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

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)

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?

[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...)