Meet Tori Lee (10 years cancer-free)

Dana and Chris Lee are certain of one thing: If not for cancer cell and gene therapy, their daughter Tori would not be alive today. 

If they had followed the standard treatment protocol at the time for treating acute lymphoblastic leukemia (ALL) – and if Tori had undergone a bone marrow transplant following her second cancer relapse – Dana and Chris believe the worst would’ve happened. 

Instead, they detoured from the standard treatment protocol and received a type of cell and gene therapy called CAR T-cell therapy through a landmark clinical trial at the Children’s Hospital of Philadelphia (CHoP). That decision – and the research that Alliance for Cancer Gene Therapy (ACGT) funded years ago, which led to the CAR T-cell therapy Tori received – likely saved her life. 

“I don’t have a doubt in my mind,” said Tori’s mother Dana. “If Tori got the bone marrow transplant instead, I don’t think she would be here today.”

Tori Lee’s diagnosis of acute lymphoblastic leukemia

Tori was diagnosed with ALL in October of 2008. Then just 5 years old, Tori had been experiencing recurring ear infections, unusual fatigue, and unexplained vomiting. A trip to the pediatrician’s office revealed an enlarged spleen, which led to blood tests. That’s when Dana and Chris learned their daughter had cancer. 

“There’s this whole flash of thoughts wondering what this means for her and wondering about the long-term prognosis,” Dana said. “It was heartbreaking to try to explain a cancer diagnosis to a 5-year-old.” 

Doctors reassured the family of the high survival rate among children with acute lymphoblastic leukemia. They said chemotherapy was most likely going to work, as it had for most other pediatric cases of this blood cancer. 

It didn’t for Tori. 

Chemotherapy struggles and two relapses

Tori was deemed a “late responder” to chemotherapy, which means the treatment didn’t work as quickly as doctors had hoped. Therefore, she received chemotherapy for longer than most children with leukemia would. Then Tori experienced the side effects that people often think of regarding chemotherapy: hair loss; nausea; loss of appetite; low blood counts and higher risks of infections; and more. 

“It was brutal,” Dana said. “She was hospitalized quite often.” 

Even still, if the chemotherapy would work, then it’d be worth it. Nearly two years after Tori was diagnosed – in the summer of 2010, right as she was finishing chemotherapy – she relapsed. 

“I felt a lot of fear,” Tori said, “but I was also frustrated. Even though I was so young, I still felt that everyone else knew what was coming but weren’t telling me. I knew whatever they were hiding was bad. So it felt like I was going through all of it alone, in a sense.” 

Tori, then 7 years old, had to start over again with chemotherapy, plus cranial radiation therapy since the leukemia had spread to her central nervous system. That lasted for two years, and in November 2012, right as they were getting close to finishing, Tori relapsed again. 

“At that point, we were feeling frantic,” Dana said. “We didn’t have a lot of options left. We were exhausted and terrified. 

“We were in a bad place.” 

Choosing CAR T-cell therapy

The recommendation from doctors was for Tori to receive a bone marrow transplant. That terrified Dana, who was a teacher at Tori’s school and watched from afar as two other children with cancer at the school struggled with treatment. One of them had a bone marrow transplant, and Dana said watching the child go through the side effects made her wish for another option. 

That option came in the form of a clinical trial for a new type of therapy: CAR T-cell therapy, a type of cell and gene therapy where a patient’s own T cells are engineered genetically to more effectively look for and find cancer. Cell and gene therapy harnesses the power of the patient’s own immune system to fight cancer more naturally than with toxins such as chemotherapy. 

Originally, the CAR T-cell therapy clinical trial was a backup plan. Tori would receive chemotherapy to put her cancer in remission, which was required for a bone marrow transplant. If her cancer didn’t go into remission, she could pivot to receiving CAR T-cell therapy. 

The Lee family had a question: Even if Tori’s cancer went into remission following chemotherapy, could she still enroll in the CAR T-cell therapy clinical trial instead of receiving the transplant? The answer was yes, and Dana and Chris had to make a choice between the two. 

It wasn’t a difficult decision. 

“Even if it doesn’t work, the patient’s quality of life is better,” Dana said. 

In April 2013, Tori became the 10th child in the CHoP clinical trial to receive CAR T-cell therapy for cancer. This trial, the first ever to use CAR T cells to treat children with cancer, was a direct result of the ACGT-funded research conducted by Carl June, MD, at the University of Pennsylvania. 

‘CAR T-cell therapy saved my life’

Five days after receiving her engineered T cells, Tori and her family returned home to Oakhurst, New Jersey. Not a single leukemia cell has been detected since then, and Tori reached 10 years cancer-free in April 2023. 

“As more time passed since receiving my CAR T cells – and I had fewer doctor visits and medications – I felt better about the possibility that my cancer would never return,” Tori said. “After years of relapsing from treatments that we were told would work, it’s tough to have faith in something that was so new at the time. But there’s no doubt that CAR T-cell therapy saved my life. 

“I feel confident the CAR T cells are still in my body doing their job.” 

Tori is one of many cancer survivors who have defeated their disease thanks to cell and gene therapy. She’s even one of the survivors who doctors consider “cured” of cancer. 

The decision to forego a bone marrow transplant in favor of CAR T-cell therapy was the turning point, but that decision wouldn’t have been possible without organizations like ACGT and the scientists conducting the research needed to develop new therapies. 

“Cell and gene therapy science is going to snowball, and there are so many different ways to use these therapies,” Dana said. “Eventually, I believe this type of treatment is going to touch everybody – not just cancer but all types of illnesses. I think it’s an investment that will benefit everyone at some point.”