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Searching for a Spina Bifida Cure Part 1 – The Promise of Stem Cells

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The first ever in utero stem cell treatment for spina bifida is the culmination of decades of work from UC Davis surgeon-scientist Diana Farmer. This novel fetal surgery at UC Davis Children’s Hospital could change the life of a developing baby with spina bifida, who otherwise could be born paralyzed from the waist down. In Part 1 of this docuseries from UC Davis Health, we meet the team behind the groundbreaking CuRe Trial and see how decades of research led to a first-of-its-kind fetal surgery.

Credit: UC Davis Health

Video Transcript:

[MUSIC PLAYING] – [NARRATOR] The Surgery Center at UC Davis Children’s Hospital sees about 5,000 surgeries per year, but never one quite like today’s. This procedure is the first of its kind in the world. You can’t tell at first glance, but there are two patients on this operating table– a pregnant woman and the developing baby inside of her. It’s the first ever in utero stem cell treatment for spina bifida, a culmination of decades of work for UC Davis surgeon-scientist Diana Farmer. Farmer is leading the world’s first human clinical trial using stem cells to treat spina bifida before birth by surgically applying the cells to the spinal cord in a still-developing fetus.

Spina bifida occurs when a fetus’s spinal tissue doesn’t close properly. It leaves the spinal cord dangerously exposed. The Spina Bifida Association estimates there are more than 70,000 people with the condition in the US.

Surgeons can perform fetal surgery and close a fetus’s open spinal cord. It can stop spina bifida from becoming more severe.

But in many cases, the patient is still left with some abnormal function, such as paralysis. Farmer wants to take the next step: to reverse spina bifida and even cure it. – I had this idea that stem cells might improve this. if we could somehow apply stem cells, give stem cells to the spinal cord, not only would we protect future injury that we were doing with the closing the back in utero, but we could reverse the damage that had already occurred.

– [NARRATOR] The name of the trial is Cellular Therapy for In Utero Repair of Myelomeningocele, which gets abbreviated to CuRe.

The CuRe Trial team has FDA approval to begin enrolling human patients and the California Institute of Regenerative Medicine gave them a $9 million grant to begin the work. – I’ve been working toward this day when we’re treating patients with stem cells for spina bifida for almost 25 years now. – Stem cells have shown promise in treating many conditions before birth. – [NARRATOR] This leading pediatric and fetal surgeon began her spina bifida work at UC San Francisco and brought it with her when she moved to UC Davis. She also brought along a bright, young postdoctoral researcher she met at Berkeley in 2009.

– When I first started as an engineer, I was always thinking about how to apply technologies into in real life patient care, how to help patients with what we know about and what we develop in the lab.

And this is what we’re doing right now! – [NARRATOR] The CuRe trial has been a massive team effort. There are more than 40 people in this lab. Over the past decade, Wang estimates more than 100 people have been involved, including lab supervisor Chris Pivetti.

– I really like the idea of helping kids. So the idea of working on something that could impact a child’s life was really appealing to me. – [NARRATOR] The academic health system at UC Davis is uniquely suited for this trial thanks to its stem cell research program, biomedical engineering expertise, and world famous veterinary school, and large animal lab. For the in utero stem cell treatment, researchers needed to determine what kind of animal model and what kind of cells to use. – We hadn’t even decided what stem cell we wanted to use yet.

So it was trial and error really. – We tried IPS cells. We tried nanofiber scaffolds. We tried all kinds of solutions, all kinds of things that didn’t work. So every story of success, if you peel back the onion, has several stories of failure.

[LAUGHS] But we never gave up. – [NARRATOR] They found what was needed in what are called mesenchymal stem cells from the placenta. Researchers believe these cells pose fewer complications than embryonic stem cells. – I think of it as delivering their magic stem cell juice and then go away once they’ve done their job. – [NARRATOR] The cells are placed on a material that mimics the spinal cord’s outer membrane called the dura.

– We seeded the cells on an already FDA approved dural patch used for brain tumors and things. So that made it easier from an FDA hurdle point of view.

And it turns out the cells loved it. – [NARRATOR] The next step, the sheep trial. In 2015, stem cells were applied to the spinal cords of lambs that would have been born unable to stand or walk.

– The lamb that was treated without stem cells cannot get up. It has complete hind limb paralysis, whereas the sibling to that lamb is actually able to stand up and walk around and move pretty much like a normal lamb. – They were able to stand at birth and they were able to run around almost normally. It was amazing. – We were in shock.

It gave us hope that maybe this could work as a therapy for these kids.

– [NARRATOR] As further proof that the idea might really work, researchers tried a similar treatment for these bulldog puppies. Darla and Spanky were born with naturally occurring spina bifida. UC Davis researchers surgically applied stem cells to the puppies’ spines when they were 10 weeks old. Within months, Darla and Spanky were running, jumping, playing.

Wang says the result shows the power of these uniquely engineered stem cells. – Stem cells are very smart, actually. When you culture stem cells in different environments, they’re going to do different things. They’re going to respond to the culture media. They’re going to be induced to do the things that they think they should do.

– [NARRATOR] With FDA approval of the cell safety profile in animal models, the search for the first human patients begins. The search for the first baby in the world to receive this groundbreaking, potentially life-changing treatment..

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