Hope and Remission in CAR T Cell Therapy

When the U.S. Food and Drug Administration (FDA) announced its approval for use of a new immunotherapy in August 2017, it was a breakthrough for precision medicine. The immunotherapy, used to treat a form of pediatric leukemia, became the first FDA-approved, commercially available chimeric antigen receptor (CAR) T cell therapy.

This particular form of therapy uses Gibco Cell Therapy Systems (CTS) Dynabeads technology, developed by Thermo Fisher Scientific, to activate and expand T cells that have been genetically engineered to recognize and fight cancers. It all starts with the patient’s own cells, which are infused back into them after the process is complete.

Life in the Lab: At what stage in your battle with cancer did you first learn about CAR T cell therapy?

Nicole Gularte: After a relapse in 2014, my doctors at Stanford University told me that I would need a transplant and that I'd be looking at 3–5 more years of additional treatment with a lot of side effects and a low percentage of success. I kept pushing because there just had to be another option, and that's when they mentioned CAR T.  But they told me that I wouldn't live to see CAR T treatments reach clinical trials and not to think about it because it would be a waste of my time. I left the hospital vowing to do my own research, and, in the meantime, I took part in another treatment that served as  a bridge to CAR T.

LITL: Can you describe the experience you had with the interim treatment?

NG: We knew it would likely put me in remission quickly, but we also knew the cancer would come back. But it served an important purpose because it bought me time, and during that time, I pushed myself to pursue CAR T. Until that point, my traditional chemotherapy and radiation treatments were doing cumulative damage to my body and I still suffer from side effects. They led to multiple knee surgeries, bone marrow biopsies, early menopause, and significant neurological damage—many side effects that still impact my daily life. There were no long-term side effects from the interim treatment, and the experience gave me confidence and hope for the CAR T therapy.

LITL: And you had to cross the country to be in position for a CAR T trial, correct?

NG: Exactly. When I was first in remission, I called the University of Pennsylvania, which had a CAR T trial on hold, but I had  a strong feeling that it would eventually come back because of the initial studies and the success rates. So UPenn granted me an appointment and although they knew I would eventually relapse, I was healthy at the time. They collected and froze my cells for when either the FDA lifted the hold on the study or I relapsed, which is what I needed to do to qualify for the study.

LITL: And what was it like for you to play the waiting game before you finally qualified for the CAR T trial?

NG: For two years, from 2014–2016, I traveled, shared my story, and raised awareness while I was getting treatments. I had relapsed several times and the leukemia had spread into my eyes and spinal fluid, which kept me from being qualified for the study. And even though I was happy and still wanted  to fight, the combination of the radiation and the chemotherapy drugs that were injected in my spine every day was toxic, and it was diminishing my quality of life. That's when I decided to go on palliative care and stop treatment. When they gave me 3–5 weeks to live, I started planning my funeral. And since I was never baptized, my family and friends arranged for my baptism. I lived those weeks thinking they would be my last.   

LITL: And then what happened?

NG: Miraculously, days before I was supposed to be dead, I had a final checkup and although the cancer didn't go away, my blood was good, and the leukemia was no longer detected in my spinal fluid, which qualified me for the study. The next week I flew out to UPenn and got the treatment. The experience was amazing and within 28 days, my leukemia was gone. It was crazy because I went from having it spread to my eyes, my spine, my lymph nodes, and my bone marrow to it being gone. I did experience some side effects—cytokine release syndrome (CRS) that led to an average fever of nearly 106 degrees over six days, and two small seizures—but they were short-term and relatively minor compared to the side effects from chemotherapy and radiation.

LITL: How are you feeling today? And what's next for you?

NG: Last October, I had my annual checkup, which confirmed that I have no leukemia cells anywhere. I've also become very involved with the Emily Whitehead Foundation and was honored to be a speaker at their inaugural gala, which included more than 30 CAR T survivors—all children, aside from me. After that event and my clean checkup, I've made my purpose in life to be a voice for children with cancer, and I'm now launching a West-Coast operation of the Emily Whitehead Foundation.

To learn more about Nicole Gularte's story, visit her blog: The Inspired Hero: Leading The Way With Immunotherapy.