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

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.

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.

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.

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).

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

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.

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

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

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

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.

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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