T Nation

Maybe SOME Good News from the FDA


Did you guys see this? Thoughts with regard to editing the germ line? “…people who haven’t been born yet and never consented to being in a clinical trial.”

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Clinical trials involving the editing of genes in sperm, eggs and early-stage embryos could be permitted under certain conditions, the National Academies of Sciences and the National Academy of Medicine recommended Tuesday in a long-awaited report.

Once technical questions are resolved, clinical trials could start with extensive oversight, the report said. But the report urged limiting trials to the “most compelling circumstances,” such as when no other treatment exists, the condition is very serious, and the genes in question are well known to cause a particular disease.

Gene editing offers the possibility of treating many diseases, and there are already trials or advanced research under way to use the techniques to try to cure individuals with HIV, sickle cell anemia and cancer. But adjustments made to the human germ line—eggs, sperm and embryos—don’t just change the health of a single person; they can be passed on to any future offspring.

For that reason, a gene-editing trial involving the germ line would have to include oversight not only of people participating in the trial but also following the long-term health of future generations, presumably including people who haven’t been born yet and never consented to being in a clinical trial.

The report was the result of work by an advisory committee, which convened more than a year ago at the request of the nonprofit National Academies of Sciences and the National Academy of Medicine. The committee studied the ethical, scientific and regulatory questions raised by gene editing, and the report weighs in on some of the most difficult and emotionally charged issues surrounding the technology.

The idea raises difficult ethical questions. Gene editing is still a novel technology and can have unintended, potentially harmful, consequences that would then be passed on to future generations.

For years, such questions remained in the realm of speculation. But the introduction of a new gene-editing approach called Crispr-Cas9, and its rapid adoption by scientists due to its comparative ease and lower cost, makes the idea feasible in a way it hasn’t been before.

“The technology has moved significantly even during the last year when we were studying the issue,” said Richard O. Hynes, co-chair of the committee that wrote the report and a professor at the Massachusetts Institute of Technology in Cambridge, Mass. “Things that were not feasible became feasible.”

The report’s cautious words aside, the findings represent a significant milestone in thinking about the topic because of the committee’s prestige and its suggestion that there is a potential ethical path to follow once the technology is further developed.

Scientists continue to wrestle with what limits to impose on using Crispr inside the lab. In 2015, a group of researchers and thinkers published a paper in the journal Science suggesting that scientists should publicly discourage preliminary experiments using Crispr to modify cells in sperm or eggs and urging greater openness in reporting and discussing research and results. Congress has also weighed in; it passed a provision that is in effect until at least April of this year that effectively bars U.S. authorities from reviewing proposals for clinical trials involving gene editing of the human germ line.

Dr. Hynes said that, despite the many concerns, he doesn’t believe such gene editing should be prohibited. He said any risks must be weighed against the possible benefit of helping “a patient who is sitting in front of you with a potential problem you may be able to solve.”

“I wouldn’t prohibit it,” he said. “I would regulate it tightly.”


Not surprising with shit like this:


The entire thread is rehashing points already addressed, so have at it.




I just love the writing in that NY Mag article lol:

"Today, a crisp September afternoon under the yawning Illinois sky, there is a far more pleasant smoke wafting about the plaza — billowing, in fact, from every crack and gap in the carapace of the Rose. That’s because Willie is onboard, wailing on a monster spliff.

Well, not just a spliff. More like a medley of miscellaneous marijuana.

On the table in front of Willie is: a vase full of pencils, an orange lighter, a smattering of pre-rolled doobies, an ashtray cluttered with the remainders of joints gone by, a dish of loose cannabis caked with kief, three slim vaporizers of marijuana extract, and a deck of black playing cards printed with the words WILLIE’S RESERVE"

In related news:

“We had two bags of grass, 75 pellets of mescaline, five sheets of high-powered blotter acid, a saltshaker half-full of cocaine, a whole galaxy of multi-colored uppers, downers, screamers, laughers… Also, a quart of tequila, a quart of rum, a case of beer, a pint of raw ether, and two dozen amyls. Not that we needed all that for the trip, but once you get locked into a serious drug collection, the tendency is to push it as far as you can. The only thing that really worried me was the ether. There is nothing in the world more helpless and irresponsible and depraved than a man in the depths of an ether binge, and I knew we’d get into that rotten stuff pretty soon…”


That sounds like a trip me and some buddies took to a Greatful Dead show somewhere around 1995ish?


That happens only when they realize they can’t stop it. But for now they are able to keep cellular medicine from the market by raising the bogus excuse of safety and helping to control the FDA. So they may hedge their bets but they do so all the while making the public suffer so they can wring out every penny of profits until they can no longer keep it up. That is how for profit healthcare is run.


What this can’t be true. Pharma fighting legalization. Why would they do that? Their main concern is making sure the public has the best option for getting relief and becoming healthy. To suggest otherwise is to admit the for profit healthcare system creates perverse incentives and that can’t be true.

The DEA ultimately rejected the HHS recommendation without explanation. This is coming from the same agency who sends officers to testify before Congress that they have no evidence that heroin is worse than marijuana. The lying one needs to commit to hold the agency official line. Much like the ideology from the right.


It wouldn’t surprise me either. Why make something that helps the suffering public, if it cuts into your profits?


Big Pharma will do what they can, including making the public suffer, until they no longer can do it. Then they will do what they can to profit from it. It was just made legal here in the state of Florida with over 60% of the vote. When Pharma realizes the writing is on the wall they are left with no choice. It is profits above all. Why is it so difficult for you to understand? That is a better question.


No, Big Pharma was not around and neither was their perverse incentive of profits above all else.


I wasn’t thinking of things like Down Syndrome, where you generally have a random mutation in an individual.

I was thinking it would be amazing if you knew you carried something in the family like the brca mutation, or Huntington’s disease. It’s just a bit frightening because you can see how things can go really wrong with just a deletion, or one little frameshift of a few important base pairs, and how that could effect not just that child, but future generations.


Road Trip!


@Zeppelin795 - Sorry, I haven’t been closely following. Do you (or someone close to you) have a health condition that would benefit from stem cell therapy, or are you talking about this from a purely intellectual perspective?


Are you questioning the year or the decade? :wink:


He’s a conspiracy theory guy. Over the years many people have tried to talk and reason with him but he just doesn’t like to hear that he might be wrong. Earlier in the thread (or was it another thread? I keep losing track) ActivitiesGuy went through and reviewed a study he posted, as did I, and his only response was effectively “Nu uuh”.

You’re a sweetheart and I love your civility, but you’re giving this one too much credit. He’s been trolling the board for years.Just thought I should warn you :slight_smile:


Ahhh. OK. Got it.

I was thinking a little bit about how the FDA will always have a priority toward caution. Safety will always win, over efficacy. It’s the nature of the beast. Paraphrasing Free to Choose a little here, but I can see why families who have an urgent medical situation get impatient with it. If you work for the FDA, there’s all kinds of downside to approving a thalidomide. People could die, you could loose your career, be pilloried in the press, become a pariah to your colleagues. And we can count the casualties of such an error. We can easily quantify lives lost, or deformaties caused from such a mistake.

On the other side, say your child has a rare and devastating neurological disorder. When she dies, we will say, “She died from complications of her disease.” We likely would never know if some slow process within the FDA made the treatment that would save her unavailable until it was too late. Nobody counts those individuals, as casualties, but of course there are lives lost anytime you have an organization that has an inherent bias toward safety.

I believe there have been studies looking back at something like the approval of say Beta blockers in Europe, and how many heart attacks might have been prevented if they’d been available in the US. I might be making that example up, but that sort of thing.


It’s not at all difficult for me to understand. In fact, my understanding is that profit is the ONLY reason for a business to exist.

What makes you think that they should exist for any other reason?


Ya and everyone died at the ripe old age of died in childbirth.


You’re not making it up - it is a reality that things can get approved more quickly outside of the US than inside the US. But, and you’ll understand this even if the resident idiot here doesn’t, the cost of that is a higher risk of “false positives” too. I’m a statistician - this is just the way it’s going to be. At one extreme, we assume every therapy works and require no evidence for its use; at the other extreme, we assume nothing works and require an extremely high bar to get things approved; moving in between those two extremes will always have the tradeoff. If you want more things approved quickly, that means we approve efficacious therapies faster but we also approve more non-efficacious and possibly dangerous therapies.

So yes, if we had a lower bar for approval, beta-blockers would have been approved faster and some patients would have benefited. The tradeoff is that we might also have approved a couple more diabetes drugs, blood pressure drugs, etc that do not work as well. Even with our rigorous approval process, there still are examples of drugs that have made it through the process and only

Very recent example of this. Last fall, the FDA approved an experimental drug for Duchenne muscular dystrophy based on VERY limited evidence (13 people, no clinically significant benefit, only a slight increase in a protein measurement that indicated the drug was biologically active, but no knowledge of whether this converted to a notable improvement in quality of life or survival). So yes, now people have a treatment option in a situation previously where there was none and (maybe) some lives will be saved as a result. The opposite side is not only the need for assessing “safety” for new treatments, despite what Zeppelin keeps posting. It’s also that we don’t actually know if the drug works yet and if the drug is approved for commercial use before it is adequately studied, it becomes nearly impossible to amass the evidence necessary to determine if it works. Anyone with even the most rudimentary understanding of study design knows this.

More than once. Many times by now. The answer is literally always the same: