FDA Approves First Cell-Based Therapy For Cancer (npr.org)

An anonymous reader quotes a report from NPR: The Food and Drug Administration on Wednesday announced what the agency calls a "historic action" -- the first approval of a cell-based gene therapy in the United States. The FDA approved Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer. The drug was approved to treat children and young adults suffering from acute lymphoblastic leukemia, a cancer of blood and bone marrow that is the most common childhood cancer in the United States. About 3,100 patients who are 20 and younger are diagnosed with ALL each year. The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients. It's also known as CAR-T cell therapy.

The treatment, which is also called CTL109, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an FDA advisory panel recommended the approval in July. The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, the FDA is requiring strong warnings.

Comments

But why that particular cancer?

[dgatwood]

Let's put this in perspective. ALL already had treatments that put 98% of affected children into remission within a couple of months, with 8% of those eventually relapsing. So 90% are completely cured with existing therapies. There are other cancers where the numbers are an order of magnitude worse. I'm puzzled why the focus seems to be on diseases that medical science has already very nearly cured, rather than the ones that kill most of the people who get them.

Also, is this actually measurably better than existing treatments? If existing treatments fail to produce remission in 2% and allow a relapse in another 8%, you'd expect only about 20% of the patients to be in that 2% group that weren't helped by chemo. So a remission rate of only 83% is probably not statistically significantly different from what they would have gotten if they had used the current generation of chemo, and doesn't necessarily indicate any benefit for people who did not respond to chemo. So this could very well be a no-op, all at a tremendous cost that insurance won't cover (because it is experimental).

I'm not saying that the research isn't valuable, because it is, but in the zero-sum game of medical research, seeing the first approved cell-based cancer treatment be for a disease that is already all but cured just seems bafflingly backwards. I would have expected the first treatments to be for things like pancreatic cancer or mesothelioma or, if you want a childhood cancer with a high case fatality rate, perhaps neuroblastoma. Brain cancers kill significantly more kids than leukemia, despite being much less common.

Re:But why that particular cancer?

[dgatwood]

I don't disagree. Clearly, chemo is a terrible way to cure disease. It's the modern medical equivalent of leeches. But if you're going to spend X dollars finding a cure for something, do you want to pick a disease that makes up 25% of childhood cancer deaths and dropping rapidly (leukemia) or a disease that has caused a consistent 30% of childhood cancer deaths for decades (brain cancer) with minimal progress? Do you want to be one of hundreds of companies in a crowded field for a disease that kills only 10% of the people who get it (and dropping) or the one company that promises possible salvation for a disease that kills 90% of the people who get it?

Re:But why that particular cancer?

[dcollins117]

First off, medical research is in no way a "zero-sum game". Finding an effective treatment for one form of cancer is a good thing. It's a net plus. It in no way detracts from the knowledge we have about other diseases. The progress we have made in this area could very well lead to treatments for all kinds of different illnesses.

There is a lot of medical research being done. Some of it pans out and some of it doesn't. Using this good news as an opportunity to lament about all the diseases we do not currently have a good treatment for gives me great concern about your ability to reason.

Re:But why that particular cancer?

[Cyberax]

Leukemia is one of the "easier" cancers, as it doesn't really form solid tumors and is susceptible to therapeutic agents. You can bet that they're now pursuing cures for more complicated cancers.

Re:But why that particular cancer?

[dgatwood]

First off, medical research is in no way a "zero-sum game". Finding an effective treatment for one form of cancer is a good thing. It's a net plus. It in no way detracts from the knowledge we have about other diseases. The progress we have made in this area could very well lead to treatments for all kinds of different illnesses.

At this point, it is largely a zero-sum game. Every dollar spent on research in one area is a dollar that can't be spent in another area. One disease's gains represent another disease's losses. The exceptions are the additional knowledge of general techniques for doing gene splicing and other similar areas, but that sort of research is sufficiently independent of what disease you're trying to cure that I doubt it is even typically done by the same organizations.

The progress we have made in this area could very well lead to treatments for all kinds of different illnesses.

In general, I would agree, but unfortunately, when you're talking about genetic modifications to human cells, as I understand it, the treatment tends to be highly specific to a single person, forget a single disease. So apart from the basic gene splicing techniques themselves (which are, I think, fairly well understood at this point), these studies basically just teach us whether you can cure a specific strain of a specific cancer in a specific person. If you're going to do an experimental treatment, if you're going to gain roughly the same knowledge either way, it seems like it would make more sense to try it on a strain of cancer that has a 90% chance of killing the person in the next year rather than a 2% chance. That's all I'm saying.

Re:But why that particular cancer?

[AmiMoJo]

One disease's gains represent another disease's losses.

Not at all. This technique for this cancer, now proven, will lead to it being adapted for other cancers. No point aiming for the hardest one first, start with an easy one to prove it works reliably and safely and then move on to the harder problems.

as I understand it, the treatment tends to be highly specific to a single person

Indeed, but the most important part of the technique is being able to quickly and relatively cheaply create such a treatment for any given individual.

Re:But why that particular cancer?

[sharky611aol.com]

IAAPHO (I am a pediatric hematologist/oncologist), so take my comments for what they're worth... TL;DR: This is a big freaking deal. To address your points, ALL was the first drug that CAR-T treatment was approved for for several reasons. 1) It's common, so easier to do the trials. 2) It has good data to support the use of immunotherapy (see blinatumumab, inotuzumab ozogamycin), and this therapy is really just the next step in a long line of improvements in immunotherapy. 3) It's got a good target. Almost all Pre-B ALLs express CD19 and downregulation (the main resistance mechanism for immunotherapy) seems rare (but has been documented in relapsed cases). 4) The cure rate is nowhere near as high as you cite for older children and young adults (currently less than 80% for anyone over 13 years.). THIS is actually where most of the use for CAR-T therapy will come from.

The bigger news is that the TECHNOLOGY for this treatment has been FDA approved. Once you have perfected the cell harvesting/transfecting/culturing/infusing process, it's trivial to plug in a different antigen target into the cassette. And in fact, this is already happening on a large scale. Hop on over to clinicaltrials.gov and search for CAR... We've already got some results from GD2 targeting (neuroblastoma), HER2 targeting (breast cancer and osteosarcoma) and IL-13R2 (glioblastoma multiforme) with promising results. And remember, this is just the first generation of CAR-T therapy to make it to the market. As the technology matures the acceptable uses of it will broaden.

Your zero-sum game argument has been sufficiently debunked below. But suffice to say, this is a true breakthrough technology which will have a huge impact on the field for years to come. But you've gotta start somewhere.

(In bigger news, I think this is the first time in my 18+ years on /. that there's been a pediatric cancer article. That should tell you something...)

Re:Just wait..

[Zontar The Mindless]

My mother is still alive today thanks to this treatment, and no thanks to you.

Re:Just wait..

[lucm]

It's amazing to think that one day cancer could be a thing of the past, like smallpox.

Re:cure or kill

[rtb61]

Sometimes retiring is the better option, rather than being a lab rat for some one else's greed. The way this stuff if being run by for profit corporations is pretty psychopathic. Lying on test results, with holding information, hiding poor results, lawyering up to keep bad treatments going profitably for as long as possible regardless of consequences. Not that this treatment is bad but they have done it to themselves, the typical US corporation is simply no longer to be trusted no matter what they claim to do or claim to be, the end result of modern PR=B$. Is it good, is it bad, yes thanks to endless lies from corporations, it is a fucking coin toss. Executives should start paying a personal price for their lies and corrupt decisions in corporations.

Re:Just wait..

[Waffle Iron]

Once it becomes FDA approved and a patent is awarded, one of the big pharma companies will come in with a blank check to the patent owner and will immediately proceed to bury this knowledge and it'll never be used again, all in favor of high-priced chemotherapy.

TFA says that the bill for this one-time treatment weighs in at $475,000. That's probably even more high-priced than almost any chemotherapy.

Re:Just wait..

[Anonymous Coward]

Gleevec, a drug used to treat CML, GIST and HES runs about $7,350.00 for a 30 day supply of 100mg tablets. I've been on Gleevec for roughly 8 years now.

Now, I know that the insurance companies don't pay full price, but if they did, then that 8 years of "treatment" with Gleevec has cost them $705,600.00, and that cost continues until the day I die.

That makes a single payment of 475,000 cheap compared to 30 years of medication at a whopping $2,646,000.00... (if the price remained unchanged)

Reference points

[DrYak]

The thing is :

- these classes of methods are generic. With very few problems (it's not relying on killing the patient slower than killing the cancer. It's about specifically targetting the cancer) Eventually this method could be adapted to other cancers as well.
so the fact that they used it against leukemia isn't a major drawback for brain cancer.
(Unlike chemotherapies which rely a lot on the general charecteristics of the cancer, to find a way to poison it with a drug faster than the drug posions the rest of the patient. Different type of cancer = different type of characteristics. Poisons will therefore work differently)

- because leukemia is well studied and has already lots of studies done with other treatment, that gives a lot data point to compare against, and to combine with.

- leukemia happens to be a slightly lower hanging fruit here. (everything happens in the blood stream, which is where you'd be injecting the modified cells).
(but again, all fruits *on the same tree*. Not an entirely different tree like chemotherapies).

These are reasons to take it as a first target, before expanding to other cancers.

Missed clickbait headline opportunity...

[Junta]

'FDA approves new treatment for ALL cancer'

Re:Just wait..

[Gilgaron]

it'll never go away just because of how cancer works, but the sorts that "aren't a big deal'" will get more broad.

Re:Just wait..

[MrHops]

Once it becomes FDA approved and a patent is awarded, one of the big pharma companies will come in with a blank check to the patent owner and will immediately proceed to bury this knowledge and it'll never be used again, all in favor of high-priced chemotherapy.

TFA says that the bill for this one-time treatment weighs in at $475,000. That's probably even more high-priced than almost any chemotherapy.

I know that this is moving the goalposts a bit, but a very common treatment for leukemia is a stem cell transplant after intensive chemo. That can go well over $1,000,000 all told, more like $2,000,000. Again, that is the listed price, the insurance companies will pay less.

(Don't ask me how I know this... :-( )