just in a dish. Without having to go in and get your neurons
out of your brains, which is definitely preferable to everyone in this room, I’m sure. Finally as I mentioned with cell replacement
therapy. If we find out that ME/CFS is a degenerative
disease of some kind. If certain cells are being damaged, then what
the possibilities are right now for macular degeneration (this is where a specific type
of cell is lost in the eye). And clinical trials have actually started
to show promise in restoring eyesight in some of these patients using stem cell-derived
versions of these cells. The idea is the same in neurodegenerative
diseases. And trials are now underway for Parkinson’s
Disease to replace those dopaminergic neurons that are lost . And we’ve just started a preclinical
program to work on macular degeneration. We’re collaborating with the Parkinson’s disease
researchers that I mentioned. So it’s a really exciting time. What we’re trying to push for at our Institute
is “disease in a dish” and “clinical trial in a dish” models to really accelerate
this. Provide an extra model system to study ME/CFS
in and understand the molecular basis of it. And understand this on a patient-specific
level so we can figure out better treatments that work for each patient. And we really have a mandate to accelerate
all of this research at a community-wide level. I was going to show you this quickly. We have a fully robotic system for generating
these stem cells from skin cells or blood cells. And I will go through that. One last cautionary note. There are a lot of clinics that offer stem
cell therapies now for a variety of diseases. I would urge you to exercise caution with
those because these treatments have not been approved yet and they can actually be harmful. These clinics are looking to financially exploit
desperate patients. So it’s important to remember that at this
stage if anybody is advertising a stem-cell therapy or an injection for ME/CFS, I would
advise you to exercise caution as none of these have been approved yet. But we’re hopeful that this is going to be
the future of medicine that we will be able to treat your disease with your cells. We will be able to study your disease with
your cells and understand it better at that level. So with that I want to thank the community
that’s made this all possible and thank you all for your attention.

1 comment

  1. It's great to know that someone with research experience in ME/CFS is learning the cutting edge technology behind stem cell research. It's so good to have you as our advocate, Dr. Aiyar! 💙

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