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.
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.
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.
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. "
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.
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.
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.
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.
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.
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.)
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.
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.
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.
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.
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.
> 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?
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.
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.
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.
Some of it also goes to pay for the R&D of other drugs that never make it to market.
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.
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.
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.
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.
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.
And insurance companies can't throw drug company officers in jail.
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. 
> 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.
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.
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.
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).
AKA the price tag on a service is usually much more than the cost of goods sold.
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.
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.
So the title is basically click-bait, as the treatment is unconfirmed.
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.'
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.”
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.
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.
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.
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.
>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'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!
And the status of a lot of people's health insurance is suddenly up in the air.
> 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.
But then those people wouldn't be able to afford other treatments either.
" 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"
With that context, it's potentially cheaper than other therapies.
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
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.
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.