FDA approves cure for sickle cell disease, the first treatment to use CRISPR
FDA approves cure for sickle cell disease, the first treatment to use CRISPR
The groundbreaking approval has been eagerly anticipated by patients and doctors alike. The treatment is priced at $2.2 million per person.
Here is an NPR article on this.
This seems like a really big deal. The obvious downside is the cost but hopefully that will come down over time.
From what I saw elsewhere, the cost of the CRISPR treatment is roughly 2 million dollars and another way to implement the cure is via a modified flu virus. That version is roughly 3 million.
If only we knew what the real costs of treatment are, not the bullshit prices the industry decides they'll say it is and then negotiate a barely more realistic real cost with insurance companies.
Guess we'll have to wait until this is approved in other countries for a real answer.
It's actually not expensive just because. They don't manufacture this stuff in a pill packing plant with an automated machine that just churns this out.
Cell therapy takes blood from a patient and manufacturers with it to make the drug. It's made manually by a team of people for a specific patient. The material costs alone are a quarter of the price in most cases.
Cell therapy ain't cheap.
It's tricky because the money, time and opportunity cost gone into development, testing and the approval process are also priced into this. Plus the fact that this needs to not only break even but make some money plus the fact that this won't be relevant for a huge market I think (not sure how prevalent SCD is). So it's an outrageous price but probably not just plucked out of thin air
wow this is huge for anyone with sickle cell that lives in a country that has universal healthcare. Other countries I guess it's great for super rich people.
I'm wondering how such a treatment will be handled in countries with a high malaria prevalence
There's a new, more effective malaria vaccine coming out right now:
Sweet
I wonder why it's so expensive.
Emergent tech always starts out very expensive.
Consider that the phone you probably posted this on is significantly more powerful than the first computers, which were several orders of magnitude larger and more expensive.
Manufacturing costs alone are insane. I work in biopharma, specifically manufacturing.
Most of these drugs you see on headlines are made from the blood of the patient. You can't mass produce that.
You can't mass produce that.
Challenge accepted.
This is the best summary I could come up with:
The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black.
In April 2021, Morning joined the clinical trial at Sarah Cannon Research Institute and HCA Healthcare’s The Children’s Hospital at TriStar Centennial in Nashville, Tennessee, a decision she initially regretted.
Dr. Haydar Frangoul, medical director of pediatric hematology-oncology for the Sarah Cannon Research Institute, said he is hopeful the therapy will provide relief to more patients.
Dr. Nicole Verdun, director of the Office of Therapeutic Products in the FDA's Center for Biologics Evaluation and Research, said Lyfgenia was given the warning after two patients who got the therapy in a clinical trial died from a form of leukemia.
It's unclear whether the gene therapy itself or another part of the treatment process, such as the chemotherapy, caused the cancer, but Verdun said the agency thought the deaths "rose to the level of a black-box warning."
Christopher Vega, 31, from Allentown, Pennsylvania, said the possibility of long-term effects aren’t a concern for him; he is happy to be living a life free of chronic pain.
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Alright real question here. I've noticed after I came on reddit and Lemmy, something I believe I've only seen in American media.
Why is it so important to mention people's skin colour all the time? This affects 100.000 Americans, and then they mention most are black?!
Why does that matter? I can't figure out why on earth they want to divide people up in race like that, like they are a different people. A different kind of Americans.
It's not hard to figure out why Americans are so divided, if they get talked about like a different group of people based on skin colour.
Because being black isn’t just a skin color. It also includes a genetic predisposition to this disease.
For one, it's a genetic disorder that's linked to ancestry.
For another, there's a huge social-justice issue around race in the US. Black people are hugely overrepresented in prisons, and there are vast, ingrained and systemic prejudices and barriers to equality everywhere you look.
This is why ferinstance efforts to enforce photo-ID requirements for voting would significantly disenfranchise Black voters (which is the real reason conservatives keep trying to push for it), because a disproportionate percentage of Black people simply don't have the means to obtain such ID - vastly more than the number of white people in a similar position.
Take centuries of explicitly racist law and policy directly excluding and oppressing Black people. Wind back the explicit racism over the last handful of decades, but quietly replace it with exclusion and oppression based on the socieconomic disparity thus created. Now you can claim to have a colourblind legal/etc system, yet continue to preserve and promote disadvantage that ends up depending on race.
And because social mobility is an absolute joke over there (as it is in most places, tbh), then the opportunities and resources available to you will depend heavily and primarily on the opportunities and resources available to your family and community for many generations back. There's not just generational wealth to deal with, but generational influence and networking; you can't be in with the Right People unless your parents were, and neither could they, rinse and repeat. And when the Right People instantly dismiss you on sight, or even just by seeing your name, then you're fucked.
So when someone says that a hundred thousand people in the US have a crippling and debilitating disease, and the great majority are Black - yeah, it means something. It means that a chunk of the population are getting a raw deal on top of the shit sandwich they've been handed just by being born, and that this is just one more thing that, for the most part, white people don't have to care about. And because the fucked up healthcare system in the US is so profit-driven, that means there's going to be vastly less done about it than if it were equally spread across demographics.
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