Emily Whitehead was born on May 2nd of 2005, a perfectly healthy baby. When she reached five, her parents noticed bruising on her legs, bleeding while she brushed her teeth, and pain in her knees. After a few doctor visits and a trip to the emergency room, Emily was diagnosed with standard-risk pre-B-cell Acute Lymphoblastic Leukemia (ALL), beginning a long and arduous journey towards remission.
Dr. Jim Powell, Medical Director, Hematology/Oncology – Medical Affairs at PRA Health Sciences, shares his experiences treating Emily Whitehead, the first Acute Lymphoblastic Leukemia patient and the first pediatric patient in the world ever treated with CAR T-cell immunotherapy.
Emily started her treatment at Penn State Hershey Children's Hospital, a two-hour drive from their home. Not long into treatment, they discovered Dr. Jim Powell, who was practicing closer to them at the Cancer Care Partnership Pediatric Hematology/Oncology Clinic, an affiliation between the Penn State Cancer Institute and Mount Nittany Health System, in State College, Pennsylvania. Dr. Powell quickly became a trusted part of Emily’s care, beginning a relationship that would last through and well beyond Emily’s journey with leukemia.
Under Dr. Powell’s care and treatment, Emily was the first pediatric patient to receive chemotherapy at the Cancer Care Partnership Clinic. Because he was local, it saved the Whiteheads two-hour trips to Hershey and provided them easy access to medical support. “Whenever Emily would have problems, we could drive over there anytime,” says Emily’s father, Tom Whitehead. “I paged Jim and he made himself available if he was in town and would take care of Emily. He was a big part of our success.”
In 2011, when treatments failed and Emily’s leukemia relapsed, the Whiteheads and Dr. Powell decided to get a second opinion from the Children's Hospital of Philadelphia (CHOP). After her leukemia relapsed a second time in February 2012, they were offered a clinical trial as a treatment option, CAR T-cell immunotherapy for leukemia that had never been tested in children before. With no better options, they decided that this was the only chance they had to save their daughter. In April 2012, six-year-old Emily Whitehead was the first child ever given bio-engineered or reprogrammed T-cells in an attempt to save her from B-cell acute lymphoblastic leukemia. She was the first ALL patient and the first pediatric patient in the world ever treated with CAR T-cell immunotherapy.
Shortly after receiving this novel therapy, she developed a severe complication, cytokine release syndrome. When Emily soon started having trouble breathing, she required mechanical ventilation and was put into a medically induced coma. The physicians told the Whiteheads that they wanted to give Emily steroids in attempt to treat her cytokine release syndrome, but they were previously told that steroids could suppress the infused engineered T-cells, which in turn may lead to therapy failure. The CHOP pediatric specialists had a very difficult decision to make. Attempt to treat the cytokine release syndrome with steroids, which may be the only option to save her life from this complication, but may then render her T-cell therapy ineffective, and Emily’s leukemia could potentially relapse again.
“I couldn't find anybody else to talk to,” says Tom Whitehead. “So, I went down in the atrium of the hospital and called Jim Powell at 3:00 AM. I'll never forget that he answered and talked me through the options and said to tell them to go ahead and do the steroids and get her breathing again.”
This choice helped save Emily’s life. Twenty-three days after her first infusion, she woke up from her coma and the Whiteheads were told that there was no leukemia remaining. “Dr. Powell was one of the first people that I let know,” says Tom Whitehead.
Below, Dr. Jim Powell, Medical Director, Hematology/Oncology – Medical Affairs at PRA Health Sciences, shares his experiences treating Emily and being a part of her journey.
How did you first meet Emily and her parents? What was your primary role in Emily's care and treatment?
I was formerly on faculty and staff at the Penn State Children's Hospital in Hershey, PA, from 2003 to 2008. Part of my role was to start a satellite clinic network throughout parts of Pennsylvania, so that children and families in more rural areas didn't always have to travel as far for outpatient clinic visits. Hershey is in central Pennsylvania, and we took care of many pediatric hematology and oncology patients in the surrounding areas, several hours-long drives from Hershey. One of the satellite clinics we set up was in State College. I eventually transferred to State College full time, where I was a part of a large multi-specialty group named Mount Nittany Physician Group, and Mount Nittany Medical Center, which is the community hospital in our area, and that’s where I met Emily.
Unfortunately, she developed several uncommon treatment complications shortly after starting therapy. One of these complications was a very serious infection in her leg, which required surgery and weeks of antibiotics. After she improved, we resumed her chemotherapy. The Penn State Children's Hospital staff contacted me and asked me to co-manage with them, so that’s when I first met Emily and her family, about a month after her diagnosis. Initially, Emily needed a lot of supportive care. I managed the administration of some of her regular chemotherapy over the next year and a half or so until she relapsed.
When she relapsed, I took over her local care and I worked closely with the staff at Penn State Children's Hospital which is about 100 miles away. Here in State College, the Whiteheads could just come travel down the road to see me. This was the exact reason why the satellite clinics were developed in the first place—to help ease the burden on families of children with serious illnesses. This particular satellite clinic ended up transitioning into a more permanent pediatric oncology hematology clinic in State College.
What was it like working with Emily? How did it feel to see a new treatment positively impact her life?
Emily and her family made my job easy. They always followed instructions precisely and never missed any appointments. She is a very strong and courageous young lady. She never complained. Emily is a remarkable young woman, and Kari and Tom are amazing parents.
I diagnosed Emily’s relapse. She actually came in for a routine blood count and unfortunately there were some suspicious cells. Unfortunately, Emily would not go back into remission despite a few rounds of chemotherapy. That's when her family continued to come back to see me—I would see them weekly or every other week. After she relapsed, I suggested that the family get a second opinion and that's how she got into CHOP. When the T-cell immunotherapy trial opened up there, the timing couldn't have been more perfect. She was running out of treatment options when the CAR T-cell therapy trial was approved to start.
It was very exciting because she was the first pediatric patient to receive this therapy. But obviously, it was also a time of great concern because Emily clearly had a highly resistant, aggressive leukemia. I would get updates often on her progress. After her complications resolved and her prolonged stay in the pediatric intensive care unit ended, she went back to regular inpatient care and then a short time later she improved to the point that she was cleared for discharge to home from CHOP. I was in close contact with the pediatric oncologists at CHOP after she was sent home, and we provided much of her care locally from that point on, with interval follow up trips to CHOP.
The experience to be a small part of the team that cared for Emily was very special. I am so thankful that this new therapy was successful. I'll always treasure the fact that I had the opportunity to work with the physicians and researchers at CHOP and the University of Pennsylvania. I really admire and respect the research team who developed this remarkable novel therapy. It was very exciting back then when CAR T-cell immunotherapy was a new type of treatment. It's gaining more momentum as a potential cure for several different types of adult and pediatric malignancies.
How did this treatment differ from existing therapies used to treat patients like Emily at the time?
This is a targeted, bio-engineered therapy, designed to primarily attack a specific protein or marker on the leukemia cells. Conventional chemotherapy attacks rapidly dividing cells, so it can also have toxic effects on the body’s native cells as well as the cancer cells, leading to unwanted side effects. Since this was a novel therapy, the doctors at CHOP were in “uncharted waters,” so to speak. The investigators at CHOP had an idea of the potential toxicities and potential response to treatment, but since Emily was the first child treated with this therapy, the overall effects were unknown.
What are some of your biggest takeaways from this experience working with Emily?
I’ve learned the importance of perseverance and never giving up. Emily and her parents never quit searching for a cure. Drs. June, Grupp, and Levine (the researchers at CHOP/University of Pennsylvania) also persevered to develop and perfect this cutting-edge therapy after working on it for decades in the lab. I also find the science behind this therapy to be fascinating and almost unbelievable. It involves basically re-training and modifying the patient’s own white blood cells to re-direct them to fight their cancer.
What do we still need to learn and strive for when it comes to CAR T and treatment advancements?
There are still some potential short-term and long-term toxicities involved with this therapy. Additionally, some patients are not fortunate enough to have a sustained response or remission of their cancer following CAR T-cell therapy, and they ultimately relapse. Ongoing trials are vital to perfecting the treatment so that it has an even higher long-term success rate with fewer toxic effects.
Can you tell us about the decision making/criteria/reasoning used to inform your decision that this treatment was the best option for Emily’s care?
Basically, there were no other options. Emily was not responding to re-induction chemotherapy after she relapsed, and her leukemia was very aggressive when it relapsed. Even if we were able to get her into remission, her best conventional chance of cure was a bone marrow transplant, but since she does not have any siblings, a related match for a stem cell transplant was not an option. For a variety of reasons, the one unrelated donor from the bone marrow donor bank who was a good match to Emily ultimately was not able to be a donor. The decision to pursue CAR T-cell immunotherapy was basically made for Emily and her family, because there were no other options available at the time, and she did not have much time to wait. The decision had to be made quickly.
How is Emily doing today?
Emily is doing remarkably well. She is a straight “A” student in her first year of high school. She is dedicated to staying physically fit and has taken up running as her primary form of exercise. She has even started running in long distance races.
Emily and her parents are amazing ambassadors for T-cell immunotherapy and the initiative to develop new types of pediatric cancer treatments. They’re dedicated to spreading the word about how critical it is to continue to develop new treatments for childhood cancers. Emily and her parents speak with families all around the world who are considering T-cell immunotherapy. They tell them what to expect and provide the families with support during their fight against cancer. They’ve helped many families over the years. The Whitehead family’s efforts were very instrumental in the FDA’s approval of CAR T-cell therapy for children. This was the first time the FDA approved a cancer treatment in children before approval in adults.
You eventually left clinical practice and decided to join clinical development. What led to your decision to do this? How has your perspective of clinical research as a care option changed since making this move into clinical development?
There were multiple reasons that led to my decision, but the primary reason was the changes that have occurred in clinical medicine over the past few decades made it difficult for me to practice medicine the way I was trained and wanted to practice. I found that it was becoming increasingly difficult for me to care for the children and their families the way that I wanted to in the current model of medicine. Additionally, I felt that I could make more of an impact in my role as a medical director. I miss and will continue to miss caring for the children and supporting their families, but for a variety of reasons, the time was right for a change.
I had some perspective regarding clinical trials and drug development during my years as a clinician because I enrolled many children in trials. I’ve done some basic science and clinical research. Since my transition to PRA and a career as a CRO medical director, what has impressed me the most was discovering the extensive “behind the scenes” work that goes into a successful clinical trial, including the amount of detail involved. I’m very impressed by the team approach, including the efforts of so many different professionals and their roles, such as business development, budgeting, safety, medical monitoring, statistics, data review, etc. I never realized how extensive the process of drug development was and the large team involved with many different roles. It’s been fascinating to learn more about the process.
How does your experience working so closely with patients for so many years impact your work at PRA?
I frequently remind myself to continue to have empathy and compassion for the patients/individuals in my new position as a medical director with hematology and oncology clinical trials. Now it’s empathy and compassion “from a distance” so to speak, but nonetheless I think it is important to keep that perspective. When reviewing data, the patients have subject numbers, so it’s easy to get caught up in the numbers and data and lose sight of the fact that each subject is an individual going through a very tough time in their lives. Because I was involved in clinical medicine for so many years, I want to always keep in mind that each subject is an individual person and not just a number.
Our experts pull meaningful clinical practice experience into their everyday clinical development work and never forget that the work we do each day is ultimately for the patient.
The Whiteheads continue to advocate for patients all across the world and continue to share their story through their book, Praying for Emily. Once costs have been covered for publishing, all profits will go towards funding the Emily Whitehead Foundation.
Missy Hansen, Pediatric Strategy Liaison, Center for Pediatric Clinical Development
We measure our HQ with actions, experiences, and intentions. Nowhere is that more evident that in the stories of our subject matter experts. We want…
The Impact Of Contraceptive Requirements On Pediatric Clinical Trials
Contraceptive requirements applied to women of child-bearing potential participating in clinical trials also apply to children participating in…
Perspectives and Stories from the Pediatric Collaboration Team
The Center for Pediatric Clinical Development (CPCD) is comprised of 11 dedicated staff members who share diverse healthcare and research…