The New York Times reports something amazing:
On Sept. 11, despite the excitement of the moment, Kendric was unable to keep his eyes open as he lay in his hospital bed at Children’s National Hospital in Washington because of the drugs he had been given in preparation for his treatment.
His life with the disease has been punctuated by episodes of excruciating pain, requiring days in the hospital as doctors tried to control it. Sickle cell eroded his hip bones. It prevented him from riding a bike or playing soccer or even going outside when the temperature was below 55 degrees Fahrenheit because cold often brought on intense pain.
Now he could see a future — in a month or so — without pain from sickle cell.
“I can’t wait to start my new life,” he told his mother, Deborah Cromer….
Kendric was lucky. His insurer was cooperative. He also rose to the top of the list because of the severity of his disease…
In a month, if all works as intended, he should be making blood cells that will not sickle — the cells will have a normal hemoglobin gene.
There are a pair of gene therapy treatments for sickle cell disease that are highly likely cures. This is stunning and amazing.
It is also wicked expensive. The cheap cure is as expensive as a replacement level veteran bullpen arm while the more expensive treatment is equivalent to the salary cap charge of a mid 1st round NFL draft pick. And this leads to problems.
One of the big hold-ups has been insurers and hospitals figuring out payment and prior authorization. Insurers want to be very careful about paying for only highly likely successes while hospitals are wary about holding a massive cash receivable that won’t be paid for months if not years. One of the challenges is that the treatment is usually a single contract period cash expenditure even as the benefits hopefully accrue in the form of bike riding and reduced hospitalizations over decades. There is a time mismatch.
As more of these types of treatments and cures come down the pipeline, these types of mismatches will be ever more important.
Balloon Juice Operation Blue Arizona
KatKapCC
This sounds amazing. I really really hope it works and is able to be scaled up to more patients. Though it does break my heart that, as with almost anything else healthcare-related in this country, too many people involved are talking about money before anything else.
(Also…why did the Times think they had to include “Fahrenheit”?)
scav
@KatKapCC: Have you ever met people who post comments to newspapers? They’re not exactly the sort to think through that only one of the two options is plausible. They’re the sort who write in objecting to the non-zero sugar amount in cookie recipes and explain how they’ve improved the recipe by using mushroom powder, liver flukes and pounded guava. Even the everloving FNYT must tire of that.
sab
I am a white woman with ancestors back to 1650 in America. I also have black grandchildren with sickle cell traits. Anything that helps on sickle cell is a miracle to me. Would be anyway, but I thought I’d toss in the grandchild just for credibility
ETA Most blacks in America (except Barack but not Michele) have older pedigrees in America than mine.
I don’t think how long you have been here matters, but other people do.
rikyrah
PRAYING that this is successful. It would be amazing.
JCJ
Another aspect of avoiding future medical care is also avoiding skeptical (perhaps hostile?) medical personnel. I remember way back when in Wishard hospital (the county hospital of Indianapolis) as a medical student being told not to give narcotics to patients with sickle cell trait claiming pain from sickle cell crisis since they were obviously drug seeking. Interestingly there is a youtube channel from a sports medicine doctor that covers mechanisms of injuries, but recently he had a video on an NFL player named Ryan Clark who had sickle cell trait who had severe pain while playing in Denver – apparently the altitude caused enough hypoxia to trigger a crisis.
https://www.youtube.com/watch?v=wMM_S-Eccg0
PAM Dirac
@sab:
Barack’s father’s line is obviously very recent to America but his mother’s line goes back to a man who came to Maryland as an indentured servant about 1657. I know this because my wife is also descended from this man. IIRC she and Barack are 8th cousins, a fact that I try to remind her Trumpy brother of as often as I can.
Nelle
My brother-in-law is the only surviving sibling out of nine children. Several died of sickle cell disease. I’m so impressed with the scientific research, so discouraged with monetized health care. How does this world hold the spectrum of people intelligent enough, creative enough to develop these therapies, these vaccines, one after another, and, in that same world, shuffling vaccine refusers and just generally blunt brained MAGAs???
Hallmark
David, can you comment on “treatment bonds”, where the treatment cost is spread over say 10 years and whoever is the insurer each year pays that year’s payment on the bond. The idea being that insurance time horizon is at least a little more closely aligned with the value to patient timeline.
Anonymous At Work
Kudos on the sports analogies. It contextualizes that the average person’s “local billion dollar business” (aka sports franchise) making this decision would have repercussions if they make a mistake but it’s not the end of their existence. A make-or-break decision for a mid-level analyst, but nothing too major for CEO.
gene108
I had a kidney transplant.
Luckily for me “the kidney lobby” was able to get Congress to agree to have Medicare pay for dialysis and transplant surgery, in the early 1970’s, when such treatments became viable but incredibly expensive for private insurance to want to pay for.
I didn’t have to worry about my insurance raising objections to treatment.
I wonder why – other than the obvious fact Republicans will oppose it – that expanding Medicare to cover other solid organ transplants and new treatments like sickle cell disease have never really been considered.
I understand taxes have to be raised, but it would make accessing life saving surgery or treatment easier for many people and the cost would be minimal to people and employers versus the lives saved.
Rugosa
@gene108:
This is wonderful news. As these new “miracle” treatments increase as we hope they will, perhaps it will spur action toward implementing government-based health care. I understand you can’t upend the existing insurance system overnight, but it may gradually become apparent that if government is stepping in piecemeal, they may as well just raise taxes and pay for everything.
Obvious Russian Troll
@KatKapCC:
The NYT does have an international audience. As an American living in Canada, I now use both on a regular basis. I also can’t spell words like “center” or “theatre” consistently any more.
Great to see that this treatment is available, and hope it becomes much more common (and affordable).
Mel
I hear the money arguments, and know that with a for-profit healthcare system, $ are supposed to always be the deciding factor and the bottom line, but it should not be. Whether a person is a child or an adult, a parent (or a childless cat/dog person!) they should be able to access life -saving and life-improving treatments without going bankrupt and without having to battle tooth and nail against corporate entities.
If insurance companies had paid for appropriate treatments, I wouldn’t have lost: 2 decades of earning ability, a career that I loved, my chance to have children, my dignity, etc.
The new treatments for sickle cell are just one example of the treatments in the pipeline that could spare kids and adults like Kendric all the loss and pain that do many chronically ill people have and continue to suffer everyday.
I hope that sweet little guy gets to ride his bike with joy, gets to experience the relief of waking up in a body that works with him, not against him, and someday teaches his kids and grandkids to ride their bikes.
Skepticat
@sab: I am a white woman with ancestors back to 1640 in America; the chances are good we’re distantly related. There also are Black people in the family line, though not recently.
This treatment is wonderful news!
David Anderson
@Hallmark: These ideas really need some collaborating and forcing mechanism to make sure that there is no free riding but it is an elegant solution and has been an elegant solution for 30+ years which means that it is not an easy solution as it would be done by now.
Elizabelle
Marvelous news. I wish Kendric a healthier and more active life.
Smart people rock. And this took a LOT of work.
(In earlier thread, TBone informs that Scientific American has endorsed Kamala Harris; its second endorsement in its 179 year history.)
Vote for Kamala Harris to Support Science, Health and the Environment
Kamala Harris has plans to improve health, boost the economy and mitigate climate change. Donald Trump has threats and a dangerous record
And their first paragraph has got me tearing up. Because of the “she.”
They rate her on healthcare, reproductive rights, gun safety, environment and climate, and technology.
Suspect another front pager will highlight this endorsement (right up Tom Levenson’s alley, among others).
Elizabelle
AND: the first candidate Scientific American ever endorsed (in its then 175 year history):
Joe Biden. In 2020. Link here.
Baud
@Elizabelle:
Good choice.
Elizabelle
@Baud: I love it. Will be a great help on the doors and talking to “undecided” voters.
Marci Kiser
I think it will be both harder and easier to start seeing approvals roll out just because (for many reasons) a significant percentage of the sickle cell population is on CHIPS/Medicaid, where the individual states will all be making their own rules.
I don’t foresee red states jumping at the opportunity to spend money on a disease that almost exclusively impacts brown/black people (have seen exactly one white person with sickle cell in my entire career). It will be interesting to compare their approval criteria for this vs. the HepC treatments – less expensive relatively speaking but also far more prevalent in their white constituents.
BubbaDave
My question is aren’t treatments like this a textbook case for some kind of coinsurance, where the Feds put a cap on one-and-done alternatives to lifetime management approaches of $X and then the cost over $X is paid by the federal government in exchange for the insurers paying the Feds $Y/insured human/yr?
karen marie
karen marie
Did my previous comment get stuck in the tubes?
Here’s the link to a study of pharma profits.
David Anderson
@BubbaDave: A reinsurance program or a federal back stop would make a ton of senses here.
narya
This is fantastic news!
On another topic: if a state has a requirement–e.g., a work requirement for Medicaid–AND they have predicted that, say, 25,000 people will become insured, BUT only fewer than 5,000 people have become insured, can the feds intervene and say their program is failing and revoke approval?
Ohio Mom
@gene108: Maybe off topic but I have long wondered how the “kidney lobby” pulled that off. Were there Congressmen who have family members with kidney disease?
Ruckus
@Nelle:
I am the only one left in my family of 5. Alzheimers got one, cancer got 2, the other one old age but had cancer, and I had a cancer that is easily treatable and has been. One of the cancer patients had it metastasize throughout the body. So the 4 having genealogy from the oldest of them got cancer.
rikyrah
@PAM Dirac:
That he’s a descendant of a slave…from his White side…is about as American as apple pie.
Ruckus
@gene108:
I wonder why – other than the obvious fact Republicans will oppose it – that expanding Medicare to cover other solid organ transplants and new treatments like sickle cell disease have never really been considered.
The cost is why. We could have a much healthier nation and how many of those that argue against better healthcare insurance so that more people would live are assholes who think they should get healthcare but screw everyone else? Some humans are just selfish fucks. I have no idea how to fix that.
Ohio Mom
@Ruckus: Most cancers aren’t related, as far as we currently know.
Even in breast cancer, where we have identified two genes that can trigger it, BRCA1and BRCA 2, only 10% of people with breast cancer have it as a result of having a BRCA gene (if you look in the other direction, having a BRCA gene can give you up to an 85% of getting breast cancer. That’s a very big risk but still not 100%).
Most cancers are the result of mistakes made in cell division. Considering how much cell division going on inside us, it’s amazing there aren’t more cancers — though one reason we don’t is because our immune system nips a lot of potential cancers in the bud.
Anyway, live long enough and it’s likely some type of cancer will gain a foothold somewhere in your body, in part because our immune systems tend to get less efficient in our elderly years.
Starfish
This cure is remittances owed to black folks by the US government. Many patients who are going to need this are going to be poor and from southern states that formerly held most of the slaves. These will include states that did not do Medicaid expansion.
Will insurers that cover those states pull out of those states before covering this? Will the paltry Medicaid plans than those states offer cover this?
Ruckus
@sab:
I have no idea how long ago all my ancestors came to the US but I know one grandfather was born in the second half of the 1800s in Sicily. I know very little about him other than what he did and why he had to leave Sicily. He passed away 13 yrs before I was born. Dad’s parents brought him to CA when he was 1 yr old, to Los Angels from Kansas City in 1918 in a horse drawn wagon.
bookworm1398
If we had single payer, the timing of cost would not be an issue. As it is, I can hope the cost will decrease as the treatment becomes more common.
Im curious about how this works, specifically will his kids still get sickle cell?
rekoob
@Hallmark: @David Anderson: @BubbaDave: Chiming in late to note that this might be amenable to a “catastrophic bond” approach, leading to some interesting implications for Insurance-Linked Securities (ILS). ILS’s can be attractive additions to diversified portfolios:
https://en.wikipedia.org/wiki/Insurance-linked_security
Noting in this case “attractive” means enhancing the overall risk/return characteristics of a portfolio.
Glidwrith
@bookworm1398: Sickle cell is recessive. Regardless of the treatment, unless he has kids with someone who is heterozygous for the trait, his kids will be heterozygous and therefore will not manifest the disease.