CAR-T Approval

Some really excellent news yesterday, one of those stories that's all over the internet, and not just getting talked about in the Lymphoma community --

The FDA approved the first CAR-T treatment. This is a big deal. It's a real victory for Immunotherapy -- using the body's own immune system to fight cancer.

Just a quick reminder of what CAR-T is. First, it stands for Chimeric Antigen Receptor T cell therapy. Basically, what happens is T cells are removed from the body. T cells are types of immune cells -- they attack invaders. But not cancer cells, which find ways to make T cells ignore them.

After the T cells are removed, they are changed so they DO recognize cancer cells as bad guys. They are put back into the body, and then the changed T cells attack the cancer cells. The treatment is personalized -- only the patient who had them removed and changed will benefit from having them put back.

(Dana Farber Cancer Institute has a nice article explaining how it works, with a very helpful video.)

The new treatment is called Kymriah, though in clinical trials it was called CTL019 or Tisagenlecleucel. It has been approved for pediatric and adult Acute Lymphoblastic Leukemia, a very aggressive type of blood cancer. Another CAR-T treatment is under review for aggressive lymphomas, including transformed Follicular Lymphoma.

How big a deal is this? I'll let Ben tell you. He runs the blog CAR-T and Follicular Non-Hodgkin's Lymphoma, with help from William -- both are active readers of Lympho Bob, and their blog does an excellent job of keeping up with new developments in the CAR-T world. Ben is a Follicular Lymphoma patient who received CAR-T, and William's wife is also a CAR-T patient with FL. Ben called it "Truly a momentous day in the long and winding history of this life-saving therapy," and he is "Excited to finally see it becoming more widely available to many more patients."

But Ben also points out that there is a high price tag for this treatment -- about $475,000, making it one of the most expensive cancer treatments ever. The company that makes it  has responded to criticism about the price by saying that it is an expensive process -- each patient gets their own personalized treatment. They have also said that any patient who doesn't get a Response within a month will not have to pay for the treatment. (Of course, if the treatment fails on day 45, you're out of luck -- in a couple of ways.)

The treatment has been very effective in trials, but it certainly is not without risks. The biggest of these is cytokine release syndrome, a response from the body to all of those T cells that results in possible fever and brain swelling. One patient in the Lymphoma trial died from this side effect.

Still, the overall news is very positive, and the approval for Kymriah is considered a positive sign for KTE-C19, the Lymphoma CAR-T treatment. It will be interesting to see how many more cancers get a CAR-T treatment, or if the cost will discourage patients and doctors, and thus pharmaceutical companies. But if not, this could be big news for all three.

And I'm always in favor of more options for us as patients.