Image courtesy of The Emily Whitehead Foundation
By Tom Whitehead
As told to writer Jennifer Clopton
Our only child Emily had just turned five when our world changed forever.
We didn’t think much of her bleeding gums or the bruises on her body until one night she woke up with excruciating knee pain that seemed like much more than growing pains. My wife called me from the pediatrician’s office the next morning to say ‘something is wrong’ and I headed straight home from work. Before I got there she called again.
Emily was diagnosed with b-cell acute lymphoblastic leukemia, or ALL, in May 2010. My wife and I felt all the emotions you would expect – worry, devastation and sadness. But we didn’t want Emily to see the fear in our eyes.
So the first thing I said to her when I saw her was, “Emily – only the strongest children in the world are picked to have cancer. You will be a hero and you will beat this.” She said ‘OK’ and believed it from that point forward.
I remember being devastated when Emily was admitted to the cancer floor at a nearby hospital. But the doctors told us if your child had to get a type of cancer, this was the one to get. With the proper treatment they expected her to be cured and live a long and full life.
But after just two outpatient chemotherapy sessions, Emily developed infections in her left thigh and right calf and suddenly cancer was no longer our biggest problem. Doctors said they hadn’t seen an infection so severe in 10 years. “You need to save your daughter’s life,” they told us.
That involved signing a release form saying we understood they might have to amputate her legs in surgery, but doctors thankfully got the infection under control without needing to do that. A week later we were celebrating when Emily was out of pediatric intensive care and re-admitted to the cancer floor that had made me so sad just days before.
I tell parents now, your perspective can change quickly.
Emily got in remission in about two months and stayed there for 16 months. But one day in October 2011, while playing with her aunt, she said out of the blue, “Aunt Kathy, I feel the blasts growing again in my bones.”
She was right. She was relapsing.
This time she was labeled high risk and given less than a 30% chance of survival. Doctors recommended a bone marrow transplant. The transplant required full body radiation, which we didn’t like because we had talked with so many other parents who said it caused life long learning disabilities, growth and organ problems.
Our search for options led us to Susan Rheingold, MD, a relapse specialist at Children’s Hospital of Philadelphia (CHOP). She told us about an experimental drug, but it wasn’t for Emily’s specific type of cancer and it didn’t feel right to us. She then told us about another experimental therapy called chimeric antigen receptor therapy or CAR T-cell therapy. This type of immunotherapy involves taking immune cells called T-cells out of a patient’s body and changing them genetically to target a specific kind of cancer. Doctors told us it was like sending Emily’s cells off to boot camp where they would be turned into a highly trained and skilled army to fight her cancer when they put them back in her.
The only problem – the FDA was thought to still be a few months away from approving the therapy for experimental trials. So we treated Emily with chemotherapy to try and buy us time for that approval. We made it a few weeks before our local doctors told us they had run out of weapons to fight Emily’s cancer.
They told us to go home and enjoy the final days we would have with our daughter.
That same day, doctors at CHOP got approval from the FDA to start the CAR T trial earlier than they had first expected.
It was such a gift. But there were challenges. Doctors said CAR T therapy had never been given to a child.
Anywhere in the world.
But they also told us they thought it might take her cancer away. We were less scared of trying this drug then we were with continuing to bombard her body with toxic chemotherapy and radiation, so we enrolled her in the trial.
A few days later, Emily became the first child to participate in the study. Doctors extracted her T-cells and she stayed in isolation at the hospital for six weeks while they modified them. The modified cells were infused into her and she received them in mid-April.
Things did not go well at first. A few days after the infusion, she was induced into a coma. The infusion of powerful cells in CAR T treatment releases chemicals known as cytokines that signal the body to produce inflammation and it can cause very serious side effects.
In Emily’s case it triggered a skyrocketing fever, plummeting blood pressure and congestion build-up in her lungs that made it tough for her to breath. The cytokine storm overwhelmed our little six-year-old’s body and she almost died.
She was in the coma for 14 days and at one point doctors told us to call our family in for goodbyes because they believed she had less than a one in 1,000 chance of surviving to the next morning.
The solution came from an arthritis drug that blocked the cytokine overwhelming her body known as Interleukin-6. The medication had never been used in a cancer patient but doctors saw it as our only hope and within 12 hours things started to turn around. Doctors said they had never seen a patient that was so sick, improve so quickly.
Emily woke up from her coma on her 7th birthday and a week later when they checked her bone marrow, I got the best call of my life from Dr. Stephan Grupp.
“I want to be the one to tell you. It worked!” he said.
The cancer was gone.
Our daughter had lived through two years of hell and suffered some of the most brutal pain you could have and still be alive. But she fought and fought and finally, she got her miracle. We got our miracle.
Emily came home June 1 and that August, started 2nd grade on time with students she had barely seen in two years.
Today she is in 7th grade and at the top of her class academically. She loves to swim and is a great artist. Her health is great and if you see her, you would never know what she has been through. She gets intravenous immunoglobulin every two weeks to help fight infections, but otherwise she is normal.
This year doctors declared her cured of leukemia because she has been cancer-free for five years.
We have worked every spare moment since Emily came home to do everything we can to get this drug approved so other families can get their miracle too. There were so many children in the hospital fighting alongside Emily and their parents were fighting just as hard as we were and their children have since died.
We travel and speak and tell Emily’s story as much as we can because we believe this treatment can change the world. Worldwide more than 300 hundred children have now been treated with this therapy and initial remission is high – approximately 90%. This October we are holding a gala called the Believe Ball for all the children who have had CAR T-cell therapy. Children have been treated in 11 other countries but most are from the United States and we have personally seen at least 20 children improve from the therapy.
But we know some children relapse and we know children who didn’t make it. And when you know the 10 out of 100 that don’t survive and don’t go home with their parents, it feels like too many.
That is one of the reasons we started The Emily Whitehead Foundation – to raise money to help research move forward to figure out how to make this treatment work for more children. Our foundation also works to help families access this treatment.
I testified at the FDA hearing in July to approve CAR T therapy because we are so thankful for every day we get to spend with Emily. We would love to see all families get to take their kids home healthy after a cancer diagnosis.
Telling my daughter’s story still always makes me cry and sometimes that worries Emily. I reassure her by saying it’s hard to talk about, but it’s also so important.
Halfway through my speech to the FDA panel, I started to tear up. Emily stood up from the audience, walked to me and grabbed my arm to reassure me. Seeing her is more powerful than anything I can say. Her presence changes people. And I believe her success is not only inspiring people, but also saving lives by helping get this treatment approved.
I couldn’t be prouder to say my daughter is the first child in the world with an immune system trained to beat leukemia.
Tom Whitehead is the father of Emily Whitehead, the first child to receive CAR T-cell therapy treatment for cancer. He started The Emily Whitehead Foundation to help raise money for cancer research. Novartis, which makes the CAR T-cell therapy given to Emily, is a sponsor of the foundation’s Believe Ball, which raises money for children’s cancer research.
Emily and Dr. Stephen Grupp were named WebMD Health Heroes in 2013.