
Two Infants Treated with Universal Immune Cells Have Their Cancer Vanish - phr4ts
https://www.technologyreview.com/s/603502/two-infants-treated-with-universal-immune-cells-have-their-cancer-vanish/
======
jackweirdy
> 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.

~~~
_ihaque
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](https://en.wikipedia.org/wiki/Kaplan%E2%80%93Meier_estimator))

~~~
woliveirajr
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.

~~~
mikeash
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.

~~~
true_religion
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?

~~~
voxic11
No, cancer does not have standardized treatments. The remission rates vary
greatly depending on the hospital and even on the doctor.

~~~
sjg007
Huh...? There are standard treatment protocols.

~~~
gregshap
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.

~~~
sjg007
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.

~~~
gregshap
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.

~~~
sjg007
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)

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blisterpeanuts
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...](http://www.timesofisrael.com/breakthrough-cancer-cure-has-deep-israeli-
roots/)

~~~
eliben
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?

------
mediocrejoker
> “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?

~~~
L_Rahman
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.

~~~
omegaworks
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.

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

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

~~~
refurb
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.

~~~
omegaworks
>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.

~~~
refurb
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.

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

~~~
chrisamiller
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.

------
rdlecler1
>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.

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

------
gt565k
'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.

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

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

------
geekuillaume
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.”

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

~~~
vivekd
Yes, chemotherapy can and has cured cancer.

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

~~~
vivekd
It can completely cure the cancer

[https://en.wikipedia.org/wiki/Chemotherapy#Efficacy](https://en.wikipedia.org/wiki/Chemotherapy#Efficacy)

------
CodeSheikh
"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.

~~~
tormeh
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.

------
guard-of-terra
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.

~~~
guard-of-terra
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.

~~~
JPLeRouzic
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?

~~~
ska
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](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.

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

------
ryeguy_24
"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.

~~~
pmiller2
I hardly think a treatment costing $4000/dose to manufacture is inexpensive.

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

~~~
gdulli
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.

~~~
popey456963
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.

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

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

