First in-utero stem cell therapy for fetal spina bifida repair is safe: study
health.ucdavis.edu217 points by gmays 7 hours ago
217 points by gmays 7 hours 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.
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.
Completely agree, this is why we need to detect bad rolls and give people the ability to reroll.
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.
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.
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.
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.
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.
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.
This affect is because of the doctor shortage though.
I am in the process of trying to find a primary care provider, and I cant find anyone accepting new patients.
Bigger places you basically see the doctor for 2 minutes when you actually need one. I went to a ortho surgeon and they had a dozen patients “seeing them” at the same time. As he just went between rooms and nurses prepped everything.
I went down a Reddit rabbit hole, a sub called /r/noctor. Basically people, mostly doctors, complaining about the prevalence of nurse practitioners, PAs practicing independently/outside of their scope, etc. The general consensus I see there is that the only people benefiting from this are private equity firms trying to squeeze more profit since they bill the same based on whether you see a doctor or an NP. This in turn has an affect where it doesn’t make sense financially to go through so much school and take on so much debt.
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.
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.
> 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.
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...
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).
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.
Best news is the stem cells used are from donated placentas. So no political footballs, just a true blessing to a child.
I did not even know it was possible to operate on a fetus. Its insane how far we have come. Very promising results!
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.
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.)
What does the stem cell treatment help with beyond the existing fetal surgery? Since it's in addition to the usual surgical treatment
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 [ . . . ].That doesn't really explain anything biologically. Just vaguely says "potentially helps heal and protect"
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.
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.
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!
Truly remarkable! Despite the darkness in the world most days, news like this lifts my spirits and gives me hope.
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.
The good news is that the rest of the world will benefit from China’s treatment philosophy.
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.
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
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There are people who do not want to have abortions. Either for religious reasons, because they themselves belong to a specific disability group (e.g. the blind and deaf) or because they tried hard already to get pregnant in the first place.
Especially for the latter, "make a new one" can mean a ticket reaching into the 6 figures, months of egg extractions, implantation attempts and spontaneous auto-abortions.
What a gross and idiotic take. “Just abort it”; i’m pro choice, but IMO life must be regarded as more sacred than what ‘ole tsss here considers. I can only conclude tsss has never dealt with any real loss in their life.