Friday, September 1, 2017

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.

4 comments:

icrazyhorse said...

Dear Friends--

Here is my personal CAT-T success story. In November 2011, at the age of 65, my wife was diagnosed with follicular lymphoma Stage 4, Grade 3A. She had 50% bone marrow involvement. She is one of the 20% of follicular lymphoma patients who progress rapidly after treatments. In 3 years she progressed after R-CHOP (6 cycles), bendamustine/rituximab (6 cycles), and Ibrutinib (12 months). Then she took Idelalisib/rituximab as her fourth treatment. It worked great for 14 months then a PET scan showed she progressed again. She is now in an NIH CAR-T trial NCT02659943 was infused on March 2, 2016. As of September 1, 2017, she has been in complete remission for 18 months. Current CAR-T clinical trial data shows that once a follicular lymphoma patient achieves a complete remission they stay in complete remission. There is more information on CAR-T for fNHL patients and people considering CAR-T at https://fnhlben.wordpress.com/

William

Anonymous said...

Hi Bob,

Thanks so much for the shoutout. Sorry I'm so late replying to your kind and thoughtful post. We were on vacation at an old farmhouse in the middle of upstate NY with no cellular and limited internet. I just saw your post last night as we arrived in Boston to drop off our son for the fall semester. I'll have a new post or two in a few days, once we're back home and I'm on my computer again. There is so much buzz around CAR-T with this FDA approval news!

Best regards,

-- Ben

https://fnhlben.wordpress.com

Lymphomaniac said...

Thanks for sharing your story, William. It's great to attach some of those trial statistics to an actual person. That should give a lot of us hope.
Bob

Lymphomaniac said...

Thanks, Ben. I hope you enjoyed the farmhouse. I'm curious about where your son is going to school in Boston -- I grew up there. And I just dropped off my own son at school last week (not in Boston, though). Email me if you don't want to put that kind of info in a comment -- bobtalisker@gmail.com. We can share some tearful stories of saying goodbye to our kids (whether they were tears of sadness or joy, we can decide for ourselves).
Bob
(And keep up the great work on that CAR-T blog.)