First in-utero stem cell therapy for fetal spina bifida repair is safe: study

https://health.ucdavis.edu/news/headlines/first-ever-in-utero-stem-cell-therapy-for-fetal-spina-bifida-repair-is-safe-study-finds/2026/02

by gmays • 5 months ago

343 68 5 months ago

health.ucdavis.edu

vpribish• 5 months ago

Interesting - they had done surgery before, but not with stem cells. 6 babies operated on, results were very good and they are recruiting for phase 2

elric• 5 months ago

Incredible to see some promising results in stem cell research. Hopefully a safe and successful application can give a boost to some other areas where stem cells might prove useful (like maybe one day we can regrow damaged heart tissue like this).

SadErn• 5 months ago

Best news is the stem cells used are from donated placentas. So no political footballs, just a true blessing to a child.

doomslayer9994• 5 months ago

The notion of "stem cells" are somewhat of a misnomer as modern biotechnology is showing us. There are an orchestration of probably 1000s of different cell types which can vary in their cell states. We don't have enough knowledge of how to use these various cell types to influence complex self-organization processes like development. But in some cases there are clever fixes without understanding the underlying process.

snyp• 5 months ago

I did not even know it was possible to operate on a fetus. Its insane how far we have come. Very promising results!

alexpotato• 5 months ago

The first episode of the Surgeon's Cut [0] on Netflix shows a doctor:

- operating in utero

- while the mother is awake

- in an outpatient/doctor's office setting

- to implant a balloon in the upper respiratory tract of a fetus with a, I believe, cleft palate so that it's lungs can develop normally.

It really is wild what modern medicine can do these days.

0 - https://www.netflix.com/title/81004466

trhway2• 5 months ago

The Grey’s Anatomy was before that with depicting Prenatal Surgery fellowship.

armadsen4• 5 months ago

Not only is it possible, fetal surgery is more or less standard treatment for spina bifida at this point. The news here is about the stem cell patch being applied during the surgery. (I have a child with spina bifida. We tried to get her into this trial, but did not qualify.)

jillsy• 5 months ago

My high school hosted the county's special day class for kids with severe cognitive and physical disabilities, a majority from spina bifida. If this stem cell method can actually repair the spinal cord before birth, the quality of life improvement is absolutely enormous.

Telemakhos• 5 months ago

Does the physical repair also help with the mental developmental effects? Children with spinal bifida often develop cognitive abilities much slower than children without it.

armadsen4• 5 months ago

The main goal of physical repair of the defect in utero is actually to reduce the incidence of hydrocephalus and hindbrain herniation, which are very common in people with Spina Bifida. The existing fetal surgery reduces the incidence of hydrocephalus from about 80% to about 40%. The improvement in leg and bowel/bladder function is actually a secondary benefit.

My understanding is that the hindbrain herniation (aka Chiari Malformation Type II) is the main cause of cognitive trouble in people with SB. But it's worth noting that it's very far from universal in causing that. Most people with SB are basically normal cognitively assuming they get good early intervention (VP shunt, PT, OT, etc.). Some early cognitive development can be slower as a knock on effect of not being able to move around as much as a baby and toddler, and thus less able to explore the environment, etc.

Source: I'm the parent of a toddler with spina bifida. She's completely on track cognitively and with fine motor skills so far. She's way behind with gross motor skills due to her inability to move her legs very much.

wincy4• 5 months ago

Another sb parent here, my kid is seven now, she’s also on track intellectually. We got the decompression surgery for the Chiari II a few months after she was born, and the VP shunt even earlier than that. Aside from some stammering (which her non SB sister also has, so I suspect it’s hereditary), and weirdness with foods (OT has helped a lot) she’s totally on track intellectually.

Our daughter was a particularly severe case too, and these interventions seem to have helped a lot. For the first four years she’d hold her breath every time she was upset, and need CPR, but we got her breathing again every time, so we don’t think there’s any brain damage. If we’d missed once, maybe I’d be telling a different story now. Thank goodness her head grew!

asah• 5 months ago

Conditions like SB affect the entire family.

Long ago, my next door neighbor's daughter had severe SB and was confined to a wheelchair, slow mental and emotion development, etc. Nobody thought she'd live, but in fact got to adulthood. It was basically a full-time job for her (single) mom.

wincy4• 5 months ago

Sadly, the divorce rate is over 50% for parents of spina bifida children. My daughter is almost 7 and my wife and I are happy, we have a good life and I’m fortunate to have a career that lets her stay at home to tend to our daughters multiple monthly doctors appointments, and one where I can largely work from home to help when she needs carrying (which is often!)

That said, our daughter is a miracle, she’s intellectually sharp, and we do everything we can to give her all the experiences any other bright and outgoing child would have. I hold her up so she can play the games she can’t reach at Dave and Busters, even if my arms get tired. We go on hikes with an expensive stroller that’s also a bike made for off road biking because we read Little House and she wanted to know what prairie looked like. We plan on getting an RV to take her to national parks.

Her wheelchair tennis coach recently tried an exoskeleton that allows him to walk at a research lab in New York, she was elated. She asks me when her robot legs are coming. I tell her we don’t know but robots in every house would certainly help that sort of technology move forward. I tell her “they’ve got to test it on adults before kids can get one!”

When she was one, they told us to make plans for the future, to get our affairs in order. Three years later the palliative care worker who had told us our child wouldn’t live past her second birthday came to visit us during a hospital visit and talk to us, so happy to see a case where they’d all been wrong. I’m so happy they were.

Intermernet• 5 months ago

You, your wife and your daughter sound like wonderful humans, and I wish you all nothing but the best.

Aboutplants2• 5 months ago

Truly remarkable! Despite the darkness in the world most days, news like this lifts my spirits and gives me hope.

jey2• 5 months ago

What does the stem cell treatment help with beyond the existing fetal surgery? Since it's in addition to the usual surgical treatment

nickburns2• 5 months ago

  The CuRe Trial is exploring whether stem cells can add regenerative power to surgery, potentially improving mobility and quality of life.
  
  “This is a major step toward a new kind of fetal therapy, one that doesn’t just repair but potentially helps heal and protect the developing spinal cord,” said Aijun Wang, co inventor of the placental-derived stem cell treatment technology and the study’s co-principal investigator [ . . . ].

jey2• 5 months ago

That doesn't really explain anything biologically. Just vaguely says "potentially helps heal and protect"

Metacelsus• 5 months ago

As a stem cell biologist: my guess is that it doesn't help much

doomslayer9994• 5 months ago

China is going to be way ahead of us in biological treatments because they are willing to actually remove the red tape and in fact encourage scientists to try these sorts of experimental treatments. Meanwhile, we have a dinosaur FDA, a bureaucratic academia second to only Europeans.

dekhn• 5 months ago

What's the plan in China for when one of these expedited treatments has a terrible side effect?

The FDA's slowness is about maintaining a low level of risk because the reputation cost of a really bad incident is huge.

doomslayer9994• 5 months ago

Well, I'm no ethicist, but I don't think they really care. They'll catalog the results and try again.

Aboutplants2• 5 months ago

The good news is that the rest of the world will benefit from China’s treatment philosophy.

khuey• 5 months ago

What does any of this have to do with China? This study was done in California.

dclowd9901• 5 months ago

As the brother of a young, amazing man who we lost due to another genetic ailment (CF), whenever I see stories like this, it makes me so hopeful for families in the future never having to see a loved one deteriorate due to a bad roll of the dice.

Hats off to everyone out there putting in the hours to make the lived experience of these folks much better than they would have otherwise been. If only we had more of you in the world.

unglaublich• 5 months ago

Completely agree, this is why we need to detect bad rolls and give people the ability to reroll.

ASalazarMX• 5 months ago

Since this is genetic theraphy, it would be amazing if it corrected the bad roll permanently. Being in-utero, this might be the case here.

tclancy• 5 months ago

Can you expand on what you mean by that? Otherwise there’s a way to interpret it that is less than lovely.

willio582• 5 months ago

Tangentially related, but it is increasingly obvious that there's an ever-growing chasm between these two aspects of medicine in the U.S.:

- What's possible for medical professionals to do for certain conditions, in large part due to the amazing levels of investment into research and implementation.

- How difficult it is for ordinary people to receive care. Primarily due to private insurance companies intentionally making it more difficult to get care.

Like the fact we're giving stem cell therapy to fetuses successfully is amazing, yet any time I go to a doctor's office or bloodwork company I hear an elderly person explain to the front desk person that they've been on the same insurance for decades and only recently started receiving bills they can't afford, or listening to the front desk person explain that now medicare no longer covers them for a routine thing.

Ideally, we could have both great research _and_ great general care in this country. I just don't know if I will ever see that day.

ecshafer2• 5 months ago

I think the largest issue with health care right now is that the US is artificially shrinking the supply of Doctors. This is due to:

1. Size of medical school classes not increasing with population

2. US has an artificially small amount of residency slots.

These are largely due to AMA lobbying afaik and bad bills. But if we allowed every qualified medical student to enroll, and gave a residency slot to every graduate. In a decade we would have really shrunk the gap.

iamtheworstdev2• 5 months ago

the largest issue in American health care is private equity and middle men raising the cost of everything.

edit if doctor scarcity were the issue then doctors would have a lot more leverage in salary negotiations than they do, which is to say they don't have much. because the hiring practices are limited by what they can bill, which they have no power over.

exhilaration• 5 months ago

Does that matter though? My impression is that most people don't see doctors anymore. Every urgent care visit I've had in the past few years has been with a physicians assistant or nurse. Same for our pediatrician, I can't remember the last time we saw her instead of one of the nurses.

I actually have a routine visit with a specialist at one of the top hospital systems in the country in 2 days, and I see in the portal I'm seeing a "CRNP, MSN", not a doctor.

watwut2• 5 months ago

AMA was lobbying for more residencies for years. And residencies are the bottleneck.

holoduke• 5 months ago

The US is a country of cowboys. There is literally nothing that can be considered fair. The only thing what is left is the kindness of it's people. If that detoriates, well...

slibhb2• 5 months ago

Does that really happen "any time you go to a doctor's office"?

That aside, what if novel therapies like this are linked to the fact that US healthcare is expensive? If you make it cheap -- as in other countries -- there's less incentive for companies to invest and you get less research and fewer breakthroughs. Also fewer doctors, hospital beds, and more rationing.

In an ideal world, everyone would have exactly the right amount of healthcare. But our world isn't ideal, it runs on incentives, and it's not clear to me that all the hand-wringing over US healthcare will lead to positive changes.

willio582• 5 months ago

Does that really happen "any time you go to a doctor's office"?

Yes. I recently made a resolution to get established with all the medical professionals I don’t have set up. So a primary care, dermatologist, etc. over the past 2 months I’ve visited and had to go back a couple of times. I’ve literally overheard insurance-related issues in all cases. Whether it was the person in line before me or just overhearing people complaining while I’m in the waiting room.

Just last week I was waiting to get my blood drawn and the woman at the front desk, after continued prodding by an elderly man frustrated with lack of coverage, out loud said “Well, that’s insurance in America for you. Go ahead and call the number on the back of your insurance card because we can’t do anything for you.” Just deeply disheartening stuff to watch a late 80s man not realize after 15 minutes of being tossed between automated insurance phone responses that he simply won’t get the help he needs.

doctorpangloss• 5 months ago

That aside, what if novel therapies like this are linked to the fact that US healthcare is expensive?

you don't have to wonder, people have been writing about this as a major factor of costs for nearly 50 years

fasbiner• 5 months ago

This point of view runs directly against mutually agreed upon matters of fact: https://petrieflom.law.harvard.edu/2022/03/15/ama-scope-of-p...

The US healthcare system is not a market system nor did it occur naturally. Do you have any conflicts of interest that could cause you to have an emotional need to misunderstand basic information about it?

Cthulhu_• 5 months ago

What may be necessary is for other countries to be better. These treatments / studies don't just affect USAmericans but everyone everywhere, and if there's enough signals of "this treatment saves kids abroad but we can't afford them in the US because of policy", MAYBE said policy will change. Maybe. Not likely because the corporations have control over the government, and the US government system is stuck in laws drafted up in the 1700s.

wincy4• 5 months ago

This is amazing to see. Spina Bifida is tough as it can range anywhere from “wheelchair bound and substantial intellectual disability” to “has a hard time with potty training”, and you won’t really know until your child has been born.

My daughter is seven now and was born with spina bifida and it’s been a long journey full of pain, but also joy. The first nine months she had breath holding any time she’d get upset (which for a baby is all the time) so she was simultaneously the healthiest and least healthy baby in the NICU.

She’s wheelchair bound, but intellectually very sharp. Getting her a spinal shunt a few weeks after birth helped alleviate spinal fluid pressure in her brain, although it elevates scenarios of “kid has an headache” to “maybe we need to go to the ER at 3am in the worst snowstorm of the season”, but she’s extremely sociable and a light to everyone that meets her.

I’m glad work is being done that can mitigate this and improve quality of life for these children. She keeps asking me when she’ll get her robot legs and we tell her they have to test it out on adults first to make sure it’s safe! Exciting times for people with physical disabilities.

armadsen4• 5 months ago

Thanks for sharing about your daughter. It's good to hear from other parents dealing with it. My daughter with spina bifida is only 18 months. It's early of course, but so far she's right on track cognitively and socially. I expect she'll be in a wheelchair full time, but don't know for sure yet. She's also just the most joyful, loving, social, happy toddler.

I know what you mean about elevating otherwise normal kid illness. She had her first shunt failure in January. It was pretty stressful going from "hmm, she's fussy" to surgery within less than 24 hours. But she was back to her happy self with hours of the surgery too, which was a pretty stark indicator of how much the shunt is doing for her.

wincy4• 5 months ago

One of the nicest things anyone ever did for me was the day after she was born, when I had just started at a new job, an older dev asked if he could talk to me and pulled me into a meeting room. I didn’t know anyone there and was confused. He’d overheard me talking about my daughter just being born, and said “I’m nearly 60, I have spina bifida, and I want you to know it’s going to be alright.” He stressed how important doing a regular catheter routine is, as the most likely thing to kill him in the next 10 years is kidney failure due to bad catheter habits as a kid and young adult. Also, overnight foley catheters with a bag has been a lifesaver, and gave us our sleep back. The doctors often just won’t mention these things, and the quality of care can vary greatly depending on your care team, as I’m sure you’re well aware. At least the doctors seem to have some respect for us as caregivers to a medically complicated child and they often trust our judgment.

Hopefully it’ll make you feel better, but the shunt revisions became far less common for us as her growth slowed down. She had one around a year old, but they put a good amount of tubing in there, and they haven’t had to go do a revision in the past 6 years.

weregiraffe• 5 months ago

My daughter is seven now and was born with spina bifida

When did you find out she had the condition?

trhway2• 5 months ago

It sounds like stem cells come from unrelated people and no rejection, etc?

ajb• 5 months ago

It wasn't so long ago that doctors assumed that it would be better for the family if babies like this died quickly - and quietly helped them off. I had a cousin with spina bifida, who only lived because her own mother was a doctor and prevented this outcome. My cousin got to live to adulthood and enjoy life, but still died early because of an infection; likely a consequence of the physical impairments of this condition. It's amazing to see hope for a complete cure.

7777777phil• 5 months ago

N=6 Phase 1, so this is purely a safety readout. But does the stem cell patch actually prevents hydrocephalus or just delays it.

lets see 35 patients in Phase 1/2a

juancn• 5 months ago

Spina bifida is usually preventable by folic acid intake (and avoiding some medications).

It pains me to see it is still so prevalent.

First in-utero stem cell therapy for fetal spina bifida repair is safe: study

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