The Return of Gene Therapy

[Waverunner]

Great article by Derek Lowe.

http://pipeline.corante.com/archives/2014/02/03/the_return_of_gene_therapy_and_more.php

The advent of such techniques as CRISPR has people thinking again about gene therapy, and no wonder. This has always been a dream of molecular medicine - you could wipe all sorts of rare diseases off the board by going in and fixing their known genetic defects.
...
A less disturbing route might be to see if the technique can be used to gene-edit the egg and sperm cells before fertilization. Then you've got the possibility of editing germ cell lines in vivo, which really would wipe these diseases out of humanity (except for random mutations), but that will be another one of those hold-your-breath steps, I'd think. It's only a short step from fixing what's wrong to enhancing what's already there - it all depends on where you slide the scale to define "wrong". More fast-twitch muscle fibers, maybe? Restore the ability to make your own vitamin C? Switch the kid's lipoproteins to ApoA1 Milano?

For a real look into the future, combine this with last week's startling report of the generation of stem cells by applying stress to normal tissue samples. This work seems quite solid, and there are apparently anecdotal reports (see the end of this transcript) of some of it being reproduced already. If so, we would appear to be vaulting into a new world of tissue engineering, or at least a new world of being able to find out what's really hard about tissue engineering. ("Just think - horrible, head-scratching experimental tangles that were previously beyond our reach can finally be. . .")

Now have a look at this news about a startup called Editas. They're not saying what techniques they're going to use (my guess is some proprietary variant of CRISPR). But whatever they have, they're going for the brass ring:


(Editas has) ambitious plans to create an entirely new class of drugs based on what it calls “gene editing.” The idea is similar, yet different, from gene therapy: Editas’ goal is to essentially target disorders caused by a singular genetic defect, and using a proprietary in-house technology, create a drug that can “edit” out the abnormality so that it becomes a normal, functional gene—potentially, in a single treatment. . .


. . .Editas, in theory, could use this system to create a drug that could cure any number of genetic diseases via a one-time fix, and be more flexible than gene therapy or other techniques used to cure a disease on the genetic level. But even so, the challenges, just like gene therapy, are significant. Editas has to figure out a way to safely and effectively deliver a gene-editing drug into the body, something Bitterman acknowledges is one of the big hills the company has to climb.

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[Bob]

A less disturbing route might be to see if the technique can be used to gene-edit the egg and sperm cells before fertilization. Then you've got the possibility of editing germ cell lines in vivo, which really would wipe these diseases out of humanity (except for random mutations), but that will be another one of those hold-your-breath steps, I'd think. It's only a short step from fixing what's wrong to enhancing what's already there - it all depends on where you slide the scale to define "wrong". More fast-twitch muscle fibers, maybe? Restore the ability to make your own vitamin C? Switch the kid's lipoproteins to ApoA1 Milano?

http://pipeline.corante.com/archives/2014/02/03/the_return_of_gene_therapy_and_more.php

Interesting idea, but I can't see it being made legal in many countries.
There are so many unknowns.
It's also similar to eugenics, or could easily turn into an unhealthy race for genetic supremacy.

 

[Waverunner]

Interesting idea, but I can't see it being made legal in many countries.
There are so many unknowns.
It's also similar to eugenics, or could easily turn into an unhealthy race for genetic supremacy.

Sorry but what you are saying is ridiculous. You wouldn't prevent people from having severe genetic diseases, even if you could, because you have the fear of genetic supremacy? What genetic supremacy do you mean? Less people with Huntington's disease pose a threat to humanity? Is it better to have some handicapped or severely ill people around, so that other people feel entertained and feel not that bad about their personal situation or their own diseases? Curing a disease has nothing to do with genetic supremacy. What would really interest me however is, if you would like more people with genetic diseases or is the balance of healthy and ill people just right at the moment? Maybe we should introduce a quota for genetic diseases.

 

[adreno]

I'd take those enhanced muscle fibers and lipoproteins, too.

 

[Bob]

Sorry but what you are saying is ridiculous. You wouldn't prevent people from having severe genetic diseases, even if you could, because you have the fear of genetic supremacy? What genetic supremacy do you mean? Less people with Huntington's disease pose a threat to humanity? Is it better to have some handicapped or severely ill people around, so that other people feel entertained and feel not that bad about their personal situation or their own diseases? Curing a disease has nothing to do with genetic supremacy. What would really interest me however is, if you would like more people with genetic diseases or is the balance of healthy and ill people just right at the moment? Maybe we should introduce a quota for genetic diseases.

It's an exceptionally complex issue. To dismiss the issues that I raised as ridiculous suggests that you haven't thought it through.

If scientists were able to test embryonic cells for potential diseases, then why would they spend money on gene therapy to alter the cells, rather than abort the cells? They'd check the cells for potential disease, choose the 'best' embryonic cells, and discard the cells that were less favoured.

If they ever perfect the technology (which wont happen any time soon), then perhaps the adjustment of a small number of individual genes may get regulatory approval. But it's not going to be approved without a huge amount of international discussion about the ethical concerns.

If we are going to change the gene pool of the human race, then it's a discussion that the entire planet needs to be involved in. It's not a casual decision. One you insert artificial changes into the gene pool, then you can't remove them afterwards without sterilising people. What if it goes wrong? What if there are unpredictable consequences?

And the process has the potential to be abused. I mentioned eugenics because many related questions will arise with such a technology. Parents will be able to pay to make their offspring more intelligent etc. That's where an ethical discussion re eugenics-related issues comes into play. Those with money will be able to create a new race of elite offspring.

So, for many reasons, it's not something that's going to get widespread approval any time soon. But I doubt that the technology will reach maturity for decades anyway.