In this first-look teaser, host Adam Rutherford introduces the focus of our first miniseries – gene therapy. Hear from scientists, experts, patient advocates and the patients themselves as they set the scene for this exciting new podcast. Launching October 2021.
Science Will Win explores the fascinating science, policy, and humanity which is shaping the future of healthcare and transforming patients’ lives for the better.
Hosted by Adam Rutherford, geneticist and Honorary Fellow at University College London, our first miniseries takes listeners on a journey behind the science of gene therapy; the next generation of medicines bringing new possibilities for patients living with rare genetic diseases.
At a time when innovative science is achieving the seemingly impossible, we’ll look at gene therapy from every angle, speaking to scientists and experts on the forefront of medical research, as well as the patients and families who are holding new hope in the life-changing potential of gene therapy.
Brenda: What we're looking for is clear, and that is to fix what is currently broken.
John: Gene therapy is a new class of medicine.
Reda: Personally it inspires a lot of hope, hope not just for patients, but for their families, their caregivers and ultimately for society as a whole.
Nikhil: To me, that potential to truly transform my life, not for a day, not for a week, but for years, that's what gene therapy offers.
Adam: Welcome to Science Will Win, a miniseries exploring the future of gene therapies. I’m your host, Adam Rutherford. I’m a geneticist, a writer, and broadcaster and also a Fellow at University College London in the UK. And this is a special teaser episode we’ve put together for you to preview what we’ll be discussing throughout this season. I’m going to be talking to experts, scientists, patients and families from around the world. And we’ll look at gene therapy from every angle.
As a reminder, gene therapy is a promising area but it’s investigational in nature. For many diseases, there is a whole load of research to be done to understand the safety and efficacy of these potential therapies. Remember, you should always discuss treatment options with your healthcare provider.
Now, there are a few gene therapies on the market today, and many more in development, all intended to treat rare diseases. We’re going to learn what it’s like to live with a rare disease from people who experience it every single day. And the people who care for them. Like Emily Crossley.
Emily: Nothing could have prepared me for the very steep learning curve of trying to become an expert in genetics and in Duchenne muscular dystrophy.
Adam: Emily is the co-founder of the charity Duchenne UK. In her work, she and her team are pushing for more access to clinical trials, and treatment options for families living with Duchenne muscular dystrophy.
Emily: I would say the challenging thing is, is it going to make a difference to my son? And I realized very early on in this journey that it's impossible to just save one child. If you want to save one child, you have to save a generation.
Adam: With each episode, we’ll also get a sense for how urgent the need is. When it comes to patients living with Duchenne, time is absolutely crucial. The disease primarily affects young boys. And as they get older, their muscles weaken. They lose the ability to walk, and eventually, it affects their hearts and lungs. For families like Emily’s, there isn’t any time to lose.
Emily: Once my son loses ambulation, it will be very difficult to get him up walking again. That will be a very difficult thing to achieve. What I'm working for is to stabilize the decline. We're fighting to stabilize the disease at whatever stage in which the boys have it and enable them to live as fuller lives as possible.
Adam: Less than 5 percent of all rare diseases have approved treatments available to them. That is why continued research in this field is so vital. And, even when there are treatment options, gene therapy has transformative potential.
Nikhil Gadre works as Pfizer’s team lead for hemophilia gene therapy and lives with a severe form of hemophilia A himself.
Nikhil: Gene therapy has the ability to truly turn science fiction into science fact. It has the ability to change the lives of people, of patients like me. So this where gene therapy to me gives such a strong motivation for my everyday life, because I want to be able to make this a reality for my kids, for my family and for others who I know and am friends with and consider my hemophilia community.
Adam: Gene therapy has the potential to transform patients' lives possibly in just one treatment. It could slow, or even stop that relentless decline in Duchenne. Or it could make it possible to stop taking frequent infusions of clotting factor for those with hemophilia. The potential is huge, and so is the need. And so, researchers, pharmaceutical companies, policy makers, and patients have to work together against the clock, to ensure that when approved, gene therapies get to eligible patients in time.
We’re going to be talking to the leading experts to understand how gene therapy works.
Dirk: Now, with gene therapy, we are not an organ transplantation. We are not at cell transplantation. We are at gene transplantation. So even deeper down into the cells.
Sonal: To me gene therapy is really the ability to think of the disease and say, okay, well there's a malfunctioning gene, so we can actually find the correct gene, introduce it into a transport mechanism, put it into the body that transport mechanism goes straight to the target organ provides that gene to become that blueprint, to produce whatever missing protein is needed in the patient and ultimately fixes their disease at that molecular level.
Adam: And we’ll also learn about the unique challenges gene therapies face. Starting with how they are made.
Bob: Each time we want to make a gene therapy, we kind of have to start from scratch. And that's where the challenges have been besides the fact that they're probably the most complicated therapeutic products that we've ever researched and developed. And so by its nature, it's just highly complex.
Adam: Then, there’s the question of value. How do you figure out how much they should cost and how to pay for them?
Erik: Today, we spend like 85 cents of every healthcare dollar managing the symptoms of chronic illness over a patient's lifetime. If there's durable treatments over a long period of time, there's value in that.
Emily: There was something far worse than having no approved treatment for your condition and that is having a treatment that has been approved, but it's sitting on a shelf and you can't get to it because no one has worked out how to pay for it.
Adam: And we’re also going to explore the ways in which policy, health systems, and society will need to quickly adapt to keep pace with gene therapy innovation.
Paige: Policy around the world really needs to kick into high gear to keep up with where the science has headed in the cell and gene therapy sector.
Simone: The job is not finished, in the sense that we have a set of policy, both soft and hard, such as in regulation, in place, but they're not yet to the point of allowing the rare disease community to progress where it should be.
Durhane: Each one of these diseases are small in numbers. In many, many cases, they're are lost in the cracks of the healthcare system, even in the very best healthcare systems.
Adam: And ultimately, we’ll hear about the passion and motivation behind it all.
John: A friend of mine reflected on the several decades of my career recently and said, goodness, John, when you first embarked on gene therapy, we all thought you were a little bit mad. And most of us thought you would not succeed in your lifetime. So these are real changes to real people's lives. And that is something that I cannot believe has occurred in my career in my lifetime.
Sonal: I keep my focus remembering those patients that I have witnessed in the emergency room or in my clinical practice back in the day, they are, those faces are my drive and force to continue to work with all of our external stakeholders to find that solution and bring the patient, the access that they need.
Bert: We deliver hope is what happens when we get this right. This is the potential to see a future where something can be addressed, that's addressable. That opens up possibility. And that's where hope it’s one of the most powerful four-letter words that exists.
Adam: Join us to explore the fascinating science, policy, and humanity of gene therapy. Science Will Win launches in October. To make sure you don’t miss a single episode, subscribe and follow Science Will Win for free on your favorite podcast app.