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The Children's Hospital of Philadelphia

Emily's parents discuss her treatment for leukemia at the Children's Hospital of Philadelphia, one of Parents magazine's choices for the 10 best children's hospital for 2013.

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-She relapsed in October 2011, and that point, we knew it wasn't kind of a standard garden-variety leukemia. Then, she relapsed again in February 2012. And so it was at that point that we made a decision that we needed to go somewhere elsewhere. We really need to try something new and something different. -We were told that we were down to a 48 hours for making a decision or she could start to have organ failure. -We knew that there were things offered here that was-- that would be different from other local hospital could offer and things that we're more, I guess, cutting edge. Newer things are being tried that weren't being tried anywhere else. -So, we're really down to CART-19 is what's left for Emily to cure her. So, we're ready-- ready to go today. -Right now, we're feeling hopeful, really hopeful and a little bit scary because Emily is one of the first patients to be treated. She's the first pediatric patient. -I've felt all along that she's gonna beat cancer and today is our day when we're gonna start feeling it. You wanna say anything else? What are we gonna do today? What we're gonna do today they're gonna get your T cells. -Uh huh. -What they're gonna do? -Do you remember? -Uh huh. -What they're gonna take away? -Cancer. -Cancer, that's right. -So, CART-19 is a treatment for patients with fairly advanced leukemia. Most patients who have the kind of leukemia that CART-19 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 CART-19 is for those patients. The essential nature of the CART-19 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. -So Emily had her-- the CART-19 therapy, April 17th and we were told to expect that she would possibly get a little bit of a fever and feel like she had a flu, but she ended up getting pretty sick shortly after and she was put on a ventilator because of how sick she was. -They had come with the 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 a line of survival Emily is almost step passed that. -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 control the immune reaction and this increase in those proteins would 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. -We weren't sure at that time if the CART-19 cells were working. If they weren't working-- -About 3 weeks after we gave her T cells, we did another bone marrow test. This young lady had not been in remission for a number of months and she now was on a complete remission. So, she completely responded to her T-cell therapy. -We were able to get home June 1st and since that time, she has been great. She has been happy and healthy and returned back to school in the fall. You know, CART-19 was really the only option like for Emily and although we weren't-- we weren't sure how was gonna 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, when we entered her into the trial, we're really hopeful. She is extremely smart. She's very creative. She's funny. She makes us laugh all the time. I mean, she's-- she never complains. -She 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 3 of us together, and we did stick together as a team and she is-- she is definitely our hero. -We checked her bone marrow for the possibility of disease at two points. We checked her 3 months and now 6 months after 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 a hospital and starting to recover to basically going to school and playing soccer and looking like every other kid is just wonderful. I think that's the best thing about doing what I do.