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Medicine Biotech

Teenager's Incurable Cancer Cleared With Revolutionary DNA-Editing Technique (bbc.com) 78

Posted by EditorDavid from the fighting-the-fight dept.
"A teenage girl's incurable cancer has been cleared from her body," reports the BBC, "in the first use of a revolutionary new type of medicine...." Doctors at Great Ormond Street Hospital used "base editing" to perform a feat of biological engineering to build her a new living drug. Six months later the cancer is undetectable, but Alyssa is still being monitored in case it comes back.

Alyssa, who is 13 and from Leicester, was diagnosed with T-cell acute lymphoblastic leukaemia in May last year.... Her cancer was aggressive. Chemotherapy, and then a bone-marrow transplant, were unable to rid it from her body.... The team at Great Ormond Street used a technology called base editing, which was invented only six years ago [which] allows scientists to zoom to a precise part of the genetic code and then alter the molecular structure of just one base, converting it into another and changing the genetic instructions. The large team of doctors and scientists used this tool to engineer a new type of T-cell that was capable of hunting down and killing Alyssa's cancerous T-cells....

After a month, Alyssa was in remission and was given a second bone-marrow transplant to regrow her immune system.... Alyssa is just the first of 10 people to be given the drug as part of a clinical trial.
Her mother said that a year ago she'd been dreading Christmas, "thinking this is our last with her". But it wasn't.

And the BBC adds that applying the technology to cancer "only scratches the surface of what base editing could achieve.... There are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia."
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Teenager's Incurable Cancer Cleared With Revolutionary DNA-Editing Technique More

Teenager's Incurable Cancer Cleared With Revolutionary DNA-Editing Technique

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  • Solid use of science (Score:5, Insightful)

    by jhoegl ( 638955 ) on Sunday December 11, 2022 @06:52PM (#63122634)
    This is a great use of science that I hope can, one day, allow for the cure to life threatening and life altering diseases. However, where one sees good, another sees evil. This type of tech will need to be monitored closely, so very closely. Imagine targeting peoples genetics in such a way as to go after melanin in the body, or goes after those with a certain hair color. Targeting genetics is a very concerning slippery slope, and I hope the human species can stick to the good side only.

    • Re: Solid use of science (Score:5, Insightful)

      by saloomy ( 2817221 ) on Sunday December 11, 2022 @07:41PM (#63122734)
      No it isnt and stop with the theatrics. I want different hair. Why not? I also want to be taller. I also want to not have history of colon cancer in my body. Everything would be true if my base pairs were editable. This is a window to that future and it is a good thing. As long as the science holds, and it does not have any bad side effects, sounds like a winner to me. If you dont want it, do not have it.

      • Re: (Score:3)

        by chill ( 34294 )

        You might want to read Change Agent [theverge.com] by Daniel Suarez. The criticisms in the linked review are certainly valid, but so are the positive points. And it is spot on for this discussion.

      • Re: (Score:2)

        by HiThere ( 15173 )

        Well, this won't work to make you taller, if you're already an adult. It *might* work for hair color or skin color.

        OTOH, I don't think the gp was thinking about optional uses of this.

      • No it isnt and stop with the theatrics. I want different hair. Why not? I also want to be taller. I also want to not have history of colon cancer in my body. Everything would be true if my base pairs were editable. This is a window to that future and it is a good thing. As long as the science holds, and it does not have any bad side effects, sounds like a winner to me. If you dont want it, do not have it.

        I'm hoping to get a 12 inch tallywhacker via this.

        The ladies tell me 16 inches is too much.

        • Re: (Score:3)

          by sjames ( 1099 )

          Poof, wish granted. Too bad you now pass out from hypotension within seconds of seeing an attractive woman...

      • Side effects are there with anything. You can't blame everything from their dog running away to their boyfriend cheating on side effects of the treatment. 2. some side effects will occur with anything. It is true, with any medical treatment, some percent of the people taking it WILL have side effects, but then a lot bigger percent of the people who don't take it will die. It's literally the equivalent of never traveling in a car because a certain percent of people who are in cars will get in an accident, th

      • Re: (Score:2)

        by jhoegl ( 638955 )
        Are you seriously suggesting I was talking about changing hair color? No... I was talking about developing a virus that targets a specific subset of people.
      • Either I'm misreading their comment, or maybe you are. I don't think they're suggesting you shouldn't be able to make cosmetic/non-essential changes with this tech. I think they were just implying that without proper oversight genetic treatments/improvements could be designed to target selective groups (i.e. only fix the cholesterol issues in white people).

    • Targeting genetics is where we were always going to end up, and you can't stop the desire of every single parent on the planet to want a healthy, intelligent good looking child. The best you could do is block it from being used by the poor but that seems the more dangerous slope.

      Face it, we are nearing the last generation of randos, within 30 years probably every single kid born will be like a genius supermodel, and then those that remain can enjoy the great works that come thereafter.

      • I agree, but I hope we find out what the genes for empathy are too. Genius without empathy is dangerous. I mean, look at post-pandemic Elon Musk .. unable to subdue his fear, hatred, and anger.

        • Genius without empathy is dangerous. I mean, look at post-pandemic Elon Musk ..

          People are already all extremely empathetic, which is why they are so easy to manipulate into believing things that are untrue/

          To me looks like we just need to bump up intelligence a lot, so people can recognize empathy [opindia.com] and not mistake it for something else or be misled b others with heavy GroupThink.

          • > To me looks like we just need to bump up intelligence a lot, so people can recognize empathy [opindia.com] and not mistake it for something else or be misled b others with heavy GroupThink.

            Curious how you define '"intelligence" and how you thing that leads to your expected outcome.

        • Genius without empathy is dangerous. I mean, look at post-pandemic Elon Musk .. unable to subdue his fear, hatred, and anger.

          Pretty sure he's actually 0-for-2 there.

    • "Curing" high cholesterol? Cookie eaters and dairy consumers rejoice I guess? LOL. Or, just don't eat those. Then you're "cured"...

  • Perhaps we will have to wait a few years or decades to see if this teenager grows a 3rd ear or develops a 6th toe on each foot.

    • Re:Any Side Effects? (Score:5, Informative)

      by c-A-d ( 77980 ) on Sunday December 11, 2022 @07:08PM (#63122676)

      I think she'd argue that it's better than dying at the age of 14.

      • I think she'd argue that it's better than dying at the age of 14.

        Unless she's a hellbent zealot on a warpath in which case she will argue that the doctors are playing God and on the path to eternal damnation before using that as a segue into some white supremacist crap. It's entirely unlikely, but I like to envision that she's a 4chan troll. :)

        • Mother will kill her because "she was supposed to go to heaven". I mean, we can ALWAYS ALWAYS ALWAYS count on the religious crazies in this world.

    • Explain how creating a T-cell, a cell which has only one purpose and that is to fight a specific type of virus [asu.edu], could possibly cause any bodily mutation to occur. Let's hear your "research".

      • Re:Any Side Effects? (Score:5, Funny)

        by rsilvergun ( 571051 ) on Sunday December 11, 2022 @07:15PM (#63122696)
        You see, it'll resonate with the 5G towers and the gay frogs to create a miniature worm hole...

        It's OK though, you can counter that with liberal doses of Brain Force (tm) and Hydroxychloroquine.

        • It's not that it COULDN'T do that. (With engineering, anything is possible.) But... why? So many more interesting things in this world to play with than people's sexuality and cell phone tower emissions.

          • So many more interesting things in this world to play with than people's sexuality

            I don't know about that. Republican senators recently told me that people are sexually identifying as dogs and want litter trays in classrooms. I think there's nothing more interesting in the world than people's sexuality. ... Or at least what Team Outrage thinks people sexuality is...

            Wait are we a protected class yet? In that case I sexually identify as someone who doesn't want to go to work today.

        • you can counter that with liberal doses of Brain Force (tm) and Hydroxychloroquine.

          Is anything safe from liberal meddling?!

      • The human body appears to run on self-modifying code. A little change in one place can have huge implications elsewhere.

        Having said that, I imagine the benefits outweigh the risks in this case.

        • Re: Any Side Effects? (Score:4, Interesting)

          by sg_oneill ( 159032 ) on Sunday December 11, 2022 @09:06PM (#63122916)

          Which is why we've been looking at this type of technology or variants of it for decades now without actually testing it. We've had to map the entire genome and get a grip with certain questions like "how does protein folding work" and "how do we trace how particular base pairs manifest".

          Cautious cautious cautious.

          The end result however is pretty damn impressive. Yeah of course we still need to proceed with caution. But on a risk versus reward basis, saving a little girls life outweighs almost any risk imaginable.

          Saving a child is the the kind of thing grown men throw themselves in front of automobiles or bullets for. A degree of scientific risk is pretty acceptable wthen the alternative is unthinkable.

      • Re: (Score:2)

        by NFN_NLN ( 633283 )

        Explain how creating a T-cell

        Explain how a cell, which isn't supposed to divide uncontrollably becomes cancerous? Shit goes wrong sometimes.

        • Epstein Barr virus, or some other gamma herpes virus.

          https://www.researchgate.net/p... [researchgate.net]

          Usually brought on by emotional stress. At some point, society needs to say that not every person is cut out to be a parent. More regulation is needed. Anyone can have a kid, with no background checks or training.

      • There are a bunch of autoimmune diseases caused by "friendly fire" from T-cells and B-cells that in theory should only be attacking pathogens. Negative selection for self-affinity is very good, but it's not perfect. The risks are very low, but they're not non-existent.

      • That's...that's not even barely true

        You have no understanding of what T cells are or what role they play in our immune system if you believe 'they only fight a specific type of virus'

    • over incurable cancer any day of the week. That said, I'm gonna say no, no side effects. What they did was train her immune system to go after cancerous cells. It's sort of like a vaccine.

      • over incurable cancer any day of the week. That said, I'm gonna say no, no side effects. What they did was train her immune system to go after cancerous cells. It's sort of like a vaccine.

        Shhh. Don't use that word. The kid could have ended up like the one in New Zealand [cnn.com] whose parents are infected with stupidity.

        • Shhh. Don't use that word. The kid could have ended up like the one in New Zealand [cnn.com] whose parents are infected with stupidity.

          While I'm glad the baby is in the care of responsible people now, I hope he does not grow up to be as stupid as his parents. Darwin would not be pleased.

    • I mean... did you want a 3rd ear or 6th toe? It seems more probable than ever with all of this gene editing. Maybe a billionaire somewhere will get some work done.

  • NHS is nice in the usa that will be $1M+

    • Yes, medical care is needlessly expensive in the US. But the NHS isn't free. They'll go broke if they hand out unlimited free cutting-edge DNA treatments.

      • They'll go broke if they hand out unlimited free cutting-edge DNA treatments.

        No, they will not: governments can print money, do you know?

      • Depends. That kid is likely now going to grow up and pay a lifetime of tax that otherwise wouldn't have been collected. Seems like a sound investment to me.

        That's exactly the kind of calculation that NICE does when deciding whether to adopt a particular treatment into the NHS. It leads to treatments being sold to the NHS at much lower cost than they otherwise would be. Seeing that most of them were developed using tax money to start with, I think that's fair enough.

        • In pure raw financial terms, no. https://www.taxpayersalliance.... [taxpayersalliance.com].
          According to the above, each British family will pay about 900K in taxes over their lifetimes. The average size of a British household is 2.4 individuals, so about $380K per individual. Further, those tax payments are largely already allocated to other government purposes besides that person's life. Only a fraction of that $380K is intended to finance healthcare. That million-plus exotic treatment will not make financial sense in terms of ta

          • I wouldn't trust The 'Tax Payers' Alliance to be a reliable source of impartial statistics for a start.

            You've missed the point about the dead paying no tax, so everything she generates in future can be offset against the cost of treating her without considering what she might have contributed in road tax, because it would have been zero if she hadn't been treated. You could even take into account that the .4 wouldn't have existed if she hadn't been treated if you like.

            Yes, NICE will have to draw a line, but

    • For the next several years, sure. At some point they'll have these machines at every major hospital and it'll be a few thousand dollars.

  • You keep using that word. I do not think it means what you think it means.

  • First of ten (Score:3)

    by WoodstockJeff ( 568111 ) on Sunday December 11, 2022 @07:53PM (#63122766) Homepage

    Alyssa is just the first of 10 people to be given the drug as part of a clinical trial.

    Assuming some of the other nine have similar results, A 10% success right is pretty good for an issue like this. Raising the rates higher will show its success.

    Testing on less-terminal people will, of course, come next.

  • Cure for very rare diseases (Score:5, Insightful)

    by Synonymous Cowered ( 6159202 ) on Sunday December 11, 2022 @08:00PM (#63122790)

    What I find most amazing about this story is just how rare her specific cancer is.

    it is thought that up to a dozen a year could benefit from this therapy.

    Normally if your illness is that rare, you are pretty much screwed unless something is discovered by accident while working on something else. But with techniques like this they can develop customized cures for people that otherwise would be out of luck.

    • Re: (Score:2)

      by HiThere ( 15173 )

      IIUC, this technique (in it's present form) only works on blood diseases. But I'm not real certain of that. (That may be from an article about a different technique.) I think you need to be able to kill off the cells that generate the problem, and then replace them with the improved version. (That's not quite what the summary implied, but much of the immune system is generated with the blood, so I think it was probably involved.)

      • According to the story, they literally killed off all the T-cells in her body (using T-cells grown in a culture). Then they are going to regrow the T-cells with another marrow transplant.

        This procedure is not for the weak-hearted.

      • Re: (Score:2)

        by sjames ( 1099 )

        I believe you are correct. This is because the treatment indiscriminately destroys all cells of the targeted type. In the case of immune cells in blood, the immune system can be reconstituted after the treatment. That, of course, wouldn't work for lung tissue.

  • Alyssa was cured of her cancer but her family has been afflicted with paying for the novel treatment, with which the "rightsholder" expects to extort millions of profits from its patients. Hell maybe monsanto should buy them and you can keep paying to stay alive, or they stop sending you updated genetic patches and you croak.

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