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FDA poised to approve first gene-editing therapy, made by local drug firms

Marie Tornyenu missed more than 100 days of high school while hospitalized repeatedly for sickle cell disease, an inherited blood disorder that causes crippling pain. Somehow, she still managed to complete at least eight advanced placement courses, play clarinet in the school band, and get admitted to Boston University.

Then in December 2021, after transferring to Cornell University, she received an experimental medicine that used a revolutionary gene-editing technology to treat inherited diseases. It freed her from excruciating bouts of pain and the frequent blood transfusions that are a mainstay of sickle cell treatment.

…

The life-changing drug, developed by Boston-based Vertex and its Swiss partner CRISPR Therapeutics, is expected to be approved by the Food and Drug Administration by Friday for people with severe cases of the disease. Called Casgevy, it would usher in a new era not only for those with sickle cell but also for medicine: The drug would be the first gene-editing therapy authorized by US regulators, and uses a tool called CRISPR.

The likely approval — Casgevy was cleared by British regulators last month — raises both the promise of cures for diseases as well as the ethical concerns that come with the power to manipulate the building blocks of human life. With an expected price tag in the seven figures, it also touches on issues of equity in medicine.

CaptainPedantic ,

“Fun” fact about Sickle Cell Disease: it gives you significant protection against malaria. If you have the sickle cell trait, you’re a carrier for the disease, but you don’t have symptoms of it. Being a carrier also gives protection from malaria.

FlyingSquid ,
@FlyingSquid@lemmy.world avatar

Severe chronic pain is one of the most debilitating things someone can go through. I know from experience because it kept me disabled for years. Mine is mostly managed through medication now, but that isn’t a hope for sickle cell sufferers, and sufferers of all sorts of other painful genetic disorders. This drug can’t be released to the public fast enough. I just hope it isn’t another situation like insulin where it is priced beyond what many sufferers can afford.

tacosanonymous ,

At a cost over one million dollars, I’m not sure it’ll be that helpful to the public.

FlyingSquid ,
@FlyingSquid@lemmy.world avatar

Depends on how much insurance is willing to cover of that. Insurance is willing to cover a surprising amount, although you will still probably be stuck with a fairly large bill (but in the thousands, not higher than that). Of course, if you don’t have insurance, you’re definitely fucked.

LastYearsPumpkin ,

Since this is treating a chronic, lifetime condition, with a single treatment, this is certainly something that you can take some time and pick a better insurance plan for a few years until the treatment is over.

It sucks that it’s even a consideration, but this isn’t a condition that suddenly comes up. You’ve probably been talking to your doctors about it for decades by the time you get to this point.

tsonfeir ,
@tsonfeir@lemm.ee avatar

The problem is that insulin didn’t need to be priced that high. Get everyone diabetes so we can sell them insulin and then jack the rate up for a while and make 1000x more money than we’ve got until someone complains and then lower it and apologize from our new vacation homes.

cheese_greater ,

Can I ask what med regime you came across that started to make a difference?

FlyingSquid ,
@FlyingSquid@lemmy.world avatar

I doubt it would help for chronic pain in general. I have atypical trigeminal neuralgia, a rare nerve disorder. After years of trials with various drugs, a combination of Lamictal, Cymbalta, Lyrica and, for a while Rexulti, seemed to work, but I stopped taking Rexulti when I got a new neurologist who wouldn’t prescribe it and I seem to be doing okay without it.

cheese_greater , (edited )

Pregabalin is such an incredible medication/substance, particularly for neuralgiac conditions. It never ceases to amaze me

Edit: this just inspired my next AskLemmy.ml series, spank-you all ;)

FlyingSquid ,
@FlyingSquid@lemmy.world avatar

It’s certainly done wonders for me. It didn’t help that I had two really bad neurologists at the start when my symptoms first showed up. The first one told me to take Gabapentin whenever I needed it, no limit. I took a huge amount and it didn’t help at all. The second one tried like 10 opioids on me and none of them helped. I didn’t get truly addicted thankfully, but I did have a pretty awful withdrawal which lasted a couple of days. Finally I found the one who suggested Pregabalin.

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