FDA Considers First CRISPR Gene Editing Treatment That May Cure Sickle Cell 39
The U.S. Food and Drug Administration is reviewing a cutting-edge therapy called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease with no universally successful treatment. "If approved, exa-cel, made by Boston-based Vertex Pharmaceuticals and the Swiss company CRISPR Therapeutics, would be the first FDA-approved treatment that uses genetic modification called CRISPR," reports CNN. From the report: CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop. [...] The new exa-cel treatment under FDA consideration can use the patient's own stem cells. Doctors would alter them with CRISPR to fix the genetic problems that cause sickle cell, and then the altered stem cells are given back to the patient in a one-time infusion.
In company studies, the treatment was considered safe, and it had a "highly positive benefit-risk for patients with severe sickle cell disease," Dr. Stephanie Krogmeier, vice president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel. Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshapen red blood cells block normal circulation and can cause moderate to severe pain. It's the top reason patients with sickle cell go to the emergency room or are hospitalized. Before the treatment, patients experienced about four of these painful crises a year, resulting in about two weeks in the hospital.
The FDA sought the independent panel's advice, in part, because this would be the first time the FDA would approve a treatment that uses CRISPR technology, but Dr. Fyodor Urnov, a professor in the Department of Molecular and Cell Biology at the University of California, Berkeley, reminded the committee CRISPR has been around for 30 years and, in that time, scientists have learned a lot about how to use it safely. "The technology is, in fact, ready for primetime," Urnov said. With this kind of genetic editing, scientists could inadvertently make a change to a patient's DNA that is off-target, and the therapy could harm the patient. [...] The FDA is expected to make an approval decision by December 8.
In company studies, the treatment was considered safe, and it had a "highly positive benefit-risk for patients with severe sickle cell disease," Dr. Stephanie Krogmeier, vice president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel. Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshapen red blood cells block normal circulation and can cause moderate to severe pain. It's the top reason patients with sickle cell go to the emergency room or are hospitalized. Before the treatment, patients experienced about four of these painful crises a year, resulting in about two weeks in the hospital.
The FDA sought the independent panel's advice, in part, because this would be the first time the FDA would approve a treatment that uses CRISPR technology, but Dr. Fyodor Urnov, a professor in the Department of Molecular and Cell Biology at the University of California, Berkeley, reminded the committee CRISPR has been around for 30 years and, in that time, scientists have learned a lot about how to use it safely. "The technology is, in fact, ready for primetime," Urnov said. With this kind of genetic editing, scientists could inadvertently make a change to a patient's DNA that is off-target, and the therapy could harm the patient. [...] The FDA is expected to make an approval decision by December 8.
lives worth living (Score:4, Insightful)
Finally an actually useful application to improve people's lives.
So far, the only genetic modifications the world has seen is to plants so they can spray more poison on them aka RoundUp-Ready crops.
So while I'm very much in favor of the former, the latter is why so many are opposed to gene editing.
I'm looking forward to more applications of CRISPR that can make very sick peoples lives worth living again.
Re: (Score:2)
Home schooled?
Re: (Score:2)
Why was this modded troll???
I realize reading comprehension isn't what it used to be, but thankfully we're here to help you out.
woketardism
Now please go back to your coloring book, just try not to eat the crayons this time.
Re: (Score:2)
We have to be careful though. That gene you shut off or modify for one disease may be crucial to other functions. Things like schizophrenia and autism may be the price we pay for having big, complex brains.
Re: (Score:2)
Like surviving malaria?
Re: lives worth living (Score:2)
Here, in the case of the OP, the choice is between living in agony day in day out or CRISPR.
You might stick to your bed and herbal tea, but in actuality there is no 'good' scenario in this case. CRISPR sure is much better than the downhill of opiods to manage the pain.
Re: (Score:1)
Its not just pain. Vasoocclusive crises can be emergent. SCD patients often have decades less life expectancy. While current SOC helps, ie hydroxyurea, the curative data from gene therapies is a huge deal, and has all but eliminated VOC.
Re: (Score:1)
possible for some genes, but is this case the therapy leads to the upregulation of a gammaglobin protein, normally expressed natally, to form fetal hemoglobin. Some people maintain expression of fetal hemoglobin already with no ill effects.
Re:lives worth living (Score:4, Interesting)
Finally an actually useful application to improve people's lives.
This is far from the first: Applications of CRISPR in medicine [biocompare.com]
So far, the only genetic modifications the world has seen is to plants
Not true. There are many GMO animals.
so they can spray more poison
Not true. Glyphosate tolerance reduces the amount of herbicide needed since it can be applied during the seedling stage instead of trying to kill weed seeds, which requires more and more potent herbicides.
Nearly all glyphosate that enters the food supply comes from its use as a pre-harvest desiccant, which is only used on non-GMO crops. Want less herbicide in your diet? Eat only GMO.
Re: (Score:2)
There really is no point to arguing with chemophobes about glyphosate. Might as well argue with chemtrailers or fluoride in drinking water; they're on the same level.
Re: (Score:2)
Finally an actually useful application to improve people's lives.
Since Greed in the Medical Industrial Complex greatly prefers supporting perpetual treatments instead of cures, I would sit down before reading the eventual price tag. Sadly I cannot even begin to imagine how expensive it will be.
Re: (Score:2)
It has been slow in coming to people because the first attempts at gene therapy failed badly.
Re: (Score:2)
Golden rice is engineered to produce vitamin A. It's saved the sight and lives of a lot of children. Many other plants have been engineered for pest resistance (so you need to use less insecticide and herbicide), hardiness, production, virus resistance, etc. If you're going with "genetic modification" then everything you eat is genetically modified and civilization couldn't exist without it.
CAR T Cell therapy is used to treat blood cancers, again prominently in children.
Genetic engineering is also used to p
Re: (Score:2)
Golden rice is engineered to produce vitamin A. It's saved the sight and lives of a lot of children.
It has?
Seems like a good idea but it sounds like it's still trying to make it into the market [goldenrice.org].
Re: (Score:2)
Ah, you're right, apparently they're still arguing about it in the Philippines. It was approved in 2019 IIRC but it seems there's been some backsliding.
Still an example of beneficial genetic modification. Also salt-tolerant rice. White rice itself is a mutation bred and propagated by humans.
UK approved it. US will eventually. Barely. (Score:1)
The UK approved this last week. Too many articles to include by link, but a simple Google search will show it.
As usual the US FDA is behind the times, disallowing anything to help people heal, and propping up big pharma's "work around the symptoms for recurring monthly revenue" vs "fix the problem for good." Same holds true for MS, diabetes, etc. We like to call it a free market, but it's not free, and it's a market where our masters tell the slaves what they're willing to offer today and the government
Re: (Score:2)
You know nothing of the regulatory process. It exists because those nice corporations will put a price on grandmothers' lives and use the free market to sell what they can without an "excessive" number of deaths, which are bad for the bottom line.
Re: (Score:2)
The FDA imagines that every new drug is thalidomide, and so it tries to kill them all by default. The result is that lots of Americans die of diseases that could be treated but the treatments are blocked. It's hard to get traction with the problem because people who die due to FDA roadblocks are buried in an invisible graveyard (to borrow [marginalrevolution.com] a phrase).
Re: (Score:2)
This is bullshit.
People imagine that every new drug is a miracle drug that's going to save lives. In reality, about 90% fail clinical trials, most because they simply don't work.
Re: UK approved it. US will eventually. Barely. (Score:2)
Which is the point of clinical trials. FDAs only purpose is to put roadblocks up. It has never blocked anything truly bad before it was too late or put anyone out of business with fines.
The FDA doesnâ(TM)t employ the best scientists, because any decent scientist would be better paid just about anywhere else. The FDA doesnâ(TM)t do any real science either so anyone whoâ(TM)s been there long enough to make a decision has been removed from the field too long to have kept up.
You literally give th
Re: (Score:2)
The FDA is the US agency that regulates clinical trials. This includes setting standards for them and evaluating the results before granting an approval. FDA approval boards are generally composed of scientists in the relevant fields whose day jobs are in labs or hospitals around the world. The FDA does employ some quite good scientists directly in labs researching things mostly not directly related to pharmaceuticals.
I don't know exactly what you think the FDA is, but it's not that.
Re: (Score:2)
Sure, if that's what you think it is. It is not. It never has been and it never will be. I think you're confusing NIH/NSF (the science funding agencies) with FDA (the bureaucrat agency)
I do deal with FDA approval quite a bit, working in the medical research field for 20+ years. Most of it is paperwork, I've never talked to a scientist or even computer scientist or programmer worth their salt from the FDA, you fill some forms, check some boxes and then you wait for some contractor that outsourced the manual
Re: (Score:2)
If you think the NIH regulates clinical trials I'm going to have to seriously question your experience in medical research. Maybe at the bench stage?
Various funding agencies in the US fund some clinical research, but the vast majority of clinical pharma trials, particularly the big, expensive, phase III ones, are funded by, surprise, pharma. And their ain't no way they'd be doing that if the FDA (and other countries' health agencies) didn't make them. In fact, before the creation of those health agencies, t
Re: (Score:2)
No, I'm talking about the staffing. The NIH/NSF is stocked with scientists that couldn't cut it in academia.
FDA "regulates" a lot of things, but they do not REVIEW clinical trials which is what I'm talking about, FDA simply isn't staffed to do that job. You can start clinical trials simply by filling in a form, notifying the FDA, effectively promising that you have read and will follow ALL their rules and regulation, which given the amount is simply impossible for even large entities to actually do, the rul
Re: (Score:1)
Re: (Score:2)
Again, you describe a bureaucratic process, not a scientific one. You don't seem to understand the difference between the two. Yes, lots of paperwork, nobody at FDA understands what you are doing, they are filing paperwork, you can fill pretty much whatever you want. As I gave my example above, run a networked blood pump (eg. Alaris) off a Windows XP machine in 2023 - approved.
Re: (Score:1)
I did not describe a bureaucratic process. The FDA has researchers and clinicians on its payroll, including the director of CBER. They conduct the review to ascertain the merits of any regulatory submission. It is not filing paperwork. Just stop. You have no clue how this works.
The FDA may not be staffed by people on the cutting edge, and sponsors have to invest time in demonstrating to the agency that any new tech is justified. Those wheels can turn slowly, and they have acknowledged this. I can't speak ab
Re: (Score:2)
Software validation is relatively trivial, again, it's a bureaucratic process and as you must admit, it's broken.
Software validation doesn't prove anything, it doesn't prove security, it doesn't prove that the software is good, because running an ancient version of Tomcat openly on the network isn't secure. It MAY have been secure for some standard 25 years ago, but it's not today. The FDA doesn't evolve with the computer or medical science.
Re: (Score:1)
Re: (Score:3)
You know nothing of the regulatory process. It exists because those nice corporations will put a price on grandmothers' lives and use the free market to sell what they can without an "excessive" number of deaths, which are bad for the bottom line.
As always, have it. Clearly you know more than the people who have spent their entire lives developing and testing medical procedures. Surely an advanced degree and decades of experience isn't needed when dealing with manipulating human genes. So go out and get our splicer and start "curing" those diseases. $10 a pop, right?
Re: (Score:2)
Clearly you know more than the regulators put there to make sure corporate profits don't come at the expense of human lives.
If you have any kind of point I would imagine you'd be able to support it with some proper examples with citations.
Re: (Score:2)
There's a new MS treatment in the pipeline - an 'inverse vaccine' that trains your immune system to ignore myelin.
It's from the University of Chicago and it looks very promising.
It's not gene editing, but I believe it is a child of COVID vaccine technology.
While the FDA can be annoying, it's not generally conspiring to watch you suffer and slowly die - a lot of the things you want medicine to do are exceptionally difficult.
Re: (Score:1)
Yay! (Score:3)
Re: (Score:2)