Faith, Science, and Miracles: A Conversation About CAR-T Cell Therapy with the Whitehead Family

December 4, 2020
Jessica Skarzynski
Jessica Skarzynski

Emily Whitehead was the first child to receive chimeric antigen receptor (CAR)-T cell therapy. Eight years later, she and her parents discuss her ongoing remission from acute lymphoblastic leukemia.

Diagnosed with standard-risk B-precursor acute lymphoblastic leukemia (ALL) in 2010 at the age of 5, Emily Whitehead experienced two relapses, and doctors told her family there was nothing more to be done. But then she enrolled in a phase 1 trial at Children’s Hospital of Philadelphia (CHOP) in April 2012 and became the first child to receive chimeric antigen receptor (CAR)-T cell therapy. The treatment turned out to be what the Whiteheads call a miracle, as Emily has been in remission since May 2012.

CURE® recently spoke with Emily, her father, Tom, and her mother, Kari, about how this treatment changed their lives and why they’re paying their good fortune forward by fundraising for CAR-T cell research through an organization they created, the Emily Whitehead Foundation.

CURE®: Can you give us some background on Emily's diagnosis?

Kari: When Emily was 5 years old, she was diagnosed with ALL. We learned that it's the most common type of childhood cancer, and it's also the most curable type, because (more than) 90% of kids get into remission with standard therapy. So, although we were devastated when we heard the diagnosis, we thought 90% was a good number, and we felt certain that she would be in that 90%.

Treatment was 26 months of chemotherapy, but 16 months into that, she relapsed. And she was scheduled for a bone marrow transplant, but she relapsed again, just two weeks before, which made her ineligible for transplant. We had gone for a second opinion at CHOP, where they told us about CAR-T cell therapy, but it wasn't quite ready for her at the time, because it hadn't gone through FDA approval (to study in people).

So when we came to the point where doctors said, “There aren't any more options for Emily; it's time to take her home on hospice,” we called back to CHOP, and they said, “It's amazing that you called us, because the FDA has just given us approval to try it on the first patient.” And so that's how she became the first child to get CAR-T cell therapy.

Can you tell us more about the therapy and what that was like?

Tom: At our first trip down to CHOP, they didn't mention the CAR T-cell therapy. And then (at) our second-second opinion when we went down, they mentioned it and said it wouldn't be ready in time for her.

I had talked to my wife, Kari, and said, “Some people say they get a sign, but I see Emily getting better down there in the future, in the bone marrow transplant hallway. (In this vision, I see that) she's beat cancer, and we're teaching her to walk again. And I've seen it more than once. I feel like some people get a sign, and that's our sign.” And then, about a month later, that actually happened.

They told us there were no more options available to treat Emily, and I said, “Well, I'm going to page CHOP, because I feel very strongly that she's going to get better there.” And they said, “The last email we read yesterday was that the T-cell (therapy) got approved.”

Then they treated her and said, “Emily’s not going to survive this cytokine release syndrome (a severe side effect of CAR-T cell therapy in which the immune system attacks healthy organs).” I said, “Please keep trying to help her, because I know she’s going to survive this.”

Then they took me in the hallway and said, “There's a one in 1,000 chance your daughter is going to be alive in the morning.” And I said, “I understand what you're saying, but please keep trying to help her.”

The next day, they tried tocilizumab (Actemra, to treat the cytokine release syndrome), which saved her life. And on the last day in the pediatric intensive care unit, they transferred us back down to the bone marrow transplant hallway, where we had to teach her to walk again. And she had beat her cancer.

So, I posted (on social media) at that time, “I believe I'm witnessing a miracle, because how would I know that last month?” I felt we were guided.

Emily, do you remember what it was like to go through that treatment or its side effects?

I always just remember my parents were constantly there for me, and my dad would always try to make me smile every day. And currently, the only side effect that I have is (that) I have to get B-cell replacement every two weeks. But that is really it. I'm just a normal teenager, and I'm pretty healthy.

Once Emily was declared cancer-free, you created the Emily Whitehead Foundation. Why did you take that step?

Kari: We created the foundation because we wanted to specifically fund pediatric immunotherapy clinical trials, like the CAR-T-cell therapy. While our primary mission is to fund that type of research, we do also provide a little bit of support to families and do a lot of advocacy work.

Emily, when you talk to other people about what you went though, how do you describe it?

Emily: It's definitely a really difficult thing to go through, and you really need the support of other people, whether that be friends or family, or even a pet, like my dog, Lucy.

But I'm just so glad that more people have access to the treatment now, to less toxic treatments. And really all that you can do is just never stop and always keep fighting and just try to keep hoping you'll get through it.

Finally, what advice would you give other children with leukemia or other types of cancer, having gone through it yourself?

Emily: I always tell them to never give up, to always keep fighting and to always believe in yourself and just to try and smile every day, because you never know when things could change in an instant.