In layman's terms: She had a particularly vicious form of cancer where the very cells that are supposed to protect your health -- your T-cells -- are the enemy. So they made three edits to donor cells and wiped out her compromised cells. Then gave her a bone marrow transplant afterwards to replenish her immune system with new cells.
I'm squeamish about genetic research, but this looks really good to me. I hope she recovers fully and there are no further complications, though I imagine it's too early to guess what her prognosis might be.
So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.
That's a big deal for people with those genetic disorders.
And I'm sad about millions upon millions of mostly well-meaning people, who died of ultimately preventable causes - if only we could push the research from the lab to clinic faster.
Everybody is prone to ageing, and very soon it will be our turn.
This is suicidal thinking - you essentially want to die, but you wish it on some future you (certainly not the you of today, whose turn it probably isn't yet). I cannot see a world in which this could be deemed healthy.
Instead you want to avoid/deny eventual natural death for ever? Fight it? To be at peace with the process of aging and the thought of eventually dying is not the same as suicidal thinking.
> I cannot see a world in which this could be deemed healthy.
I cannot see a world in which fighting and avoiding natural death can take healthy forms. All matter decays. All things recycle. We have material bodies, hence we are part of the cycle of life. The old giving way to the new is a necessity for growth & evolution. I would even say it's beautiful. And healthy.
To what extent is your view an adaptation to how things are currently (and I agree it can be good to have a positive outlook on things you can't change) and to what extent do you think it's a strong analysis of how things should be?
Let's say a mysterious virus spreads through the population, and edits all our DNA with the only effect that we all suddenly gain negligible senescence - people still die from accidents and acute diseases, but everyone retains the same level of general healthiness as a young adult with no aging. In that situation, would you be advocating that everyone take medicine to turn aging and death from aging back on?
Look, if you want to die, go right ahead. But leave me and my family out of it. We love our lives and each other and I'd like for us to live as long as we can. Proclaiming your disdain research in this field because "death is good" without elaborating on that (and when the obvious conclusion is the exact opposite, see: The deaths of children with cancer, the deaths of world-changing intellectuals like Von Neumann, the eventual deaths of every good person/pet that you know), does not come off in a flattering light for you.
The fact that you think dying is good is a feeling. It is based on an article of faith - it must be, because you cannot make coherent arguments for the necessity of death that aren't either based on faith or other issues that are solvable in ways that don't kill people.
I'm not "just being offensive". Death is harmful, and insisting it must happen is perpetrating death. If you insist this, you are perpetrating a harmful ideology that interferes with research into senescence and therefore contributes to suffering.
You're afraid, I get it. So am I. We'll all die someday. So will everyone and everything, at some point. It's not good or bad. It's not a matter of faith or ideology. It's scary and we don't want it and we fight it with everything we got. That's also a part of being alive.
I'm just concerned with the possibility of us "winning over death" and how it would transform everything. It's not another cure for an illness or another 100 years to live. It's no one ever going away without someone else making them. For better or worse. That's not a part of life. That's an entirely different existence.
I 'm totally with you. Most of human misery, suffering and malice is due to decline, loss, grief and death, and the awareness about them.
People who claim it would be bad to live long are either coping or had a chance at a decent life or believe it would somehow cause unsolvable problems.
Lmao. If we extrapolate your logic to few hundred years ago - you shouldn’t survive over 40 yrs. But I’m sure you can’t wait to retire at 60. So your perspective is simply a result of past scientific work in defeating ‘natural death’. And suddenly you think it’s a bad idea.
while a rude response, guy above does raise the point of well, yes I'd like to not die at 40. Medicine is good.
But the talk of imortality is hubris. Is is to judge oneself the best or most worthy thing, and to deny our children and the nature of progess itself.
As medicine has brought us extra life, death and re-generation have brought us us. And as us which have been given, we give to others through our death.
So whats the limit? Its a big question that dosen't need to be answered now, because immortality is not on the table. Another 20 or so years maybe. So maybe lets take care of the world we got before we fall into some delusional sense of godhood. (Just upload yourfselfs bros)
You don't need to ban, say, clinical trials of gene therapy (especially with inert lipid nanoparticle carriers which do not replicate), to ban a quite narrow and not really clinically useful subfield called "gain of function research".
Why can't our legal system deter the obvious high-level publicly visible perpetrators of the recent "gain-of-function" fiasco is really some food for thought.
It’s more than broad weaponization of viruses that are a danger though.
Take for instance what we’re doing with plants. We make designer plants that provide food, and are immune from particular pesticides. We then spray the fields and all the other plants and bugs die (besides the immune plants).
What happens when it turns out some genetic inserts in the corn creates something like mad cow disease (but in humans) 3-5 years later? Turns out some protein the corn produced we thought was harmless caused massive issues.
Genetics has massive implications even if well meaning.
I think the ability to eliminate disease is massively powerful and a net good. That said, I’m concerned about the long term tail end risks of such work.
I don’t think you can ban it, because someone will do the work. And those that do the work will get a competitive advantage (eventually letting them “win”). I think it’s about mitigating risks by reducing / limiting mass adoption of genetic based technology without a very long window of analysis.
> I think the ability to eliminate disease is massively powerful and a net good.
One real-world example of this is genetically modifying the American Chestnut by splicing in genes of the Japanese Chestnut, which is resistant to the blight since it co-evolved with it. The resistance comes from how Japanese Chestnut trees respond to oxalis acid by walling it off, preventing the fungus from spreading.
> That said, I’m concerned about the long term tail end risks of such work.
One bleak science fiction novel that takes place in this kind of future, specifically an amped-up version of the corporate GMO food crop meddling you referenced, is The Windup Girl by Paolo Bacigalupi.
> What happens when it turns out some genetic inserts in the corn creates something like mad cow disease (but in humans) 3-5 years later? Turns out some protein the corn produced we thought was harmless caused massive issues.
Do you require similar safeguards with plants created by "classic" genetic engineering and random mutations occurring all the time? Such methods usually involve making tons of random mutations, which nobody tests for anything, until we manage to also get the one that we want.
Of course you rarely make a completely new protein by random mutations (different story with naturally evolving viruses), however, current genetic engineering of plants and animals for food is AFAIK not about completely new proteins too -- it's about turning off existing pathways, or about transferring existing proteins from one species to another.
Eh, life forms can have a exponential growth curve, so virologist and bacterial genetic research can easily kill a large part of the population. But then we are well on the way for that, so who cares?
Probably not. Genetics isn't nearly as clean a people imagine it to be.
Much easier to target one individual (and their whole host of genetic factors) than to target one genetic factor that can be associated with an ethnic group and pray that it doesn't either also express itself in an undiscovered way in other ethnic groups or have knock-on effects or the potential to mutate.
We aren't as genetically different as people imagine, and this sort of approach would be absolutely playing with fire.
One could even craft a virus that is targeting a certain behavioral anomaly like sociopathy, if its genetically clearly identify-able. All one would need is a proof-able potent carrier virus and once could reign in the destructive potential of humanity.
"No more bad boy behavior, or the bad boys vanish from the surface of the earth." behavioral deterrence via a biologic weapon.
What a fascinating nash equilibrium, the unsocial monsters, forced to be social, including applying gentle pressure, so they do not become history like there victims of the past. What a concept..
>One could even craft a virus that is targeting a certain behavioral anomaly like sociopathy, if its genetically clearly identify-able.
It isn't, and I'd be surprised if it ever could be. There will always be statistical correlations (especially when looking at multiple genes in combination) but you can't conclude what a person's brain and mind are like just from their genome.
Nonono that would be lovely, finally some evolutionary pressure. What's the real threat is breeding obligately obedient citizenry and immortal leaders. Which is the first thing practical genetic engineering will probably be used for.
It looks to me like a somewhat unique opportunity to risk it because without this treatment, she was dead anyway and in fairly short order. So it was a means for them to learn something with very little potential downside. Worst case scenario: The treatment kills her. And she was dying anyway.
From the article:
In Alyssa's therapy, each of the base edits involved breaking a section of genetic code so it no longer worked. But there are more nuanced applications where instead of switching an instruction off you can fix a defective one. Sickle-cell anaemia, for example, is caused by just one base change that could be corrected.
And I already quoted (in my previous comment) the next paragraph that indicates they are already doing trials. This is potentially a big deal and they seem to be pursuing it in a very reasonable and conservative fashion, which is always something that concerns me with this type thing. There's a lot of potential for terrible and horrifying abuse and that is not what I am seeing described here.
Yeah, those are valuable opportunities. You have to take risks to learn, but human life is to valuable to risk.
I remember looking into the state of the art around in utero and neonatal health when we got pregnant. I was astonished at how limited the knowledge is. I quickly realized the big gaps are just because it’s too risky to study. You can’t morally experiment on pregnant people and infants.
Seems like this particular version of gene editing only works with blood/marrow disorders? If someone had a single mutation affecting their colon or eye, would the same technique be appropriate?
Blood/bone marrow is relatively easy as you have a very clear delivery vehicle for your modified genes. Actually getting to the colon cells or retina is a lot harder, can’t just inject it into the blood and expect it to end up in the right place.
A "problem" with this treatment is that, for most patients, the current standard of care works. To the extent that this treatment is effective, it is likely effective for most leukaemia patients, including those who would respond well to current treatments. It is very plausible that this treatment (or a refined version of it) will eventually be shown to be superior to the current standard of care, in which case it would become the standard of care, and the current treatment will be relegated to the niche patient that does not respond to gene edited t-cells.
More broadly, the technique of precisely editing DNA of select cells to perform specific engineered tasks is very broadly applicable. But that is a technique for developing treatments, not a treatment itself.
Remember what "works" means in this case. Chemotherapy, or for other cancers, radiation, surgery that removes large, healthy parts of organs that might have a tumor. Chemotherapy has hosts of side effects, of course (hair loss, nausea, ruined immunity, CIPN, etc) some permanent for some people. It "works" when you look at what KPIs oncologists like, very reductionist things like "5-year overall survival" whilst you ignore things like quality of life both in the short term and long term.
We should be moving away from chemotherapy. It really is poison (cytotoxin) that has "treatment" of cancer as a side effect, just because it will readily kill cancer cells more than healthy cells whilst also taking a lot of the healthy cells with it. But, it's the "established" standard of care, which many doctors built their careers on, so I suppose it won't go away soon.
Surgery to remove part of an organ, followed by chemotherapy gave my mother a few more years of life. Are you suggesting there is some other alternative, currently accepted and understood, that would've worked better? Because while those side effects are terrible they are usually more temporary than the extension of life that they afford people. Face the question, would you rather chemo for a couple months and then live a couple years, or die in a couple weeks?
Isn’t this treatment simply accomplishing the same thing that the current treatment does but in a targeted way with hopefully much fewer side effects? When you have cancerous T cells the only option is to eradicate all of them and start over with a bone marrow transplant. Normally that would mean frying them with radiation or poisoning with chemo which also effects a lot of other tissue. This treatment is supposed to only kill the T cells.
That's my noob understanding too. This is a more precise form of autologous transplant.
I assume quick adoption of base editing for trickier cases, eg for T-cell acute lymphoblastic leukemia, like this patient had. Note that she had a previous transplant, using the more conventional treatment of her T cells. Just starting with base editing will be much less onerous to future patients.
This is one tough person. Surviving chemo then two transplants is amazing. Just thinking about it crushes my heart.
Source: Had bone marrow transplant 30+ years ago. My then experimental treatment became the default option (as applicable). Don't know much about biology or medicine, but I can repeat what I've heard and read.
It starts with researchers convincing their ethical review board that they should be given the opportunity to experiment with the different approach.
That’s why new procedures almost always are done on patients who are as good as dead (gene editing to change one’s hair color won’t soon get past ethical review, for example, but if gene editing gets used in mainstream medicine, enough data may be collected to make a review board say it’s acceptable to give it a try)
The first experiments typically only prove the medicine or procedure works. Long term survival of the patient the exception. From there, it’s step by step towards treating healthier and healthier patients.
If it turns out the approach is better for the typical patient, patient groups (who may even have paid for part of the research) start lobbying for its use. If the new procedure is both better and cheaper, insurers also will lobby for it.
Wiping out your immune system and replacing it has something like a 50/50 survival rate even if you use your own cells. It's a hail Mary than usually either kills or cures.
I knew a guy with cancer that had this done. He survived the process, but unfortunately the cells they restored him with were not cancer free like they thought. He died anyway.
I think increasing the survival rate for these things could be very useful.
Wiping out the recipients immune system and transplanting a donor system is in itself a common (advanced) practice since long, so the new part here is the tweaking of the donor system. Very interesting.
Resetting the immune system can be already done and it seems to help against auto-immune diseases such as lupus, but it is very dangerous. Once you destroy the immune system of the patient, and before it grows back from donor cells, the patient is fatally vulnerable even to normally banal pathogens such as the common cold. Keeping the patient isolated and alive in a hospital full of germs is a major challenge.
That is why this kind of therapy is only used when the patient faces death anyway.
Some people are interpreting that more broadly than I intended it. I'm squeamish about this kind of medical gene editing in specific.
There is a genetic disorder that runs in my family. I used to be on a bunch of email lists for the condition. People with the condition routinely volunteer for drug studies in a desperate attempt to get help that current medical science can't provide them and/or to get free care for a condition that's very medically expensive.
People with serious medical conditions are all too often treated like lab rats whose lives don't really matter. Treating people like me humanely isn't a real high priority. Quality of life is not much part of the discussion. Keeping us alive a little longer is the only metric that seems to count and horrifying things get done in the name of it.
Not OP, but something just feels unnatural and unsettling about changing humans, and especially for changing yourself. It feels like the uncanny valley where it just doesn't feel right. I'm agnostic so it's not for a particularly religious reasons, and it's purely an emotional response for me that's not grounded in logic at all, so I also just describe it as squeamish for no reason.
Thank you for a honest response. I think this is an underexplored topic - how many of our broad social policies were shaped mostly on the basis of sufficiently broadly shared visceral feeling.
Should we base ultimately coercive society-wide regulatory framework on mere "squeamishness", appropriately rationalized, or should we instead embrace a non-discriminatory, laissez-faire approach in this domain?
Have you ever tried making changes to a large, incredibly complex codebase with zero comments? Sometimes the changes you make don't have the expected results.
Not the OP, but I think there are ethical concerns about genetic manipulation that do not amount to catastrophizing (e.g. 20th century eugenics is back!).
The problem with old school eugenics was that it wasn't consensual. Like forced sterilizations and abortions.
If gene tech can make future generations healthier and smarter with informed choice by the parents, those future generations will look back in horror at the barbaric pro death & disease lobby of the 2020s.
Forcing no, but those who could afford to do it would engineer superior children, and society would rather quickly devolve into a caste-type social system or worse.
It is inevitable that more people will be able to exert more control over their reproduction. I agree that this is likely to amplify the human tribalism that already causes so many problems. I still don't see how that inevitably leads to “20th century eugenics” nor why we should discourage or fear research.
You haven't had children, have you? They don't neatly fit into plans or develop to top down desires.Thinking the children will be superior is extreme arrogance even if it all works out and they are born immune to cancer. It puts too much faith in one advantage determining all.
Under that catastrophic social model the elite should have been descendants of the elite from before the Bronze Age collapse, and heavy cavalry should have maintained their combat effectiveness over the masses even during the Infantry Revolution.
Simple put people think "its a slippery slope". Every step closer to the evolution of a new technology makes people think about all the bad ways it could be used. Many past leaders in chemistry probably thought their advances would lead to the holocaust.
Its not bad to think/talk about the possible negative consequences of bringing a new technology into this world.
>Can you elaborate on what makes you squeamish about research?
Yes, I for one can't imagine why editing the genes of humans would cause alarm. After all, who wouldn't want to have children that are smarter, more docile, and healthier? Or, for other people's children, who have qualities that make you uncomfortable and can't be edited out, like dark skin or homosexuality, you have the option to edit their reproductive ability so that such qualities can come to an end humanely, without violence.
In the same way, I can't understand are those people against longevity research. Who wants to die? Not me, and certainly not those in power. You know, the people in power earned their positions, they are good at it, and mortality just means someone new and inexperienced will fumble around at the job. For continuity and safety, immortal leaders will do a better job and keep us safe. I don't see why anyone would consider this a bad thing.
The detective work going on with MS is fascinating. The blood work from the US military and what it has shown is really interesting - it’s not new news but was to me when I stumbled on it this year.
I have long suspected that viruses are the underlying cause of most of the diseases that have eluded us for the last 50 years. HIV gave us insights into a novel way that viruses can operate. It makes sense to think that there are more ways than we imagined.
E/B virus is suspect in Type 1 Diabetes too.
E/B Vaccination could eradicate a group of diseases potentially.
> The team at Great Ormond Street used a technology called base editing, which was invented only six years ago.
Fun fact: J M Barrie who wrote Peter Pan donated the copyright of both the book and play in perpetuity to Ormond Street children's hospital. It's been a significant source of funding for them over the years.
Thanks very much for posting this tidbit, very interesting!
After seeing your comment I did a little googling, and fascinating to me is that a special law was passed in the UK specifically to make one of the Peter Pan copyrights held by Great Ormandy Street never expire: https://www.plagiarismtoday.com/2015/10/21/peter-pan-and-the...
Taking away freedom is always about "saving children's lives". It's so common in fact that the instant one hears about "saving children's lives" we can be certain something very bad is going to happen.
I am not a big fan of copyright myself, but calling being unable to use someone else's story for yourself "taking away your freedom" is a bit too much.
I mean, no? There is absolutely no indication that that particular slippery slope is being slid down. Can you even point to anyone advocating for infinite copyright on all works on the basis of the Great Ormond Street example?
The proper solution would have been for the state to fund the hospital as needed, while freeing everyone else to create content based on Peter Pan if they wished so.
Civilization is made of unsavory compromises... Funding by taxation and committee spending, or by fetishisation of conspicuous consumption are also pretty bad and yet quite prevalent.
In this case the copyright seems to have expired essentially everywhere, but a special UK extension for Peter Pan allows for royalty collection for performances or adaptations of the play there in spite of the copyright's expiry.
> It's been a significant source of funding for them over the years.
TFA:
> Still, best estimates are that it’s not a hugely important part of the hospital’s budget.
which links to The Guardian [0]:
> The hospital has abided by Barrie's wishes expressed in his will and always kept the proceeds from Peter Pan a closely guarded secret. With copyright expiry looming, De Poortere is at pains to point out the revenues are "significant", but not as great as many suspect.
> "People who talk about millions a year have a strange idea of what a character is worth," she says. "On the whole, it's been significant but I wouldn't call it huge. I am on my own doing the Peter Pan administration, but I am part of a charity that has 100 people."
> If the newspapers of 1929 were right about proceeds of £2,000 a year, that would be £90,000 a year in today's money, according to the Office for National Statistics.
In response to the multiple people asking if base editing and CRISPR are the same, the answer is: not quite. Same idea, different enzyme.
The enzyme used by CRISPR is Cas9 (which is why you'll often see it referred to as CRISPR-Cas9), which acts like a pair of molecular scissors and cuts strands of DNA at a specific point in the genome.
Base editing is a newer technique and utilizes an enzyme called deaminase, which can change the chemical structure of a base pair without doing the "cutting" part. There are specific deaminases based you use depending on which base pair you're trying to modify.
To oversimplify a bit: base editing is a more precise version of CRISPR, which is less likely to cause more changes than actually desired, as is a problem with CRISPR. That doesn't make CRISPR obsolete though, because if your goals actually is to remove entire portions of a genome, CRISPR is a more effective technique. Regardless, there is a lot of overlap between the methods: both usually use adenoviruses as the delivery vehicle, you can use both enzymes in tandem, etc.
I took it to mean doing an in place patch / removal of code (CRISPR) vs doing a more general refactor that results in cleaner code (base pair editing).
Great to see medical techniques slowly starting to resemble engineering design:
1. Donor T-cells added with receptors to destroy the recipient's cancerous T-cells.
2. Donor T-cells' markers removed to prevent them from attacking each other.
3. Donor T-cells' existing receptors removed to prevent them from attacking the recipient's other cells (since the donor T-cell will see them as 'foreign').
4. Donor T-cells altered to resist chemotherapy.
It seems each of these changes were achieved by editing specific base pairs to break the relevant gene, except perhaps for (1)
To even have something like this actually respond, in the way they planned it, is a marvel. I know they have worked with T-Cells before, but this is putting it to precise use.
Sadly, cancer is vicious. It only takes one rogue cell to keep building, about 2-years for it to surface again. Luckily, this process seems repeatable.
I can't imagine the euphoria that family feels right now.
In heroic medical science history fiction it's a bit of a trope to write "the operation was a success but the patient died"
I sincerely hope she survives at least 5 years, and significantly more. If she gets only a single year of treatment -free high quality life outside of respite care, that would be a bitter pill outcome but might still be held a success. Sometimes giving people a year of quality life at end-of-life is a good outcome.
The article as written is light on for data and time info. She's had at least 3 post treatment checks one of which was 3 months, the other 2 not stated but 6monthly might not be unusual.
Recurrences in blood borne diseases are not unheard of. She had some signs of recurrence at 3 months but it went away. Maybe fragment traces? Non viable remainders flushing out of the marrow? (I don't know if this is even plausible. I'm not in medicine)
It's great news, but so was Christian Barnard's first transplant. In modern terms the survival wasn't very long. (200 days in the first 4)
A better outcome would be like Steptoe's work on assisted conception and birth. (IVF) and I certainly hope she and future patients have that.
The fact that she developed cancer in the first place means that she is more likely to continue developing cancer, via a plethora of mechanisms. Cancer is always just a matter of time, for all of us. Subtle mutations accelerate the timeline, and they accumulate over time.
A) We have already largely solved infant mortality in the developed world, and are making progress on the issue globally. Societies seem to adapt over time by decreasing birthrates to compensate [0]. In fact, in most of the developed world, birth rates are below replacement rate.
B) Despite all of the medical advances we have made, we have hardly put a dent in old age. Medical science has made great progress in stopping people from dieing young, which has pushed up the average lifespan. But a 100 year old today is still likely to die within a few years even with the most advanced medical treatment.
Having said that, at some point, we are likely to start making breakthroughs in maximum lifespan, which could very quickly put us back into rapid population growth. Eventually, we would probably see society adjust to the point that having any children is no longer expected of a typical human, but that could be a painful transition depending on how quickly we we go from a lifespan of ~100 to unbounded.
[0] Although it is not clear if this is directly related to infant mortality, or a response to broader economic changes,
Where I live in the EU, people get old; 100 here is not so rare and there are many 90+. Most of them are healthy; walking the mountain for shopping daily and going to the bar for a shot or two in the morning. 0 of them want to live longer. Not because physical but because the mental agony of yet more time, see more horror. Think people who don’t have this issue underestimate how bored and ‘done’ most older people are. My grandparents (all 4) made close to 100 in good health and wanted out since 70. When you are young you want to live forever, when older I don’t think many people do.
I seem to remember reading that there is some limit to the age of the brain, separate from the mortality of the body. So even if you were to not die from any cause you’d be pretty much done after 150-170 years.
Setting aside any specific mechanism, the overall point still seems plausible. Assuming we can prevent the primary causes of aging (e.g. telomer shortening), we will likely start to find other parts of human biology that start to fail, either because of additional aging mechanisms, or just limitations of the body's self-repair mechanisms.
Human biology is incredibly complex, and there has been no evolutionary pressure for it to sustain itself beyond its natural biological clock. These problems are probably solvable eventually, but not until we live long enough to see them. Which problems means we will have at least a few generations of people die after the initial old-age cure.
Oh, absolutely. Once we "fix" dying of the normal reasons, I'm sure we'll start dying of all sorts of weird things. It just seems suspect to put a number like 150-170 on anything right now.
Past projections of human population growth were generally overblown, because nobody expected e.g. China's population to (officially) peak this year.
Meanwhile world population growth peaked in the late 60s and has been in decline ever since. The 21st century will likely see the global population reaching its limit at less than 11bln.
That's still a lot, but not nearly as much as 16bln.
If 8 or more story-falls are fatal...
8-Town limits hi-rises to 8-Stories
20-Town limits hi-rises to 20-Stories
Which Town and what Height of Buildings would you rather be a tenant of?
...I don't know about Human Population, but I know that "more code" is certainly not "better code", just as "more sub-systems" does not mean "more quality of code"
Stop embarrassing yourself. You started by talking about population growth in a post about a kid's cancer, and now you are continuing with making stupid analogies that barely make sense.
Getting rid of cancer will alleviate a huge source of suffering and despair and it will do nothing of consequence to the population. Even if it did we should not care.
I disagree with the notion of benevolent DOCTORS "Killing people off with cancer".
Was the patient body full of DOW Chemical or DDT or Big Gulps or Pharmaceuticals[0]? industrial Society and Old Age are what "kill people by means of cancer"
[0] USA being the only country I know of to allow public Advertisement of Controlled medicines
I'm not sure what you're talking about. Choosing to not cure cancer is killing people off with cancer. The little girl in the article had leukemia. It sounds like you would prefer to let children die of cancer because of your weariness of population growth.
I thought the same thing initially, then I realized that rare forms of cancer are... rare. Not that many people die from them to cause a significant increase (or lack of decrease) in the population.
You arent anti-natalist, so your view is that people should be born and die from preventable disease or starvation ("medicine and agriculture saved them")? Isnt anti-natalism preferable to that?
The idea that we should not progress because we might have more populace is repulsive. A simple reversal of the idea is insightful. Should we minimalize the human population? Seems absurd.
I propose the DEFAULT be no growth or no decay. Of course it's natural for animals to spread and be fertile. Of course. But we already conquered and inhabited everywhere with land and water.
Obviously Technology will advance or stall (or collapse) regardless of my small whims -\o/-
Don't worry, there's no shortage of ways to prevent getting to 16 or 32 billion people.
First among those is that very few countries have birth rates that exceed replacement rates! And the others are on a very strong trend to follow over the next 10-20 years.
>"What happens when there are 16 or 32 Billion people?"
Not sure about other part but in the western world birth rate is gong down and is not enough to offset decrease of the population. Having people leave longer will not change it in any way. If we ever grow to this number it will not be because of curing cancer.
>"Even the idea that "GDP should rise 2% every year" makes me nauseous."
You think anybody responsible for GDP growth gives a hoot about what "makes you nauseous"?
I believe the world population is expected to peak this century, not because of people dying or starving at higher rates, but because when countries become more developed, their birth rates plummet. I don’t think there’s any reason to think curing all diseases and ensuring no one ever starved again would change that.
This looks pretty great, hopefully a huge step forward for the medical industry and finding cures for cancer. Unfortunately don't know too much about it; wish there was something that allowed me to learn about medical problems easily, without needing to understand the jargon
We are at the "assembly" stage of genetic tinkering. Manually changing over the 'machine code' of the cell, the linear sequence of physically bound instructions. I wonder what the future might bring in terms of higher level programming of cells.
But we can’t just start writing new code, the system is already running millions of lines of undocumented code. Also it can’t be halted. Also, there are no backups.
> “Base editing allows scientists to zoom to a precise part of the genetic code and then alter the molecular structure of just one base, converting it into another and changing the genetic instructions.”
It's explained in the article that the second transplant is necessary to replace the white blood cells which should have been fully wiped out by the first transplant of modified cells.
But her body ought to start manufacturing her original T-cells at some point, right? Or are the stem cells producing them cancerous too? Doesn't say that in the article, and it doesn't seem like a reasonable inference, so there's a bit of detail missing there.
Even if the self-manufactured cells were healthy, the patient is vulnerable to infection with a compromised immune system, and adding more white blood cells reduces that vulnerability.
Tangent: before undergoing some kinds of chemotherapy, patients are injected with drugs that stimulate white-blood-cell production. Most of your body's WBC production happens in the long bones of your body, and the sudden burst of "factory" activity makes your legs ache, the way growing pains hurt in children.
Usual tactic is chemo + bone marrow replacement and hope new tcells mop up cancer cells.
Didn't quite work so they started with new marrow and made some changes to a supply of tcells they flooded her with. Once they couldn't find cancer they put the marrow on.
Could be. It would be nice if you could maintain the modification though because then you don't have to worry about some cancerous cells surviving the first wave.
The article states “The first base edit disabled the T-cells targeting mechanism so they would not assault Alyssa's body”. Not an expert but I presume the ‘targeting mechanism’ is the T-cell receptor, whose activation is necessary (but not sufficient) to trigger immune response.
I'm talking well beyond my competency, but short of one of those T-cells spontaneously becoming cancerous itself, I don't see how. T-cells do not duplicate themselves. Instead, new T-cells are creates in bone marrow. Since they are not editing the genes in the bone marrow, there is no mechanism within the body to create new cells using the modified DNA.
No. The only DNA that gets passed to offspring is what is in the sperm/eggs. Collectively these are referred to as the "germline". Research into germline editing is illegal in the US, and frowned upon internationally.
The most famous case is from 2018, when Chinese researcher He Jiankui created the first genome-edited babies. Using CRISPR to give the babies resistance to HIV-1. [0]. This change will propagate to the decedents of the subjects [1]. The Chinese Academy of Science, along with the global medical community, denounced this experiment; and He Jiankui was arrested.
[0] The necessary mutation, while rare in humans, is naturally occurring, and generally thought to confer HIV-1 resistance.
[1] Subject to normal genetics that arrises from sexual reproduction, where a given parent is responsible for only half of a child's genes.
> Attempting to cure Lieutenant Reginald Barclay of his Urodelan flu, Dr. Crusher used synthetic T-cells to activate the gene that would fight off the infection. However, an anomaly in Barclay's genetic structure caused the T-cell to mutate and activate all of Barclay's dormant genes, including his introns. The T-cells then became airborne, spreading throughout the ship like a virus and causing the crew to "de-evolve."
This is a wonderful outcome and amazing science and all involved are to be commended for saving this girl's life.
However would it not make more sense to try and prevent the disease in society, since there is a viral component to it? It is a retro-virus and was first discovered in 1977. Is there even research on a vaccine? We have one for cervical cancer. (Hit's a nerve since I have a child with Lymphoma)
Tangential, but I wonder how long it'll be before we start combining these recent advances in AI with a dataset like Medikanren and identifying really custom treatments.
Noone is forcing you to accept anything. If else the girl and parents had to make that decision and accept the risks. They would probably had to sign a legal document as well. Maybe you need to chill out of the conspiracy mindset.
I'm not sure it counts as "revolutionary" if it has helped only 1 person, and maybe as many as 10-12 per year. It's really the opposite of revolutionary, though I am very happy for this family and their daughter.
This is like the ultimate form of Unimpressed Internet Guy.
The article even talks about other trials also underway, so the parent comment doesn't even make any sense:
> Dr Robert Chiesa, from the bone-marrow transplant department at Great Ormond Street Hospital, said: "It is extremely exciting. Obviously, this is a new field in medicine and it's fascinating that we can redirect the immune system to fight cancer."
> The technology, though, only scratches the surface of what base editing could achieve.
> Dr David Liu, one of the inventors of base editing at the Broad Institute, told me it was "a bit surreal" that people were being treated just six years after the technology was invented.
> In Alyssa's therapy, each of the base edits involved breaking a section of genetic code so it no longer worked. But there are more nuanced applications where instead of switching an instruction off you can fix a defective one. Sickle-cell anaemia, for example, is caused by just one base change that could be corrected.
> So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.
Actually, it's the other way around. The treatment described is an adaption of CAR-T (Chimeric Antigen Receptor T-cell) therapy, which was approved for use in the United States in 2017, to the patient's specific form of leukemia. It's a challenging adaptation because the cancer cells being targeted are the same type of T-cells that are used in CAR-T therapy, thus requiring the use of donor T-cells, and a number of modifications that are unnecessary when CAR-T is used for other cancers. Base editing makes that easier than earlier forms of CRISPR. But base editing isn't the therapy here, CAR-T is, and while CAR-T is certainly relatively, this patient is not the first to receive, just the first with her particular leukiemia.
No, the parent comment is totally fine. The technique is indeed new and yes will be applied in the treatment of other diseases in the future. Don’t know why you went to open with a contrarian tone.
My point was simply that this is an adaptation of an existing therapeutic tool (CAR-T) to a particular cancer, not something completely novel. It's a leaf on a tree of therapies, not a new tree per se.
I'm squeamish about genetic research, but this looks really good to me. I hope she recovers fully and there are no further complications, though I imagine it's too early to guess what her prognosis might be.
So there are already trials of base editing under way in sickle-cell disease, as well as high cholesterol that runs in families and the blood disorder beta-thalassemia.
That's a big deal for people with those genetic disorders.