Gene-Editing Trial on Three Individuals for HIV Cure Yields Uncertain Result 16
Antonio Regaladoarchive, reporting for MIT Technology Review: The gene-editing technology CRISPR has been used to change the genes of human babies, to modify animals, and to treat people with sickle-cell disease. Now scientists are attempting a new trick: using CRISPR to permanently cure people of HIV. In a remarkable experiment, a biotechnology company called Excision BioTherapeutics says it added the gene-editing tool to the bodies of three people living with HIV and commanded it to cut, and destroy, the virus wherever it is hiding. The early-stage study is a probing step toward the company's eventual goal of curing HIV infection with a single intravenous dose of a gene-editing drug. Excision, which is based in San Francisco, says the first patient received treatment about a year ago.
This week, doctors involved in the study reported at a meeting in Brussels that the treatment appeared safe and did not have major side-effects. However, they withheld early data about the treatment's effects, leaving outside experts guessing whether it had worked. "This is an exceptionally ambitious and important trial," says Fyodor Urnov, a genome-editing expert at the University of California, Berkeley, who believes it "would be good to know sooner than later" what the effect was -- "including, potentially, no effect."
A failure wouldn't come as a surprise to anyone familiar with HIV. It has proved a devious adversary: there is still no vaccine, even 40 years after the virus was identified in 1983. Still, pharmaceutical companies did develop antiretroviral drugs, which stop the virus from copying itself. Taking these pills lets people with HIV live normal lives. But if they stop, the virus will quickly rebound and, if left unchecked, cause the fatal syndrome of infections and cancers known as AIDS. One reason the virus can't be fully wiped out with drugs alone is that it inserts its genetic material into the DNA of our cells, leaving behind hidden copies that can restart the infection.
This week, doctors involved in the study reported at a meeting in Brussels that the treatment appeared safe and did not have major side-effects. However, they withheld early data about the treatment's effects, leaving outside experts guessing whether it had worked. "This is an exceptionally ambitious and important trial," says Fyodor Urnov, a genome-editing expert at the University of California, Berkeley, who believes it "would be good to know sooner than later" what the effect was -- "including, potentially, no effect."
A failure wouldn't come as a surprise to anyone familiar with HIV. It has proved a devious adversary: there is still no vaccine, even 40 years after the virus was identified in 1983. Still, pharmaceutical companies did develop antiretroviral drugs, which stop the virus from copying itself. Taking these pills lets people with HIV live normal lives. But if they stop, the virus will quickly rebound and, if left unchecked, cause the fatal syndrome of infections and cancers known as AIDS. One reason the virus can't be fully wiped out with drugs alone is that it inserts its genetic material into the DNA of our cells, leaving behind hidden copies that can restart the infection.
Good stuff! I hope it worked (Score:3)
If they can cure aids then there are a long list of other nasty things they should be able to correct as well.
We live in amazing times.
Re: (Score:3)
The gobn't is changin ma dna! Ain't no way I'm lettin 'em near me no matter how sick I is.
Sadly, stupidity is something which will never be cured.
Re:Good stuff! I hope it worked (Score:4, Interesting)
I wonder if herpes and Porphyromonas gingivalis could be targeted this way, too. Both can hide from the immune system by tucking themselves into human body cells. They've been linked to Alzheimer's disease, and the latter may play a role in arterial plaque buildup.
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You said it. "If".
Why withholding: how long te treatment takes (Score:2)
My wild guess is although they know the new drug acts against the viral sequences hidden in the human cells, they cannot tell yet if the treatment works as a cure from a limited number of doses, or if patients will need to take it continuously for a very long period of time until the body is clean. They want to tell the audience they have a cure and become instant billionaires. If it's Just another drug that reduces viral load that you need to take for a lifetime, the value is much more limited. We already
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I doubt it. If they had any indication at all that it had worked, they'd be trumpeting it - loudly.
Re:Why withholding: how long te treatment takes (Score:4, Insightful)
Maybe? Depends on if they've vetted out their process. Lots of folks are currently in the industry on this with lots of patents abound. If they have a method that's not been vetted, they'd risked lawsuit explaining too deeply.
Remember that a lot of greed (A LOT) is in medicine. And so scientist that discover things need to be careful because every pointy haired demon wants their pound of flesh.
Re:Why withholding: how long te treatment takes (Score:4, Insightful)
We already have drugs that do that, another one based on another concept is a nice-to-have but they'd still need to convince doctors to change their practice, which is difficult.
Cost. Current antiretroviral therapy (ART) is expensive, especially in the US. So it depends on what the cost of this is. While usual CRISPR stuff is in the millions of dollars for treatment, that's because it's usually tailored made to target codons specifically in the patient. Whereas, the HIV codons are similar in all infections for people, so no tailor made payload is needed. But again, it really depends on what these folks are doing. But tons of room for more ART in the market as costs for the medicine is just insane per month.
and the only way to know is to tell to stop taking their medications and make weekly analysis to see if the virus shows up again (which I speculate could pose a risk for the patient).
I mean that IS how it is done. Monitoring of really new things is usually daily. Does indeed pose risk, patient is usually informed of this. But that's how it gets done.
Re: Why withholding: how long te treatment takes (Score:2)
Well if left wing conspiracy theories are to be believed, drug companies don't work on cures because ongoing therapy is more profitable.
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drug companies don't work on cures because ongoing therapy is more profitable
I've only heard idiots spout this. Political affiliation is usually not a determining factor. I have heard the same argument from ultra-Conservative hillbillies in Coalmont Tennessee and Liberal "free thinkers" from the Green Hills area in Nashville. So while you're trying to attribute political bias to further, well whatever it is you're trying to further, most people understand this kind of garbage is more closely associated with the attribute "idiot" and less any political attribute.
Re: Why withholding: how long te treatment takes (Score:2)
Right here on slashdot is where I hear crap like this the most. rsilvergun is full of these nuggets. I haven't heard this particular one come from the other side.
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They say it's about custom tailoring. But they also are trying to say it's about the "value". Most firms use ICER [icer.org] to determine the price of a drug relative to it's value. With the recent sickle cell therapy, they have stated it's $2.05M, because "The treatment is preventing more attacks and it's worth more" [reuters.com]. While there's a certain logic to it, that's ignoring many fundamental facts of reality.
ICER is in my mind controversial; they use extremely basic economic
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CRISPR edits DNA differently in different cell types. If there's HIV in your blood cells, your skin cells, your bone cells, etc, it has different levels of success on the same DNA. And unfortunately success is often defined by what's called an Indel score or a Knockout score (there's subtle differences), and often a "good" editing chemistry results in 80% of cells having a successful "INsert/DELetion" of the target sequence and proper repair of the genome, but it's in that % of cel
Caution is warrented. (Score:2)
Too Soon (Score:2)
Bone marrow transplant (Score:3)
Is, to the best of my knowledge, the only treatment that has been 100% successful at wiping the virus from the body, and even then I'm only aware of two occasions. It may have been tried lots more.
Bone marrow transplant is difficult, unpredictable, unreliable, requires a very close DNA match, and is by all accounts extremely painful. So I can understand doctors wanting some other options on the table.
The lack of announced results would suggest the CRISPR method isn't as successful as hoped, or that it's hard to know whether it has been successful or not.
I'm going to assume that it's used on people absolutely loaded on the antiviral, because otherwise cells would be reinfected as quickly as they were cured.
My guess, based on previous articles on the unreliability of CRISPR to make major changes in low doses, is that the trials didn't factor this in. Biohacking with CRISPR is reportedly very difficult which is why the experts keep saying the publicity stunt biohackers probably aren't having any effect at all. But this logic must apply to single low dose medical trials too.
In that case, the technique might work better if it wasn't a single dose and if the dose was a lot larger.
However, I am but a mere Slashdotter who only knows what he's read.