This Viewfinder episode is supported by UC Davis Health, where doctors, nurses, and researchers share a passion for advancing health.
Learn more about their latest medical innovations at health.ucdavis.edu.
[Lara Hawthorne] My first thought was, "What is Duchenne?"
And then, when I looked it up, death sentence.
[Hunter Horkheimer] What's the point, right?
I want to live- enjoy my life because I don't know when's the last day I'm going to have.
♪♪ But I obviously lived way past that mark.
I'm doing really good.
I'm considered healthy with Duchenne.
[Dr. Criag McDonald] The trial results were really spectacular, and patients actually, uh, experienced complete stabilization in the deterioration of their heart function.
[Hunter] The stem cells allowed me to see that I have a future.
- Coming up, discover how cutting edge stem cell treatments are transforming the lives of those facing unimaginable health challenges.
[Emily] She shouldn't be able to move anything below the hips.
[Dr. Farmer] The most exciting thing was the baby came out kicking her legs and wiggling her toes, which really exceeded expectations.
[Dr. Johnathon Anderson] What we actually saw is a year out from the treatment, there is actually an improvement in their ability to use their tongue and to swallow.
- This could change lives forever.
♪♪ - Our bodies are made up of trillions of cells that have different functions, like blood cells, heart cells and nerve cells.
And then, there are stem cells.
Stem cells are undifferentiated cells.
In other words, they are cells that do not have a specific job.
However, some stem cells do have the potential to become other kinds of cells in your body.
And under the right conditions, they can multiply, they can divide, and they can even transform into other kinds of tissue.
Stem cells can be found in various tissues throughout the body, like bone marrow, blood and fat.
But they can also be obtained from embryos, which are called embryonic stem cells.
The purpose of stem cells is to help our body repair and regenerate tissues and organs that have been damaged due to injury, disease or aging.
- Every one of us has experienced the healing power of stem cells.
Some stem cells- like those in our follicles that create the hair, those in our gut, those that make our skin- they're continually dividing and making new tissue.
Others respond to damage.
If we get a cut, the stem cells will quickly create the new cells that need to heal that over.
So, we really need the stem cells to regenerate all of our tissues.
- Scientists say stem cells have tremendous potential.
That's why they're working hard to utilize this healing power by creating a very specialized kind of medicine known as regenerative medicine.
[Dr. Nolta] Regenerative medicine is... is pretty much a... a catch-all phrase for using the... the cells from your body to heal yourself.
What we do in the laboratory is we take some of the stem cells out of the patient and we grow them up to huge numbers in bioreactors, and then we give them back to the patient for a certain disease or injury.
- Some researchers believe that stem cell-based therapies may one day be the key to treating many types of injuries and different illnesses.
But some are already making a difference.
♪♪ One such groundbreaking therapy holds particular promise for Lara and her son, Hunter, who lives with a debilitating disease.
[Lara] He was a really happy, easy baby, but at age one, we really knew that something was going on.
- Lara's son, Hunter, was able to walk but displayed signs of fatigue and difficulty standing up from a sitting position.
[Lara] My son would kind of, from that position, lean forward onto the floor, stretch out his legs, and then walk his arms up his legs.
They said that he would grow out of it, um, that, you know, he was just slower to develop than other children.
But it took another two and a half years to get a diagnosis.
- It was at Shriners Hospital in Sacramento where Lara finally got the diagnosis.
Her son has Duchenne muscular dystrophy.
[Lara] My first thought was, "What is Duchenne?
I have no idea."
And then, when I looked it up, death sentence.
[music fades] [Dr. McDonald] Duchenne muscular dystrophy is the most common muscular dystrophy of childhood.
It's a genetic disorder caused by a defect in a gene causing production of the protein dystrophin.
Dystrophin is important for protecting muscle cells, uh, from really normal wear and tear injury.
And if a patient lacks the dystrophin protein, they actually, unfortunately, will progressively lose muscle fibers.
So, essentially, they'll develop a progressive weakness of their arms and legs and eventually require power wheelchair for... for their mobility.
- The affliction doesn't stop there.
Duchenne muscular dystrophy also gives rise to heart dysfunction and weakness in the muscles responsible for breathing, ultimately, ending lives too soon.
[Lara] Duchenne is a beast.
At the time, the life expectancy was really 16, mid-teens.
You really have to use your muscles to do that, don't you?
[Hunter] Yeah, I do.
[Lara] Gotta use your body.
There were different answers at different ages.
We described it, um, as though, uh, his muscles just have to work so much harder than other kids.
- Hunter became aware of the severity of his condition when he attended Muscular Dystrophy Camp.
[Lara] That brought him a lot of questions coming home, because he saw a lot of older children that were using ventilators and that even were in wheelchairs.
You know, we talked about how, uh, we didn't know what tomorrow would bring and that, um, if he did end up using a chair, it was going to be OK. Ah, the Hawthorne lap dog.
- When Hunter was ten years old, Lara decided it was time to transition her son into a wheelchair.
[Hunter] Going into a wheelchair was probably the best thing that has happened to me for a while because I was constantly falling over and over and over and over.
And I was just tired of it.
The wheelchair literally saved me a bunch of pain and suffering because of my disorder.
It helped me get through a lot of life.
[Lara] It was this incredible tool of independence.
It gave him wings.
- Despite the challenges imposed by the disease, Hunter's life took a positive turn when Lara introduced him to "power soccer."
[Hunter] It's soccer using, uh, a power chair.
Anybody with any disability can play it.
I'm here!
I'm here!
I got it!
Even though I'm in a wheelchair, it gives me the freedom like I can forget about my disability.
[Lara] It's so fun watching him because his face lights up.
[Hunter] I don't know where I'd be without it.
I've definitely gotten a lot of smiles out of it.
- However, shortly after experiencing this newfound passion, Hunter's health declined sharply.
That prompted Lara to turn to steroids- at the time, the only available treatment option.
[Lara] When you are knowing what's going to happen, uh, you are willing to look outside the box for different therapies.
Um, we... we knew that the... the steroids were buying him time.
It gave us the time to get into the stem cell trial.
- In 2018, Lara received word from Dr. Craig McDonald of UC Davis Health about an upcoming clinical trial using stem cells to treat Duchenne muscular dystrophy.
- Patients in the HOPE-2 trial were the... the most severely affected patients with Duchenne muscular dystrophy.
- The HOPE-2 clinical trial included 18 young men and children, conducted at UC Davis Health and other sites around the U.S..
The objective of the trial was to evaluate the safety and effectiveness of stem cell therapy for heart failure caused by the disease.
Ultimately, physicians hoped the stem cell therapy would improve heart function and overall quality of life for the patients.
[Dr. McDonald] These stem cells are actually derived from human hearts that were destined to be transplanted in human individuals.
We actually then use those, uh, cardiosphere-derived stem cells in a, uh, therapeutic sense to infuse these stem cells intravenously into these, uh, children and young men with Duchenne muscular dystrophy.
- The stem cells are injected into the bloodstream, where they circulate throughout the body and reach the affected muscles.
The cells, themselves, produce exosomes, which promote muscle fiber regeneration and repair.
[Dr. McDonald] The infusion, itself, is... is only about, uh, an hour and a half long.
It's just a simple IV inf- administration.
♪♪ [Lara] We were on pins and needles once he actually received the medicine.
♪♪ Every day, "You feel different?
You feel different?"
"No, mom.
No, I don't."
And about a week, ten days in, he's like, "I don't know if I'm going to feel anything."
And I'm like, "Oh, we just need to hold on a little longer."
And on day 12, 13, he woke up and he said, like, "Mom, I'm different."
He knew.
[Hunter] The first thing I noticed was arm control, and just my entire feeling of my body.
I didn't feel, like, achy and slow.
I had energy again.
[Dr. McDonald] The trial results were really spectacular.
The patients actually, uh, experienced complete stabilization in the deterioration of their heart function.
So, this, uh, definitely, uh, will have a... a huge impact on... on, uh, survival and life expectancy.
Hey, Hunter.
How are you?
[Hunter] Good.
How are you?
[Dr. McDonald] Yeah, good.
Good to see you.
How ya doing?
[Lara] Hi.
Good.
Good afternoon.
[Dr. McDonald] How have you been?
[Hunter] I've been- I think I've been getting a bit stronger.
- Today, at the age of 21, Hunter continues to receive stem cell infusions and routine checkups from Dr. McDonald and his team.
[Dr. McDonald] Good.
Spread out...
Spread out your fingers.
Good.
[Hunter] I'm doing really good.
I'm considered healthy with Duchenne.
[Dr. McDonald] Straighten out your elbow.
[Hunter] I think the stem cells are really doing it for me.
They're... they're making me stronger, more... more agile, more mobile.
It's given me confidence to have a life.
[Lara] I feel that this has added decades to his life, and I see more and more joy from him and more confidence in who he is.
And, uh, so, that's been a true gift.
[Hunter] The stem cells allowed me to see that I have a future.
I have something to look forward to.
♪♪ [Dr. McDonald] Based on our exciting results of the HOPE-2 trial, we've now, uh, embarked on a late stage phase three, HOPE-3 trial.
You think they have good reliability to their testing?
- Yeah.
Yeah, absolutely.
[Dr. McDonald] Patients are being enrolled in this trial all over the United States, and we're hopeful that this trial will confirm the exciting results of the HOPE-2 trial and, uh, perhaps lead to an FDA approval of this stem cell, uh, therapeutic approach.
- The HOPE trial is just one of many UC Davis Health trials dedicated to harnessing the potential power of stem cells.
Another trial currently underway aims to use stem cells as a treatment for patients with swallowing problems related to tongue injuries inflicted by cancer treatment.
♪♪ Bill Souza of Carson City, Nevada, was one of the patients who participated in the clinical trial.
In 1999, Bill's life took an unexpected turn when he was diagnosed with neck cancer.
- While the invasive surgeries and radiation treatments successfully eliminated the tumor and left Bill cancer-free, it came at a cost.
Bill's cancer treatments impacted his speech, ability to swallow and to easily consume solid foods.
All of the procedures caused a permanent effect.
- In 2017, Bill decided to participate in the trial.
- For the groundbreaking stem cell treatment, a small biopsy was taken from the patient's thigh.
Within this tiny piece of muscle tissue lies a handful of muscle stem cells.
The stem cells were then extracted and placed in a specialized medium that triggers them to grow exponentially, becoming hundreds of millions of stem cells.
Once they've multiplied, physicians inject the stem cells into the patient's damaged tongue.
[Dr. Anderson] Even if we had a therapy that kept them steady state, with no decline, that would be a... a huge win for the field.
[Dr. Anderson] The data from the phase one looked really promising.
What we actually saw is a year out from the treatment, there is actually an improvement in their ability to, um, to use their... their tongue and to swallow.
If that trend of working that well that we saw in the phase one continues to the phase two and phase three, these clinical trials are potentially a game changer for these patients.
Hands down, no other treatment would come close to working as well as these cells do.
Having said that, uh, it's not clear if that's actually going to be the case.
We actually have to do the hard work and go through the trials.
There's very good reasons why we have these clinical trials in place.
Uh, but yes, we're... we're very excited about the... the data we saw in the phase one.
- Continuing their pursuit of groundbreaking stem cell therapy advancements, UC Davis Health researchers and physicians have embarked on yet another unprecedented clinical trial- the world's first in utero stem cell treatment for spina bifida.
On the operating table lies a pregnant woman and her unborn baby.
[Dr. Diana Farmer] It's really just been an amazing journey.
- Dr. Diana Farmer is the surgeon scientist leading a clinical trial which aims to reverse the damage caused by spina bifida by surgically applying stem cells to the still developing fetus' spinal cord.
[Dr. Farmer] So, I had this idea that stem cells might improve this.
If we could somehow apply stem cells, give stem cells to the spinal cord, we could reverse the damage that had already occurred.
- Spina bifida is a condition where the spinal tissue of a fetus does not close properly, leaving the spinal cord exposed.
Without treatment, spina bifida can lead to a range of lifelong issues including problems with mobility, muscle weakness and sometimes, total paralysis of the legs.
[Dr. Farmer] The real manifestations don't show up until later in life.
It's only when these children sometimes reach two or three and families start to realize they're not making those same milestones.
- Launched in the spring of 2021, the clinical trial is known formally as the Cure trial.
The trial received FDA approval and a grant from the California Institute for Regenerative Medicine.
I've been working, uh, toward this day when we're treating patients with stem cells for spina bifida for almost 25 years now.
- Over the years, the team experimented with different types of stem cells and found that mesenchymal stem cells from the placenta worked best.
[Dr. Farmer] We tried all kinds of solutions, all kinds of things that didn't work.
So, every... every story of success, if you peel back the onion, has several stories of failure.
[chuckles] But we never gave up.
- The stem cells were manufactured at UC Davis Health's Good Manufacturing Practice facility.
The cells were then placed on a small patch made of a material that mimics the spinal cord's outer membrane, known as the Dura.
[Dr. Aijun Wang] We have the stem cells.
We have the right medical device that can carry the stem cells to the location where you want them to go to.
That actually get us to the animal model of spina bifida.
- Researchers conducted successful trials on lambs born with spina bifida.
[Chris Pivetti] The lamb, um, that was treated without stem cells cannot get up.
Um, it has complete hind limb paralysis, um, whereas the sibling to that lamb is actually to- able to stand up and walk around.
That gave us hope that, you know, maybe this could work as a therapy for these kids.
- Success with the animal trials paved the way for the first human patient.
[Emily] As soon as we knew about it, we knew that it was probably the route we wanted to take.
- Expectant mother Emily volunteered to be the first patient in the unprecedented surgery.
[Emily] We didn't know what spina bifida was before we got our diagnosis.
So, that was crazy.
We had no prior indications that we'd be experiencing any sort of a complicated pregnancy whatsoever.
And when we got those ultrasound results, it's like everything took a 180.
We decided that fetal surgery was going to be our best bet.
[Dr. Farmer] What I might suggest that I would put something in the carrier.
Emily was one of three folks that reached out to us.
She had the right gestational age of her fetus- because there's a window, a small window of a few weeks where we can operate- and she's just turned out to be the perfect patient.
- Emily was 26 weeks pregnant when the first-of-its-kind surgery took place.
[Emily] I feel like we've got a pretty even mix of excitement and nerves.
- The operation was led by Dr. Hirose and his surgical team.
[Dr. Shinjiro Hirose] We open Mom's abdomen and we pull the uterus up and out of her belly, making sure that the baby's in a good position, figuring out where the placenta is, because we have to stay very far away from that so we don't injure it, because that's the lifeline for the baby.
And then, we identify where the baby's sitting.
And we don't pull the baby all the way out, we just sort of float the baby up to that incision and we expose just the part that we need, which is the myelomeningocele - Using a microscope to perform the delicate work, the patch containing the stem cells was then placed directly on the fetus' exposed spinal cord.
[Dr. Farmer] Emily's surgery went flawlessly.
By the time we actually did the operation, it was almost anticlimactic.
It was unbel- It was perfect and perfectly smooth.
- As the weeks went by following the surgery, things looked promising for Emily and her baby she named Robbie.
[Dr. Farmer] The ultrasounds suggest that she- that there is more leg movement in the fetus than they would have expected, giving- given her level of lesion, which is pretty high.
Um, and Emily, herself, is describing what she says is lots of kicking now.
I'm trying to manage her expectations, too.
But we are pretty excited, um, and pretty anxious for this delivery.
♪♪ - It's delivery day.
Emily is scheduled for a caesarean section.
- We hoped and prayed that her arrival wouldn't come too soon.
And now, it's finally time.
And I'm- It's the most beautiful day ever.
[baby crying] - Hello, hello, hello!
- The first baby in the world to undergo in utero stem cell therapy for spina bifida arrived on September 20th, 2021.
[Emily] She shouldn't be able to move anything below the hips.
[Dr. Farmer] The most exciting thing was the baby came out kicking her legs and wiggling her toes, which really exceeded expectations.
It was amazing.
Like, am I seeing that?
Is that real?
[Baby cries] - Robbie, who was destined for a life of paralysis, defied all odds, representing a milestone in the treatment of spina bifida and offering hope to others for reversing damage caused by the condition.
[Dr. Farmer] This could change lives forever.
- Since then, more infants have been participants in the Cure trial.
The team will continue to monitor Robbie and the other babies' progress until they're six years old.
[Emily] It's larger than life.
It's enhancing quality of life for so many patients to come.
It really- It blows my mind, and it's hard to understand just what it means and just how special it is that we got to be part of this.
[Dr. Famer] We could not do any of this work, any of this discovery without the partnership of the women who have been willing to, frankly, in a way, risk their lives for the advancement of their child and science.
[Dr. Anderson] These trials would not go forward, uh, if it wasn't for these brave patients.
They're stepping into unknown waters.
They don't know what to expect.
This hasn't been done before.
[Hunter] I wanted to take that risk, no matter what, to make sure drugs like this can actually get to the light and people can use it.
[Dr. Nolta] We are so grateful to each patient who volunteers to be in a clinical trial that might or might not help them, but will definitely help science and help the patients to come They are moving science forward by leaps and bounds.
This Viewfinder episode is supported by UC Davis Health, where doctors, nurses and researchers share a passion for advancing health.
Learn more about their latest medical innovations at health.ucdavis.edu.
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