Episode 4: Cerebral Palsy Health. Iona Novak, PhD and Madison Paton, PhD: What's the Deal with Stem Cells Anyway?

Episode 4: Cerebral Palsy Health. Iona Novak, PhD and Madison Paton, PhD: What's the Deal with Stem Cells Anyway?

About this Episode
Photo of Jennifer Lyman and her son with the copy Cerebral Palsy Health by Jennifer Lyman across to the top of the image

On this episode I talk with two of the world’s leading researchers… Dr. Madison Paton and Dr. Iona Novak on stem cell treatment for cerebral palsy. Stem Cells have been a hot topic in Cerebral Palsy for at least 15 years now, with many parents and researchers hoping that at the least, stem cells will lessen the impact of CP and at most hold the key to a cure. Dr. Paton and Dr. Novak will share their insider knowledge into this subject and help us sort through the hype and so we can hold onto our hope.

We discuss the various types of stem cells and how they might benefit those with CP.  We also discuss current research, Jen's experience with her son's stem cell trial, how to learn about other stem cell trials and where to find resources.  We also talk about stem cell tourism and issues to be aware of with these operations. 

Professor Iona Novak is the Head of Research for the Cerebral Palsy Alliance Research Institute, located at the Brain Mind Centre, in the University of Sydney (in Australia). Iona is a Fulbright Scholar, and has won the Western Sydney University Chancellor's Award for Alumni of the Year and The University of Sydney Award for Professional Achievement. She co-founded the Cerebral Palsy Alliance Research Institute for the purpose of: prevention, cure and treatment of cerebral palsy. Driven by an internal belief that healthcare truly has the potential to change lives, Iona has pursued projects and roles that will have the greatest possible impact on children and families in today’s and tomorrow’s world. She is passionate about evidence-based practice; knowledge translation, neuroplasticity; stem cells and neuroregenerative clinical trials. Iona leads a collaborative research team that is currently conducting the world’s largest ever, cerebral palsy early intervention trial, exploring whether harnessing neuroplasticity can reduce the severity of disability.

Dr Madison Paton is a Research Fellow at the Cerebral Palsy Alliance Research Institute. She received her PhD in 2018 investigating stem cell therapies to protect the developing brain. She has been working in the field of cell therapies and cerebral palsy for more than 7 years and has transitioned from working in the lab, to helping develop clinical trials that drive the research pipeline. Madison’s early work focussed on stem cells collected from the placenta and how we can best protect from brain injury occurring during pregnancy or around the time of birth. She now is interested in applying these therapies to treat babies and children with a risk of brain injury or cerebral palsy. Dr Paton is passionate about science communication, engaging with consumers, and sharing the best available evidence on stem cells.

TRANSCRIPT:

Jen Lyman (00:00:00):

Welcome to Cerebral Palsy Health, conversations that count with experts who care. On today's episode. We'll be talking about stem cell treatment for cerebral palsy with two of the world's leading researchers, Dr. Madison Paton and Dr. Iona Novak. Stem cells have been a hot topic in cerebral palsy for about 15 years now with many parents and researchers hoping that at least some cells lessen the impact of cerebral palsy and at most hold the key to a cure. Dr. Paton and Dr. Novak will share their insider knowledge into this subject and help us sort through the hype so that we can hold onto our hope. And I hope you enjoy the show.

Jen Lyman (00:00:38):

Welcome to the Cerebral Palsy Health podcast. We dive deep into health topics that impact people with cerebral palsy, such as stem cells, genetics, neuroplasticity, exercise and fitness, nutrition, accessibility. Issues that could be confusing or controversial and those that offer hope, but might not live up to the hype.

Jen Lyman (00:00:55):

I'm your host, Jen Lyman. Join me in conversations with leading experts as we separate fact from fiction, tackle tough-to-understand topics and try to shed light on how best to maximize and optimize health participation and quality of life for those with cerebral palsy.

Jen Lyman (00:01:15):

Professor Iona Novak is the head of research for the Cerebral Palsy Alliance Research Institute located at the Brain Mind Center in the University of Sydney in Australia. Iona is a Fulbright scholar and has won the Western Sydney University Chancellor's Award for Alumni of the Year and the University of Sydney Award for Professional Achievement. She co-founded the Cerebral Palsy Alliance Research Institute for the purpose of prevention, cure and treatment of cerebral palsy.

Jen Lyman (00:01:43):

Driven by an internal belief that healthcare truly has the potential to change lives, Iona has pursued projects and roles that will have the greatest possible impact on children and families and today's and tomorrow's world. She is passionate about evidence-based practice, knowledge translation, neuroplasticity, stem cells, and neuro-regenerative clinical trials. Iona leads a collaborative research team that is currently conducting the world's largest ever cerebral palsy, early intervention trial, exploring whether harnessing neuroplasticity can reduce the severity of disability.

Jen Lyman (00:02:18):

We also have Dr. Madison Paton. She is a research fellow at the Cerebral Palsy Alliance Research Institute. She received her PhD in 2018 investigating stem cell therapies to protect the developing brain. She has been working in the field of cell therapies and cerebral palsy for more than seven years and has transitioned from working in the lab to helping develop clinical trials that drive the research pipeline.

Jen Lyman (00:02:42):

Madison's early work focused on stem cells collected from the placenta and how we can best protect from brain injury occurring during pregnancy or around the time of birth. She is now interested in applying these therapies to treat babies and children with a risk of brain injury or cerebral palsy. Dr. Paton is passionate about science communication, engaging with consumers and sharing the best available evidence on stem cells.

Jen Lyman (00:03:05):

Welcome Iona and Madison from your homes down under. I'm thrilled to have you guys on the show and I hope you both are doing well.

Madison Paton (00:03:12):

Hi, thank you so much.

Jen Lyman (00:03:14):

Let's start with a personal question. Madison, I don't know you nearly as well as I know Iona, so I figure let's get to know you first and then we can switch over to Iona. When did you get into this field, or why did you get into this field and why did you choose to research stem cells for people with cerebral palsy?

Madison Paton (00:03:31):

I guess I was very lucky early on. I'm a bit of a science geek and I knew that I had a very caring and nurturing personality. This kind of always started when I was a lot younger and I wanted to set up fake doctor clinics in my lounge room with my parents and really only a real stethoscope would do. So, I was really lucky when I was five years old that my parents actually gifted me a real stethoscope. So I knew I wanted to care for people and look after people and provide in some way. That was always kind of inbuilt in me.

Madison Paton (00:04:04):

Along with that came that science nerd side and I studied science all through university. Then I was given the opportunity to join a lab where they focused on stem cell therapies and cerebral palsy, and really their passion rubbed off on me. All of their hearts were in the right place and we got to work for the most incredible research projects that were people-focused, looked at brain injury and really had a lot of these personal stories really built into them. Most of them were funded by philanthropy and I got the opportunity to meet different families who my research really affected firsthand. So that was really important to me and inspired me all the way through all my research up until when I joined the CP Alliance.

Jen Lyman (00:04:50):

Wow. Well welcome. Amazing to have you here. I'm so thrilled that this is what your path has taken you on, and that we have you here researching stem cells.

Madison Paton (00:05:00):

Thank you. I'm very lucky.

Jen Lyman (00:05:01):

You certainly are and so are we.

Madison Paton (00:05:03):

Good.

Jen Lyman (00:05:05):

Iona, besides being a fantastic babysitter for my son, I kind of have a funny story about that. Years ago, I think it was about six or seven years ago, we were both at a conference and I, all of a sudden lost my helper and had to run to go do my own presentation. You were standing next to me and I said, "Here take him." I think you did. So I know you... Well, I don't know you as well as I would like, but I would like to know from you, how did you get into this field and choose to work with people with cerebral palsy?

Iona Novak (00:05:38):

My mother was an early childhood teacher, and so she had a belief system that we had zero television in the house because it would ruin our intellect. I had two lovely sisters and the minute she left the house, we had a roster going. One person would lie on the floor, would come to the curtains and wait for mom to come back and the other two would watch TV and we would rotate.

Iona Novak (00:05:58):

On one of those sort of banned, unauthorized watching days, I saw a film that in Australia won film of the year. It was based on a true story about a child with cerebral palsy. In the era, she was in an institution and she had severe cerebral palsy with a severe physical disability but was misunderstood to have an intellectual disability. When the teacher came to the school, she suddenly realized very quickly that this young woman was actually very bright and actually taught her to read and communicate and ended up adopting here and this young woman with cerebral palsy became a lawyer.

Iona Novak (00:06:35):

I had watched this film while my mother was out at some point, and I decided I was going to work for people with cerebral palsy. I still haven't told mom, but you know, it was a pivotal day for me on the television. So you never know what you can learn on television.

Iona Novak (00:06:50):

As my career progressed, I took that on and I had the privilege of meeting the late Christopher Reeve, an actor who had a spinal cord injury and became a stem cells advocate. And [inaudible 00:07:02] physicians came to Australia for a fundraising effort, which was sponsored by the [inaudible 00:07:06] Premier, which is like the head of state in the United States and I was lucky enough to be part of that [inaudible 00:07:12].

Iona Novak (00:07:12):

And I noticed I was the only one talking about stem cells for the brain. Everyone was talking about stem cell for spinal cord or joint conditions and it really got me inspired. I was talking to scientists and they said, "Well, I don't see why not it couldn't help the brain." So, I got on this journey and when we interviewed families about their research priorities, it turned out, of course I wasn't the only person thinking about this. There were families who were thinking about this and this was the second highest research priority. Yet I found myself in a situation where I couldn't find any scientists in the world that were actually focused on stem cells for cerebral palsy. So that actually led me to work with some of the best people in the world and build a network to accelerate bench-to-bedside research for people with cerebral palsy.

Iona Novak (00:08:00):

I have an inner belief system that if you look hard enough and work hard enough, you can find answers and people with cerebral palsy of course have great insight and have the best insight into what they need. So it's been a really exciting journey for me to be on.

Jen Lyman (00:08:15):

It seems like you've really pushed the initiative forward and took the ball and ran with it. I'm so fortunate to know you and have you on the show.

Jen Lyman (00:08:23):

It seems like, talking about Christopher Reeves and the spinal cord injuries, and I remember when people were first starting to look at it and now it seems like they're everywhere. I go to Whole Foods and just about every product in Whole Foods has stem cells in it and somehow they're going to make my hair not look the way it does or make my skin regenerate so it doesn't have any more wrinkles, or you hear about celebrities injecting them in their lips. How did it go from... I know it wasn't you. You weren't pushing this, but how did go from being a spinal cord injury to all of a sudden, everybody can benefit from stem cells? Is this true? Is there anything behind this?

Iona Novak (00:09:06):

Well, I think there's a lot of hype and there's a lot of hope in the stem cell [inaudible 00:09:10]. So of course, when things are not researched, they're very easy to sell. If they have a hope component to them. We disliked treatments that are sold to people, especially vulnerable people and families with a child with cerebral palsy without any evidence, but it doesn't mean there couldn't be evidence. So, our philosophy has been to further evidence.

Iona Novak (00:09:29):

Of course, the cosmetic industry is very good at revolutionizing new treatments and here in Australia, here in the coronavirus season, the Australians [inaudible 00:09:39] the product most purchased is skincare products. So, honestly we're looking in the mirror more, Jen, or something in social isolation, but these are things that are easy to market and sell.

Iona Novak (00:09:49):

One of my favorite stories is one of the Kardashians getting a vegan stem cell transplant. When you walk that backwards, how is that even scientifically possible? Because don't stem cells come from humans or animals. I mean, they can be in plants, but it's really kind of a interesting half concept.

Iona Novak (00:10:07):

So, I think what's important here is that we cut through hype and hope. There is actually both in the field. What we'll talk about today is some ways forward for doing that.

Jen Lyman (00:10:18):

So, with CP, we've said it already, I mean, the parents, I know we all want try it and, and I'll talk later. Bauer's been part of an FDA-approved stem cell trial. What are the different ways that stem cells might actually benefit people with cerebral palsy?

Madison Paton (00:10:34):

So, classically stem cells can go on to form many different tissues or cells in our body, and they can also self-replicate and make even more stem cells. So that in itself is such a fascinating concept. You might think that you need to replace lost cells or damaged cells, but actually when we start to look at them as a cell therapy, we're looking at them more kind of like a drug.

Madison Paton (00:10:57):

So, what can they do when they enter our system and try and change our environment to make it more reparative or support better function? I guess that's the way we start to look at it when we want to apply it, say in cerebral palsy and for brain conditions. So they have the potential to do a lot of things and it really depends on what cell type we're looking at to understand what outcome we might want to actually try and target.

Madison Paton (00:11:21):

I guess that also comes in with pairing it with the right kind of injury, if I can say that. So if we can understand what's going on, say in cerebral palsy, we can understand how to best apply a stem cell to target the type of injury. That's what makes it so exciting is, because I guess in me being quite vague is the idea that they can do so many different things and it's about harnessing those different effects for the target that we want.

Jen Lyman (00:11:45):

So maybe we can step it back a little bit. Help me understand what are the different types of stem cells?

Madison Paton (00:11:51):

We can break up the different of stem cells depending on where they're originally sourced from. So we have embryonic stem cells, fetally derived stem cells, including cells from the placenta and adult tissue stem cells. We also have this new, incredible form called induced pluripotent stem cells. This is kind of the laboratory stem cells where you can take a, say a skin cell and drive it all the way back to behave like an embryonic stem cell.

Madison Paton (00:12:21):

But as I said, when we actually start to think about the different types of stem cells from each of those different sources and types, there are so many more and they in itself and have a different target and a different function. So really we can be talking about hundreds of different types.

Jen Lyman (00:12:36):

So they come from all over the place in your body and they're as well as fetal and embryonic?

Madison Paton (00:12:44):

And adult.

Jen Lyman (00:12:45):

Yeah, and adult. Then how do you extract them?

Madison Paton (00:12:48):

They're so different depending on the type. My favorite example, I guess, comes from my PhD and some of the work now I do at Cerebral Palsy Alliance. A simple example is say from the placenta. So you've got a physical tissue or say umbilical cord blood and from each of these tissues or blood sources, you can try and break down from a whole unit, whole tissue and you get lower and lower until you can actually try and isolate the different cell types.

Madison Paton (00:13:16):

We know actually what these stem cells, what they express and what's on the outside of their cells. So we can begin to actually understand what they look like try and isolate them out in different ways. This is very complex depending on the type of stem cell, but really what you're trying to do is, extract them from their original source and purify them so you know what they are, and you can get to one or a few cell types and then be able to give those cells back as a treatment.

Jen Lyman (00:13:44):

How do you give them back as a treatment?

Madison Paton (00:13:46):

So, again, it comes down to what cell you're looking at. I guess as a scientist and Iona and I try and understand how we could best give a cell so that they can have their best effects. So most cells or some stem cells that we can apply as a therapy, we can give intravenously, so via a drip, and this enters your circulation. This is one way we're looking at actually administering certain types of stem cells, because we know that they don't necessarily have to go to your brain to have effects. Instead, they can be in your circulation and create this pro-reparative environment, which then in turn helps your brain.

Madison Paton (00:14:27):

Alternatively, you can give it say into your spinal cord. You can implant certain types of stem cells directly into the brain. This becomes, I guess, even more important when we try and understand how we could apply the stem cells. We would want to give cells directly into the brain that would form new brain tissue. So it's about applying certain cells which we know would be appropriate for that. So we can give a neural stem cell say, into your brain, which would then regenerate tissue which might be missing or damaged. That's another example, but there are many different ways you can give stem cells as long as they're paired appropriately with the cell type.

Jen Lyman (00:15:06):

One of the things that I've heard about stem cells is that they can help reduce inflammation. How does that work? It seems like it's not making something new. It's actually reducing something that does exists.

Iona Novak (00:15:19):

So, stem cells can have three main actions and a different cell line to do all of those things. It might do some of them. So the three main actions we think about number one, as you're saying, reducing or stopping inflammation. We know when you have inflammation, it's much harder for your body to repair and other bad things can happen. So in a brain injury, if you're able to reduce inflammation, you reduce the size and the severity of the injury. So that's an important role that stem cells might play for cerebral palsy.

Iona Novak (00:15:52):

In addition, that they might, what most families are thinking about hoping they could repair or replace tissue in cerebral palsy. You'd be thinking about neural stem cells, which are brain stem cells, which would therefore have the capability of making new brain cells, whereas if you had a blood stem cell, it could help with repair, but it's not going to make new brain. Only brain stem cells will make whole brain stem cells. So that's another mechanism of action.

Iona Novak (00:16:18):

Then the third one we think about is something I like to call the Hugh Jackman effect, which is if you have a kind of... The technical science term is trophic, but if you have a barbecue going at your house and you did it every week with your friends, and then say one week, Hugh Jackman just dropped into the barbecue, everyone would start talking differently. "Is that Hugh Jackman? It can't really be Hugh Jackman." "Well, you go and say hello." "Well, I'm going to get an autograph. I'm getting a selfie." All of a sudden, everybody behaves differently because there's someone different at the barbecue. That's what we call a trophic effect with stem cells, that they can actually show up and make other cells and other natural processes in the brain start to do things differently and they can initiate self-repair.

Iona Novak (00:17:03):

So, any of those reactions can happen with a different stem cell, but some cells can only do one action, some cells can do multiple actions. So what we need to understand in cerebral palsy, what is the cause of your brain injury? What things need repair? What processes do you have underway that we could use a cell to initiate repair or work in your case? Because as we all know, no two children with cerebral palsy are the same. So, we need to understand the biologics in order to choose the right cell for you that's going to get the most repair in your circumstances.

Jen Lyman (00:17:40):

Is there any concern about the stem cell coming in and doing something bad, causing the other stem cells to behave badly? Is there evidence to that effect?

Madison Paton (00:17:51):

I guess one of the main concerns that we get asked about a lot, can stem cells come in and form tumors, or much like you said, can they come in and kind of make processes that would normally be okay into a dangerous process and have an effect on other cell types? The risk of this is really low. Again, it comes down to what type of stem cell you're applying. If you give a stem cell such as an embryonic stem cell, they have such great potential to grow into many different cell types. There is an increased risk of that.

Madison Paton (00:18:23):

But when you give cells which are well-defined and have a single purpose or a number of purposes, which aren't to form tumors, which aren't to form those risks, then it's very, very low chance of this occurring. So when applied in a research setting or in a controlled environment where the cell is well-defined, it's well manufactured, we know exactly what it is, that risk can altogether almost be eliminated completely. So, when we start to think about applying stem cell therapy, say for CP, the real concerns actually come down to just general medical risks and how we can actually give the cells rather than the cells themselves.

Iona Novak (00:18:59):

People assume these cells integrate into your body and they're coming from a donor and they integrate. Most of the time for most stem cells, your immune system knows this as a donor cell, and will act to clear the cell. So in some places, some stem cells can do a job really quickly and then your immune system will clear them and there's no lasting, what we call engraftment, meaning integration of that cell into your body. But other cells like a brain cell, if you want it to make new cells, brain cells you'd want it to engraft. So in that case, then your body's going to try and get rid of that cell and you might have to use an immunosuppression drug. So if you thought about like a heart transplant, people will always take the medication so they don't reject the heart.

Iona Novak (00:19:44):

If you were using a brain stem cell, and you want it to make more, a new brain, you'd need to use immunosuppression so it doesn't reject the cell as well. So again, it comes back to the type of stem, what it's capable of doing and what you actually want the cell to do therapeutically.

Jen Lyman (00:20:02):

So, let's talk about the trials that are currently underway that you guys are involved in and we also know about. I'll talk a little bit about a trial that Bauer was in here. We were at, I think it was Texas University with Dr. Cox. It was the first FDA-approved stem cell trial in the U.S. where it was autologous bone marrow. Bauer received his own bone marrow stem cells and we did pre and post MRIs over a two-year period, as well as GMFM and cognitive testing.

Jen Lyman (00:20:32):

It was fascinating because the results were... After he received... and it was a double blind study. We found out about after a year, we found out whether he had received the stem cells or not, but all the children that were in the trial ultimately did get the stem cells. So in his case, the MRI showed that the bilateral white matter increased in his brain after receiving the stem cells. That was at one year, but at two years, it actually went back down a little bit. It didn't go back down to baseline, but it showed a volume increase in the white matter in his brain. It also showed increased myelination.

Jen Lyman (00:21:13):

We thought that was pretty cool, but I didn't really see any other effects. I didn't see any cognitive implications or improvement. I claim to this day, I saw that he was sitting up a little bit better after the trial, but beyond that, not much more. But I've got to say, it gave me hope and I thought, "Hey, we're on the front lines. Maybe there's something to it." And it was nice to have the MRIs to show that, yeah, you know what, there was an impact. I don't know if that study is still going on, or if there's others like it that we could compare it to, but I do know that that's one that we've participated in here locally.

Iona Novak (00:21:52):

Yeah, so Charles Cox's study was a very important study because it compared two stem cells, umbilical cord and stem cells from your bone marrow, just as you were describing, but also compared them to placebo. The reason this study was so important is, there's a lot known about the effectiveness of umbilical cord blood for cerebral palsy. But in most cases during the pregnancy, you didn't know you were going to have a child with cerebral palsy and so you didn't think about storing umbilical cord, which means you're going to need a donor cord.

Iona Novak (00:22:25):

That's one thing why this study was really important because it looked at a source of new brain cells from bone marrow that could be compared. He was aiming to find out, head-to-head, if we can get an umbilical cord, you might be lucky enough to have this as a source of cells that come from your own body that we could readily harvest. So, it's important that studies are listed as finished. They didn't fill all the recruitment and we're waiting for the results first, though we haven't seen them yet but it's really important. Yeah, [crosstalk 00:22:53]-

Jen Lyman (00:22:52):

I know. I know I've been waiting for them too.

Iona Novak (00:22:54):

Oh well, so if you're out there, Charles, we want to know what you've done.

Iona Novak (00:23:00):

So, if we go to thinking about, Madison and I can do a double act to you, but the different types of stem cells from the many that have been considered for cerebral palsy. So the best known one is umbilical cord blood, which is morally and ethically uncomplicated because most people are going to [inaudible 00:23:17] umbilical cord blood away. It's a great product because it contains a lot of different types of stem cells and they might all be acting and working together to produce a result.

Iona Novak (00:23:29):

So, we know there are over 1000 patients with cerebral palsy that have had umbilical cord blood in at least four clinical trials and there are a number more underway. We can see, when we look at children's movement skills, children of all severities of cerebral palsy that have had umbilical cord blood plus rehabilitation have better movement skills than children that have had rehabilitation alone. The effect is small, but it's definitely worth doing and it's definitely worth more research.

Iona Novak (00:23:58):

So, we're now in a place with umbilical cord blood which we Madison and I used to traffic light to code different interventions. Umbilical cord blood is a green light intervention. It's been proved to have benefit to children with cerebral palsy. It's a very safe product. There's over 50 years of safety data and it's one that gives a small benefit.

Iona Novak (00:24:18):

So, of course the next areas with clinical trials that are really important with umbilical cord blood [inaudible 00:24:24]. If it worked once, you're thinking and it wears off, as you were saying, do you need repeat doses for example? Would it be better at higher doses? And no two umbilical cords are the same so some of them naturally have a high dose, some have a naturally low dose. It would depend which cord you're using.

Iona Novak (00:24:42):

So, there are some things about dose, repeat doses, and then of course timing. So if you get a result maybe six years after your brain injury, what would happen if it was six hours after your brain injury? Would the result be even bigger? So there are now researchers working hard on clinical trials on babies born on time with brain injury, but also thinking about [inaudible 00:25:03] babies born [inaudible 00:25:04] premature birth, whether we could give cord blood to them. I think umbilical cord blood is here to stay. It's really basically a proven treatment for cerebral palsy, but there's more work to be done in that space.

Iona Novak (00:25:17):

Then we think about the cells you were talking about, hemopoietic stem cells, which come from bone marrow. They have a number of advantages too because as you experienced, you could use your own cells. For example, there is a clinical trial as well, showing that you get an improvement in movement in children with cerebral palsy using hemopoietic stem cells like a bone marrow transplant.

Iona Novak (00:25:38):

Of course, this is important and that's why Dr. Cox was trying to compare hemopoietic stem cells to umbilical cord cells. But we don't know the fatigue points of the bone marrow [inaudible 00:25:51]. Like how many times, again, if you want to repeat it and higher doses. How many times can you put your body through that regimen of building up the number of cells just to extract them and reinfuse them? Is there a safe number of repeat doses? That's something we've got to think about that, but more research in that space is certainly warranted.

Iona Novak (00:26:12):

Then a third cell we think about is something called mesenchymal stem cells or MSCs. Mesenchymal cells is actually the American pronunciation so I should do that for you so we talk in the same language too. They're actually cells, which come from a number of sources, maybe the fat tissue, [inaudible 00:26:29] tissue, et cetera.

Iona Novak (00:26:31):

They're a great cell because they're what we call immune privileged. So if I give you my cells, you don't reject them. If I take yours, I don't have an immune response. There's no donor problems with them so we don't need immunosuppression, but these cells don't stick around too long in the body. They are very good at building up and cutting down inflammation. So, in cerebral palsy, they might be very helpful or stroke, for example, where you have a big bleed and then a lot of inflammation. If we could give them quickly and that's where researchers are thinking [inaudible 00:27:03] and there's a clinical trial, of course at the moment.

Iona Novak (00:27:05):

Lots of researchers are also thinking about these for COVID-19, where there's a lot of inflammation and these cells could be very quickly given in repeat doses. So that's an exciting space as well for people with cerebral palsy, because it might be a life-saving treatment, but it also is going to cross track regulatory approvals for cells for clinical trials.

Iona Novak (00:27:25):

Then the last couple we might think about, amnion epithelial cells. There is no test at the end of these long names, but [inaudible 00:27:33] the placenta. They're also very good at dampening inflammation and therefore can help protect the brain from injury and they will act on the white matter a little bit like the cells you were talking about. So that's the connectivity pieces of the brain.

Iona Novak (00:27:48):

And are being used in lung diseases in preparation for cerebral palsy it could affect [inaudible 00:27:54]. We're just about to go to trial soon in Australia, trying those with newborn babies in the first week of life to see whether they can protect the brain from an injury.

Iona Novak (00:28:04):

Then the last one, which we alluded to earlier, so neural stem cells or brain cells, and these are key cells for cerebral palsy because they're the only ones that can make more brain cells. They have the capability to build the three building blocks in the brain of the different types of cells that we use to make nerves and connections. So there are a wonderful cell for thinking about brain repair.

Iona Novak (00:28:27):

But the problem with these cells is, of course, I might donate a kidney to someone and can live through that experience. But if I donate brain, I'm going to experience a brain injury. So how do you source these cells? We have [inaudible 00:28:39] thinking about getting them from somewhere else, so maybe a family who had fertilized embryos from IVF that they're not going to use, and they donate them for research scientists. Never ever make embryos to make stem cells.

Iona Novak (00:28:53):

Or maybe from an organ donor, you could get these cells. So this sort of cells we're thinking about using from an organ donor and we can expand them. [inaudible 00:29:01] have been working with a company and expanded one donor cells for the last 15 years and they're still expanding them. So just one single donor is helping hundreds of patients.

Iona Novak (00:29:10):

So, there has been trials, two trials of using neural stem cells for cerebral palsy and have shown preliminary effects. Now, but what we learned from these trials is that these cells are just not good travelers, that if you would use a less invasive administration method, like an IV line or drip, or you put them into the spinal cord and then hope that they circulate, they just don't move very fast or very well. They only move when they're on brain structures, which means... and they can't cross the blood-brain barrier very well.

Iona Novak (00:29:45):

So, we all have a gate around our brain that keeps bad things out, but in this case, it's keeping the neural stem cells out. So you actually have to directly place these cells into the brain. So a neurosurgeon has to do that, taking an MRI and building a brain model and very carefully, right to the millimeter practice... they can practice on a robotic model and then place the cells exactly where you need them to be. Then once they're in the brain making [inaudible 00:30:12] around it and get to the right places and start making repair.

Iona Novak (00:30:16):

So, we are in the process of activating a trial using these stem cells where they're placed into of children with cerebral palsy and this is important work. As you can see with each cell line, whilst it had some benefits, there are some limitations. So Madison and our philosophy in the network we work with, we shouldn't put all eggs in one basket. We should be pursuing all these different cells and it's why head-to-head comparisons of different cells is really, really important.

Jen Lyman (00:30:49):

Back to the placenta cells. Are you seeing that families now, when they're going into the NICU or when they're having premature babies, they're more and more requesting, "Hey, we need to save this. We need to save the umbilical cords." I would kill to this day, had we saved Bauer's. After he was born, we had my sister-in-law save hers, my sister saved hers, everybody in the family's got theirs and I don't have mine. Are you seeing that now families are aware of this and NICUs are aware of this and they're taking action and it's becoming a frontline of defense?

Madison Paton (00:31:24):

Yeah, definitely. I actually used to work in hospitals collecting umbilical cord blood for research. But if it wasn't for research, we'd always get families asking like, "Can I give it some way? Can I give it to the hospital? Can we put it in a bank? Can I bank it myself?" So you can speak to all the private banks as well. Business is booming. The more and more this research is coming out, the more people are wanting to say, look at cord blood as an insurance policy, say as a treatment down the line.

Madison Paton (00:31:53):

But I guess the place that Iona and I are working in is that once cord blood, as we're seeing, is being proven as safe and effective, then it's really not fair for families to have to feel this regret that they never collected or stored cord blood. It should be made available to everybody. That's the way it is really when a new drug gets proven safe and effective. It's just given to you because it's safe and effective.

Madison Paton (00:32:19):

So, I'd like to see us moving to a different place where we can say access samples which have been donated or that we start to store more routinely so that this is an option and people don't actually have to fork out the money themselves or for this to be a consideration around the most stressful time in your life when you are pregnant and expecting a baby. And that's where I would like to see it go.

Iona Novak (00:32:42):

And the point Madison was making we've been doing advocacy work to loosen up the rules. So of course, when the umbilical cord for cerebral palsy, [inaudible 00:32:50] are going to use it in their children's own [inaudible 00:32:53] because that was of course deemed safe, no rejection problem. Then there was studies that tried a donor cell, and a lot of them started with a sibling or a relative so that there were a good match.

Iona Novak (00:33:04):

Then of course, now there's some studies looking at more remote donors or unrelated donors. Those studies are still showing good results. I think what's complex about this cell is, it was originally used in the cancer field and so you had to take away someone's immune system so they didn't reject these cells. So everyone's concerned about people's immune system with cerebral palsy, but we have to remember, these children are healthy. They have a healthy immune system. They can manage this. So really we think donor cells is the way to go and that will improve access for everyone.

Iona Novak (00:33:38):

So, there's a real need to improve the legislation about cord blood administration around the world. It's sold a lot privately in clinics and in high quality clinics, it's probably a great product. However, it really should be as Madison is saying a universally available product like a drug, because there are enough samples worldwide in stock for this to be used as a product.

Jen Lyman (00:34:02):

Fascinating. So I'll tell my sister to keep her as where they are because there's a chance, right?

Iona Novak (00:34:09):

Absolutely.

Jen Lyman (00:34:09):

Where can parents and where can families find trials if they want to enroll their children in clinical trials? How would they go about finding them and getting them in?

Iona Novak (00:34:20):

Yeah, there's a couple of good places to look. Something called clinicaltrials.gov is the major American website that lists registered clinical trials. There is also a website called patientslikeme, which pulls from all the clinical trials around the world. So if you put cerebral palsy in there, then it will pull from all of the different websites what trials are available all the way around the world.

Iona Novak (00:34:47):

I think what people often find is that it's still scenarios where they would be willing to be trial participants but there aren't still enough trials. And that's part of the advocacy work we're doing to try and get more funding available. Happily in Australia, 150 million was dedicated to stem cell research to accelerate clinical trials here. But we still all need a lot more money to make these things go to trial.

Iona Novak (00:35:14):

But I think it's time for a [inaudible 00:35:17] shift in the science and healthcare community who are very good at protecting the lives of people with cerebral palsy, but this is a new frontier medicine. People have been skeptical because of like you said, the hype that has existed as well that maybe we're selling snake oil. I think good quality trials that are free for participants to enroll in, that have good solid measures that are scored by people that don't know whether you got the placebo or don't know if you got the cell and all of these sorts of things that remove what we call bias in research and improve the likelihood of us being able to believe the results. Those are incredibly important for people with cerebral palsy. We're learning so much from these previous brain pioneering trials about how to do trials well. The space is wide open now and a lot more trials need to be done.

Jen Lyman (00:36:11):

This is exciting. This is exciting. So let's move on to the opposite of that, stem cell tourism. Let's talk about that for a little bit, because I do know families also who have traveled to Honduras. My son's physician has told me a story of a family that went to China and came back and the child passed away because of whatever procedure occurred with the stem cells there.

Jen Lyman (00:36:33):

I know that families spend a lot of money to do this. How do we tell the difference between stem cells tourism? I think you just explained to us what the science does. What does the tourism industry do that differs from science?

Madison Paton (00:36:46):

When people will travel overseas for stem cells, and that's what you refer to as stem cell tourism, they're often seeking treatment from a private clinic and they function really quite different to research. So the key differences are with stem cell tourism, often, as I said, it's a for-profit private clinic that are offering a therapy. This therapy usually has quite limited oversight, regulatory oversight. It's charging for a product and so you're buying into an industry much like you are when you go shopping.

Madison Paton (00:37:23):

This is really contrasted to the research, which is built on trying to answer rigorous research questions, which then contribute to the greater good. These are free of charge. They try to enroll people into trials to answer questions, which then build upon the next answer and the next answer in hopes of getting a safe and effective treatment, cure, therapy out into the world.

Madison Paton (00:37:47):

we understand that this process, this scientific process is slow. So, therefore I guess people opt into that stem cell tourism branch, because they need other options and this is their way to do that and explore those options. I guess, in that though, people are putting their lives or engaging in some sort of risk because there are so many unknowns around that. We have many different ways and strategies that we can help direct families to try and identify the differences between research and then stem cell tourism and then also then weigh up that benefit risk so that they can make better informed decisions.

Iona Novak (00:38:25):

I always like to send families to the Australian Stem Cells Handbook, which is a free download off the internet, which has a lot of really good, open-ended questions. The [inaudible 00:38:35] Stem Cell Center has one as well. What it allows you to do, if you're thinking about buying from one of these private clinics, it's asking you questions because the marketing is pretty good from these clinics. But sometimes as a scientist, if you pick through the marketing texts, they use a lot of times words, which make you as a customer assume that their product is proven and think because they're using scientific language, it sounds like a highly efficacious product.

Iona Novak (00:39:06):

But in most of these cases, these clinics are not producing any research. So these open-ended questions good things to ask before you consider buying from these [inaudible 00:39:16]. For example, are the cells you're giving me human cells. So it's important not to make assumptions about [inaudible 00:39:24], right?

Iona Novak (00:39:25):

And it goes through a range of cells. Will you monitor me afterwards? If I had this cell overseas, and I have an adverse event or a side effect when I get home, will insurance coverage or Medicare Medicaid coverage to cover the results? Or what I put my family in further financial risk?

Iona Novak (00:39:44):

So, these are important things to weigh up in addition to what the hope of the cells might be, [inaudible 00:39:51] what the consequences might be as well.

Jen Lyman (00:39:54):

Absolutely. That's great. I'm going to put both of those resources on with the show notes, as well as earlier, you mentioned clinical trials.gov and patientslikeme. That will all be on the show notes for the show.

Madison Paton (00:40:06):

I was actually going to bring up then how wonderful that paper is then on crowdfunding because I guess in that is that we often talk theoretically in this space because we don't often get to hear from many families that do travel overseas and engage in this stem cell tourism. They are taking some sort of risk or they are investing financially and they don't often want to come out and openly speak about this.

Madison Paton (00:40:30):

So I was able to speak with all of you guys and present actually at a conference last year and I brought up this wonderful paper that we found. It was by Snyder et al that came out last year. Really the only kinds of evidence that we're getting in the CP space for stem cell tourism comes from crowdfunding. So I'm not sure if you wanted me to bring this up, but as a researcher, I got to read this. I was like, "Oh my goodness, people are analyzing stuff."

Jen Lyman (00:40:54):

This crowdfunding paper was really exciting to hear about when you talked about it last year and it was remarkable to me how much money people were raising.

Madison Paton (00:41:02):

Yeah, I'll say this wonderful paper, it analyzed all posts that came up from YouCaring and Go Fund Me and so in the crowdfunding space. They wanted to understand for brain conditions, what were families seeking treatments for and also what were they trying to fundraise and where were they going? So, this is kind of our best early evidence of an analysis of say stem cell tourism in the CP space.

Madison Paton (00:41:29):

What they found is, that to date there have been over a thousand posts and people reaching out to generate crowdfunding for many different types of brain conditions and that cerebral palsy is actually the fourth, most popular brain condition to seek crowdfunding for, which just floored me. I don't know about you, but when I read that fact, I was like, "Okay, I need to keep reading this paper."

Madison Paton (00:41:54):

I guess the biggest standout was that on average, people who were seeking crowdfunding for cerebral palsy were looking at trying to raise over $27,000 American. That's just a huge amount. I don't know about you, but I don't have $27,000 in there. I can't even imagine what I would buy with $27,000.

Madison Paton (00:42:16):

When I actually read this and sent it to Iona and we chatted about it, the very first thing we thought about is, "Do we even spend $27,000 for each participant in a research trial to receive cells?" So I know that's a bit of a science mind and put my science hat on, but that's what we thought. Imagine if people could be guaranteed a safe treatment for that price where they didn't have to go online and fundraise, it really puts it all into perspective and the lengths that people will go to try new treatments for that hope.

Jen Lyman (00:42:45):

You're absolutely right. If insurance would pay for this and we could go through and have the cells that we need when we need them and for the right reasons, that's why we do the research., or that's why you do the research. It was a remarkable paper when you first discussed it. I was blown away and I've had that $27,000 in my head for about a year now.

Madison Paton (00:43:04):

Yeah, I think about it all the time every time when we sit down to try and budget for a trial or a new treatment. It really puts things into perspective.

Madison Paton (00:43:14):

I guess it also just shines a spotlight that more work needs to be done like this and that's something in which we're investing a lot of time in as well as to explore people's experiences and understand if they felt that it was also worthwhile. Then after investing all of this money, if they saw some benefit or if there was an adverse event or there were risks, because I think we also need to just generate more dialogue fullstop.

Jen Lyman (00:43:38):

Absolutely.

Iona Novak (00:43:38):

Yeah, and the economic argument is important because not only the burden on families, but if we don't invest in cerebral palsy research, there's a chance really that different places will find effective treatments and our governments will be forced then to buy cells from private companies overseas in order to license those products. So there's an economic loss for the country not investing in research as well as a financial loss to families. Because it could turn out that these cells have worked up in scenarios where the price point for the cell is too high for our health system or an insurance. So it's a collective project that needs to work at a governmental level as well, for ensuring that stem cells are found in a way that return a commercial benefit to the country so that they're an affordable product longterm as treatments.

Jen Lyman (00:44:29):

Well, that's a fascinating point because it makes me think about the pharmaceutical industry and the amount of money that they put into researching drugs and creating drugs. This is something that comes from our own bodies so it's almost competing with the pharmaceutical industry in a sense.

Iona Novak (00:44:45):

Yeah, I think about it like a parallel universe really. As Madison said, you could think of stem cells almost as a drug. The thing is that they're newer. And so we find in the research base some of the legislation was written around drugs, so assuming for example, that there was a lot of financial backing from the pharmaceutical company when you go to trial and it's their responsibility to check safety [inaudible 00:45:10] but stem cells are not really owned by pharmaceutical companies. They're actually usually developed by scientists. So they don't have a large backing behind them to get to trial.

Iona Novak (00:45:20):

I think all the assumptions that's in operation around research is actually holding it back because how is a scientist supposed to find a couple of million dollars to go to trial? So it's a complex area that needs really rethinking.

Jen Lyman (00:45:34):

Well, let's move on. I have some questions from families for you guys. I'd like to start out with the term regenerative medicine versus stem cells.

Jen Lyman (00:45:43):

The question was, is regenerative medicine an umbrella term and stem cells part of it? Can you elaborate a little bit on that?

Iona Novak (00:45:51):

So, that's how I think of it is, regenerative medicine is an overarching term for repair. Repair of the brain is particularly what we're interested in in cerebral palsy so that might be done through a stem cell, might be one type of regenerative medicine. It could be done through gene therapy. So if you have a genetic pathway to cerebral palsy, then a gene therapy might be even more important than a stem cell therapy.

Iona Novak (00:46:16):

It could be through a pharmaceutical agent. So for example, [inaudible 00:46:21] regenerative medicine trial where babies that have a brain injury at birth and we're giving them a drug called erythropoietin or EPO, some that Lance Armstrong was a little bit famous for. It helps you make red blood cells and start the repair process in the brain. So, there are different categories of things that could be regenerative medicine. So yes, I'd say stem cells is one type of regenerative medicine.

Jen Lyman (00:46:44):

Got it. Thank you.

Madison Paton (00:46:45):

I was going to say too, for cerebral palsy, we start to actually talk, or our field, we are now moving actually more to the term just cell therapies, because as Iona said, it's starting to encompass so many more things that are outside of just regen med. That's the terminology we're starting to move towards, even dropping out the regenerative aspect and even the stem of the stem cell aspect broadens it out even more because that's really where we're working now.

Jen Lyman (00:47:11):

So, a new question and on top of the research. How are the studies at Duke going that Dr. Kurtzberg has been involved in and she was early famous for it early on with the cord blood studies? I saw her present years ago, but this came from one of the families and what have they learned?

Iona Novak (00:47:32):

So, Duke University and Dr. Joanne Kurtzberg is undoubtedly the world leader in umbilical cord blood for cerebral palsy. Her pioneering work has made extraordinary difference in this field. The first people that actually published were in South Korea using a seminal cell structure, and they had some beautifully high quality design trials and then had two studies that showed a clinical benefit.

Iona Novak (00:47:54):

Dr. Kurtzberg's study had even more patients in it and they didn't find as big a benefit as they found in Korea. However, they made an important finding about dose. They found that cords that had naturally a higher dose, there was a threshold dose of where children with cerebral palsy start to experience a benefit. If the cord was naturally under that dose then the children didn't have a very [inaudible 00:48:18].

Iona Novak (00:48:19):

So that's probably the most important finding from their cerebral palsy study to date, which allow us as researchers if we're starting to think about repeat doses for cerebral palsy and high dose, and how many cords do you need depending on the child or the adolescent's body weight. It tells us where [inaudible 00:48:37] therapeutically start. So that's really an important finding that they've made in the field. They continue to pioneer new areas with stem cell. There are many people also working in that space around the world now.

Jen Lyman (00:48:48):

Well, this one's a little one-off. How about baby teeth? For those of us that saved our baby teeth, can we do anything with them?

Madison Paton (00:48:56):

Yeah, so actually what you're referring to is dental pulp stem cells. So they're the cells that kind of hang off the bits of tissue, which are left on the teeth that fall out after you lose a tooth. Iona brought it up before actually, they're what we refer to as a mesenchymal stem cell. They're the most well-studied stem cell across a range of different brain conditions.

Madison Paton (00:49:20):

There's lots of different research that's gone into mesenchymal stem cells. Quite honestly, those dental pulp or those baby teeth cells aren't necessarily the best kind. They're in such low numbers and we can get them from other leftover waste, say from pregnancy, from placentas in such huge amounts rather than those cells in your teeth, but they're still being explored because they're thrown in the bin as well and so they're definitely something which is still being explored and researched. But I don't know if I would pick them out of the bin and keep them in my back pocket any time soon.

Jen Lyman (00:49:53):

So, I shouldn't feel bad that I bleached Bauer's down and I have them in a little thing next to-

Madison Paton (00:49:58):

No, please don't feel guilty.

Jen Lyman (00:50:02):

Then the sibling and the cousin cord blood. That was another question, but I think we answered that earlier. What about age limits to effectiveness? Are you guys seeing age limits? I know, I think about puberty a lot and that being a time of brain growth and brain development. And can you still have stem cells?

Iona Novak (00:50:20):

Yeah, so age limits is a really important question. The first one Madison, I think [inaudible 00:50:24] answering that question is, it depends on what type of cerebral palsy you have, what injury is in place and what mechanisms are there for a stem cell to work on. And how long after the initial injury is it still in an injury [inaudible 00:50:39]?

Iona Novak (00:50:40):

So, what do I mean by that? So if you thought about inflammation and you wanted to use a stem cell that has anti-inflammatory properties, how long did the inflammation last? Well people thought initially it only lasted a very short time, like a week or so after a brain injury. But we now know it persists for many years with cerebral palsy. So if you have a type of cerebral palsy that has inflammation that is persisting for many years, you might well respond for a long time to a stem cell that has anti-inflammatory properties.

Iona Novak (00:51:11):

But if your pathway to cerebral palsy was genetics and there's no inflammation present, then you wouldn't respond to the later point. You'd be much better pursuing something that actually is maybe going to work on conductivity because low conductivity is one of the issues that happens from a genetic malformation. So again, it's going to come down to what kind of cell and what kind of injury you have.

Iona Novak (00:51:33):

But we do think neural stem cells, the ones we talked about that are [inaudible 00:51:36] those may well be the best candidates for use in the long term, because they are going to work at any age for reactivating and building new tissue.

Jen Lyman (00:51:46):

That's very exciting. Iona this one is for you. And it's actually from Dr. Amy Bales. She and I were talking about the GMFCS on the last podcast. One of the questions that we had was, what percentage of individuals with cerebral palsy could be a GMFCS I and have quadriplegic CP? She said you wrote a paper that had a beautiful pie chart and we were curious if you remembered this answer. Totally off the topic, but had to ask.

Iona Novak (00:52:16):

Yeah, great. So GMFCS [inaudible 00:52:19] is the type of cerebral palsy where you don't usually walk independently as your main form of mobility. Accidentally for a long time, people have assumed people with the motor type, spastic quadriplegia, so where four limbs are involved, or dyskinesia where all four limbs are involved, automatically would mean because the brain injury is so big and often severe that you therefore can't walk.

Iona Novak (00:52:44):

However, 25% of people with four limb involvements, spastic quadriplegic, or dyskinesia actually do walk. So what that tells me is that it's really, really important, especially in the early years when we can't tell if any baby is going to walk. We know humans take 12 to 14 months to learn to walk. It's a skill that they get from practice unlike horses and dogs that can walk on day one of life. So, it's really important when we're thinking about early intervention for children with cerebral palsy and stem cells that we start to think, "Okay, so it looks like four limbs are involved. So, would you be in the 75% that can't walk? Or maybe [inaudible 00:53:20] one of these people in the 25% that could?" So, early intervention opportunities are really, really important because we don't know that answer definitively on day one which group you're in.

Jen Lyman (00:53:35):

We were going back and forth with it on the last one and we just decided to go to the source. I appreciate it.

Jen Lyman (00:53:40):

I wanted to talk about your amazing systematic review. It just came out about a month ago. It was the second one. I think the first one was in 2013 and I remember reading it back then and being blown away, based on your traffic light system. It's called State of Evidence: Traffic Lights 2019. Systematic Review of Interventions for Preventing and Treating Children with Cerebral Palsy. It's so beautifully written. It's, I feel like a must-read for all families and every single therapist and doctor out there. Both of you guys are authors. Could you all talk a little bit about it and I will include it with the show notes?

Iona Novak (00:54:15):

Thank you for this introduction. This is a topic that Madison and I are very passionate about. We find that there is so much information and treatment choices out there for families. How do you know which one's the right one for your child? Should you be doing all of them? And I meet so many families that feel guilty, like when we say how's that going? That often the person says, "Well, I haven't done enough," in this sort of apologetic way with parental guilt.

Iona Novak (00:54:39):

But in fact, maybe you have done enough. You need answers, but depending on what type of treatment, how much of it should you be [inaudible 00:54:46]. So the aim of the systematic review was to have a look at everything ever published about interventions for cerebral palsy and transect them using a traffic light. So green means that there's good quality evidence proving that this intervention works. Red is the exact opposite. So a good quality evidence proving that either it has no benefit over no treatment or actually causes harm.

Iona Novak (00:55:11):

Yellow is everything in the middle, could be like stem cell that's just being sold, but we don't know if it works, if it doesn't work. It could be that there's promising evidence, but it was a small number of patients and we would like more research to feel confident about that treatment. Or it could be that it's not really showing any gain, but there's flaws in the research and more research would help us understand. So it could be lots of different reasons.

Iona Novak (00:55:35):

What we found was, there's over 180 different treatment options for people with many, many different things. So some treatments aimed to do one thing, some treatments aim to do many things. So for example, one treatment might aim to just reduce the spasticity [inaudible 00:55:51]. Another study might aim to reduce spasticity plus improve movement. So, we tried to have to look at, can one treatment do one thing, or does it do many things to try and clear the story a little bit for families? So, we hope it provides a bit of a mind map or a helicopter view of what to do.

Iona Novak (00:56:10):

I would say to families out there, as we said before, no two children with cerebral palsy are the same. So when you think about this paper and have a look at it, if that doesn't fit with what you know, we are no way making any criticism of the choices parent are making. And it is possible when you look at the data that this is the average of how people with cerebral palsy performed in a clinical trial. There are always children that do better than the clinical trial and there are children that sadly, and unfortunately don't do as well as the average in the trial. So there are people on both ends of the continuum. So if it doesn't fit your story where you, we make no judgements.

Iona Novak (00:56:48):

But on average, if you're thinking about, should I do some of these things, the paper gives you some guidance. What's my goal for my child? What is it my child is saying they want to learn to do? Then you can look through the list of options and you can hopefully just pick a green light that's there that will help you reach your goal. That should speed up the amount of time that you're spending on intervention and therefore give your child and you more time to have a leisure and play time, do the things that matter in your lives.

Jen Lyman (00:57:15):

Absolutely. And decrease the anxiety and anguish and help you participate. It's such an exciting article. I've really enjoyed it.

Madison Paton (00:57:23):

And it includes stem cells now.

Jen Lyman (00:57:25):

It's exciting. It's very exciting. So I want to close out asking you guys, and I think I know the answer, but Iona, what do you think is going to have the biggest impact on cerebral palsy in the future? Is it legislation? Something in research? Education? Where do you think we're going that's going to have the biggest impact?

Iona Novak (00:57:44):

Well, when I trained I was told cerebral palsy was unpreventable, incurable and practically untreatable. I think we're starting to put a line through every single one of those words. First of all, in Australia, just a year and a half ago now, we announced a reduction in the rate or the number of people with cerebral palsy by 30%. If you thought about a 30% reduction in cancer, that's extraordinary, but a 30% reduction in a permanent brain injury is medically extraordinary. So the idea that it's unpreventable is simply not true. We now know some ways to do it, and we'll be learning more.

Iona Novak (00:58:20):

Madison and I are working hard in this space about whether a cure is even possible. There isn't one right now we have no promises and no treatments to offer. However, there are a number of things that look worth pursuing with more research and stem cells is certainly one of those. And if we were looking for a way to repair brain, stem cells is one of the best candidates for doing that.

Iona Novak (00:58:40):

As you talked about before, I think with the traffic light paper, there are many treatment options for cerebral palsy and 16% of them got a green lights and including one of them was a stem cell. So, there are a number of things out there that help people managing their lives to have full lives.

Jen Lyman (00:58:56):

Madison, how about you?

Madison Paton (00:58:58):

Yeah, well, I think Iona said it best, but I guess mine is a little bit different in that it's on the same line, but I guess to fast track these new treatments, it's all about collaboration. So to me, I think what will have the biggest impact on CP in the future is actually all of us pushing forward with the clinicians, the scientists, the consumers, our larger communities.

Madison Paton (00:59:21):

As you say, you've made this podcast to try and kind of make sure that families are kept up to speed with things. That's becoming more and more prominent and it's excellent. I guess it's important to make sure the clinicians aren't left behind as well, and that we're all doing this together at the same pace and this is how we're going to create the biggest change at the most rapid pace that we can.

Jen Lyman (00:59:44):

Well, I hope we can all work together to influence that. I'm certainly doing my part, trying as hard as I can. I think the world of you both and all of the researchers out there. I'm just in awe of everything that is happening in research. To know that you guys are there for my son, it's just heartwarming to me to know that y'all are over in Australia thinking about these things that will ultimately impact Bauer and impact so many people with CP and I'm so grateful for everything you guys are doing.

Iona Novak (01:00:12):

Well, it's a good thing for us to say thank you to you, Jen. You're an amazing mother and advocate and person that brings evidence to families and without people like you, this filed wouldn't continue. So thank you for everything you do.

Jen Lyman (01:00:25):

Thank you.

Madison Paton (01:00:26):

And I hope I get to see you guys in New Orleans in September, if y'all are allowed to leave your country.

Iona Novak (01:00:33):

We hope the travel ban gets lifted.

Madison Paton (01:00:36):

Otherwise virtually. We'll see you on Zoom.

Jen Lyman (01:00:38):

Right. Well, I would also thank my producer, Greg Tilton. He will take out all these ums and clean it up for us and make us all sound a little bit smarter. And I really appreciate Madison and Iona for joining me today and I hope you guys find this to be a conversation that counts with experts who care. Thank you.

Jen Lyman (01:01:03):

Thanks for listening to the Cerebral Palsy Health podcast with me, Jen Lyman. If you enjoyed the show, please subscribe wherever you listen to your podcasts and follow me on Twitter and Instagram. You'll find the links in the show's description.

Jen Lyman (01:01:15):

Please feel free to email me with comments, questions, and topics you'd like to learn more about at jblyman@mac.com. This podcast is for educational purposes only. This podcast is not a substitute for a medical doctor or any other medical provider. This podcast is provided on the understanding that it does not constitute medical advice or services. We encourage all of our listeners to have an open, honest discussion about the topics presented on this podcast and or any other medical concerns with their personal medical team.

 

Madison and I are working hard in this space about whether a cure is even possible. There isn't one right now we, have no promises and no treatments to offer. However, there are a number of things that look worth pursuing with more research and stem cells is certainly one of those.

Iona Novak