Spina bifida is the most common cause of lifelong childhood paralysis in the United States; approximately four children are born with this spinal defect every day. Standard care usually involves surgery, but it still leaves more than half of children unable to walk. But a combination of surgery and stem cell treatment may offer hope to children. In this episode of Unfold, we examine the world’s first human clinical trial using stem cells before birth to treat the most serious form of the condition.
In this episode:
, pediatric surgeon and chair of surgery at UC Davis Health
, medical director of the Shriners Spina Bifida program and associate professor in the department of Physical Medicine and Rehabilitation at UC Davis
Michelle Johnson, mother of baby enrolled in spina bifida clinical trial
Jeff Maginnis, father of baby enrolled in spina bifida clinical trial
Transcript
Transcriptions may contain errors.
Amy Quinton
Michelle Johnson was 20 weeks pregnant with her second child, when she got a phone call about her ultrasound that left her shaken.
Michelle Johnson
Just in the clinician’s voice. You know, it was not a good call. And he had just said, "Is this a good time? You know, are you available to talk?" And then, he said he wanted to talk about the results of the ultrasound and that based on the abnormalities they were seeing it was indicative of spina bifida.
Amy Quinton
In tears, she immediately called her boyfriend and the baby's father, Jeff Maginnis, at work.
Jeff Maginnis
I could hear in her voice that she was concerned and, you know, very upset. And so, I left work and came home, and you know, we talked about it. And that's, I think, when I learned that it was a lot more severe than the phone call might have led on to be.
Amy Quinton
The Portland, Oregon couple spent the next few days researching what the diagnosis meant, and what treatment options they might have. What did you know about spina bifida?
Michelle Johnson
Nothing. Very little to nothing.
Jeff Maginnis
Nothing for me, yeah.
Michelle Johnson
Yeah, I think I knew, I knew it was a defect of the spine, and that it could impact walking. So, I guess I knew briefly of spina bifida but not specifics. And I was just in shock. You know, I, I had no idea what the right answer was or what outlet to pursue. So, it was it was surreal.
Amy Quinton
Michelle and Jeff's developing baby had myelomeningocele, the most severe form of spina bifida. Paralysis, incontinence and excess fluid in the brain were all possibilities. But they soon heard about a trial testing a new stem cell treatment and prenatal surgery at UC Davis Health. They learned it might prevent the most severe conditions. While it had never been tested on humans, they decided to participate.
Michelle Johnson
What won't you do for your child, unborn or born? You're going to do everything you can.
Amy Quinton
In this episode of Unfold, you'll hear about their journey through the world's first human clinical trial that's providing hope for a spina bifida cure. Coming to you from UC Davis. . .
Marianne Russ Sharp
and UC Davis Health
Amy Quinton
this is Unfold, a podcast that breaks down complicated problems and unfolds curiosity-driven research. I'm Amy Quinton.
Marianne Russ Sharp
I'm Marianne Russ Sharp. This story is about the groundbreaking work of pediatric surgeon Diana Farmer with UC Davis Health.
Amy Quinton
It's also about the courage of families taking part in the world's first clinical trial to treat spina bifida through prenatal surgery and stem cell treatment.
Marianne Russ Sharp
Spina bifida is when the fetus’s spinal cord doesn't develop properly in the womb. Part of the spinal cord and the surrounding tissues are exposed through a gap in the backbone.
Amy Quinton
Farmer says four children in the United States are born with spina bifida every day, and it can leave children paralyzed.
Diana Farmer
The severity can be variable based on where in the spinal cord the defect develops. So, if it's in the cervical, in the neck area, then a person might be paralyzed from the neck down. If it's in the back area, you might just be paralyzed from the legs down.
Marianne Russ Sharp
Farmer actually pioneered the prenatal surgery to close the exposed spinal cord. It doubled the chances a child would walk and decreased the likelihood that a child would develop excess fluid in the brain, called hydrocephalus.
Amy Quinton
Now researchers have designed the first human clinical trial to see whether engineered placental stem cells combined with prenatal surgery can do even more.
Marianne Russ Sharp
Surgeons will close the developing baby's exposed spinal cord and then use a special stem cell patch. The stem cells are engineered to protect neurons, or nerve cells, from damage.
Amy Quinton
And as we mentioned in the last episode of Unfold, the procedure was successful on lambs with spina bifida. It allowed them to walk.
Marianne Russ Sharp
And in 2017, UC Davis veterinary surgeons were able to successfully treat spina bifida in two English bulldogs named Darla and Spanky, using this same procedure.
Amy Quinton
Darla and Spanky can now walk and run. Farmer says the bulldogs shed light on a few things about this procedure, especially given the dog's age much faster than humans.
Diana Farmer
The pets Darla and Spanky allowed us to see that there appears to be durability to this therapy.
Marianne Russ Sharp
The FDA has given doctors approval to conduct a safety trial also called a phase one trial
Amy Quinton
And a safety trial probably sounds a bit scary.
Marianne Russ Sharp
It does sound scary, but it's actually standard.
Amy Quinton
Well, it was scary for Michelle Johnson and Jeff Maginnis. Before Michelle even considered participating, she had to weigh all the risks and options and she had several, including a much less invasive laparoscopic surgery, without stem cells.
Michelle Johnson
Laparoscopic has much less risks to the mom. You're not going to be at high risk for ruptured uterus, you can have subsequent pregnancies, but laparoscopic didn't offer the stem cells. And if you do any research on the last decade of stem cell studies with animals, it's really black and white. You know, the animals that receive stem cells are able to walk at birth and the ones that don't, don't walk at birth.
Marianne Russ Sharp
So, Michelle had really done her homework on the lamb studies. Did she know about Darla and Spanky, too?
Amy Quinton
Yeah, Michelle says the animal studies gave both of them hope that the surgery could work.
Michelle Johnson
I knew there were more increased risks. But with what we knew, we felt that it was just the best, the best decision to go forward. I don't think we felt like we had anything to lose.
Amy Quinton
Jeff says they just wanted to do what was best for their baby.
Jeff Maginnis
It's scary. You know, I mean, "what happens if" is always, you know, in the back of your mind, but I think that, you know, as soon as we spent those four days kind of doing our research and learning, I think that we decided that, you know, there was no question in our mind that that was the