Did We Just Cure High LDL for Life?

[RetCurious]

Fascinating results from the latest gene therapy trial. The same proven platform is now being tested against triglycerides.

https://www.nejm.org/doi/full/10.1056/NEJMoa2601283

"For the first time, scientists have edited a single letter of DNA inside living people to switch off a cholesterol gene, and in the first human trial, LDL cholesterol fell by up to 62% from one infusion. This is a full, evidence-first breakdown of VERVE-102 and the Heart-2 trial: what it is, how base editing actually works, what the data does and does not prove, and why it might matter far beyond cholesterol."

View: https://www.youtube.com/watch?v=s0Sp8kM-p2o

 

[CathyGoesFar]

So freaking cool.

If I became aware of such a trial in my area I would sign up in a heartbeat. I get my first blood test since starting Repatha(also a type of PCSK9 inhibitor) and ezetimibe on Monday. Hoping for a big drop.

 

[fatbegone]

Here's a great article on cholesterol.

"For young adults in relatively good health, aggressive LDL reduction will permanently protect them from developing cardiovascular disease with nearly zero downside. Consequently, cardiovascular disease is effectively a fully solved problem."

Cardiovascular disease is a solved problem – Total Health Optimization

totalhealthoptimization.com

 

[Meritocrat]

Then, a mild rosuvastatin and ezetimibe combined use, until the gene therapy becomes more accessible, should be a good choice?

 

[lessthanhalf]

As far as I know the only currently approved and in actual use gene therapies cost millions, a very long way away from something that could be used to lower lipids. Permanent gene editing requires very strict safety evidence, errors in editing or location can cause cancers, which might be an acceptable risk in treating fatal genetic disorders, but less so for something that already has perfectly decent drug therapy, I doubt there is any realistic chance of this being a thing in the next decade even.

 

[RetCurious]

As far as I know the only currently approved and in actual use gene therapies cost millions, a very long way away from something that could be used to lower lipids. Permanent gene editing requires very strict safety evidence, errors in editing or location can cause cancers, which might be an acceptable risk in treating fatal genetic disorders, but less so for something that already has perfectly decent drug therapy, I doubt there is any realistic chance of this being a thing in the next decade even.

We may not live to see it but we do see the dawning of the hope that will drive medical research professionals.

 

[lessthanhalf]

It seems ridiculous that there are so few gene therapies actually used , when most animal studies routinely create specialised gene editing tools to knock out genes in mice or create new strains with genes missing or even knockout genes in specific tissues only or only in response to light or chemical signals. Some studies might do 10 different gene edits to test different aspects of what they are researching, and it seems very routine. There was a good Asianometry video on youtube ( great channel ) about gene therapy and why it is so expensive.