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?

[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:Just wait..

[lucm]

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

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.