Regenerative Medicine Fights Congenital Heart Disease with Dr Timothy Nelson in 14 minutes

Introduction (one innovative approach to treat congenital heart disease )

In this interview, Alan Olsen, CPA, MBA discusses how regenerative medicine can fight congenital heart disease with featured guest Dr. Timothy Nelson, MD, PhD. Dr. Nelson is a general internal medicine practitioner, with a specialty interest in regenerative medicine and the director of the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome at Mayo Clinic, Rochester, Minnesota

Transcript:

Alan Olsen

Hi, this is Alan Olsen and welcome to American Dreams. My guest today is Dr. Tim Nelson. Tim, welcome to the show. Hey, Alan, great to see you. Thanks for having me today. So you’re working on some very interesting technologies, state of the art bleeding edge. And for the listeners here, can you give us a part of your, what brought you up to where you are today? And in your current projects and research?

Tim Nelson

I’m a physician scientist that has always dreamt about taking care of kids with congenital heart disease and rebuilding hearts. And I have been able to be part of a team that has now developed some world’s first technology, that we can actually envision a day, because we’re doing it today, where we can start rebuilding patients hearts from their own tissue.

And so this is science fiction stuff, Alan, that the idea of being able to create organs and tissues and transplant them into your body to make your body younger and healthier and stronger, is literally what we’re doing. So as a physician scientist, being part of a journey like this with a team of talented researchers and scientists, it’s really a remarkable time to be in biomedical innovation right now.

Alan Olsen

Can you give a an overview of what congenital heart diseases and why it has been such a significant medical challenge?

Dr. Timothy Nelson

Yeah, so you and your listeners may not fully realize that congenital heart disease is the definition of it is, babies born with missing heart parts. So babies are born with a defective heart because of a valve because of a heart mass, something is missing when they’re born. What people don’t realize is that is affects one in 100 live births in the United States. And that number is one in 101, and 110. These are the numbers across the globe for centuries, if you will, that heart problems are relatively common one in 100, that means you know somebody else and whether you know it or not.

But it’s also a rare enough to the number that big pharmaceutical companies, device companies, the entrepreneurial community doesn’t necessarily get behind it. Because it’s a small market, there’s bigger markets to solve in healthcare. So that has led to a Rare and Neglected disease like congenital heart, not ever been the focus of new technology. And that’s what our group at heart works is fundamentally changing.

Alan Olsen

And you mentioned in past interviews, that congenital heart disease has been a neglected field of medicine. Why is that?

Dr. Timothy Tim Nelson

Yeah, I would say largely, it’s because of the market size, right? Largely, it’s because there’s too few of patients to be able to generate enough corporate interest to get behind the product development. But it’s also true, if you look at the NIH and other funding sources, from governmental agencies, that pediatrics is a small population percentage wise, and gets a smaller percentage of funding, then then more common diseases like chronic heart disease. So you know, there’s multiple things that stack up.

But ultimately, it’s probably simply because of the market pressures that it’s, it’s rare enough that, you know, like most rare diseases, they don’t get funding. Now, what’s really scary, Alan is that if you add up all of the rare diseases, all of the rare diseases together, one in 20 of us, one out of every 20 people listening to your podcast, are personally affected by a rare disease. So they’re rare as individual diseases.

But we as a population are affected by more and more of these. And so this is really important to not only develop the technology, to cure diseases, but to develop a system that allows us to focus on on rare neglected diseases as a pathfinder to bigger markets.

Alan Olsen

What is spare you to pursue research in regenerative medicine? And, you know, basically as a potential solution for congenital heart disease?

Dr. Timothy Nelson

Yeah. So I was trained in to be a heart surgeon. And I’ve told this story a couple of times before that. I remember in medical school, doing a very complex surgery that had only a 50% survival of a three month old baby, and I was part of the team as a student. did the surgery we got out of the O R, and the baby did well. And I remember being part of that team very, very proud and very excited that that this child did so well in surgery, despite the odds being stacked against them.

And then within that same week, I was in the clinic with another care team, seeing a four year old that four years previously had the same surgery done that we had just done. And now that four year old, we were telling them that we had no options that your heart was failing, the pump was failing. Despite everything we did for the last four years, there’s nothing more we can do. And I and I can remember to this day like it was yesterday, that sense of guilty feeling I had in my gut, that, what did we really do? Did we really cure this disease?

Or did we just delay an inevitable, and I’m not taking anything away from those four years for any day that we can give back to a family with severe congenital heart. But as a provider, I can tell you, what became very motivating to me is we have to fundamentally think differently, to have a different outcome. And just doing traditional surgical care models is simply not good enough for patients that we’re taking care of today. And how are we going to do it differently, we have to get involved in product development, research and development.

And if we’re going to make this sustainable, we got to build a business model around it, to be able to bring it to patients, we can’t let this just be an academic exercise. And so that really at the core of who I am, has driven me to to be in a role in a position of at heart works to be able to do what we’ve dreamt about doing.

Alan Olsen

Are there any recent breakthroughs or promising developments in regenerative medicine at Hope, particularly promise for congenital heart disease?

Dr. Timothy Nelson

Yeah, I know the big one is, in this last year, we’ve got the world’s first FDA permission to be able to take a piece of your skin and train it to become your heart muscle. And so it’s literally a nine month process that we put a piece of your skin through in the lab, we converted into stem cell like cells first, we then train those cells to become your beating, contracting heart muscle.

So we literally today for the world’s first time ever can create heart tissue that’s genetically identical to your body, we can make it in the lab, and we now have a clinical trial that is open for enrollment at Mayo Clinic in Rochester, Minnesota, to be able to transplant those cells back into your heart. Think of it as as a rebuilding a small engine, right? Put in a new cylinder and, and putting the new rings and pistons into the engine. To get more horsepower out of that old motor, we literally are building the cells that we can rebuild the patient’s heart.

And this is only possible because of the work that’s happened in the last decade. And then it’s now available for the first time in the US within the last couple of months.

Alan Olsen

It seems like the miracle here. When you hear how harder heart works and number beats per per minute, day, you know, weeks and, and and a lifetime. You know, we often take for granted as we live through life, how hard the heart works, but that first beat you know, how did that how did you guys come across not only creating the DNA, you know, the tissue, the heart tissue, but getting that first beat to stay in place?

Dr. Timothy Nelson

Well, you know what, what it really comes down to is embryology and we all did this some better than others as an embryo. And in our mother’s womb, we all created the heart muscle beating for the first time that created the blood flow for the rest of our embryonic development to happen normally. So the cells that we’re making in the lab are reminded to look and feel like we did when we were an embryo. And with that we are we’re stacking the odds in the favor of these cells becoming heart muscle. And when you look under the microscope and see the beating heart muscle for the first time.

It’s It’s remarkable. And I can tell you, I’ve seen it 100 If not 1000 times and every time I see it from a new patient sample, that beating heart muscle under the microscope. It’s pretty creepy. And it really allows you to imagine what we can do it those basic building blocks of the heart muscle cells that we can now re muscular arise and rebuild Have the hearts even from potentially 89 year olds that have failing heart muscles. So this technology of regenerative medicine and rebuilding the heart is going to change dramatically over the next 10 years.

Alan Olsen

At what stage? Are you now with clinical trials? And what are the expected outcomes? Yeah.

Dr. Timothy Nelson

So as some people might not know that, you know, to develop a technology with the FDA, you go through three phases. Phase one, phase two, phase three, phase one is really just showing that it can be done safely and effectively. That’s where you start. These are the patients that we are enrolling that have never been before, right. So Alan, I don’t know, have you ever been asked to do a medical procedure that’s never been done by anyone ever before?

Alan Olsen

And no, no?

Dr. Timothy Nelson

What would you imagine feeling like when I am talking to you about being part of of a medical procedure, a surgical procedure of transplanting a part of your heart back into your heart to make it stronger? And you ask me the question, Is this safe? And I tell you, I don’t know. I’ve never done it before. What would that make you feel?

Alan Olsen

Well, I guess if I was on my last leg, I would say yeah, you might as well it no other options here. Yeah, but I bet the points well taken. So

Dr. Timothy Nelson

so what we’re doing right now is we’re asking for those pioneer families, to consider doing something that nobody’s ever done before in the history of mankind. And so we are in discussions with families right now. We are we are dependent on their contributions and participations to be able to move it forward. They’re dependent on the trust of our team and the experience of our team to be able to be willing to do that. And we’re working through that we literally are working through that right now, as we speak, Alan, to be able to offer this for the right patient at the right time.

But you got to start somewhere. And you can imagine that if this goes as well as we hope it could go, that there’s millions of patients out there that could potentially benefit from this type of technology.

Alan Olsen

What advice would you give to patients and their families who are interested in regenerative medicine as a potential treatment option for the congenital heart disease? I mean, right now, the likelihood of success is unknown. So sir, is there a certain type of candidate that you would consider another type that you would not?

Dr. Timothy Nelson

Yeah, so the patient’s just like what you said, if you were, if you had no other options, we are we are searching for those patients that have heart failure, because they were born with with a bad defective heart. That wasn’t normal. And those are the patients that we can enroll in that clinical trials today. And so if you do have a listener, if you know, somebody, we would just encourage you to reach out to us. And let’s have the conversation because everybody’s unique, everybody’s different. There’s probably about 2000 of these patients living in the United States right now that we are trying to engage in this type of research.

So it takes all of us. And I’m appreciative of you asking that question, because all of us know somebody that knows somebody, but it takes all of us to try to identify the right patients to move this forward. So you know, they can contact me, I’m sure you’ll provide them with my contact information, or you can email us at hlhs@mayo.edu HLHS at Mayo mayo.edu. And that’s our research study team at Mayo Clinic that is partnering with us to be able to make this clinical trial happen. And we’ll

Alan Olsen

have more information on the transcript here for the playback for people reaching out to you, Tim. It’s been an absolute pleasure having you with us today.

Dr. Timothy Nelson

Thanks Alan. It’s great to be with you.

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