Scientists Edit Gene in 15 Patients That May Permanently Reduce High Cholesterol (cnn.com) 21
A CRISPR-based drug given to study participants by infusion is raising hopes for a much easier way to lower cholesterol, reports CNN:
With a snip of a gene, doctors may one day permanently lower dangerously high cholesterol, possibly removing the need for medication, according to a new pilot study published Saturday in the New England Journal of Medicine.
The study was extremely small — only 15 patients with severe disease — and was meant to test the safety of a new medication delivered by CRISPR-Cas9, a biological sort of scissor which cuts a targeted gene to modify or turn it on or off. Preliminary results, however, showed nearly a 50% reduction in low-density lipoprotein, or LDL, the "bad" cholesterol which plays a major role in heart disease — the No.1 killer of adults in the United States and worldwide. The study, which will be presented Saturday at the American Heart Association Scientific Sessions in New Orleans, also found an average 55% reduction in triglycerides, a different type of fat in the blood that is also linked to an increased risk of cardiovascular disease.
"We hope this is a permanent solution, where younger people with severe disease can undergo a 'one and done' gene therapy and have reduced LDL and triglycerides for the rest of their lives," said senior study author Dr. Steven Nissen, chief academic officer of the Sydell and Arnold Miller Family Heart, Vascular & Thoracic Institute at Cleveland Clinic in Ohio.... Today, cardiologists want people with existing heart disease or those born with a predisposition for hard-to-control cholesterol to lower their LDL well below 100, which is the average in the US, said Dr. Pradeep Natarajan, director of preventive cardiology at Massachusetts General Hospital and associate professor of medicine at Harvard Medical School in Boston...
People with a nonfunctioning ANGPTL3 gene — which Natarajan says applies to about 1 in 250 people in the US — have lifelong levels of low LDL cholesterol and triglycerides without any apparent negative consequences. They also have exceedingly low or no risk for cardiovascular disease. "It's a naturally occurring mutation that's protective against cardiovascular disease," said Nissen, who holds the Lewis and Patricia Dickey Chair in Cardiovascular Medicine at Cleveland Clinic. "And now that CRISPR is here, we have the ability to change other people's genes so they too can have this protection."
"Phase 2 clinical trials will begin soon, quickly followed by Phase 3 trials, which are designed to show the effect of the drug on a larger population, Nissen said."
And CNN quotes Nissen as saying "We hope to do all this by the end of next year. We're moving very fast because this is a huge unmet medical need — millions of people have these disorders and many of them are not on treatment or have stopped treatment for whatever reason."
The study was extremely small — only 15 patients with severe disease — and was meant to test the safety of a new medication delivered by CRISPR-Cas9, a biological sort of scissor which cuts a targeted gene to modify or turn it on or off. Preliminary results, however, showed nearly a 50% reduction in low-density lipoprotein, or LDL, the "bad" cholesterol which plays a major role in heart disease — the No.1 killer of adults in the United States and worldwide. The study, which will be presented Saturday at the American Heart Association Scientific Sessions in New Orleans, also found an average 55% reduction in triglycerides, a different type of fat in the blood that is also linked to an increased risk of cardiovascular disease.
"We hope this is a permanent solution, where younger people with severe disease can undergo a 'one and done' gene therapy and have reduced LDL and triglycerides for the rest of their lives," said senior study author Dr. Steven Nissen, chief academic officer of the Sydell and Arnold Miller Family Heart, Vascular & Thoracic Institute at Cleveland Clinic in Ohio.... Today, cardiologists want people with existing heart disease or those born with a predisposition for hard-to-control cholesterol to lower their LDL well below 100, which is the average in the US, said Dr. Pradeep Natarajan, director of preventive cardiology at Massachusetts General Hospital and associate professor of medicine at Harvard Medical School in Boston...
People with a nonfunctioning ANGPTL3 gene — which Natarajan says applies to about 1 in 250 people in the US — have lifelong levels of low LDL cholesterol and triglycerides without any apparent negative consequences. They also have exceedingly low or no risk for cardiovascular disease. "It's a naturally occurring mutation that's protective against cardiovascular disease," said Nissen, who holds the Lewis and Patricia Dickey Chair in Cardiovascular Medicine at Cleveland Clinic. "And now that CRISPR is here, we have the ability to change other people's genes so they too can have this protection."
"Phase 2 clinical trials will begin soon, quickly followed by Phase 3 trials, which are designed to show the effect of the drug on a larger population, Nissen said."
And CNN quotes Nissen as saying "We hope to do all this by the end of next year. We're moving very fast because this is a huge unmet medical need — millions of people have these disorders and many of them are not on treatment or have stopped treatment for whatever reason."
Inflammation? (Score:2)
I thought inflammation was the new bogeyman when it came to heart disease. Are we treating the wrong thing here?
Re:Inflammation? (Score:4, Informative)
https://www.ncbi.nlm.nih.gov/b... [nih.gov]
Lotta work being done in this area.
Re: (Score:2)
The kinds of foods that trash your LDL predate the FDA and the United States.
Re: (Score:2)
Humans didn't die until the 21st century?
Re: (Score:2)
Not an engineer, I see. Some problems have multiple "causes".
Re: fuck diabetes and cholesterol (Score:1)
You do realize your butt isnâ(TM)t supposed to be in the toilet water, right? Thereâ(TM)s a reason the toilet seat exists, use it!
Re: (Score:1)
Some group of zillionaires on a private island are gonna stretch the limits on this. You youngbies are gonna see some weirdass shit
Re: (Score:2)
[X] Irrational paranoia about the above
Re: (Score:1)
[x] Combination of above factors
Re: (Score:1)
Potential issue (Score:1)
From TFA: out of 15 patients, one patient died during the study. Hope it wasn't due to the treatment...
Re: (Score:3)
Gene editing for adults (Score:5, Interesting)