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FDA Halts One of the First Human CRISPR Studies Before it Begins (technologyreview.com)
A trial planning to use the gene-editing tool CRISPR on sickle cell patients has been put on hold due to unspecified questions from US regulators. From a report: CRISPR Therapeutics, which is developing the therapy, sought approval from the US Food and Drug Administration in April to begin the study. The therapy involves extracting stem cells from a patient's bone marrow and editing them with CRISPR in the lab. Once infused back into the patient, the idea is that the edited cells would give rise to healthy red blood cells. But according to a statement on Wednesday from CRISPR Therapeutics, the FDA ordered the company not to proceed with its study until it answers questions about its CRISPR treatment.
If I want to turn myself into some kind of vampire bat-man that feeds on human blood to sustain my nocturnal life, I have every right to do it, and then to turn to a life of vigilantism for the fun of beating people up and stealing their vital fluids.
No government will stop me, because I am vengeance, I am the night, I am the MAN-BAT!
FDA once in a while does its job, amazing
I disagree, in this case I think using CRISPR on humans might be a legitimate use. I don't agree with using it to try and edit the human genome to make taller offspring, or blue eyes, or 15 inch ding-dongs; but to cure a genetic disease... I think that's wonderful. Using CRISPR to cure certain defined genetic diseases is a far cry from designer-babies.
This is an area that needs careful oversight but we shouldn't just dismiss a technology because we fear a slippery slope or because we think a beneficial technology is creepy.
"That's the way to do it" - Punch
I'm generally not big on layers of bureaucratic red tape, but for an emergent technology like this it seems absolutely appropriate to take an extra cautious approach for the moment.
no you have improper understanding.
Medicinal use of CRISPR sequences is in clinical trial stage only, in China and USA.
It is not a proven or approved treatment anywhere on planet earth, nor will it be for a very long time
Having the FDA raise the proper questions is not "dimissing". You seem to imagine they're holding up a line of dying patients
I'd hate to conduct animal testing on this procedure.
I'm sure these guys could come up with a name that doesn't make people instantly think of the electric chair.
whats ur deal wierdo
So if the "cure" causes the patient to grow a third hand, or lose their eyesight, or cause bones to start growing in their brain, you are good with that eh? CRISPR is editing the sw for the body. A bug could be a very bad thing. We barely know how this stuff works even though we think we do. Would you have your 6 year old start making edits to the linux kernel?
That is true, and hard to accept.
CRISPR brings promises of curing diseases that are right now causing terrible suffering and early death to billions of people.
Everyone is in a hurry. Many people are desperate enough to submit to experimental treatments, because they are at the end of their rope. In such a situation, getting in front of such experimentation "feels" morally wrong.
The common tendency to confuse morality and legality makes people experience confusion when they see the guardians of food and drugs standing in the way of important medical breakthroughs.
I think they should allow Human trials on terminal patients
Aren't trials like this the very way we figure out how it works?
One problem with these gene-editing treatments is that it is very hard to measure the safety of the treatment. It could be that the company tried to show how it would measure safety, but FDA wasn't satisfied with the process.
Chemical and Engineering News (probably behind a paywall) has an article about how companies are trying to come to a consensus on how to measure safety. https://cen.acs.org/pharmaceut...
A huge problem here is that DNA breaks all the time in our cells and gets repaired. That is the exact process CRISPR leverages to make its edits. So, how do you tell a natural break and mis-repair from a misdirected CRISPR edit. Not an easy thing to tell. FDA wants the applicant to show safety, not for someone else to show dangerousness. Proving a thing that is very difficult to measure in the first place is a great challenge, and may keep these treatments from advancing at FDA.
The Europeans have a similar issues with their beta-thalassemia trial. https://www.bionews.org.uk/pag...
I knew several people that were diseased and desperate, and so participated in clinical trials for deadly ailments over the decades. Guess what happened to 100% of them?
CRISPR may not be a breakthrough at all for humans
OK
You misspelled you're
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Life is a sexually transmitted disease with a 100% mortality rate.
yeah but it can take 80 years or more to die for most in the USA
meanwhile, going to clinical trial instead of normal treatments for deadly cancer will shorten your life even more in most cases.
FDA once in a while does its job, amazing
But is FDA going to be forthright about its 'questions' right away so that a corrected trial can begin without undue delay, or is it going to drag its feet until we see this tech changing lives on other continents?
Don't forget that another reason many patients are desperate enough to opt for clinical trials instead of normal treatments is because they can't afford the cost of the normal treatments while the cost for the trials is often waived.
FDA once in a while does its job, amazing
That depends entirely on the line of questioning. If it is something along the lines of "we have reason to suspect this may pose a risk to patients due to scientific evidence A, B, and C (citations provided)" then yeah, it's a good thing.
If it is something along the lines of "Aren't you violating God's mandate by editing His(tm) perfect genome and the fate He(tm) has decreed for these patients," which is not something I'd put past this administration (and how interesting that the question(s) are secret), then no, this is anything but a good thing.
Either way, we can expect China et al to step into the breach and progress the technology, leaving the US in their dust like they did with Stem Cell research under W.
OK, this is weird, but bear with me.
My dog has experienced a total loss of nasal turbinates, cause unknown, he is a rescue. Subsequent to this he was diagnosed with nasal angiofibromas, benign, but ultimately destructive of the nasal cavity and probably will end with infiltration of the cranial cavity and euthanasia.
Now the oncologist would very much like us to pursue treatment, focused radiation, and then monitor for recurrence, primarily because there is very little known about these conditions. Few dogs receive documented treatment, and so the vet industry doesn't really know how the treatments work, outcomes are indeterminate, and such.
All they want is for us to pay $8,000+ for treatment, with neither any assurance of success, nor any prognosis, and certainly no assurance that a relapse or recurrence won't come, and soon.
And he's the most lovely dog, but I would not pay this for a car repair with similar conditions - *might* fix it, far exceeds objective value, might happen again.
It seems as if this canine oncology practice would love for us to pay to advance the current understanding of this condition. But we were close to asking, if it's so interesting to them, could they perhaps discount the treatment? But nothing about the appointment led us to believe this was anything but a business decision. We've decided no.
We decided to not make our dog the test case. He's probably going to have a shortened but happy life, and we will not let him suffer unnecessarily, which is, to say, very little. He could have been put down in the shelter, so this is a win, and he's such a lovely dog.
While the lure of CRISPR therapy is undeniable, the risks are substantial. The FDA has good reason to demand substantial safeguards, information, and explanations. Look at some of the recent revelations regarding lab tests (Theranos), multiple drug trials ending in failure, fabricated results, etc. Gosh, it seems as if you can't readily trust the industry in general.
This will bump into the 'right to try' movement, but I'm inclined to accept the FDA's reluctance as reasonable. This is a big deal, not as simple as trying it to 'see what happens'. No, it is not.
deleting the extra space after periods so i can stay relevant, yeah.
I don't agree with using it to try and edit the human genome to make taller offspring, or blue eyes, or 15 inch ding-dongs; but to cure a genetic disease... I think that's wonderful.
Can we define stupidity as genetic disease? Just look at the consequences of it...
If you're not at all amusing in person - and we both know you're not - what the fuck makes you think any of that would be funny in print??
Various psychological conditions are suspected of being hereditary. And there is no more dangerous condition than to be diagnosed with one that renders you incapable of being a mentally functional human being. For that, they can take your liberty for your lifetime. Without question. And never permit you to be cured.
deleting the extra space after periods so i can stay relevant, yeah.
Life expectancy for sickle cell patients is something like 20 years. Most are lucky to make it to what we consider middle age.
See: https://patient.info/health/si...
But to be fair, I trust the FDA about as much as I trust the FTC, FBI, DHC, CIA, IRS, and DOJ, which lately is 'less than none, or negative trust'.
I think that is too much trust. Can you go exponential on your negative?
I don't understand in today's world of multi billion dollar corps why they don't buy a decommissioned cruse ship and park it 25 miles off the coast of CA and do whatever the hell they want on the boat. Want to risk the CRISPR treatment, take a boat ride out to international waters and make it happen.
If CRISPR is a miracle one and done cure, expect lots of dragging of feet. How can you charge for a lifetime of pharmaceuticals when the cure is a DNA edit away? Companies that paid their dues to the FDA and various politicians will find rules they can throw in the way of progress.
This may not be the miracle cure, but I am hopeful that the diseases that age us and destroy our minds will be solved in the next 20 to 30 years. If only we can get the roadblocks out of the way of innovation.
FDA once in a while does its job, amazing
That depends entirely on the line of questioning. If it is something along the lines of "we have reason to suspect this may pose a risk to patients due to[...]
The FDA is a bureaucracy responsible for the safety of patients, but bears none of the costs of that safety.
It is in the FDA's interest to make the safety bar so high that medical progress is stifled, so long as safety is its primary goal. Medical companies are always at odds with the FDA, trying to negotiate more sane procedures and reliances.
Taking this article as an example, the patients are completely cut out of the equation. The patients might have a terminal disease with a projected survival of a few years, or might have an awful quality of life - and yet, they cannot choose to try this treatment. They cannot bear the risks from their own choices. The FDA lets patients die rather than have the possibility of an unsafe treatment.
It's ridiculous.
(And for a suggested alternative, let the FDA regulate *products*, not tests. Let the companies do studies and experiments on people willing to take part, and only step in when the treatment becomes publicly available. Add in a bit of legal protections and a standard value of human life - $5 million, say - and medical treadments would advance very quickly.)
This is the reason it exists as an organization, to protect the public and our health. I haven't agreed with everything they've done, and I'm not a huge fan of over-regulation. But, stopping and asking for more details before proceeding on something as new and potentially dangerous to the entire species as CRISPR is the correct approach.
Meanwhile Chinese researchers, not bothered by any of those morality concerns, carry on perfecting the technology. I wonder who's going to be first to market...
Exactly. The first thought I had was whatever this study is might cause an established pharma company to lose profits. After all, we're talking potential cures here, not profitable "treatments".
True? Probably not, but the corruption in the medical industry pretty much pushes money as the top reason anybody does anything, actual patients be damned.
Wasn't this the same thing that someone claimed they were going to stop looking into because "the GMO activists" could send a LOT of nasty emails and put them off their work? If so, I guess it proves that the complaint was a load of bollocks from the off, then. It was they were going to be asked by the FDA about it, and didn't want to answer.
IF the FDA uses its usual deliberate speed, we'll have CRISPR treatments, oh, after I die from something that could have been treated with CRISPR.
If they have questions, ASK THEM. QUICKLY. Get answers, get out of the way.
The FDA is a massively expensive formality, an obstacle of saving lives with allegations of corruption all over the place.
If the FDA told me to eat undercooked meat would I do it. No way !
Did the FDA write The Jungle expose that uncovered the unsanitary practices of food handling? No, it was a private citizen, Upton Sinclair.
How many medical/pharmeceutical breakthroughs happen in over regulated Europe? None.
Does it do anything? Maybe, but better off without it.
I don't think rational thought has much to do with it. Humans pattern match, they don't really think much.
The FDA is covering its ass, and in so doing will cause the deaths of...millions? With the capabilty here, they will literally make millions of people live and die in agony, to cover their mandate and "save" us.
I think people should be able to design their children. A five foot three inch man and a five foot even woman may want more for their child.
See, height matters in ALL aspects of human society. It's a subconscious reaction because deep down, we're monkeys and we respond to the alphas. And height makes one an alpha.
And intellect too. The US Army won't take anyone with an IQ lower than 85. Why? Because people with that low of an IQ cannot be trained for anything. That's 10% of the population.
A world like that great sci-fi movie Gattaca? Let's go!
Usually you have to be relatively sure there are not crazy side effects before you can start human-trials.
Shouldn't that choice be ultimately up to the patient in question?
It is their life, it should be their choice....
Light travels faster than sound. This is why some people appear bright until you hear them speak.........
I'm holding out for the CRSPN-GLOVR treatment. It is our density.
Good luck with your pup....great story to hear!!
I lost mine a few short years ago...she lived to be almost 16yrs.
I"m contemplating if I'm ready for another one...they DO bring such joy into your life.
Enjoy yours while you have him...
Light travels faster than sound. This is why some people appear bright until you hear them speak.........
I don't agree with using it to try and edit the human genome to make taller offspring, or blue eyes, or 15 inch ding-dongs
I suppose the FDA are only able to interfere with this study BECAUSE this is a study related to a product for curing a disease or treating a medical condition, and the FDA approval is required for medical treatments.
Using CRISPR to design offspring traits or interesting ideas that someone is going to pursue, and i'm sure it could be a "thing" eventually after we as a civilization actually learn enough about genetics to reliably and repeatedly make such child customizations... Fine to disagree and not use the tech yourself, since when developed everyone with a lot of cash to burn would have their own personal choice to choose to Use it or Not use it for themself, when having their kids. Also --- since "editing the genome of offspring to have blue eyes in the lab" is not a product or procedure designed to remedy any disease or address any medical condition (Not a "medical treatment"); the FDA will likely have zero authority to discourage or regulate it, since tailoring eggs and sperm in the lab to achieve certain desired attributes for your baby is not medical --- And i'm sure the lobbyists for companies that will emerge before this becomes a concern will not allow the situation to change, providing the corps. do a sufficient job at quality control to ensure they won't have tailored babies born with "defects".
Why not?
I mean, one of the primary driving goals of human parents is to do their best to give their offspring the BEST potential to do well in their life....
If they couldn't start ahead of the curve and give them physical traits (height, brain capacity, disease resistance, heck even traits towards attractiveness)....why not?
It is just taking what parents do now to a higher step.
Light travels faster than sound. This is why some people appear bright until you hear them speak.........
Medicinal use of CRISPR sequences is in clinical trial stage only, in China and USA.
Perhaps.... but what counts as "Medicinal use" ?
Is using the tool to alter cells in a test tube and then re-inject only altered cells back into the body a "Medicinal" use?
Don't think so..... Medicinal treatment would suggest injecting
a CRISPR formulation to modify DNA in the cells that are inside the human body while being modified.
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Meanwhile Chinese researchers, not bothered by any of those morality concerns ...
China has moral concerns. They just reach a different conclusion. After weighing the potential benefits to millions of potential beneficiaries against the risk to a handful of patients (who are informed consenting adults), I don't think their conclusion is wrong.
The West is cautious about medical experimentation because of historical abuses such as the Tuskegee experiments, and Nazi experiments. The Chinese don't have the same cultural baggage.
i have crohns, i would happily take one of the current experimental treatments if i lived close enough to a testing center... anything beats my current treatment regimen even if it shorts my lifespan considerably its still a chance to not be bound to a toilet.
You compare your dog to a car, and then expect to be taken seriously?
You don't, eh? So much for the:
In Soviet Washington the swamp drains you.
FDA once in a while does its job, amazing
I disagree, in this case I think using CRISPR on humans might be a legitimate use. I don't agree with using it to try and edit the human genome to make taller offspring, or blue eyes, or 15 inch ding-dongs; but to cure a genetic disease... I think that's wonderful. Using CRISPR to cure certain defined genetic diseases is a far cry from designer-babies.
And your vision of this future utopia is a far cry from reality. It's cute that you still assume modern medicine is being used to cure disease, or increase life expectancy. That's not the goal of any country that is overcrowded already and fighting to sustain enough resources for the current population. I more expect CRISPR being abused to create genetic diseases. We're doing OK today with shit like HPV spread very well across the masses which will ultimately funnel trillions into the Cancer Industrial Complex over the next few decades and help cull the population, but if greed ultimately determines it wants more and faster, manufacturing disease that creates faster results is certainly an option.
Go ahead. Call it impossible. Call it delusional. Call it fucking batshit insane. Call it whatever you want. Then look at history.
This is an area that needs careful oversight but we shouldn't just dismiss a technology because we fear a slippery slope or because we think a beneficial technology is creepy.
Need oversight? Take a number and get in line. You'll be assigned appropriate resources right after the snow-cone maker is delivered to Hades.
Meanwhile Chinese researchers, not bothered by any of those morality concerns, carry on perfecting the technology. I wonder who's going to be first to market...
Morality concerns would be the only reason to pursuing these kinds of treatments at all.
If amoral, you let the less fortunate suffer and die, replace them with new humans, and trust evolution to reward the winners by their genes propagating more.
Personally, I'm midway between the two, which some might find extreme enough. I'm all for offering saving of lives and reducing severe suffering, as long as it is always conditional on mandatory sterilization, and as long as suicide and infanticide are also presented as options.
One of the few times pet insurance is a really great idea.
You compare your dog to a car, and then expect to be taken seriously?
Rickb928 did not compare his dog to a car.
Why not?
I mean, one of the primary driving goals of human parents is to do their best to give their offspring the BEST potential to do well in their life....
If they couldn't start ahead of the curve and give them physical traits (height, brain capacity, disease resistance, heck even traits towards attractiveness)....why not?
It is just taking what parents do now to a higher step.
Well, for one, that would be creating a permanent over-class and servant-class, unless it was available for everyone. For another, it would subject children to the whims and fashions of the day. Thirdly, and most importantly, if this were available for everyone it would impact the diversity of the human genome which could have long lasting consequences beyond our possible knowledge. A fashionable gene could end up putting the entire human population at risk at being wiped out by a single disease.
"That's the way to do it" - Punch
FDA once in a while does its job, amazing
I disagree, in this case I think using CRISPR on humans might be a legitimate use. I don't agree with using it to try and edit the human genome to make taller offspring, or blue eyes, or 15 inch ding-dongs; but to cure a genetic disease... I think that's wonderful. Using CRISPR to cure certain defined genetic diseases is a far cry from designer-babies.
This is an area that needs careful oversight but we shouldn't just dismiss a technology because we fear a slippery slope or because we think a beneficial technology is creepy.
I also would be concerned about a mother that wanted her son to be gene-edited to have a 15 inch ding-dong.
The problem is that depending on the stage of a trial, a single death (regardless of it being caused by the new medication/procedural or just the fact that the patient is already dying from the condition), can derail the entire trial and prevent or delay a new treatment from coming to market (did patient X die from the treatment or the condition). Yes the person is rolling the dice hoping that it comes up box cars, but if it comes up snake eyes thousands of others can be affected
Nope. Not only is their decision making capacity impaired, the vast number of con artists and frauds in the industry endanger all of us.
Sorry, but Law and Order exposed the truth about the alleged truth over three decades ago.
as society we've already made the choice that doctors, medicines and treatments will be vetted. so no, the patient doesn't get to support a quack (legally). yes there are shortcomings and abuses in the system, different topic
eh, "medicinal" only means healing. The mechanism and protocol could be anything.
It is unknown at this point if CRISPR use can heal *anything* in humans.
we're far, far from what the headlines imply. I see silliness that are variation on "Governments not keeping up with the wonderful power of CRISPR" . Oh noes, this live-saving tech is only restrained by powerwork!
er no, there is no treatment for humans using CRISPR sequence. no known benefit. no healing of any disease.....only clinical trials exist and the results not published yet. nor peer reviewed,
So the article is just about some bureaucratic mumbojumbo like _always_ when such trials start.
I guess form 643a got stuck in the mail and now we get a crappy story here.
Tell us when they cured the guys but not every step to get the authorization to begin.
> yeah but it can take 80 years or more to die for most in the USA
"for most in the USA"? Please, most Americans can't actually afford to live that long. America's health care model is so fucked compared to the modern world.
shades of "Cancer. The Forbidden Cure".
https://www.youtube.com/watch?...
This is totally incorrect, and the link you gave doesn't support your claim at all.
Sickle-cell is an evolutionary adaptation that protects an individual from malaria, so in some areas (before modern medicine) it would increase a person's lifespan and chances of reproducing. In modern times, it's a chronic condition that does come with health risks but is in no way a death sentence.
Yes, they do, they just choose to ignore it. They were subject to Japanese scientists experiments which were just as ghastly as the Nazis. Then there is the abject horror of the Mao years.
The more interesting part is the following, from a CRISPR press release: The planned initiation of a Phase 1/2 trial of CTX001 in Europe in adult patients with transfusion dependent Beta-thalassemia is unchanged, and the companies expect to initiate the trial in the second half of 2018. Therefore, ex vivo CRISPR gene-edited therapies are actually about to go into trails. The SCD treatment has hit a very small bump in the road, which is expected and less of a story.
Havenâ(TM)t we learned anything from the last four zombie apocalypses? No? Nothing?
Shouldn't that choice be ultimately up to the patient in question?
It is their life, it should be their choice....
Nope. Too many quacks and frauds involved who will sell poison and claim it is a cure under the auspices of choice. Law and Order covered this over three decades ago.
Besides, all of those "clinical trials" want the government to sanction them, and prevent me from executing the bastards in the streets.
Now who's choice is that?
Sorry, but unless you want to give me the right to rid the world of these pests, you'll have to accept the FDA.
This company and their claims sounds exactly like Theranos. Anybody remember Theranos? The company that FAKED the capabilities of their product?
FDA is 100% right when halting the "experiment" until they answer questions and show proof that can be duplicated.
BTW, Why is the CEO of Theranos still walking on the streets? She committed fraud in the most heinous of ways and possibly caused many deaths due to the fake results the company provided.
So rather than fix the process just let people continue to die out of fear of fucking it up for everyone else? That's ridiculous.
This is a confusing comment. Medicine has advanced, and many effective treatments were first validated for efficacy and safety through a clinical trial. That's really the only way we know how to do this at the moment. Of course many clinical trials have not had the desired effect and many have had a net negative impact on the patient. That sucks, but what alternative is there?
> And he's the most lovely dog, but I would not pay this for a car repair with similar conditions - *might* fix it, far exceeds objective value, might happen again.
Sorta.
I think the size of the investment is coloring the decision here. What if it was a $75 treatment? Would anyone be morally opposed to funding experimentation then?
The more coupling there is between corporations and the federal agencies that are supposed to regulate them, the less you need a tinfoil hat to wonder if influential companies might use the FDA to obstruct research that could result in products that threaten their business. We live in an age of medical maintenance, not cures. How long has it been since the last eradication of a major disease? I have to wonder if we'll ever see cures for diseases such as diabetes that support multi-billion dollar maintenance industries.
not saying there is alternative, my point only that this type of gene therapy is at that early stage where unknown if it will even benefit a human at all for any disease for which it is in testing. The headline makes it sound like there is a magic cure with hordes of dying people being pushed away by FDA bureaucrats, which is nonsense.
Shouldn't that choice be ultimately up to the patient in question?
It is their life, it should be their choice....
Nope. Too many quacks and frauds involved who will sell poison and claim it is a cure under the auspices of choice. Law and Order covered this over three decades ago.
Besides, all of those "clinical trials" want the government to sanction them, and prevent me from executing the bastards in the streets.
Now who's choice is that?
Sorry, but unless you want to give me the right to rid the world of these pests, you'll have to accept the FDA.
nothing changes in over 3 decades?
So what are the reasons?
What about "Right to try"?
Self-importance and self-indulgence is the root of ALL evil.
It's not a process issue, its a case of potentially adding a whole bunch of extraneous and erroneous data into a trial that could derail, or delay the introduction of a new treatment E.g. A team is in the process of trialing a new treatment for pancreatic cancer. A desperate patient suffering from stage 4 pancreatic cancer wants access to the treatment because, after all, what have they got to lose. The patient gets the treatment, but they still unfortunately die. Now the trial team has a problem. Did the treatment kill the patient? Did they just get the treatment too late? Was their some other factor?. Since much of the data generated during a trial is statistical in nature, you can't just say, "well ignore this guy" because maybe it did identify a legitimate issue with the treatment.