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Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish (technologyreview.com)
318 points by phr4ts 354 days ago | hide | past | web | favorite | 134 comments



> Although the cases drew wide media attention in Britain, some researchers said that because the London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids.

Could someone explain what the process for proving it would be in this case? I presume some kind of animal testing?

Seems pretty immoral to give someone a wildcard treatment and not back it up with something known to work. Not disparaging this treatment but given we don't know if it works yet (and how aggressive cancer can be) it seems totally fair to administer it alongside chemo.


Standard approach would likely be to choose a population of patients, randomize them between "chemo" and "chemo+immune" treatment groups, and then test for a significant difference in the metric of choice such as response rate or progression-free survival. The graphical analysis of these data is often done using Kaplan-Meier curves (https://en.wikipedia.org/wiki/Kaplan%E2%80%93Meier_estimator)


So, can't simple take some (or all) other children who are going through "just chemo" in any other hospital, or even in the same hospital? I think there are plenty of children in the world that are doing (suffering?) chemo everyday.


You'd want to eliminate as many potential confounding variables as possible. If they took the control group from another hospital, maybe that other hospital does something that improves or hurts their chances. Even if you pull from the same hospital, just being in the study might change the treatment they get in subtle ways.

There's also the issue of blinding. If the control group is some outside group, then you know that everyone in the study group is receiving the new treatment. It would be much better if everyone involved didn't know whether any given patient was getting the new treatment or not until after all the results were in. For that, you need to recruit patients into the study, then randomly assign them to the new treatment or the conventional sort.


With something like cancer which has a wide geographical coverage, and more or less standardised treatments, couldn't you get a good result from including everyone in hospital X in a treatment and comparing to the general public?


No, cancer does not have standardized treatments. The remission rates vary greatly depending on the hospital and even on the doctor.


Huh...? There are standard treatment protocols.


Speaking from very recent experience, some things are obviously standard, like "take these pills twice a day" but cancer often comes with lots of ancillary issues, and most treatments come with side effects.

There was a lot of variance in how doctors and nurses treated the side effects, how they spoke to the patient, what other resources they provided, the quality of bedside manner, their attentiveness to lab results and patient health over time, whether they consistently washed their hands, etc.

People don't really die "of cancer" they die of things like blood loss, toxic build up from renal failure, blood clots, or infections acquired in the hospital that attack a weakened immune system, to name a few.


Sure. Anyone with cancer should go to a high volume academic and designated cancer center. The hospital should also rate high in us news and reports and score top marks in nurses, safety and cleanliness. But you can't deviate from FDA guidelines except in very exceptional cases.


What guidelines?

Doctors have tremendous latitude in how they treat patients and, as far as I know, there are no "official" guidelines for most diseases (very communicable diseases might be one major exception).

There are guidelines for developing, manufacturing, advertising, and selling drugs. However, doctors can use them off-label. From the FDA (here: http://www.fda.gov/ForPatients/Other/OffLabel/default.htm ) "From the FDA perspective, once the FDA approves a drug, healthcare providers generally may prescribe the drug for an unapproved use when they judge that it is medically appropriate for their patient. "


That was my experience of variability for a family member treated within a particular high volume academic hospital and designated cancer center.

But my point is in response to the original parent comment. If you give treatment Y at one top hospital and your control group is everyone getting treatment X at all the other top hospitals, you still have basically anecdotal data.

If I'm very sick and the standard treatments aren't working, would I want to try a protocol that was anecdotally very successful in some clinic? Absolutely. Just because it's not scientifically proven to work doesn't mean it doesn't work.


They do run multi center clinical trials... but yes if your current treatment doesn't work or is likely out of date then you should seek another treatment option (usually a phase 1 or 2 trial)


The point still stands in practice- it's an interesting sociology of science topic. There are often unwritten rules and protocols that have a huge impact.


You can get A result, but not a good one.

You want to eliminate anything and everything that can (in)directly effect the outcome of treatment in a study. Ideally, the only difference at all would be the variable you are testing, but real life doesn't make things that easy.


You'd also have a host of confounding factors outside the treatment. Trace minerals in local water, different pollution, different seasons, etc...


You don't need blinding for cancer trials


No, they can't. To do so would introduce confounding variables in the inevitably unique way they treat their subjects, even if it's just knowing you're part of the experiments.

For maximum assurance of conclusions, such studies need to be double-blind: neither the subjects nor the researches who interact with them are to know who's taking just chemo and who's taking the new treatment.


You need a larger sample size than 2 to differentiate between the odds of "success due to just chemo" vs "success due to chemo + x treatment."


Its not immoral.

Right now I'm in a cancer treatment study. (my particular study is a "shotgun" type of study. They have a drug that they know works on certain types of cancer, but they have a guess that it might work on my weird/rare type.)

The previous studies on this drug I'm on, were done on patients they had exhausted the conventional treatments, chemo, radiation etc. And they were put on this drug as a last chance after the conventional treatments had failed. I'm lucky in that I'm getting this drug as a first line treatment.

In this case, with these kids, it sounds like they were given the traditional chemo, and when that show results, they tried this. As a result, they don't know if the chemo caused the remission or it was the T-Cells, or the combination.


Give both the normal treatment and (new thing), and compare it to just the normal treatment and see which group has better outcomes. For serious issues you can stop the trial early and give it to everyone if it's shown to be a dramatic improvement.


The full paper states that they did this prior to even phase I of clinical trials:

http://stm.sciencemag.org/content/9/374/eaaj2013.full

Therapy was provided for two infants under the UK. special license arrangements after institutional ethical reviews.

Named-patient, open-label, nonblinded, compassionate therapy was undertaken in two infants ahead of planned phase 1 clinical trials.

I guess the proceedings of the ethical reviews would be quite interesting.


Compassionate therapy basically means "it seemed super likely that these patients are on track to die, so even if this therapy is awful for them, you can't make the situation much worse, and we should take a shot at curing them."


> Could someone explain what the process for proving it would be in this case?

You can have a look at NICE who do the cost:benefit approvals for meds in England.

Here's what they said about Abraxane, a medication for pancreatic cancer. (TL:DR it's not good enough to replace current first treatment, and it has too many side effects to replace current second level treatment. I don't think cost, a bit over £5000 for the average patient, was a factor.)

https://www.nice.org.uk/guidance/ta360

If you walk through the complete document you can see what they think is important; what the manufacturers think is important, and what evidence is used.

It's written for a specific audience, and it's a bit medical, but it's (IMO) very readable.


Animal testing is used for some initial toxicology and indicators that a treatment is worth testing in humans but you can't use animals to "prove" a treatment is effective for people.

Once a treatment is through animal safety trials, they will give it to patients with very advanced stages of cancer, who are considered unlikely to survive very long. This is both to minimize harm, and make it easier and cheaper to tell whether a treatment worked.


> Seems pretty immoral to give someone a wildcard treatment and not back it up with something known to work.

I think they're giving the treatment to patients for whom pretty much all options have already been pursued and they're still terminally ill. Of course that still doesn't "justify" necessarily a wildcard treatment completely, but I guess one last wildcard attempt might be better than nothing.


It depends. If the usual treatment for a terminal disease has big side effects and is unlikely to pay-off it might be more ethical to use a promising treatment whose effects are uncertain.

However, I expect a lot is known about chemo-only survival rates so it may not even be a legit criticism as you can compare the combination survival rate to the baseline. That said, n=2 is too small to draw conclusions.


Happy babies! As a point of interest, this type of engineered T-cell treatment has its roots in research at the Weizmann Institute in Tel Aviv. They demonstrated the use of CARs modified T-cells in curing leukemic disease in rats and mice several years ago.

Their research was expanded to human trials at Univ. of Penn. where 27 of 29 patients with incurable leukemia and most of whom had a prognosis of death within a few months went into remission and showed no sign of the disease.

This modality may work with other forms of cancer; engineered T-cells that can enter every capillary in the body could potentially wipe out entire colonies of cancer cells. The potential is enormous, as are the challenges; cancers can be very difficult to differentiate from healthy tissue.

[1]http://www.timesofisrael.com/breakthrough-cancer-cure-has-de...


Thanks for the extra info - very interesting. I'm always curious about whether these trials were followed later on as well. I mean those 27 of 29 patients, what is their state now? Still healthy? And how long ago did this trial occur?


> “We estimate the cost to manufacture a dose would be about $4,000,” she says. That’s compared to a cost of around $50,000 to alter a patient’s cells and return them.

> Either type of treatment is likely to cost insurers half a million dollars or more if they reach the market.

Can someone more familiar with the pharmaceutical industry explain this?


Because it's not about how expensive it is to actually make the drug. To use a software analogy, Oracle doesn't price their database software by what it costs in bandwidth for you to come to their site and download the latest version. Or in the days of shrink-wrapped software, the cost of software was far from the cost of the materials (cardboard and discs).

Books similarly aren't priced based on the cost of printing and distribution.

The cost is directly related to recouping R&D costs as well as the clinical trials. In addition, with these therapies specifically, you actually have pay lots of highly trained people to administer them in the first place.


In some cases (and you left out marketing, which often exceeds the R&D costs). In other cases, the cost is directly related to 10% less than other treatments. Or to whatever the CEO thinks he can screw out of patients. See: Shkreli; Epipen.


I disagree with your statement "in some cases". It's "most cases" and where it is not, those are the ones that get the attention and screaming headlines. Shrekli is in the news precisely because this behavior is not normal. But generally, much like other goods, drugs are priced based on how much the consumer values them. I will also add, that for really big ticket things like oncology drugs, I believe every company offers payment assistance programs which often have very high income cutoffs that provide co-pay help or outright free drugs when insurance doesn't cover it.

Marketing is a red-herring. Sales and marketing should bring in more money than you spend on them or they're not worth doing. They exist to drive money This makes the company more profitable in the near term than R&D expenditure which can take decades to materialize.


I can explain the first quote.

In a universal donor scenario, the T-cells are engineered, error corrected and tested once. From there, they can be multiplied pretty cheaply. Most of the cost is in the initial engineering.

In a patient cell scenario, each individual patient's cells must be engineered, tested and delivered. This is obviously much more expensive.

A very crude computer science analogy would be the difference between scaling up a load balanced application relative to adding a new server from bare metal without automated scripts.

Why the treatment would cost half a millions dollars at the market is outside of my purview, so I'm eager to get an explanation as well.


Mostly profit to pay back the investors for billions of dollars in research and development, and baked in liability insurance.

Some of it also goes to pay for the R&D of other drugs that never make it to market.


This is propaganda. Spending on research and development at private pharmaceutical companies is dramatically eclipsed by marketing and sales spend.

https://www.google.com/amp/s/www.washingtonpost.com/amphtml/...


Yes, but sales and marketing has a positive ROI. If you spend $100M on sales and marketing, you'll get $150M back (for example). Otherwise, why would you do it?

So if pharma companies cut out all of their sales and marketing, they'd have even less revenue than they do today. And that would lower R&D spend.


Why do drugs need marketing?


Because it's one of the key ways that physicians get new information about drugs. Not every doctor has the time or the proclivity to do research on every newly launched drug. Having a rep come in and answer questions goes a long way to getting doctors up to speed on the latest information.


This... I feel that we (US) should go back a bit on this, and restrict all advertising not in specific magazines/journals that target doctors, and restrict office visits of pharma reps to dr offices.


Why does anything need marketing?


The treatment also requires a bone marrow transplant (which requires destroying the patient's immune system). It's a lot of medical care.


Are you sure? I don't find that claim in the articles or report. The bone marrow procedures seem performed in the context of the chemotherapy treatment - which failed in combination. They dont seem to indicate how necessary or unnecessary that combination was to this treatments success here.


The "Subject 1" and "Subject 2" sections of the paper both speak of persistent malignancy in bone marrow.


I have not the experience to read this report with any confidence, but your comments are so far too brief to convince me that you have. Fig 2 in the report includes a chart which appears to indicate a bone marrow reading clearing in response to application of allo-SCT. How do you ascertain that this treatment does not in itself improve malignancy in bone marrow?


These “off-the-shelf” CAR T cells were then used to treat two infants with relapsed refractory acute lymphocytic leukemia and bridge them to allogeneic stem cell transplantation.

I don't have much knowledge of the subject but a stem cell/bone marrow transplant seems to be a common treatment in aggressive cases of leukemia.


Private insurers are too fragmented and small to negotiate a reasonable price for treatment. They lack the leverage that something like a national single-payer system would have.


The largest insurer in the US covers more people than the NHS. The second and third largest insurers rival other continental health services.


Please offer an alternative rationale for why they aren't as effective. I'm trying to understand why US healthcare is so broken.


It's easy. The UK can say "no" to paying for certain drugs. They take some political flak for it, but overall they've been able to keep spending down.

Even mention not paying for something in the US and holy hell happens. Even Medicare and Medicaid can't say no for the most part.

If American's want cheaper healthcare they are going to have to get used to having less of it and not get all the fancy new technology right away.


>It's easy.

No, clearly it's not.

I work for a startup that's delivering health services. Insurance companies have a very high bar for what they cover.

They are very conservative about covering new therapies. There's a lot of snake oil in medicine (acupuncture, homeopathy) and they don't want to pay for what doesn't keep their members healthy.


It's not easy to execute, but it's easy to figure out one of the key drivers of increased costs in the US.

Compare drug coverage in the US vs. Canada or the UK. New drugs are often covered immediately post-FDA approval in the US. Heck, look at Exondys 51 for Duchenne's. The FDA barely approved it and pretty much every US insurer is paying for it. That doesn't happen in other countries.

A great example is the diabetes drug called Jardiance. It has better efficacy than older drugs and every US insurer covers it. It's not covered in many Canadian provinces.


Insurance companies only pay for what they consider usual and customary.


If by that you mean they can't dictate the price of drugs, sure. But that has more to do with not being an arm of the state than being "fragmented and small".


The CHS and NHS are able to negotiate drug prices even though they can't coerce US companies with force.


"You give us this price or we don't use your drug" isn't coercion?


The drug company can walk away from the negotiation. Either party can say no. That's the difference between coercion and negotiation.


The difference is governments don't have competition. If you play hardball with a drug company in the US, one of the reasons they can walk away is there are other insurance companys to which they can sell their drugs.

And insurance companies can't throw drug company officers in jail.


> The difference is governments don't have competition.

There are many governments in the world. If one government plays hardball with a drug company, drug company can go find a different government, or negotiate with private insurers in that same country. Yes, private insurers exist even in countries that provide basic healthcare to all their citizens. [0]

> And insurance companies can't throw drug company officers in jail.

Show me where this has happened. This scaremongering and black-ops has nothing to do with the practice of negotiating price. There are many people that wish Martin Shkreli went to jail for gouging AIDS patients, but that's not happening.

0. http://thehealthcareblog.com/blog/2012/01/16/the-awkward-wor...


The cost of $4,000 doesn't take into account R&D costs, which must be recuperated in the drug's price.


It also doesn't take into account marketing costs, which also must be recuperated in the drug's price, and are much bigger then the R&D costs...


Simply not true.

Most drugs never make it past phase 3 trials, so fail in R&D before marketing.

Gilead didn't need a lot of marketing for its HepC cure to become a blockbuster and propel them to be the 6th biggest pharma.

DTC is also illegal in all countries except the US and NZ, so it's not even possible to spend that much. Sunshine Act in various countries, China putting execs in jail, etc. The world has moved on.


Nobody invests in marketing unless the ROI is >100%. The marketing activity funds itself unless somebody screwed up.


Yes, of course. However, consider this dillema.

You can sell 1 million pills, at a cost of $5 each. That's a 5 million profit. Or, you could spend 80 million in marketing, and sell 2 million pills, at the cost of $43 each. That's a 6 million profit.

This is absolutely not beneficial to the first million customers.

Also, consider that the United States is the only country in the world that allows direct-to-customer medical marketing... Which is one of the reasons why the cost of healthcare is so damn high.


This is absolutely not beneficial to the first million customers.

Drug companies don't increase the price to pay for marketing. Prices are set based on what the market can bear. A small drug company that decides not to spend a lot on marketing is going to charge the same as a big company that does spend on marketing.

How the math works is as follows:

$5/pill; 1 million pills sold with no marketing = $5M profit

$5/pill; 2 million pills sold with $3M in marketing spend = $7M in profit (66% ROI on marketing spend).


You think that is also true for leukemia drugs/treatments?


Why do they need marketing?


One dose costs $4,000. A full treatment is more than one dose. That sounds like over 100 doses if all you do is pop some pills. If I had to guess it probably isn't 100 doses but you have to do other things like stay in a hospital during the course of treatment, other tests, or something else that adds to the price tag.

AKA the price tag on a service is usually much more than the cost of goods sold.


Does anyone here know which companies are looking for software engineers to help with this kind of stuff?


There are lots of companies in the immunotherapy space. Don't limit yourself to industry, though - lots of academic labs working on this that are in desperate need of good coders and data scientists.


Novartis, mentioned in the article would be one. Companies that partner with those like Novartis would be some more. The needs would be both "data-ish," running databases and studies, as well as medical device control software. Lots of breadth and depth, start looking around.

Follow press releases from company to mentioned company would be one way to start looking.

Another way would be to ask the FDA: https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfPMN/pmn...

Searching in that form for "immunology" in the Panel field yields 500 results, and lots of companies.

That link also points to other databases.


Check out http://www.parkerici.org/. Their head offices are in San Francisco, but they work with a lot of labs across the US.


I just got an email from a colleague that the Roswell Park Cancer Institute is looking for software developers and cloud developers. They're a good group doing good things.


>And I guarantee you even if things were equal, which they are not, you would want your own stuff, not someone else’s cells.

This sounds like wishful thinking from VC who just put a lot of money into the Bespoke solution. The idea of immunotherapy is that your injecting cells that can attack the cancer. What does it matter it it's your own cells especially when the cost is an order of magnitude bigger.


Yep. In medicine "other people's stuff" is used quite often...blood transfusions...sperm donors...organ transplants...etc.


'Although the cases drew wide media attention in Britain, some researchers said that because the London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis. “It would be great if it works, but that just hasn’t been shown yet.”'

So the title is basically click-bait, as the treatment is unconfirmed.


There's no way they don't also give them chemo. Doing otherwise would be hugely unethical. Just need more data points before we would know more...


True, but it could still be the case the combination of traditional chemo and the experimental therapy would work on other patients. Obviously it'd be preferable if it worked without chemo (given the side effects), but the cure is the goal.


'says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis.'

I don't know if I would trust the opinion of somebody that 'collaborates' with one of the companies that has already lost millions trying a different approach and has the most to lose if this actually works. It even points this out earlier in the article - 'The ready-made approach could pose a challenge to companies including Juno Therapeutics and Novartis, each of which has spent tens of millions of dollars pioneering treatments that require collecting a patient’s own blood cells, engineering them, and then re-infusing them.'


Point taken, but the title doesn't actually say the universal immune cells cured the disease.


Most clickbait isn't outright false, just highly suggestive in a misleading way.


There is some scrutiny.

Abstract: The London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis. “It would be great if it works, but that just hasn’t been shown yet.”


The odd thing: does standard chemo ever totally cure anybody? If not, then what did cure them, if not the new treatment?


Yes, chemotherapy can and has cured cancer.


Not like this as I understand it - remission isn't the same thing as "zero cancer cells detectable" - total eradication of the disease.


It can completely cure the cancer

https://en.wikipedia.org/wiki/Chemotherapy#Efficacy


"Rights to the London treatment were sold to the biotech company Cellectis, and the treatment is now being further developed by the drug companies Servier and Pfizer."

And that's when it will get sunk into an abyss of bureaucratic processes and this treatment probably won't see sunlight for another ten years.


In fairness, that is mostly the fault of the world's various regulatory agencies. I mean, I understand that it's easy, when asked "How many people should die yearly of insufficiently tested medicine?" to answer "zero!", but that's kind of how medicine became so stale.


It's hard to cure cancer definitely because cancer cells will always be trying to out-evolve any treatment. Anything that quickly kills 99% of cells, unfortunately, lets them select for survival traits really fast.


However, given that cancer cells do not evolve between individuals (as opposed to bacteria and virii), if we are to find a efficient immunological treatment it's going to stay efficient. If anything, we will evolve for our cancers to respond better to it.


Well, until you deal with super cancer.

https://en.wikipedia.org/wiki/Devil_facial_tumour_disease


Wow, that's an actual transmissible cancer, not a transmissible trigger.


Somehow it is strange that cancer cells do not try to propage to other organisms, given they try hard to invade their host? Is there any known reason for that (absence of) behavior?


See Retric's comment and link. Short answer is, can.

A more common transmission mechanism is itself a virus (https://en.wikipedia.org/wiki/Oncovirus) .

But, remember unlike bacteria or a virus, cancer isn't typically "invading" and you aren't "hosting". It's a malfunction of your own cells resulting in unregulated growth. For the most part it isn't really something you "get", so much as a failure mode of you as a system.

We do use the terminology "invasive" but not because it came from outside the body, rather that it is leaving one organ or tissue and entering another.


Well, it's because it's not "cancer cells" as something different then the host, it's just "here's some cells that, for whatever reason, decided to start growing and multiplying indefinitely, rather then being restricted by what their programming says". Comparatively, cancer is far "less threat" then most diseases, except for the fact that your immune system treats it like part of your body. Thanks to that "leg up", it can do a lot of damage. If your cancer got into another person, their immune system would treat it as foreign (modulo transplant factors and such), and attack and destroy it.



I imagine a big part of it is that they kill the host pretty quickly.


Although they may evolve from parent to child, so what works for one generation may be less effective on following ones.


Only if dormant cancer cells get passed from cured parent to child, which generally should not happen.


Children saved from deadly illness by using T-cells(virus?)... I think we all know where this is going...


"The ready-made approach could pose a challenge to companies including Juno Therapeutics and Novartis, each of which has spent tens of millions of dollars pioneering treatments that require collecting a patient’s own blood cells, engineering them, and then re-infusing them."

Is author serious with this statement? Two kids lives were saved with a potentially inexpensive technique and the author think's this point is relevant? If 500 companies go bankrupt because cancer is cured, that is a monstrous win.


> Is author serious with this statement?

Whether or not this author is serious, there are many people who are deadly serious about this sort of thing. It's one of the main reasons that, for example, marijuana has been kept illegal despite the overwhelming evidence that it is less dangerous than either alcohol or cigarettes: legalizing pot is bad for established business interests. It's the reason that credit card transactions cost 2-3% when the technology to process payments for an order of magnitude less has existed for years. It's the reason "low fat" became a thing when sugar is actually much worse. It's reason you can't buy an open-source mobile phone. I could go on and on.


Farther back in the history of the demonization of marijuana you'll find it's connected to an argument used by fundamental Christians, Evangelical Christians, related to demons, spirits and the spirit world. Now combine that with fundamental republican right.


Prohibition may have been pushed using a Christian narrative but only because it made it an appealing message for the wider population that wasn't going to just take "look, we can sell way more paper and cigarettes now" as justification.


And that technique continues to be effective. If anything, it has become more viable over the years. I honestly don't understand how anyone can look at the prosperity gospel (https://en.wikipedia.org/wiki/Prosperity_theology) and not see the blatant cynicism behind it.


Don't forget racism - reefer madness was invented to further joint stigmatize minorities and MJ (pun intended).


Though none of those things are life and death so not sure relevant.


They certainly are matters of life and death. People die from obesity, diabetes, and cancer caused by sugar and cigarettes. And medical marijuana is a matter of life and death for some ALS and cancer patients who can't tolerate chemotherapy otherwise. For example:

http://www.chicagonow.com/soapbox-momma/2016/10/illinois-man...


I don't see it as the author complaining about the danger to the companies but merely stating it. It's not only just OK, but also necessary to throw light on this.


Agreed, but it's still the 3rd of 16 paragraphs. I think it's far the most relevant item.


Think of it as a warning.


I agree, especially considering that the author also mentions relations to expose potential bias or conflict of interest (italics are mine for emphasis):

>Although the cases drew wide media attention in Britain, some researchers said that because the London team also gave the children standard chemotherapy, they failed to show the cell treatment actually cured the kids. “There is a hint of efficacy but no proof,” says Stephan Grupp, director of cancer immunotherapy at the Children’s Hospital of Philadelphia, who collaborates with Novartis.


Yes, the author is serious that investors worry about losing billions of dollars. If your house or savings became worthless overnight, I suspect you'd be worried too.

It's a statement of fact. If UCARTs come to prominence on the market, Kite & Juno no longer appear to be the only game in town (or even the only CAR-T game in town!). Toss in some grade IV/V events in their clinical trials, and an investor in these companies might have reason to worry.

Individuals and societies can have conflicting priorities. Sometimes the same individual may be conflicted (plenty of oncologist friends invest in various companies, and are distressed if a competing company's drug blows their holdings' product out of the water, even as they consider switching patients to the more effective drug). Put differently, you can own a Tesla and hold Exxon stock ... and that does not make you a bad person.

Infant leukemia is (thankfully) rather rare. Nobody who's ever seen a cold, gray infant in a morgue drawer would ever say that those lives are worth less than some company's profits. But they might regret investing in the company!


I read that as a caution that these companies could try to fight this technique, or that it could face problems getting to market, not saying that it was somehow sad that these companies would face trouble.


I hardly think a treatment costing $4000/dose to manufacture is inexpensive.


To cure cancer and live? Seems pretty cheap to me.


I didn't say it wouldn't be worth it, just that it's expensive. There are certainly access issues to consider here if this comes to market.


Some people don't have $4000 so it's not a matter of deciding whether parting with that much money is worthwhile.

And the status of a lot of people's health insurance is suddenly up in the air.


First result on Googling the normal cost of cancer treatments

> Newly approved cancer drugs cost an average of $10,000 per month, with some therapies topping $30,000 per month, according to ASCO, which discussed the costs of cancer care at a recent meeting. Just a decade ago, the average cost per month of new drugs was about $4,500.

It seems $4,000 is rather a small fee in comparison.


Nitpick: it's $4000 per dose, and you're comparing to the monthly cost


> Some people don't have $4000 so it's not a matter of deciding whether parting with that much money is worthwhile.

But then those people wouldn't be able to afford other treatments either.


The insurance market and the government providing medicaid loves these types of cures. An effective cure that costs a few thousand dollars is obviously worth the healthy cured people living to pay premiums for the rest of their healthy life. Much preferable to a costly patient needing many treatments in the tens of thousands of dollars in the insurance pool. The economics are much better than what the system currently bears.


It appears the treatment was a single dose.

" Two infants with relapsed refractory CD19+ B cell acute lymphoblastic leukemia received lymphodepleting chemotherapy and anti-CD52 serotherapy, followed by a single-dose infusion of UCART19 cells"[1]

With that context, it's potentially cheaper than other therapies.

[1]http://stm.sciencemag.org/content/9/374/eaaj2013


If I understand that the article is claiming a new treatment could cure cancer for $4000, then this is orders of magnitude cheaper than all the combined treatments that are administered now to treat cancer.


I agree the manufacturing cost of $4,000 doesn't seem unreasonable, but that's the manufacturing cost. The article also states either of these competing treatments would cost insurers half a million $ or more, so it will be much more expensive than today's treatments. Still worth it, but absurdly expensive especially compared to the manufacturing cost.


If you read a little more carefully, it says the full treatment costs about $50k.


doubt it would stay that expensive if proven, mass manufacturing surely would drop it quickly.

with regards to the mention of other companies more complex methods, perhaps its merely reminding people that complex solutions are not always the only way


How much were the first large-screen LCDs? How much are they now?


Unfortunately the market for therapies does not follow the same market scaling model as consumer electronics due to the various financial models of insurance companies, government subsidies, etc.


Technology products often decrease in price. But for cancer treatments it's the opposite. New treatments used to cost $4,500 a decade ago and cost $10,000 to $30,000 now.


That's a function of perverse incentives, rather than some sort of immutable truth. If there were stronger penalties for (e.g.) cartel-like pricing (e.g. TKIs), and more price transparency for the true costs of care (ibid), we might see less of this. Or if, say, the primary market for such drugs adopted a single-payer system where the payer negotiated on behalf of the patients, rather than on behalf of its shareholders.

Gleevec and Sprycel are the canonical examples of "competition makes the price go up" but if a single payer dominated the market and used this as leverage, it would be simple enough to just say "not at that price" and force manufacturers to play ball. The real problem is regulatory and legislative capture which prevents this from happening.


Agreed. Patent and insurance reform are crucial.


It seems somewhat inline with epipens being 600 dollars..


> Two kids lives were saved with a potentially inexpensive technique

Potentially saved. They did get traditional therapy first. This type of conclusion is really premature. Don't get me wrong, this is super exciting. But the reason we have standardized clinical trials is to ensure treatments work reliably and consistently across as many humans as possible.


Isn't this how 'I Am Legend' started?




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