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 The scientists are writing up their findings, which have not yet been peer-reviewed or published.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: -1, Troll) by Anonymous Coward on Tuesday June 30 2020, @09:13PM (4 children)
Die already. Haven't you done enough?
(Score: 0) by Anonymous Coward on Tuesday June 30 2020, @09:17PM
Show me in the doll where your parents have touched you.
(Score: 1, Interesting) by Anonymous Coward on Tuesday June 30 2020, @09:21PM
Our work here is not yet done :)
(Score: 0) by Anonymous Coward on Tuesday June 30 2020, @09:51PM
The sad excuse of life called millenials are attacking me.
Boomers and millenials, the perfect cocktail.
(Score: 2, Funny) by khallow on Tuesday June 30 2020, @11:13PM
(Score: 1, Funny) by Anonymous Coward on Tuesday June 30 2020, @10:04PM
Gonna have me a Big Mac to celebrate!
(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.
(Score: 0) by Anonymous Coward on Wednesday July 01 2020, @02:04AM (12 children)
I thought people got heart disease because they made bad choices. Now you're telling me it's just genetics??
(Score: 2) by maxwell demon on Wednesday July 01 2020, @10:08AM
That's a false dichotomy.
As an analogy: If I walk without need on a visibly unsafe bridge, and consequently fall down and die, it clearly is the result of a bad decision of myself (I shouldn't have walked on that bridge), but it is also a result of the state of the bridge. If then someone makes the reasonable decision to make the bridge safe so that people don't die by falling from it, it doesn't imply that the people who previously walked on the bridge despite it obviously being unsafe didn't make a bad decision.
The Tao of math: The numbers you can count are not the real numbers.
(Score: 2) by legont on Wednesday July 01 2020, @12:11PM (10 children)
People get heart diseases because of propaganda. Most can't realistically resist corporate PhD's constantly looking for biological buttons to make one consume in general and overeat in particular. We have to kill them first.
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 1) by khallow on Wednesday July 01 2020, @01:09PM (9 children)
Or we could just do nothing. It's not a real problem after all.
(Score: 2) by legont on Wednesday July 01 2020, @03:53PM (8 children)
Well, I am not sure what you consider a real problem, but let's see a pure business point. Population grows at what - 2-3% rate? Therefore food business can't really grow faster. There is no investor who would go in with such a grows rate. So, either food industry becomes subsidized utility or we eat more and more.
People are talking about government healthcare. Government "foodcare" makes way more economic sense simply because medical care is expanding area while food is stable and stagnant.
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 1) by khallow on Wednesday July 01 2020, @05:48PM (7 children)
The food market doesn't work that way. Many avenues for growth were found, such as a wider variety of food, and advancing technologies for food transportation, storage, and preparation. Reducing that amazing progress to "PhDs" or some sort of hose with a single faucet means you're missing a lot.
(Score: 2) by legont on Wednesday July 01 2020, @08:15PM (6 children)
Nevertheless, the inflow of money is proportional only to the number of people and as such is not attractive to investors unless people are reduced to gluttony which is exactly what we observe throughout the world. PhD's reduce food to feed and stuck it into us using unethical methods.
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 1) by khallow on Wednesday July 01 2020, @11:00PM
And there are currently a lot of people, growing increasingly wealthy.
People reduce to gluttony quite readily, all by themselves. For example, a lot of restaurants in the developed world (ok, the US in particular) get customers by offering a lot of food. It's not PhDs deciding that this is the secret sauce that will pull in customers, it's just human nature.
And I already noted many ways that one can handsomely profit from food production by other means than merely increasing consumption through psychological woo.
(Score: 2) by maxwell demon on Thursday July 02 2020, @12:23PM (4 children)
No, it's also proportional to the average income. You know, you can spend vastly different amounts of money on the same amount of calories.
The Tao of math: The numbers you can count are not the real numbers.
(Score: 2) by legont on Thursday July 02 2020, @11:22PM (3 children)
Within one country, this is called inflation which is usually eliminated from the investment analysis.
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 1) by khallow on Friday July 03 2020, @04:29AM (2 children)
No, it's not. People don't merely care about the calorie content of food. Inflation only applies when the price increases more than the value of the product. If you choose to spend more and acquire something which you consider of that increased value, then there is no inflation.
(Score: 2) by legont on Friday July 03 2020, @03:47PM (1 child)
Yes, I know the modern "theory" that the government uses to calculate inflation and consider it a total horseshit. According to it say a TV set from 80s has deflated to about $1 today. This little trick allows them to report inflation at just above zero; crooks they are.
BTW, similar theory replaces steaks with burgers because see, consumers don't eat steaks any more for price reasons. They eat burgers. Hence burgers go into statistics instead of steaks so inflation goes down.
In the end, consumers eat shit and die of obesity and diabetes.
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 1) by khallow on Friday July 03 2020, @05:44PM
(Score: 3, Interesting) by maxwell demon on Wednesday July 01 2020, @09:54AM (8 children)
One would think that negative genes would tend to get eliminated over time by evolution, unless they also bring a positive effect. For example, it is no accident that sickle cell anaemia is most prevalent in malaria regions: While having two of the genes is disastrous, having only one of them gives some protection against malaria. Also, the reason why humans aren't immune against HIV is a genetic mutation that helped against another viral decease that got extinct millions of years ago. Also, I remember having read that people who are more likely to get diabetes when on a sugar-rich diet are better adapted to lack-of-food scenarios.
So the question is: What is the positive effect of those “heart-decease genes”, and is that positive effect still relevant today? That is, if we switch off those genes, do we indeed get a net improvement, or do we pay the protection with a side effect that's worse?
The Tao of math: The numbers you can count are not the real numbers.
(Score: 0) by Anonymous Coward on Wednesday July 01 2020, @10:17AM (3 children)
By the time it's a problem, you should have already reproduced.
(Score: 2) by maxwell demon on Wednesday July 01 2020, @10:45AM (2 children)
If the genes were selected for, that is unlikely. Because otherwise they would not have been selected for to begin with.
The Tao of math: The numbers you can count are not the real numbers.
(Score: 2, Insightful) by Anonymous Coward on Wednesday July 01 2020, @12:36PM (1 child)
The point is they wouldn't be selected for, but they also wouldn't be selected against, since it only manifests after you've already reproduced. Humans have hit more and more of those as average lifespan has increased, and will surely continue to find more in the future.
(Score: 2) by maxwell demon on Thursday July 02 2020, @12:25PM
Since production of the enzymes costs energy, their production would be selected against even if they had no effect at all.
The Tao of math: The numbers you can count are not the real numbers.
(Score: 2, Interesting) by Anonymous Coward on Wednesday July 01 2020, @10:55AM (1 child)
The issue is cholesterol and triglyceride. Those are required for your body to function. However when they're oxidized they damage blood vessels which causes the heart issues. Sadly this 'cure' is completely the wrong approach. From my non-medical understanding, it aims to reduce the amount of cholesterol and triglyceride in your blood. Thus the levels of oxidized cholesterol will also decrease thus vessel damage is decreased. Sadly this is treating the symptom not the cause, like trying to fix a wobbly ride from a flat tire by reducing the air pressure of all the good tires to match the flat one. The correct fix is to fix the damaged tire. For cholesterol, the correct fix is to stop oxidizing it. That is done by reducing stress, avoiding carbohydrates, and absorbing your fat soluble vitamins. Thus you need to eat more meat and fat and cut out sugar and carbs. But pizza and cake are too exciting so no one does that. Thus we're going to screw up our body's ability to move nutrients around (what cholesterol does) because we're too scared to eat fat. This 'cure' is going to have horrible long term effects. It's a "feels good fix" like skim-milk with added Vitamin D. You need fat to absorb Vitamin D which isn't present in skim-milk, so that added D is mostly unabsorble to your body.
In terms of all those studies you hear about how eating meat is going to kill you, that's true. If you eat tons of meat and tons of carbs you'll get a ton of oxidized cholesterol. However if you cut out those carbs and just eat a ton of meat, you'll feel the best you've ever been in your life.
The main point being those genes are only considered bad because we're eating improperly. Having more non-oxidized cholesterol is a good thing. Instead of eating like we evolved to we're going to perform genetic experiments on ourselves. Humans are crazy.
(Score: 0) by Anonymous Coward on Wednesday July 01 2020, @04:06PM
Red meat comes with mostly saturated fats, which is not optimum. To improve on that, people should in addition consume monounsaturated fats (e.g. olive oil) and cis-polyunsaturated fats, both omega-6 and omega-3 (salmon, walnuts).
(Score: 2) by legont on Wednesday July 01 2020, @12:13PM
The carriers fuck and fight better until sick. Having them makes lots of sense with life expectation below 50.
"Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
(Score: 0) by Anonymous Coward on Thursday July 02 2020, @12:41AM
Oops, I posted this as a response to another post when I meant to post it here.
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] [slashdot.org]
(Score: 0) by Anonymous Coward on Wednesday July 01 2020, @12:46PM
(Score: 0) by Anonymous Coward on Wednesday July 01 2020, @04:00PM
https://www.nature.com/articles/d41586-020-01906-4 [nature.com] "CRISPR gene editing in human embryos wreaks chromosomal mayhem"
Sure, those are embryos. But do we have evidence that should the gene editing technique used in the "cured" mice be used in humans there won't be "chromosomal mayhem" that might jeopardize any health benefits of such a proposed therapy?
Yes, of course understand, this is indeed why it takes time for such proposed treatments to be refined for human use.