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CRISPR eliminates HIV-1 infection in live animals

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Re: CRISPR eliminates HIV-1 infection in live animals

#131

Post by blacksmith_tb »

Teknoman117 wrote: post #93
ChuckMcM wrote: post #66

Earlier quoted context omitted.

Given billionaires running their own space programs it makes me wonder why there aren't more billionaires running biologic research companies. Between life extension and immunity to (or protection from) really pathological conditions it seems like their interests would be aligned with folks like this.

I almost wonder if running your own space program is cheaper. As in the amount of regulatory BS you have to go through due to the monopolistic behavior of the large pharmaceutical companies...

That wouldn't slow down a self-respecting multi-billionaire, though, would it? Couldn't you just set up your lab in Malaysia / Brazil / Belarus? You don't even need a country without regulations against your research direction, just one that doesn't (consistently) enforce any such laws...

Re: CRISPR eliminates HIV-1 infection in live animals

#132

Post by db48x »

ChuckMcM wrote: post #103
Teknoman117 wrote: post #93

Earlier quoted context omitted.

I almost wonder if running your own space program is cheaper. As in the amount of regulatory BS you have to go through due to the monopolistic behavior of the large pharmaceutical companies...

And yet, if you're not really planning on selling the techniques you find on the open market could you save yourself a lot of time by ignoring the FDA?

You can spend hundreds of millions of dollars before you even get to the point where you can ask the FDA if you can start human trials. Even having spent all that time and money, you still won't have any guarantee that your drug will prove to be safe, effective, or better than any existing drugs.

Re: CRISPR eliminates HIV-1 infection in live animals

#133

Post by smitherfield »

colordrops wrote: post #48
Animats wrote: post #32

Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure. But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be th…

This antiviral is extremely promising, and it boggles the mind how this has not gotten real funding. My cynical side thinks it would cut very deep into a lot of profits for all the various viral medications for lifetime diseases such as the HIV cocktail, herpes meds, and other viral illnesses, so there isn't much upside for a large company to fund this.

Of course there's upside — if it really does cure all viral diseases it could be a trillion-dollar pill. Not to mention they'd want to introduce it before the competition.

What's probably working against it is it sounds too good to be true.

Re: CRISPR eliminates HIV-1 infection in live animals

#134

Post by notyourwork »

ithinkinstereo wrote: post #16
ChuckMcM wrote: post #9

Wow, I had not connected the idea of using CRISPR genome editing with targeting virus dna segments. If I understand the technique correctly (and I am not a biologist for sure!) they used an adeno virus modified with a specific Cas9 setup to elide HIV DNA from cells it infected (normally I associate viruses with adding DNA rather than removing it :-) Is that even close to a correct interpretation? It sounds like the t…

Correct. You can also use CRISPR to edit a person's own cells in a lab for injection back into the person's body to combat a slew of diseases, including cancer. China is already experimenting on live humans: http://gizmodo.com/china-is-racing-ahead-of-the-us-in-the-qu...

Question: Do different counties have different governing bodies on qualifications for human experimentation? I assume so but am not well versed in this domain.

Re: CRISPR eliminates HIV-1 infection in live animals

#135

Post by TaylorAlexander »

Animats wrote: post #107
dmix wrote: post #53

Earlier quoted context omitted.

This is the type of innovation we exchange for a sense of absolute security in having highly-vetted decade-long bio R&D and extensive backlogs at the FDA for approvals to sell products on the market. It's go big or go home because the regulatory risk/timeline is so extensive. Most of these things die quietly or never get started so we never really see the true costs of what it takes to push the pharma world forward.…

No, this hasn't gotten anywhere near human testing yet. The FDA isn't involved.

FDA rules increase barriers to entry to the market, resulting in fewer companies (especially fewer smaller companies) who would be interested in funding such a project.

Re: CRISPR eliminates HIV-1 infection in live animals

#136

Post by alevskaya »

Animats wrote: post #32

Back in 2011, there was an announcement from MIT about a new approach to a broad-spectrum antiviral that appeared to work.[1] This goes way beyond an AIDS-specific cure. But it was at MIT Lincoln Labs, which doesn't usually do bio. So the researcher moved to Draper Labs, but didn't get much funding. Then that funding ran out. Now the guy behind this is trying to get funding on Indiegogo.[3] The problem seems to be th…

The reason this isn't getting funded is because it's extremely unlikely to work in reality. They've engineered synthetic protein sensors of dsRNA that induce apoptosis on detection - this mimics the natural viral detection systems that all cells use to prevent viral infection. The argued benefit is that this artificial protein avoids the common hacks that many viruses use to circumvent the natural detection pathways. For this to work you have to (1) inject a ton of this foreign protein into the blood, (2) have it persist and be taken up by many cells in an organism, (3) not elicit a negative immune reaction against cells expressing this foreign beast on their MHCs (4 - for prophylaxis) have it persist long enough to provide realistic protection against future viral infection events (5) hope that the distribution of protein-transduced cells overlaps enough with the natural viral targets to provide some clinical utility.

The costs of trying to turn a novel therapeutic approach into a real therapy are extremely high - hundreds of millions of dollars. I'm unaware of any approved therapy that utilizes protein transduction of cells - I also suspect existing protein transduction methods aren't very efficient. There is a tiny pile of evidence that this method "works" in vitro in cultured cell models of infection, I can imagine a hundred ways it will fail in bodies.

There's a reason some ideas are left unexplored by industry.

Re: CRISPR eliminates HIV-1 infection in live animals

#137

Post by WalterBright »

barrkel wrote: post #68
lend000 wrote: post #62

Earlier quoted context omitted.

> sense of absolute security Well said -- the keyword is "sense" of security.

There's also the small matter of profiting from desperation with unproven remedies that do unknown, possibly permanent damage. There are good reasons and historical precedent for strong medicine safety regulations; it's not just bureaucracy.

This was covered in Sam Peltzman's "Regulation of Pharmaceutical Innovation" book, where he goes over the statistics before/after the 1962 FDA Amendments. Statistically, the percentage of drugs that later prove to be ineffective or damaging is no different, just the aggregate amount of new drugs, good and bad, is way less.

Re: CRISPR eliminates HIV-1 infection in live animals

#138

Post by gasbag »

tejtm wrote: post #60
mandude wrote: post #2

Gene therapy (and CRISPR) is ultimately going to eliminate most if not every ailment/issue/genetic problem that we have, including aging and eye problems. Very thankful we are reaching the point where we can go into our cells and fix ourselves.

I do agree, but that is just the good news. In other news although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form, but there is not a force on heaven or earth that is going to stop parents from trying to give their children every advantage.

> In other news although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form

Why is this the right thing to do? What are you signifying by putting "right" in quotes?

I am reminded of the Hyperion Cantos from Dan Simmons, where most of the human race has declared that nanotech modification of the human organism/germline is a cardinal sin. Meanwhile there is another segment of humans who decided that maybe it wasn't, and they're off colonizing deep space by remaking themselves into a wild range of body types.

In fact if we don't stop dirtying up the planet, we may feel a lot more pressure to adapt our bodies to an environment whose rate of change we can't keep up with. In any event I am sure there will be a segment of humanity that does want to preserve human life in a currently recognizable form, and they should be able to choose that for themselves. That should not stop those of us who want to vary it wildly from doing our thing too!

There are a number of ethical concerns when it comes to these kinds of changes, of course. There is the world of The Windup Girl, where competing agricorps target each other's crops with tailored viruses to wipe them (or their consumers) out. So we will need to figure out how to rebalance our societies in the light of this vast new power. Yet it looks like it may also be the start of a whole new stage in treating human diseases. Imagine a world where mass-produced medicines are mostly replaced by taking some host cells, gene sequencing the target diseases, then programming and re-injecting the cells to eliminate them. I'm not well-versed enough in this stuff to know how far-fetched that is, of course. It sounds like something out of Star Trek. But it's hard not to be optimistic about what it can enable.

Re: CRISPR eliminates HIV-1 infection in live animals

#139

Post by WalterBright »

devoply wrote: post #50
colordrops wrote: post #48

Earlier quoted context omitted.

This antiviral is extremely promising, and it boggles the mind how this has not gotten real funding. My cynical side thinks it would cut very deep into a lot of profits for all the various viral medications for lifetime diseases such as the HIV cocktail, herpes meds, and other viral illnesses, so there isn't much upside for a large company to fund this.

That theory would work except that if that were the case they would've bought him out and shut it down. Everyone wants to make money, as it stands someone will and they can be from any sort of background. Why hasn't a silicon valley head given this guy a few million though is beyond me.

In the 1970s the conspiracy theorists were all convinced that oil companies bought the patents to 100 mpg carburetors in order to keep them off the market. My father (ex military) laughed at the idea, as a huge problem for the military is fuel consumption, and they would not let a little old thing like patent rights stand in the way of using it.

Re: CRISPR eliminates HIV-1 infection in live animals

#140

Post by devoply »

WalterBright wrote: post #139
devoply wrote: post #50

Earlier quoted context omitted.

That theory would work except that if that were the case they would've bought him out and shut it down. Everyone wants to make money, as it stands someone will and they can be from any sort of background. Why hasn't a silicon valley head given this guy a few million though is beyond me.

In the 1970s the conspiracy theorists were all convinced that oil companies bought the patents to 100 mpg carburetors in order to keep them off the market. My father (ex military) laughed at the idea, as a huge problem for the military is fuel consumption, and they would not let a little old thing like patent rights stand in the way of using it.

Yeah that's true too it's difficult to stop technology once it's been discovered, someone will make use of it. Genie out of the bottle so to say.
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