Tori Lee: Beating Leukemia Thanks to CAR T-cell Therapy

Our youngest daughter Tori was diagnosed with acute lymphoblastic leukemia (ALL) in October 2008. After four years of grueling chemotherapy and radiotherapy, the leukemia relapsed a second time. Fortunately, Tori was able to take part in a clinical trial testing a new type of treatment called CAR T-cell therapy. It is now more than five years since she received the treatment [now called tisagenlecleucel (Kymriah)] and she remains in remission. There is no doubt in our minds that CAR T-cell therapy, and the research that led to it, saved Tori’s life.

Tori’s diagnosis came just weeks after her fifth birthday. She had been having a lot of ear infections that required multiple courses of antibiotics. So, when she woke up one Saturday with all the symptoms of yet another ear infection, we took her to her pediatrician expecting nothing more than another prescription for antibiotics.

However, during the exam, the pediatrician felt that Tori’s spleen was enlarged and did a rapid blood test. The results concerned the pediatrician enough to recommend that we go straight to the hospital for further tests.

At first, we had no idea what was wrong with Tori but we knew it must be bad; you don’t get sent to the hospital on a Saturday unless it is serious. Then we started thinking about other symptoms Tori had been having “general exhaustion and unexplained vomiting.” We began to be fearful that it might be leukemia. It was an excruciating two-day wait until the Monday, when a bone marrow biopsy confirmed our worst fears. Tori had ALL.

Tori began the standard treatment – six months of intensive chemotherapy followed by two years of maintenance chemotherapy – immediately. Unfortunately, Tori was a delayed responder, meaning the chemotherapy did not work as quickly as expected to eliminate the leukemia cells. This meant her treatment plan had to be lengthened, and she went through a year of very high-dose chemotherapy.

The chemotherapy was brutal. Tori had every side effect imaginable and was hospitalized many times.

Then, just before Tori was due to finish the maintenance therapy, when we thought it was nearly over and she was beginning to resume some activities, a routine spinal tap detected leukemia cells in her spinal fluid.

Tori had relapsed.

This time, Tori needed cranial radiation in addition to high-dose chemotherapy. Going through this second round of treatment was pure torture for us. There were moments when we didn’t know if Tori would make it but she did, and her leukemia went into remission again.

Then, in November 2012, Tori relapsed for a second time. We were told that a bone marrow transplant was her only option. Her local oncologist recommended that we travel to Children’s Hospital of Philadelphia (CHOP) for the procedure.

The oncologist at CHOP told us that to be eligible for a bone marrow transplant, Tori had to have more high-dose chemotherapy to get her into remission. She also told us that she had a back-up plan. The plan was to collect Tori’s T cells before the high-dose chemotherapy so that if Tori did not get into remission she could take part in a clinical trial testing CAR T-cell therapy.

After the T cells were collected, and while Tori was beginning to prepare for a bone marrow transplant, we began researching the CAR T-cell therapy clinical trial. What we learned led us to ask the oncologists at CHOP if Tori could take part in the trial even if she got into remission. We felt CAR T-cell therapy would give Tori a better quality of life than a bone marrow transplant.

Thankfully, Tori entered remission and was accepted into the trial. She received the CAR T-cells in April 2013 and returned home five days later.

The difference between chemotherapy and CAR T-cell therapy was like night and day. The side effects of chemotherapy caused Tori to spend more than 300 nights in the hospital. The CAR T-cells were engineered to target the leukemia and Tori has not been hospitalized since she was discharged after the treatment.

Since receiving the CAR T-cells, there has been no sign of Tori’s leukemia and she is living the life of a typical 14-year-old. She likes sports and going to the beach, and received a leadership award at her eighth-grade graduation.

Tori’s experience has made us advocates for research into pediatric cancers. We want to do all we can to ensure that more money goes toward research because Tori is living proof of the amazing things that researchers can do.