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HIV used to cure 'bubble boy' disease - Printable Version

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HIV used to cure 'bubble boy' disease - Kyng - 04-18-2019

https://www.bbc.co.uk/news/world-us-canada-47969367

US scientists say they used HIV to make a gene therapy that cured eight infants of severe combined immunodeficiency, or "bubble boy" disease.

Results of the research, developed at a Tennessee hospital, were published in the New England Journal of Medicine.

The babies, born with little to no immune protection, now have fully functional immune systems.

Untreated babies with this disorder have to live in completely sterile conditions and tend to die as infants.

The gene therapy involved collecting the babies' bone marrow and correcting the genetic defect in their DNA soon after their birth.



This is clearly a very severe disease, which prevents those suffering from it from living a normal life, so I'm glad to see there's a cure now!

Though, I'd like to ask: is the use of HIV necessary, and is it going to leave the patients with any lasting side-effects? If so, is there anything else which can be used which might be safer?


RE: HIV used to cure 'bubble boy' disease - Lurkerish Allsorts - 04-18-2019

(04-18-2019, 09:35 AM)Kyng Wrote: This is clearly a very severe disease, which prevents those suffering from it from living a normal life, so I'm glad to see there's a cure now!

Though, I'd like to ask: is the use of HIV necessary, and is it going to leave the patients with any lasting side-effects? If so, is there anything else which can be used which might be safer?

1) The kids rarely live that long who have the disease, even the case that put it in the public conscious only lived to 12 and that was a long time for it.
2) the current treatment [per article] is a bone marrow transplant, which can be extremely painful for both donator and receiver. (I had to get my bone marrow tested once....)
3) They tried gene therapy before but it carried a 10% risk of leukaemia [per a linked article] that the BBC disheartnly calls a "Bloody disorder", plus other risks, plus needing to also go though chemo (to wipe any trace of an immune system I guess?)
4) I am guessing they use the modified HIV because of how the virus spreads in the body since it targets white blood cells.

Also the hospital is St. Jude's a very well respected children's hospital that runs off of just donations (and well I assume grants, they don't charge for their services) I doubt they would have injected the modified HIV virus in the kids unless it was their best hope.


RE: HIV used to cure 'bubble boy' disease - Kyng - 04-22-2019

(04-18-2019, 10:40 AM)Lurker101 Wrote:
(04-18-2019, 09:35 AM)Kyng Wrote: This is clearly a very severe disease, which prevents those suffering from it from living a normal life, so I'm glad to see there's a cure now!

Though, I'd like to ask: is the use of HIV necessary, and is it going to leave the patients with any lasting side-effects? If so, is there anything else which can be used which might be safer?

1) The kids rarely live that long who have the disease, even the case that put it in the public conscious only lived to 12 and that was a long time for it.
2) the current treatment [per article] is a bone marrow transplant, which can be extremely painful for both donator and receiver. (I had to get my bone marrow tested once....)
3) They tried gene therapy before but it carried a 10% risk of leukaemia [per a linked article] that the BBC disheartnly calls a "Bloody disorder", plus other risks, plus needing to also go though chemo (to wipe any trace of an immune system I guess?)
4) I am guessing they use the modified HIV because of how the virus spreads in the body since it targets white blood cells.

Also the hospital is St. Jude's a very well respected children's hospital that runs off of just donations (and well I assume grants, they don't charge for their services) I doubt they would have injected the modified HIV virus in the kids unless it was their best hope.
Yeah, I'm definitely not suggesting that there's any kind of conspiracy here, or that the hospital didn't do this with the children's best interests at heart - because, clearly, they did. Nor am I suggesting that any of the current alternatives would be better than this HIV-based treatment (because, again, that doesn't appear to be the case).

I'm just wondering why they used HIV (your hunch makes sense); whether there are any problems or risks associated with this method (I don't know whether or not there would be, but it wouldn't surprise me); and, if so, how much potential there is for improvement in the future.

Sure, I agree that this treatment is better than anything else currently available - but if it does have any side-effects, then that doesn't mean they shouldn't continue trying to develop anything better.


RE: HIV used to cure 'bubble boy' disease - Lurkerish Allsorts - 04-22-2019

If there are side effects either they are not known, or were deemed not sufficient enough to prevent treatment. My limited understanding is that with side effects is a "Risk vs reward" model of weighting. "Are the risks worth the cure".


I wasn't accusing you of anything, I was just giving the facts I found and possible logic that I could work out.