First in-utero stem cell therapy for fetal spina bifida repair is safe: study
health.ucdavis.edu
health.ucdavis.edu
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.
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.
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.
When did you find out she had the condition?
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.
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.
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.
- 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.
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.
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.
None of that is efficiency in any reasonable sense.
> “As our investigation revealed, these financial entities are putting their own profits over patients, leading to health and safety violations, chronic understaffing, and hospital closures. Take private equity firm Leonard Green and hospital operator Prospect Medical Holdings: documents we obtained show they spent board meetings discussing profit maximization tactics—cost cutting, increasing patient volume, and managing labor expenses—with little to no discussion of patient outcomes or quality of care at their hospitals. And while Prospect Medical Holdings paid out $645 million in dividends and preferred stock redemption to its investors—$424 million of which went to Leonard Green shareholders—it took out hundreds of millions in loans that it eventually defaulted on. Private equity investors have pocketed millions while driving hospitals into the ground and then selling them off, leaving towns and communities to pick up the pieces.”
https://www.grassley.senate.gov/news/news-releases/private-e...
No, private equity is not the reason healthcare costs in the US are out of control, you can even ask chatgpt.
PE is a 3rd tier mild symptom in certain niche health markets that sits downstream of all the structural root issues created due to the twisted public/private incentive misalignment nightmare of US healthcare.
It's been poisoned by the hysterical climate of US politics like everything else on the internet, so there are no thinking men left.
It's a lost cause. If I were to explain the situation rationally I would get downvoted for not cheering on the shooting of CEOs.
Upvote communities are all dead and dying. There are no more interesting conversations happening in them anymore.
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.
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.
The primary utility of most medical professionals is to diagnose and treat a condition correctly. In the ER and elsewhere, the correct diagnosis is indeed often "drug seeking behaviour". And this is also a major aspect of medicine that many relatively healthy people interface with and remember. They are in pain for whatever reason, they desire to be relieved of said pain, and that desire puts them into contact with the skepticism and hesitancy around opiods that physicians have built up out of unfortunate necessity. It's often a hurtful and protracted experience, and so they remember it and form opinions like yours.
But this area of contact with medicine is a tiny, very visible tip of a much larger iceberg. Your description of "security guard around medication" is not strictly wrong for my field, seeing as internal medicine is largely about administering the right drug at the right time, but the 99% of the drugs we guard are not desirable at all for any drug-seeker. They are potent, full of side effects, are sometimes potentially deadly. But they do work. And you do not see any of this until you get properly sick, which to most people does not happen very often often (at least until they approach 70). And when it does happen, most people tend to focus on the one little side of the ice berg they come into contact with. But it is there, and it is about much more than distinguishing you from a drug seeker.
There are limits, naturally. I don't really expect to fit the percutaneous pins into my hand myself, even if I had third hand capable of equal dexterity. But if I have to sing a song you can be sure the song is sung. It's no different from selling B2B SaaS. You just need to make the sale.
My point was not about the emotional experience of being presented with a certain viewpoint of the function of physicians. My point was simply that if you look at the details of what physicians actually do, the stated viewpoint is wrong.
Of course, "primary function" is a somewhat subjective concept that you could define however you'd like, so it is more or less unfalsifiable as a standpoint.
Others need to be told to “advocate for themselves”. I simply get what I want and it always works.
Let us say they try to simulate an acute ruptured appendicitis. If they do this convincingly, they will get an acute CT with contrast. In my hospital system these machines and interpretation of resulting images is expensive and resource constrained, especially during evening and night time, meaning that the prioritisation of one patient will generally mean that another, let us say a patient in the process of having a very real stroke, might get delayed if traffic is high.
This is beyond the fact that roughly 30-120 minutes of the physicians time in the ER will be wasted in examining the patient, ordering blood work, the imagery, writing notes, and so on, which means that another patients time, who is often literally waiting in line for your time, is being wasted. Furthermore this kind of clientele have an unfortunate tendency to become unpleasant when you tell them that you can't find any reason for their pain or giving opioids, which is an extremely unpleasant and frankly often traumatic experience for green eyed doctors that enlisted in this career with the goal of aiding the sick. You can only get threatened, spat upon or assaulted so many times and maintain your professional enthusiasm. Many quit for this reason. And for the ones that don't, the experience of being forced to take on the role of distinguish between drug seekers and non drug seekers will generally turn you into a more unpleasant human being.
In summary, mostly due to unfortunate societal circumstances, you really, really, really do not want to encourage drug seekers to try their luck. It is an expensive waste of everyone's time, in circumstances where both money and time is tight.
Conversely, you really cannot predict in advance which ones of your opioid-naive patients will become addicts because the opioids that you gave them, which effectively means that you've fucked their life forever. Opioids are really, really dangerous. Sometimes people are obviously in pain and you open the tap quickly. But there's a name for the historical consequence of playing fast and loose with pain relief, it's called the opioid epidemic.
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.
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.
you don't have to wonder, people have been writing about this as a major factor of costs for nearly 50 years
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?
- 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.
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.
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!
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 [ . . . ].lets see 35 patients in Phase 1/2a
It pains me to see it is still so prevalent.
The FDA's slowness is about maintaining a low level of risk because the reputation cost of a really bad incident is huge.