Prenatal Stem Cell Treatment Improves Mobility Issues Caused by Spina Bifida

(SACRAMENTO, Calif.) – The lower-limb paralysis associated with spina bifida may be effectively treated before birth by combining a unique stem cell therapy with surgery, new research from UC Davis Health System has found.

Diana Farmer (front), a pioneer in fetal surgery, and her research team are testing placental stem cells as a treatment for the paralysis associated with spina bifida. Diana Farmer (front), a pioneer in fetal surgery, and her research team are testing placental stem cells as a treatment for the paralysis associated with spina bifida.

The study, conducted in an animal model, was led by Diana Farmer, the fetal surgeon who helped pioneer in utero treatment for spina bifida — a congenital birth defect that occurs when the spinal cord does not close properly, leading to lifelong cognitive, urological, musculoskeletal and motor disabilities. Farmer’s chief collaborator was Aijun Wang, co-director of the UC Davis Surgical Bioengineering Laboratory.

“Prenatal surgery revolutionized spina bifida treatment by improving brain development, but it didn’t benefit motor function as much as we hoped,” said Farmer, chair of the UC Davis Department of Surgery and senior author of the study, published online today in Stem Cells Translational Medicine.

“We now think that when it’s augmented with stem cells, fetal surgery could actually be a cure,” said Wang.

Farmer and Wang are the first to combine fetal surgery with a placental stem cell treatment to reduce the effects of spina bifida, which in children can range from barely noticeable to severe. The most common and disabling form of the disorder, called myelomeningocele, causes the spinal cord to emerge through the back, often pulling brain tissue into the spinal column and causing cerebrospinal fluid to fill the interior of the brain. Permanent shunts are required to drain the extra fluid.

View video of Dr. Diana Farmer as she discusses her research

Farmer was senior author of the landmark Management of Myelomeningocele Study (MOMS), which showed that prenatal surgery could improve cognitive outcomes for the 1,500 children born each year in the U.S. with spina bifida. A majority of treated children in the study, however, were unable to walk independently at 30 months of age.

For the current research, lambs with myelomeningocele received standard fetal surgery to return exposed tissue to the spinal canal. Human placenta-derived mesenchymal stromal cells (PMSCs) — known for their neuroprotective qualities — were preserved in hydrogel and applied to the site of the lesion. A scaffold was placed on top to hold the hydrogel in place, followed by traditional surgical closures to complete the repair.

Six animals that received the stem cell treatment were able to walk without noticeable disability within a few hours following birth, while six control animals that received just the hydrogel and scaffold were unable to stand.

“We have taken a very important step in expanding what MOMS started,” said Wang. “Next we need to confirm the safety of the approach and determine optimal dosing.”

Farmer and Wang will continue their efforts with funding from the California Institute for Regenerative Medicine. With additional evaluation and FDA approval, the new therapy could be tested in human clinical trials.

“Fetal surgery provided hope that most children with spina bifida would be able to live without shunts,” Farmer said. “Now, we need to complete that process and find out if they can also live without wheelchairs.”

Additional authors were Erin Brown, Lee Lankford, Benjamin Keller, Christopher Pivetti and Nicole Sitkin of UC Davis, and Michael Beattie and Jacqueline Bresnahan of UC San Francisco. Their study — titled “Placental Mesenchymal Stromal Cells Rescue Ambulation in Ovine Myelomeningocele” — was funded by the UC Davis Department of Surgery. It is available online at http://stemcellstm.alphamedpress.org/content/early/recent.

More information about UC Davis Health System is at www.healthsystem.ucdavis.edu.

http://www.healthcanal.com/medical-breakthroughs/62792-prenatal-stem-cell-treatment-improves-mobility-issues-caused-by-spina-bifida.html

5 comments

  • They talk about this being a “cure” which sounds great. Prenatal closure corrects hydrocephalus, stem cells promotes nerve healing, providing better mobility.

    But I was left asking the question, what about tethering?

    Nate Payne had prenatal surgery, which corrected/stabilized his hydrocephalus and he is very mobile (even without stem cells), but he still required a detethering, which then led to an ETV and a shunt (if I remember correctly).

    I wonder what this “scaffold” is and whether, perhaps, it prevents tethering. Wouldn’t that be amazing in and of itself!

  • I don’t disagree with you. But…

    I always have mixed feelings about this sort of thing. It always carries with it a trace of implication that our kids are universally “other/broken”, and I dislike that. They have medical needs, yes, as do we all. And I certainly am appreciative of anything that aids them in living a full life. I think this might be amazing. But I also think there is such a tremendous, dramatic lack of appreciation for how perfect they are, just as they are.

    We live in an age so driven by the physical… How we look. How healthy we are. How free of disease we are and can guarantee ourselves to be. There’s this unspoken suggestion that some sort of “get fit” effort is a near moral obligation we owe others. I reject that utterly. And, I think that this is, in its own way, kin to that.

    The problem is this idea that all things can be, or ought to be, “fixed”. Which leads to a very deficient ability by society at large, and often very acutely in certain medical and other circles, to actually treat people as people and have grace and compassion for what actually is.

    I’m not sure that all makes sense. It sounds a little rambly, but I’m running on 3 hours of sleep, so I beg your pardon for that.

  • I guess I look at it a little differently.

    God originally made us in his image and perfect, but because of the fall and the effects of sin, things get broken. My thyroid, for example – das kaput! So my goal for Pooka is to restore function to its original unfallen state as much as possible, just like I would desire that for my own thyroid.

    But I do agree that our culture is OBSESSED with looks and are always trying to “fix” things that aren’t broken at all.

    The Bible explains it this way – people now worship and serve the creature rather than their Creator.

  • I understand your perspective. I would agree with you on the why of it.

    But I also think that caring for the woundedness of others is caring for Christ whose wounds were what made the difference, not a thing to be avoided or abandoned in favor of something more “normal”. That it is a gift to be given any child, and to serve whatever their needs may be. Too often that idea is unobjectionable only when the needs are things we find acceptable. When they move outside of that category we suddenly are desperate for a cure.

    So, I cringe when I see mom’s complaining about “Why don’t they just get with the stem cells already”. I do see and hear with many of these kinds of stories a real rejection of the fullness and beauty of what is. And what is, is a wonderful person also made in His image, also full of potential for complete union with Him and for amazing love in this life. So often those things are dismissed or not even noticed in these conversations, and the flaws of the body are seen as primary. To me, that is disordered.

    This *is* exciting. I’m also not suggesting you fall into the comments above. But I always crave a little balance when I read these. This article seems a little over focused on all the potential physical negatives (which are not automatic) without any focus on the actual people, or even the risks involved. That’s really all.

  • Ah, I see where you’re coming from now. I’ve seen this negativity a lot, full of regret and/or wistful thinking about what could have or even could be. I don’t have any of those feelings, so I missed what you were saying. I get you now.

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