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A ‘Cure for Heart Disease’? A Single Shot Succeeds in Monkeys:
In the first gene-editing experiment of its kind, scientists have disabled two genes in monkeys that raise the risk for heart disease. Humans carry the genes as well, and the experiment has raised hopes that a leading killer may one day be tamed.
“This could be the cure for heart disease,” said Dr. Michael Davidson, director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine, who was not involved in the research.
But it will be years before human trials can begin, and gene-editing technology so far has a mixed tracked record. It is much too early to know whether the strategy will be safe and effective in humans; even the monkeys must be monitored for side effects or other treatment failures for some time to come.
The results were presented on Saturday at the annual meeting of the International Society for Stem Cell Research, this year held virtually with about 3,700 attendees around the world. The scientists are writing up their findings, which have not yet been peer-reviewed or published.
The researchers set out to block two genes: PCSK9, which helps regulate levels of LDL cholesterol; and ANGPTL3, part of the system regulating triglyceride, a type of blood fat. Both genes are active in the liver, which is where cholesterol and triglycerides are produced. People who inherit mutations that destroyed the genes’ function do not get heart disease.
[...] Not only did the system work in 13 monkeys, the researchers reported, but it appeared that every liver cell was edited. After gene editing, the monkeys’ LDL levels dropped by 59 percent within two weeks. The ANGPTL3 gene editing led to a 64 percent decline in triglyceride levels.
One danger of gene editing is the process may result in modification of DNA that scientists are not expecting. “You will never be able to have no off-target effects,” warned Dr. Deepak Srivastava, president of the Gladstone Institutes in San Francisco.
In treating a condition as common as heart disease, he added, even an uncommon side effect can mean many patients are affected. So far, however, the researchers say that they have not seen any inadvertent editing of other genes.
Another question is how long the effect on cholesterol and triglyceride levels will last, Dr. Davidson said. “We hope it will be one-and-done, but we have to validate that with clinical trials,” he said.
(Score: 4, Informative) by stretch611 on Tuesday June 30 2020, @10:32PM (8 children)
Being able to afford it will be another.
How much will it cost? I guess no less than $250,000.
Before you call me cynical remember this: The drug companies will want a huge profit. And they will claim all the costs of research for the drug. Not to mention all the costs of research of all the failed drugs. They will also conveniently "forget" how much of those costs were paid for by the government (and by extension, you) through research grants and research done in public universities.
And you can be damn sure that not only will they want profit on the new drug, but they will also want to replace the revenue on all the existing drugs that will no longer be needed..., like all the statins and blood thinners that will no longer be prescribed.
In a related topic: Remdesivir Priced At More Than $3,100 For A Course Of Treatment [npr.org]
The referenced article goes on to mention that consumer advocates argue that the drug should be free because of the substantial public investment, while investment bankers argue that the price should be roughly $40,000 per treatment because that is how much money the drug is expected to save for each treatment. Essentially arguing that Remdesivir should cost as much money as it saves from normal treatment, instead of actually costing anything close to what it cost to develop and make.
So when it comes to pricing, that $250,000 is quite low compared to what investment people would want to charge for it.
Now with 5 covid vaccine shots/boosters altering my DNA :P
(Score: 1, Funny) by Anonymous Coward on Tuesday June 30 2020, @10:41PM
To be fair to the pharma VCs, Wu Tang Clan albums don't come cheap.
(Score: 2) by takyon on Tuesday June 30 2020, @10:49PM
The potential market is huge since this is a treatment for one of the most common diseases. Off target effects could keep it off the market for decades though.
As for the price, it might be helpful if countries refuse to allow gene therapies to be patented. Or if anyone with the right equipment and the known genes can just make the gene therapy themselves.
[SIG] 10/28/2017: Soylent Upgrade v14 [soylentnews.org]
(Score: 4, Informative) by krishnoid on Tuesday June 30 2020, @10:50PM
That's a treatment you'd definitely want to get outside the U.S., then.
(Score: 0) by Anonymous Coward on Wednesday July 01 2020, @12:16AM
Everyone could afford a single shot that get them rid of heart diseases. On the low cost range of the scale, a gun shot is all it takes.
(Score: 1) by gmby on Wednesday July 01 2020, @09:32AM
Time for the open dna coders to put the fix in the Corona19 and let it spread to all for free! You don't want the treatment then wear a mask! Of course you code the CV19 so it only releases the DNAFIX subroutine when it detects x?
Bye
(Score: 2) by legont on Wednesday July 01 2020, @12:05PM
Perhaps both - timing and price - are grossly overestimated similarly to earlier computers. We've done it once, we can do it again.
https://en.wikipedia.org/wiki/Body_hacking [wikipedia.org]
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 0) by Anonymous Coward on Thursday July 02 2020, @12:38AM (1 child)
There was a whole discussion about this on Slashdot. The discussion is actually quite similar to the one here but might still be worth reading about.
https://science.slashdot.org/story/20/06/28/1510204/a-cure-for-heart-disease-a-single-shot-succeeds-in-monkeys [slashdot.org]
(Score: 0) by Anonymous Coward on Thursday July 02 2020, @12:43AM
(same poster)
oops, I responded to the wrong comment by mistake. I meant to respond to another comment.
Regardless, the Slashdot discussion is worth reading through as well. Not that there is anything wrong with the discussion here either.