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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
21 comments
[ 2.6 ms ] story [ 45.5 ms ] threadLong 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.
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.
- 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.
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.
lets see 35 patients in Phase 1/2a
It pains me to see it is still so prevalent.