Gene Therapy Cures "Bubble Boy"

bofh31337 writes "NewScientist is reporting that Welsh boy Rhys Evans has been cured of the fatal severe combined immunodeficiency ("bubble boy") disease. The medical team, lead by Adrian Thrasher, was able to take the stem cells that give rise to immune cells from his bone marrow and add a normal copy of the gene to the stem cell using a retro virus. Seven months after treatment, Rhys was cured."

Question

[Da Masta]

The potential power of stem cell research is clearly evident in this case. My question, not to troll, is whether this type of research could have been possible/allowed in the US. AFAIK, the laws in the states allow a restrictive amount of stem cell research -- would this have been enough for similar treatment here?

Re:my experience

[Anonymous Coward]

They took this guy's stem cells from his own body. Not from some embryo. There are no laws against this in the U.S. What they did was the almost the same thing as how people are treated for Hodgkins disease every day. In simplified form: 1) Extract bone marrow (and thus, stem cells) from patient, 2) Submit patient to massive radiation to kill existing stem cells in his body, 3) Put patient on IV drip of his own bone marrow (Yes, the stem cells "magically" end up where they are supposed to be in the marrow).

The only difference here (AND IT'S A REALLY COOL DIFFERENCE) is that before they put the stem cells back in this guy, they used genetic engineering techniques to insert a good copy of the Interleukin-2 gene (a "bad" il-2 gene was causing his disease to begin with) into the DNA of the stem cells.

The majority of the genetic engineering (e.g. recombinant DNA) techniques that made this possible were developed in the US over 20 years ago (and funded by US tax dollars). (These guys appeared to have done some cool things to make the stem cells more likely to be "infected" by the vector.) So don't use this story to make a case against the US policys on embryonic stem cell research. This work has nothing to do with embryonic stem cell research.

The reason why this stuff probably didn't happen in the US is that our FDA officials are a bunch of overprotective suits. That said, there have been many uses of genetic engineering in the US (treatment of cystic fibrosis comes to mind) to date- although not all have been successful.

Personally, I think it's awesome that these former bubble boys will have the chance to lead "normal" lives. Everyone involved in getting this done, from the patients themselves to their families to the doctors to the researchers to the governments to whoever paid for it (this must have cost millions including everyting), deserves much congratulations for their vision, courage and hard work.

Re:How do Retro Viruses work?

[professortomoe]

Nope, you're pretty much right about a retrovirus. My Bio teacher came up with a really great analogy about retroviruses, HIV in particular: Ok, say you've got eight men in a tank. These guys each have a set of blueprints. They drive their Panzer tank through the wall of the nearest Ford factory and tell the workers to make more tanks instead of those fruity Ford Taurus things. The workers construct 7 more tanks and the guys each hop into their respective tanks, driving out of the building through the walls, bringing it to the ground. You've got the tanks as the delivery system, the men as the viral RNA, and the factory as the cell. Mind you, I may have skewed it slightly because it's been a good three months since we covered that. Oh well, it's also tired time. Heh.

Other points

[Faux_Pseudo]

While this was going on there is a couple in California that is hopeing (as in activly looked for sperm donner who was deaf) to have a kid that is deaf so that he will be like the rest of the family (minus the cats). So while we have gene experaments going on to inhance the lives of people and potentialy bring a brave new world kind of classism effect [BadThing(TM)] we have also got people who are actively trying to set the pace of progress back.

Re:Bahh!

[Anonymous Coward]

> I think the solution to your fear is to become an atheist =)

What is true isn't changed by what you think is true. You can believe war is not going to occur, but it very well may. Telling yourself it won't can't change that. Ignorance is not always bliss.

> I mean, serously, if science brings us to the point where it's commonplace to "play God" then what's the point of worshipping a diety?

Well, what's the point of worshipping at all? To bring glory. God being worthy of glory doesn't change regardless of what man accomplishes. Also, "playing God" is not at all close to "being God". A young boy can "play cops and robbers", but he's no closer to being a police officer than a young girl "playing doctor".

Being God

[Sean Clifford]

We're not going to use this technology responsibly - we don't use any technology responsibly. Of course, we'll develop "good" uses for the technology - treat and cure diseases, discover new forensic techniques, grow new organs, and blah blah blah.

But we'll also develop targeted bioweapons to kill "terrorists","Dangerous Radicals", Saddam Hussein, or other enemy-of-the-year. We'll do horrible shit with this, possibly doomsday our species along with our environment.

Re:God's Biotech Lab...

[dragons_flight]

Well technically, manipulating all reality through the power of will alone is a pretty good functional definition of what it means to BE God. Only important thing missing is knowing everything, but that probably comes along with all events playing out according to one's will.

As you no doubt know, "playing god" is what the disdainful call it every time man gains control over an aspect of reality that was previously ruled by chance/God alone.

Since God created us with intellect, reason and the ability to learn moral judgment, I for one, believe that we should "play God". Man is created in the image of God, and if we are to fulfill that destiny than it means learning to act with as much wisdom, knowledge and moral judgment as humanly possible, which certainly includes scientific exploration.

Re:retrovirus information

[Ian Bicking]

However, it is questionable that AIDS is caused by HIV at all [duesberg.com]

It is strange that not everyone with AIDS has HIV, and not everyone who has HIV seems to get AIDS, even after many years. Barring other evidence, this should lead one to think HIV does not cause AIDS. Other evidence doesn't seem to be forthcoming.

Basically, AIDS is just a disease of definition -- when you die of particular diseases, and happen to have HIV, you are declared to have died of AIDS. In Africa you can die of TB, have HIV, and be declared to have died of AIDS. The AIDS epidemic in Africa is in fact just a horrible amount of traditional (and often curable) diseases -- it is indeed a tragedy and deserves a great amount of attention, but they need antibiotics, clean water, basic medical care, and mosquito control. They don't need AZT.

They've been talking about a 30%-50% HIV infection rate in Nigeria for 10, maybe 15 years. They should all be dead! They aren't. A lot of people are dead, but it's from all the same old reasons -- all the problems we, as a world and as a species, haven't been willing to attack since the 60s.