Emily Dumler, a 36-year-old mother of three, is petite, energetic and appears to be the very picture of health. To look at her, you’d never know that four years ago she was at death’s door.
Her symptoms struck out of the blue in the middle of the day. It was lunchtime and her kids were with her. Her husband, a medical device salesman, was at work. Dumler called him and told him she needed to go to urgent care right away.
“My blood wasn’t clotting properly and so I went into urgent care and then ended up in the hospital for 43 days,” Dumler says.
Initially, she was diagnosed with ITP, a disorder that leads to excessive bleeding caused by low levels of platelets. It turned out she had something else: non-Hodgkins lymphoma, a blood cancer.
Dumler wound up undergoing six rounds of outpatient chemotherapy, one round every three weeks. The initial results looked good. The cancer appeared to be gone. But it wasn’t. It came roaring back.
In January 2015, she underwent a stem cell transplant using her own stem cells at M.D. Anderson Cancer Center in Houston.
“They just basically wreck your body so much that you almost are basically dying, and then they give you your stem cells back just to keep you alive,” Dumler says of the stem cell therapy.
She lost 20 pounds in a matter of weeks. And then came the bad news: the transplant failed. At that point, she was pretty much out of options. Her only hope was getting into a clinical trial at M.D. Anderson for an experimental treatment. It had just begun and luckily she qualified.
“I was the third patient in the world to be in this trial, which is called CAR T-cell therapy,” Dumler says.
Reengineering the body's own cells
CAR: It’s a mouthful, but it stands for chimeric antigen receptors. The therapy involves removing a patient’s T cells, a type of white blood cell, and genetically engineering them to recognize and attack the patient’s tumors. The T cells are then put back into the patient’s body.
Dr. Joseph McGuirk is director of blood cancers and stem cell transplants at the University of Kansas Cancer Center. McGuirk oversaw a CAR-T clinical trial at KU that showed promising results.
“When patients develop cancer, the T-cells have failed to do their job, because part of their job is surveillance for misbehaving cells or abnormally shaped cells – recognize that and attack and destroy them,” McGuirk explains.
Unlike traditional intensive chemotherapies or radiation, CAR-T re-arms patients’ immune systems, allowing them to do the work. It is, says McGuirk, “a revolution in cancer medicine that’s ongoing right now.”
Just last week, the U.S. Food and Drug Administration for the first time approved a CAR-T therapy, in that case to treat kids and young adults with leukemia.
KU’s clinical trial involved patients like Dumler with an extremely aggressive form of non-Hodgkins lymphoma. One of those patients, a 47-year-old Australian who traveled to KU to undergo CAR-T therapy in late 2015, died last year. But a significant percentage of patients – nearly half – achieved complete remissions.
“It is a keyhole view into this extraordinary future that’s coming down the line in cancer medicine,” McGuirk says.
Costly treatment
The KU clinical trial is no longer accepting patients. But now that the FDA has approved CAR-T therapy, KU is expected to be one of the few facilities to offer it.
While CAR-T therapy has delivered impressive results and may eventually be approved for solid tumors, it’s extremely expensive.
“When we talk about the cost of treatment, there's going to be the cost of the drug itself, there's going to be the cost, from the hospital side, of preparing the patient and then giving the treatment, and then there's going to be the cost of actually administering the treatment,” says Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.
In fact, Novartis, the drugmaker behind the FDA-approved treatment, has priced it at $475,000. And some patients, after showing promising results, have died from the side effects of CAR-T therapy.
“Because as the CAR-T cells begin to get into the body, as they begin to attack the cancer, then there's the release of various proteins and other substances into the bloodstream that can actually cause, sometimes, fatal events,” Lichtenfeld explains.
Emily Dumler got her CAR-T treatment on July 13, 2015, just over two years ago. And she did have side effects. One of them was neurotoxicity: basically her brain fogged up.
“And it's just a little unnerving to be outside of what you can, I don't know, cognitively understand, so I did experience not knowing anything,” Dumler says.
But the side effects eventually dissipated. And now, Dumler says, she feels great.
“I actually just had my two-year scans in Houston a couple of weeks ago and I’m still in remission today,” Dumler says.
Before her brush with death, Dumler had been a stay-at-home mom. Now she’s director of religious education at the Catholic church she attends.
She says she might have wound up taking the job anyway. But having gone through what she’s gone through, she now says she felt called to do it.
Dan Margolies is KCUR’s health editor. You can reach him on Twitter @DanMargolies.
