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Gene Therapy Could Soon Be Approved In Europe

Soulskill posted about 2 years ago | from the i-will-take-xray-vision-please dept.

Medicine 44

another random user writes "According to the BBC, 'Europe is on the cusp of approving a gene therapy for the first time, in what would be a landmark moment for the field. ... The European Medicines Agency has recommended a therapy for a rare genetic disease which leaves people unable to properly digest fats. The European Commission will now make the final decision. The idea of gene therapy is simple: if there is a problem with part of a patient's genetic code then replace that part of the code. The reality has not been so easy. In one gene therapy trial a U.S. teenager, Jesse Gelsinger, died, and other patients have developed leukaemia. There no gene therapies available outside of a research lab in Europe or the U.S.' They have considered the use of Glybera to treat lipoprotein lipase deficiency, which leads to fat building up in the blood, abdominal pain and life-threatening pancreatitis (inflammation of the pancreas). 'The therapy uses a virus to infect muscle cells with a working copy of the gene.'"

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44 comments

reaction (0)

Anonymous Coward | about 2 years ago | (#40716001)

The dawn of a new age

Re:reaction (4, Interesting)

GameboyRMH (1153867) | about 2 years ago | (#40716189)

A few years ago, Gattaca was rated the most realistic sci-fi movie by NASA. Keep that in mind everyone.

Re:reaction (1)

Teresita (982888) | about 2 years ago | (#40716413)

Europe does have a lot to bring to the table, what with their extensive mid-20th Century research in hereditary hygiene.

Re:reaction (5, Insightful)

fuzzyfuzzyfungus (1223518) | about 2 years ago | (#40716485)

For reasons of fairness and/or perverse nationalism, I'd like to point out that the US was a bastion of eugenic progress and enthusiasm until those Germans ruined it for everyone... A few states were still sterilizing the unfit for a couple of decades after the war!

Re:reaction (1)

jamstar7 (694492) | about 2 years ago | (#40716743)

And yet politicians, lawyers, and bureaucrats were allowed to reproduce. You'd think they'd be the first ones chopped at the genetic block.

Re:reaction (1)

tnk1 (899206) | about 2 years ago | (#40718717)

They have the will to power. Those groups sound like genetic supermen to me, in the same manner as Khan Noonien Singh. Being able to take care of your own and have as many choices to mate with is actually encouraged by the whole natural selection thing.

Nothing about being genetic "superiority" means you are an altruistic, non-asshole. Indeed, it's more likely to make you an asshole than the alternative.

Douche from US, Ignorant of His Own History! (0)

Anonymous Coward | about 2 years ago | (#40722277)

How's that American Nazi Party, Douchebag?

Re:reaction (5, Informative)

simula (1032230) | about 2 years ago | (#40716629)

Gene therapy creates the opportunity to prevent Gattaca like scenarios. Within the Gattaca universe it was possible to sequence a person's DNA, but everyone was stuck with what they were born with. If you were luckily born with "good" genes, or if your parents selected for the sperm and eggs with the "best" genes with which to make a test tube baby, then your life was set. If you were born with less than "stellar" genes you were deemed inferior and discriminated against.

What is so exciting about this advance is that if you are born with a defective gene that results in illness, for a certain spectrum of genes, it is now possible to insert a non-defective version into a virus, inject that virus into muscle cells, and you are now as good as new.

This advance is about changing what genes you have at run-time, rather than being stuck with what you are born with. At the moment the changes we make are only additive, but give it time :)

Re:reaction (1)

denis-The-menace (471988) | about 2 years ago | (#40717403)

Give it time and we will have Gattaca-like scenarios.
We have them right now for pills and vaccines.

When H1N1 broke out what was stopping us from getting Vaccines beside the technical issues: Greed via patents

Until that is dealt with, we can have all the gene therapies we want but only if someone gets paid a king's ransom, everytime.

Re:reaction (0)

Anonymous Coward | about 2 years ago | (#40722569)

One key problem with this is: Not all mutations are diseases! Not everything we deem "bad" is actually a disadvantage.

I see the next step as not "fixing" but *improving* the genes. :)

Re:reaction (1)

MozeeToby (1163751) | about 2 years ago | (#40716785)

So you're saying the guy in Gattaca could have had his faulty heart gene repaired and lived his dream legally?

Re:reaction (1)

voidphoenix (710468) | about 2 years ago | (#40719003)

So you're saying the guy in Gattaca could have had his faulty heart gene repaired and lived his dream legally?

Yes, but his parents chose to have him naturally, without screening or augmentation. (IIRC, it's been a while since i last saw it.)

Re:reaction (0)

Anonymous Coward | about 2 years ago | (#40717227)

A large group of physicists rating a movie mostly dealing with biology regarding realism. Sounds legit.

then 28 days later... (0)

Anonymous Coward | about 2 years ago | (#40716357)

then 28 days later...

Rare? (1)

LifesABeach (234436) | about 2 years ago | (#40716031)

rare genetic disease which leaves people unable to properly digest fats

My first reaction was, "I thought that Diabetes was common, maybe not in Europe?"

Then I thought, "I hope it works, it would mean a proven therapy that could easily be applied to other issues like celluar break down of skin tissue." I wish these folks success.

Re:Rare? (0)

Anonymous Coward | about 2 years ago | (#40716219)

The problem of patching genetic defects using a viral infection is massive. It can include as happened infection with a real disease. It can also include a serious problem of the payload. While the gene may be desirable for those without it, what happens when somebody with a good working copy of the gene now has 2 working copies....? Another genetic disease. Will we start a business of extracting gene copies? Where does this house of mirrors end?

Re:Rare? (1)

0123456 (636235) | about 2 years ago | (#40716449)

Where does this house of mirrors end?

When we can rewrite DNA directly without having to rely on viruses to do it for us.

Re:Rare? (1)

Githaron (2462596) | about 2 years ago | (#40717053)

How do you propose going about modifying trillions of cells manually?

Re:Rare? (1)

paintballer1087 (910920) | about 2 years ago | (#40717547)

How do you propose going about modifying trillions of cells manually?

That's up to the programmer, though it will most likely involve a vi vs Emacs flame war.

Re:Rare? (0)

Anonymous Coward | about 2 years ago | (#40722275)

Why, nanobots, of course.

In the future, not next tuesday.

Re:Rare? (0)

Anonymous Coward | about 2 years ago | (#40722689)

You dont need to modify trillions of cells just specific stem cell groups. You think you are a static system in which the cells arent continually replaced?

Re:Rare? (2)

Antipater (2053064) | about 2 years ago | (#40716493)

"Use a viral vector to administer the treatment" does not equate to "just tack it onto the common cold and release it into the wild." Nobody with a working genome is going to get this, because nobody's going to be injected with it unless they need to (I'm sure the cost of treatment will see to that, even without governmental restrictions). And even if they did, it wouldn't do anything - it would replace a working copy with a working copy, kind of like cut-and-pasting the same block of text into the same spot.

Re:Rare? (2, Interesting)

Anonymous Coward | about 2 years ago | (#40716515)

You would be insane to use a fully working virus - Instead you package your pay-lode in viral proteins either within a partly functional virus, or as a replacement for a non functional one. For safety reasons the second is always preferable. The first is more risky but you would usually use a virus with no transmission ability outside of the cell line you bread it in, or in the worst case at least no ability to spread between humans. Note that neither of the TFAs differentiate at least as far as I can say so I had to look it up, and normally this info is skipped or even unintentionally lied about in the media due to ignorant journalists.

In this case "most therapeutic genes require the complete replacement of the virus's 4.8 kilobase genome"[1] as it uses a Adeno-associated virus vector[2], so likely it can not spread, this should also be the case due to the normal modifications made before use as a vector. This mean that it will act closer to a genetic drug than a actual vius, affecting the cells it enters permanently but only ever half of the offspring (does not integrate can not divide or piggyback on genomic division).
[1]http://en.wikipedia.org/wiki/Adeno-associated_virus#Advantages_and_drawbacks
[2]http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2012/07/news_detail_001574.jsp - "Glybera uses an adeno-associated virus vector as the delivery vehicle to add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells."

Re:Rare? (4, Informative)

pahles (701275) | about 2 years ago | (#40716231)

Diabetes has to do with the inability to digest sugar, not fat... (sorry for my English)

Re:Rare? (1)

Gordonjcp (186804) | about 2 years ago | (#40716325)

There's nothing wrong with your English, there.

It's funny how so many people say "sorry for my bad English" when they speak better English than most Americans - or even English people.

Re:Rare? (0)

Anonymous Coward | about 2 years ago | (#40716517)

Maybe, but trying to correct an Englishman's English is about as futile as trying to tell a Frenchman how to cook.

Re:Rare? (1)

jpapon (1877296) | about 2 years ago | (#40716599)

when they speak better English than most Americans - or even English people.

It's funny that you think English people speak "better" English than Americans. I've heard some pretty horrid gobbledygook coming out of the mouths of Englishmen (just as I've heard it coming out of Americans).

Re:Rare? (1)

triffid_98 (899609) | about 2 years ago | (#40718253)

Woss this about English not speakin' English proper? I'll get out me spoons. We invented it yer damn Yanks

Re:Rare? (1)

pahles (701275) | about 2 years ago | (#40717893)

Thanks, just wasn't sure... It is not my mother tongue.

Re:Rare? (1)

Anonymous Coward | about 2 years ago | (#40716471)

Diabetes has to do with the inability to digest sugar, not fat... (sorry for my English)

To clarify, diabetes results when cells' ability to import glucose from the bloodstream is impaired by insulin deficiency or insulin resistance. Once the sugar gets into the cell it is metabolized normally.

There no (0)

AaronLS (1804210) | about 2 years ago | (#40716069)

There are no? If you have a southern draw like me though, you just run it together and it sounds the same.

Re:There no (1)

GameboyRMH (1153867) | about 2 years ago | (#40716215)

Still it should have been spelled "There're no"

Re:There no (1)

PolygamousRanchKid (1290638) | about 2 years ago | (#40716843)

No, it should be written as it is spoken,

There ain't no

Quality of life vs. acceptable casualty rate (1)

sl4shd0rk (755837) | about 2 years ago | (#40716359)

When you just exist in life without really enjoying it because of disease, illness or other condition out of your control, every avenue of possibility to improve your quality of life looks appealing. It's unfortunate there were casualties but I'm sure there were also advancements made despite that fact.

Re:Quality of life vs. acceptable casualty rate (0)

Anonymous Coward | about 2 years ago | (#40716899)

Yeah, reading the article says they fear their next meal. It leaves me wondering how much they have to eat to get that extreme pain to end up in hospital.

It's hard to say, if I would take it... the chance of either dying of complications or having a better life. Ofcourse both also help medicine research but that won't do a lot for you unless you're near death anyway.

But ghod forbid we use gene modded plants (1, Insightful)

alispguru (72689) | about 2 years ago | (#40716633)

Messing with the human genome, in a way that could very likely propagate? OK, then.

Hypocrites (assuming Greenpeace isn't protesting this advance).

Re:But ghod forbid we use gene modded plants (2)

MozeeToby (1163751) | about 2 years ago | (#40716887)

So many things...

A) Infecting reproductive cells isn't quite the same as infecting muscle cells.
B) I doubt they are infecting a statistically significant number of cells in the person's body, so in the unlikely event that the virus can also infect reproductive cells it's still statistically unlikely to happen.
C) They are repairing a faulty human gene using the correct version of that human gene, as opposed to taking a gene from one species and inserting it into another
D) Only the people who elect to take part in the treatment are affected, as opposed to gene tailored crops which will find their way into your food supply no matter how hard you try

Hell, I'm not even against modded food crops (I am against the way companies like Monsanto have prosecuted people who, by some arguments, were the ones being wronged) and even I call your argument BS.

Re:But ghod forbid we use gene modded plants (1)

Internetuser1248 (1787630) | about 2 years ago | (#40722491)

You are right that this is a highly unlikely scenario, especially given that your point C multiplies against A and B such that it is highly unlikely that a dangerous or detrimental genetic change could occur, and also highly unlikely that any change would be passed on to offspring. The chances of both are extremely small. I am forced to disagree with your conclusion though. There are seven thousand million people in the world. In terms of medical treatment unlikely is synonymous with daily occurrence, very unlikely is the same as doesn't happen very often, and extremely unlikely is the same as bound to happen eventually. The question is not will something go wrong, the question is what are the potential consequences if something does go wrong. Given that the consequences might go unnoticed for many generations and spread to large numbers of people, and given that we know so little about the way genetics works, the answer is: We have no idea, it could be anything.

Disclaimer: I am not against this treatment at all, I support and celebrate advances in medical science. I merely point out that this is not a decision to be rushed or taken lightly.

Re:But ghod forbid we use gene modded plants (0)

Anonymous Coward | about 2 years ago | (#40722577)

THIS

And fuck Monsanto! Nobody else in the world deserves a worse punishment. Literally NOBODY. They put even Dr. Mengele to shame.

genetically enhaced olympic games? (1)

Fpdx (2689069) | about 2 years ago | (#40716881)

the real possibility to have genetically modified athletes in the future (via the same principle of targeted virus infection) is discussed in Nature (pay per view #@!) http://www.nature.com/nature/journal/v487/n7407/full/487297a.html [nature.com]

Immun response (1)

Anonymous Coward | about 2 years ago | (#40717165)

Gene therapy with some modified virus will always have this little problem: Immun systems responding in a not so predictable way. There's no way around it. If you introduce a virus the immun system will respond. In every person in a slightly different way.

Re:Immun response (2)

tnk1 (899206) | about 2 years ago | (#40718845)

The immune system can be tricked. It is also not unstoppable. Indeed, in some cases, it is possible to seriously compromise or nearly eliminate the body's immune defenses on purpose these days. If the gene was a critical one that needed to be replaced, you'd probably put them in a bubble, compromise the immune system, administer the treatment, and then allow the immune system to regain its potency after the virus did its job. It's not risk free, but if it was the difference between possible life or certain death, it may be warranted.

And I don't think that even that will be necessary forever. Once we know enough about how things work on that level, we will probably be able to manipulate the immune system more specifically, or be able to tailor the virus to be accepted. The flip side of that is that we could then weaponize that knowledge to ensure biological weapons with almost zero immune response. Improvements in applied science can definitely be a two-edged sword.

sooo... (1)

superwiz (655733) | about 2 years ago | (#40721681)

they approve gene therapy on people, but not on the plants that people eat. We do it the other way around. Pretty sure we still win the race to be the least dumb.

How do they keep the virus in the body? (1)

Hentes (2461350) | about 2 years ago | (#40722913)

How do they make sure that the virus they use doesn't spread to others?

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