In April 2012, 7-year-old cancer patient Emily (Emma) Whitehead became the first child to be enrolled in a clinical trial for patients with B cell cancers such as acute lymphoblastic leukemia (ALL), B cell non-Hodgkin lymphoma (NHL), and the adult disease chronic lymphocytic leukemia (CLL). Emily received this experimental treatment at The Children's Hospital of Philadelphia in April 2012 after her cancer relapsed for the second time. Her cancer has been in remission since her treatment.
Emily Whitehead's Story: T Cell Therapy for Acute Lymphoblastic Leukemia
Kari Whitehead (mother of Emily): She relapsed in October 2011 and at that point we knew it wasn't kind of the standard, garden variety leukemia. Then she relapsed again in February 2012. And so it was at that point that we made the decision that we needed to go somewhere else where we needed to try something new and something different.
Tom Whitehead (father of Emily): We were told that we were down to 48 hours for making a decision or she could start to have organ failure.
Kari Whitehead: We knew that there were things offered here that was — that would be different from what our local hospital could offer, and things that were more, I guess, cutting edge. Newer things were being tried that weren't being tried anywhere else.
Tom Whitehead: So we're really down to the CART19 as what's left for Emily to cure her. So we're ready to go today.
Kari Whitehead: Right now we're feeling hopeful, really hopeful. It's a little bit scary because Emily is one of the first patients to receive this. She's the first pediatric patient.
Tom Whitehead: I felt all along that she's going to beat cancer and today's our day when we're going to start doing it. You want to say anything else, Em? What are we going to do today? What are we going to do today? You're going to get your T cells?
Emily Whitehead: Uh-huh.
Tom Whitehead: What are they going to do?
Kari Whitehead: Do you remember?
Emily Whitehead: Nuh-uh.
Kari Whitehead: Nuh-uh?
Tom Whitehead: What are they going to take away?
Emily Whitehead: Cancer.
Tom Whitehead: Cancer, that's right.
Stephan Grupp, MD: So CART19 is a treatment for patients with fairly advanced leukemia. Most patients who have the kind of leukemia that CART19 treats, which is the most common kind of childhood leukemia, it's called B cell ALL, that is a disease where most patients are treated very successfully with standard chemotherapy. But there's a subset of those patients, and a substantial number of children in the United States, for whom chemotherapy does not work, or it works temporarily. So for that group of patients who have very treatment resistant disease, we need something brand new, and CART19 is for those patients. The essential nature of the CART19 treatment is that we have to get cells from the patient. These cells are called T cells. They're cells of the immune system that need to be engineered. We can engineer these cells to go after cancer cells and kill them.
Kari Whitehead: So Emily had her — the CART19 therapy April 17, and we were told to expect that she would possibly get a little bit of a fever and feel like she had the flu. But she ended up getting pretty sick shortly after, and she was put on a ventilator because of how sick she was.
Tom Whitehead: They had come with a team of doctors in the PICU every morning and did rounds with us, and we were always first. And they would come to us and kind of show us a line on the floor and say, "If that's the line of survival, Emily is almost stepped past it."
Stephan Grupp, MD: During the few days when she was quite sick, she was receiving the most advanced medical care that's available on the planet. So the people in the hospital were taking care of this child during the period she was quite sick until such time as we learned what was wrong, had a chance to intervene, and made her better. One of the results of her T cells growing in her body was that she had an increase in various proteins that controlled the immune reaction. And this increase in those proteins was actually making her ill. We actually found that one of these proteins was quite elevated, and as it turns out there's a drug that targets that exact protein. We gave her that drug and she had a dramatic response. The ICU doctor on that night told me that he had never seen a patient that sick get better that quickly.
Kari Whitehead: We weren't sure at that time if the CART19 cells were working, if they weren't working.
Stephan Grupp, MD: About three weeks after we gave her her T cells, we did another bone marrow test. This young lady had not been in a remission for a number of months and she now was in a complete remission. So she completely responded to her T cell therapy.
Kari Whitehead: We were able to go home June 1 and since that time she's been — she's been great. She's been happy and healthy and returned back to school in the fall. You know, CART19 was really the only option left for Emily. And although we weren't — we weren't sure how it was going to work because she was the first child, we still felt like by enrolling her in the trial, even if it didn't work, it would give them a little bit more information, and from that hopefully they would learn something and be able to help other children. You know, but we entered her into the trial really hopeful. She's extremely smart. She's very creative. She's funny. She makes us laugh all the time. I mean, she's — she never complains.
Tom Whitehead: She had told us, you know, from the beginning that she would continue to fight and do what we ask as long as we were there with her. So we've both been there through this entire fight, all three of us together, and we just stick together as a team. And she's definitely our hero.
Stephan Grupp, MD: We checked her bone marrow for the possibility of disease at two points. We checked her at three months and now six months out from her treatment. She has no disease whatsoever. We need to treat a larger number of patients before we understand what the success rate might actually be. It seems like the cells stick around for a long time; that actually might provide long-term disease control. That is of course the most exciting potential part. But we need more time to know if that's actually where we are. To see her go from leaving the hospital and starting to recover to basically going to school and playing soccer and looking like every other kid is just wonderful. I think it's the best thing about doing what I do.