S5, E01: Emily Whitehead: Pioneer and Hero

Tom Whitehead (00:02):
Emily and I went to the European Parliament and spoke there to share our story, just to put some pressure on these people that make the decisions. When these treatments work and you save a child, you save the entire family.

Emily Whitehead (00:16):
I always like to say, to always smile every day. My dad did that for me and I think it made a really big difference in my attitude and my outlook.

Todd Poley (00:36):
Imagine your daughter, age five has been diagnosed with acute lymphoblastic leukemia. Doctors were initially hopeful about her prognosis, but after nearly two years of chemotherapy, she's relapsed and you're told she's not likely to survive. You have the option of trying a leading edge immunotherapy, but it's only been tested in adults, so doctors can't predict its effects on a child. Would you take the leap knowing the risks involved and allow your daughter to become pediatric patient 1? I'm Todd Poley, and in this episode of Vital Science, we speak with Tom Whitehead and his daughter Emily, about her brave journey as a CAR-T therapy pioneer, the uphill battle pediatric cancer patients face and how the Emily Whitehead Foundation supports cancer families around the world.

Gina Mullane (01:28):
Well, welcome to Vital Science, Tom and Emily. We're honored to have you both here. Would you tell us about yourself and your family?

Tom Whitehead (01:36):
Yeah, I'm Tom Whitehead and I'm Emily's dad and the president of the Emily Whitehead Foundation. And this is my daughter Emily with me.

Gina Mullane (01:45):
Hi Emily. And how would you describe Emily? What are some of the things that make her so special?

Tom Whitehead (01:54):
Well, I would say first of all, all that she went through as a child and to come out of that thriving in life now I would describe her as our hero and very smart, very funny. She likes to have a good time. She does take her grades very serious though when it's time to study. She's very well-read. She's an excellent writer. I'll stop there because she'll get mad at me if I keep going, but every day I wake up thankful that I'm still her dad and Carrie's still her mom and that we are together as a family.

Gina Mullane (02:36):
So tell us a little bit about how the Emily Whitehead Foundation got started. I understand it's of course very near and dear to your heart.

Tom Whitehead (02:47):
So when Emily became the first child in the world to have her immune system trained to beat her cancer, and then they put picture on the cover of the New York Times, just about every news magazine or newspaper in the United States in December of 2012, it gave international media to us overnight and along with all that media attention with a new way to fight cancer, it brought us phone calls from parents and patients from all over the world saying, how did you do that? And how can I do that? And that kind of inspired us to start the Emily Whitehead Foundation because a lot of people had heard about her name and associated her with this new way to fight cancer, and we wanted them to have a way to get more information that we didn't have whenever we were looking, whenever Emily needed it and advocate for those patients to get treated.

Gina Mullane (03:45):
Yeah. And when you envisioned the foundation in those early days, what was your ultimate kind of dream and where have you landed now? Have you kind of met what you set out to accomplish or has it surpassed your expectations?

Tom Whitehead (04:04):
Well, I would say in the beginning we just had good intentions and no idea what we were doing other than we wanted to help people. It's completely gone past our early expectations. And for us, it's amazing that it's still gaining traction and growing and we've been lucky to surround ourself with a great board of directors to show us how to run it like a business because we raised over a million dollars with basically just volunteers. And now we're getting a lot more support because we have infrastructure in place and we're learning how to treat it like a business so we can have a bigger impact on more patients. And again, every day our goal is to help other families have the same outcome that we've had.

Gina Mullane (04:52):
I'd love to hear more about that. So what drives your mission at the Emily Whitehead Foundation?

Tom Whitehead (04:59):
In the beginning, we were just raising funds to put towards more research for less toxic pediatric cancer treatments, and it turned into so many more phone calls from adult patients and just parents from all over the world that we've transitioned now because in the beginning there wasn't much funding for research and now there's a whole lot of funding for research but now the patients that need the treatments can't get to them or can't find them or don't have access. So we've transitioned more now into saying who's the next Emily and how can we advocate to make sure it's available in their country or advocate for them to find a treatment before it's too late.

Todd Poley (05:48):
Is estimated that between 3000 to 3500 children are diagnosed with acute lymphoblastic leukemia or ALL each year in the United States alone. ALL is a type of fast-growing blood cancer that arises from immature white blood cells. These cells multiply quickly crowding out the healthy ones in bone marrow and blood. Diagnosis is typically determined through a combination of blood tests to check cell counts, bone marrow samples to scrutinize cell characteristics and genetic tests to understand the underlying mutations. Although it can be found in adults, ALL is the most common childhood cancer. Children tend to have more resilience against ALL with a higher chance of remission than adults. However, prognosis isn't set in stone. It's more like a weather forecast that can change. Let's hear more about the shifting winds of Emily's ALL experience.

Gina Mullane (06:46):
Emily was diagnosed with acute lymphoblastic leukemia, ALL when she was just five years old. What led to the diagnosis?

Tom Whitehead (06:56):
So really she was healthy the week that she was diagnosed up until the day before, and we were going into Memorial Day weekend of 2010 and early in the week my wife Carrie noticed that Emily had some blood on her gums when she was brushing her teeth. And then the night before she was diagnosed, Carrie called me and said, "I was putting Emily in the bathtub and she has 21 bruises on her body and some of them have been there more than a week and they're not getting better." And she actually said, "I Googled that today and it came up leukemia."

And we were like, oh, it couldn't be leukemia, so if we have to, we'll take her to the pediatrician tomorrow and get her checked, but let's not panic. And then overnight on Thursday, May 27th, she had so much pain in her knees and her legs that she woke up sobbing in the morning saying, "There's something wrong with my legs." And by that afternoon she was on a morphine pump at the Penn State Children's Hershey Medical Center diagnosed with cancer. So that's how we got started. It was feeling good Thursday and by Friday she's on a morphine pump and our lives changed forever.

Gina Mullane (08:11):
So your lives all changed overnight and you probably didn't picture your life taking the shape it has as a patient advocate now, how has this affected your family?

Tom Whitehead (08:27):
Well, I'd say it brought us a lot closer and we decided from the beginning to focus on the positives because the negative stuff could be so overwhelming. So each day we tried to make each other smile and lift each other up and just stick together. And I'd look at it as for the two years, she pretty much spent in the hospital at the age of five and six until she turned seven that we got to spend all of that time together when really we would've been busy at work and she would've been in school. So even though she had some really horrific times, we really became a lot closer as a family and learned what it's like to be together every day and strengthen our bond with each other.

Gina Mullane (09:10):
Emily, you've turned 18 and kind of grown up before our eyes and your childhood journey, witnessed by so many of us, has been one of such remarkable resilience and strength. How has this all shaped who you are today?

Emily Whitehead (09:30):
I think it's a really big part of who I am and a lot of the passions that I have I wouldn't have today based off of all of the experiences we've had. I'm really passionate about the environment and I hope to major in environmental studies in college, and I never would have been interested in that had we not visited all of these different labs and biotech companies, so I think it's really shaped who I am today and I wouldn't change anything that happened.

Gina Mullane (09:56):
And I heard that you met your genetically engineered model, is that true?

Emily Whitehead (10:02):
I did.

Gina Mullane (10:03):
What was that like?

Emily Whitehead (10:05):
It was quite a few years ago, and I had always known that there was a mouse at the Children's Hospital of Philadelphia that they modeled after me genetically, and I had always wanted to meet it because I thought it would be pretty cool. They said there's no way that they could get me into the lab because people aren't usually allowed in. So one day, whenever they were transporting it up the elevator, they stopped on the floor that we were on and just took a little pit stop and they introduced me to it and they were carrying it in a little takeout container and it had some grass inside. So it was pretty cool. And I actually wrote about it in my supplemental essay for UPenn, which is where I'm going this fall.

Gina Mullane (10:48):
Very cool. Did they name it?

Tom Whitehead (10:51):
I think they just called them the Emily mice because they're heard genetically.

Gina Mullane (10:57):
Yes. Oh, that's so cool. Good for you. Well, aside from having a mouse named after you, is there anything else you want listeners to know about you?

Emily Whitehead (11:08):
I think, and this doesn't just apply to me, but a lot of other cancer survivors that we are not just our stories and what happened to us and not just patients, we're people too and we have other interests as well. And being a cancer survivor is just part of what makes us whole.

Gina Mullane (11:26):
Well said. And what is your advocacy role in either raising awareness or providing support for others battling childhood cancer?

Emily Whitehead (11:39):
Yeah, so a lot of the pediatric patients look up to me, and I like to advocate specifically for pediatric patients and adults too. But I understand the pediatric patient experience more, but I think it's important to advocate for less toxic cancer treatments as well, because I spent so much time in the hospital receiving chemotherapy. I had 23 months of failed chemotherapy and 22 days after CAR-T I was cancer free. So I think it's really important to advocate for these new immunotherapy treatments, which is what I'm really passionate about, because as children, I think it's really important to spend less time in the hospital so that you lose less of your childhood as well.

Gina Mullane (12:25):
So Tom, I know the journey wasn't all kind of perfect and predictable along the way. There were some relapses and there were some treatments that maybe didn't have the outcome you were expecting. Can you tell us about that time in her life?

Tom Whitehead (12:42):
Yeah, when we got started we were told this is the garden variety kind of leukemia. It's the most curable kind of childhood cancer that you can get. If you have to have a child with cancer, this is the one you want to have. That gave us a lot of hope from the beginning, but it made it even harder when she relapsed. They had told us if you do 26 months of chemotherapy for a girl Emily's age, that was this protocol. And they said, if you just do what we tell you, she'll grow up and become a grandmother someday and have a happy life. So we tried that in the beginning, just the first couple doses of chemotherapy. Emily developed infections in her legs and almost lost her legs on June 11th, 2012 when we had just got started on... I'm sorry, 2010 when we had just got started on May 28th. So she made it through there and went into the intensive care unit, had a really rough start, and it changed my perspective right away.

And I tell people, because I was devastated the first day I walked onto the cancer floor thinking we shouldn't be here. And then a few weeks later we were celebrating getting back to the cancer floor with Emily and with her legs still intact. So I realized pretty quickly that if you think things can get worse, they really can. But she did get in remission then in the first month, and it lasted 16 months. So we were 10 months away from thinking we were going to be done when she relapsed in October of 2011. So we had routine blood work, she relapsed, and then they told us that day that she would need to get a non-related donor because she didn't have any siblings, and that there was a less than 30% chance of survival. So that's what got us into trying to go to bone marrow transplant, which ended up not happening. And then instead of going home on hospice, we transferred to the Children's Hospital of Philadelphia and the CAR-T cell trial had opened the day before.

Gina Mullane (14:48):
Interesting. So I believe that Emily was the first pediatric patient to receive the CAR-T therapy. Could you tell us about that type of therapy that Emily did receive?

Tom Whitehead (15:01):
Yeah, when I paged them down at CHOP, Dr. Susan Rheingold was the oncologist that took my call and she said this has never been tried before on a child. It's never been tried before in this cancer at all for acute lymphoblastic leukemia, but the science shows it should work, and if you would like to try it, we'll introduce you to Dr. Stephan Grupp who's in charge of the trial, and they'll extract Emily's T-cells, send them off to the lab and train them to recognize and then kill her cancer. So that gave us hope because we knew standard treatment was not going to help her anymore and we weren't ready to go home on hospice. So that led us to transferring down from Hershey to Philadelphia on March 1st, 2012, and that's when they extracted her T-cells and they gave her the harshest round of chemotherapy, which was clofarabine that she ever received, and that completely wiped out her immune system. So she had to stay in her hospital room in isolation for six weeks while they took her cells off to the lab and trained them to become the serial killer cells.

Todd Poley (16:17):
Did he say train them to become serial killer cells? Let's take a moment to review exactly what this means from a scientific perspective. To create CAR-T cells, scientists collect some of a patient's immune cells, genetically modify them in a lab and then infuse them back into the patient. In this process, scientists equip the immune cells to have special receptors called CARs. These CARs act like homing devices. They guide the cells to the leukemia cells and stick to them. Once inside your body, these CAR-T cells or serial killers as Tom called them, go on a mission. They attach to the leukemia cells and activate the immune system to attack them. The best part, CAR-T cells remember the fight and are ready to defend in case the leukemia tries to come back. And that's why after 22 months of failed treatment, it took just 23 days for Emily to achieve remission with CAR-T therapy. Let's hear more from Tom on what this experience was like for Emily.

Gina Mullane (17:20):
And was Emily an ideal patient in some way? Was there something that kind of raised her to the top of this being a good potential treatment for her?

Tom Whitehead (17:32):
Well, what they had told us was they actually did think she was the ideal patient to try it on first because she did not have a failed bone marrow transplant yet, she had all her organs intact, and did not have an infection at the time when they treated her. So they said they really thought the first patient they would ever get to try it on might be in an intensive care unit already dying whenever they tried it. So Emily was actually pretty healthy and walked in to the room the day they gave them to her and Dr. Grupp come in with them all in one big syringe and just pushed it in over maybe less than 10 minutes and said, "Now we wait." And we were like, is that it? And he said, "Yeah, that's all it is. Now, let her immune system take over and we think it'll work."So it was like, the first day they gave her 10% of the dose to make sure there wasn't a catastrophe.

She had really no effect at all and felt fine. And we went in day two and she got 30% of her dose and felt fine. And that evening around midnight, she got a fever. And then the next morning when Dr. Grupp showed up, she felt fine and had no fever so we went in and got the final 60% of the dose and they really said, we just split it up because we didn't know what was going to happen. But then when she got the final 60% of the dose, it was probably the worst part of her treatment that we witnessed because three and a half pounds of her body weight was cancer, and each one of those genetically modified CAR-T cells can kill a thousand tumor cells. And it was killing her cancer so fast that it overwhelmed her system and her kidney shut down and her lungs failed and they ended up putting her in an induced coma in the intensive care unit, and she stayed in that coma for 14 days.

Gina Mullane (19:35):
Oh, wow.

Tom Whitehead (19:35):
So it was a really brutal time, but they worked round the clock to fix it, and that's when they figured out that tocilizumab medicine would slow down the side effects of the CAR-T cells, and that's helped many other patients survive it. And most patients now do not end up in the intensive care unit when they get the treatment.

Todd Poley (19:59):
Although there is a happy ending to this chapter of Emily's story, it was not a foregone conclusion. Emily's parents knew that three adults had been treated with the same CAR-T therapy at Penn the year before, but doctors advised not to put too much stock into this. The patients had a different type of cancer, and children often react differently to treatment. Knowing the risks involved, the Whiteheads decided to proceed making Emily the first pediatric patient ever to receive the treatment and the first patient of any age to receive it for ALL. And although it was not without complication, Emily's experience ultimately led to the first FDA approval of the drug, now known as Kymriah. No other child has received this regimen of Kymriah. According to Tom, the FDA approved 40% of Emily's total dosage or what she received on days one and two of treatment. But Emily served as a brave pioneer as the first of many children that would go on to achieve remission with this groundbreaking treatment.

Gina Mullane (21:02):
It's just incredible to hear that after such a long and brave struggle. Emily is where she is today. What does her prognosis look like now?

Tom Whitehead (21:12):
Well, what we've found out since then is when kids qualify for this treatment now that are going to die, they can usually get them into remission around 90% of the time. And the patients that are done like Emily after this treatment is somewhere between 50 and 60%. But when you're a parent living through that, that's a much higher success rate than a bone marrow transplant. I just wanted to say that we are hoping as a family and Emily especially, that this becomes a frontline treatment someday for standard risk kids with ALL, so they don't have to go through the chemotherapy. When it works, it's much [inaudible 00:21:50].

Gina Mullane (21:50):
I can understand why, given what these kids go through. What do you think is needed to ensure that patients, children, and adults really have access to new therapies like this?

Tom Whitehead (22:03):
Well, we try every day to help improve that. In the beginning, all of the patients had to come to Philadelphia. We've done everything we can to collaborate with other foundations to get it to other hospitals to start with. And then we found when we're in the media, it actually helps get it approved in other countries that might have a national healthcare system. So Emily got to travel to Oslo, Norway and meet the first child that was treated in Norway. We've really advocated too, because I've talked to a lot of parents in South America who are just sobbing because their kids would absolutely qualify for the treatment and they just can't get to it. But again, the United States too, we did everything we could do to help get that FDA approval in five years because then the insurance companies are covering it, which makes it much more accessible to patients within the United States as well.

So from our end, not enough people are getting it yet, but we're going to continue to do everything in our power to try to make a difference and we do that. Sometimes it's just help an adult patient get a flight from San Francisco to Philadelphia to get treatment or help someone line up on Angel Flight with a corporate jet so they can travel not on a commercial flight where they could get sick to get access wherever the treatment is. So we continue to try to do everything in our power and meet with congressmen and meet with government officials from other countries.

Emily and I went to the European Parliament and spoke there to share our story, just to put some pressure on these people that make the decisions to... when these treatments work and you save a child, you save the entire family, and it's a great investment because if she went to bone marrow transplant, there's very little chance that she would've been 11 years since she spent a night in the hospital. And that's where we're at now. So on the backend, it saved money there as far as other complications and possibly other cancers coming from the radiation she would've received to go to bone marrow transplant.

Gina Mullane (24:12):
Over the last decade, you mentioned lots of collaborations that have had to be part of this journey and the successful outcome. Can you tell us a little bit more about the scientific collaborations that you witnessed or were aware happened to enable Emily to get that treatment and for the continued development of drugs that can help children with cancers?

Tom Whitehead (24:41):
Yeah, so I would say what we wanted to see is all these researchers instead of competing to do the same research to get to the treatment first, work together and collaborate. And we've seen that with our collaboration with Stand Up 2 Cancer with Sean Parker's Parker Institute, where they're bringing these top minds together and saying, let's all figure this out together rather than compete to see who can become the first one to get it to market. That way we're not wasting research funds, and maybe this center works on a different cancer than this center's working on, which in the end leads to more people getting treated.

But then on the other hand, we've seen companies like yours that step up because when they do have a success, they can't make enough of the treatment, they can't get it to the patients, and there's not enough people working in the industry to create these new treatments if they even solve one or two of the solid tumor cancers. So we've seen companies collaborate, we've seen mergers happening, scientists working together that didn't work together in the past. And in the end, we've got to meet some of the top oncologists in the world. And I think when you meet them, all they want to do, their families have been affected by cancer too, and they just want to make a difference. And the companies that are all working together and making these treatments are saving lives every day.

Todd Poley (26:06):
The Emily Whitehead Foundation helps further these efforts through fundraising. The foundation hosts a gala where patients and industry professionals can forge connections, swap stories, and celebrate the progress together. In addition, the foundation accepts donations in honoraria for patients to travel and share their stories. Foundation supporters can participate in marathons, attend car races, and purchase treats at local bake sales, all sponsored by the foundation. Donations go toward ALL patients and their families, be it to help a patient travel to a long distance appointment or further ongoing research. Let's hear more from Tom on his vision for the foundation.

Gina Mullane (26:45):
And so what do you hope the legacy will be for the Emily Whitehead Foundation?

Tom Whitehead (26:52):
Well honestly, we have the name recognition internationally as the place where patients go and they're looking for a new less toxic treatment. We hope someday to bring in the funds to back that up so we can have an impact on everybody that reaches out. I still work full-time on the power lines. My wife works full-time in research at Penn State University, and I still take all the phone calls I can, but I'm hoping someday that at least I could be able to do this after I retire from the power lines and do it full time because there's so many more patients that need help.

Gina Mullane (27:30):
Wonderful. So Emily, you're doing well, yes?

Emily Whitehead (27:34):
Yes.

Gina Mullane (27:35):
Feeling good? Well, we're very proud and happy to speak with you. And I'm just so curious if you... and maybe you've had this opportunity with the children that you're supporting, but if you could go back in time and give advice to the five-year-old you or to other children going through similar issues, what would you say?

Emily Whitehead (27:59):
I always like to say to always smile every day. My dad did that for me, and I think it made a really big difference in my attitude and my outlook, and to also just keep advocating for yourself because nobody knows your body like you. And whenever you're younger, I think that can be a little hard to understand but I still think it's important for younger kids and five-year-old me to know that everything was so out of her control, but you still have control over your body and how you feel as well.

Gina Mullane (28:34):
It is so important to focus on what you can control. Did you encounter a lot of things you didn't expect during your treatment and recovery?

Emily Whitehead (28:43):
I don't know if there was anything that I didn't really expect. The only thing that still affects me today is that the treatment removed all of my B-cells, not just the cancers. So I do have a suppressed immune system, and once a month I have to get B-cell replacement, which is just an infusion in my abdomen where it just kind of gives you an immune system to fight viruses. So I didn't know about that whenever I was going through the treatment, but it's just something that I have to do today and I've done it for so long that it's just become a part of my life, but I'm really happy that that is the only thing and I'm super healthy otherwise.

Gina Mullane (29:25):
That's wonderful. Do you have any words of wisdom for those family members who have children battling a childhood cancer?

Emily Whitehead (29:32):
Yeah, I think it's really important that for parents of these children to maintain a positive outlook. I know that it can be hard to kind of keep up that front around your kids, but my parents did that for me and I think it was important that I saw them be strong for me, and I think that that really helped me out. And just to never give up and to always keep fighting as well. I think that's really important.

Gina Mullane (30:00):
Wonderful. So what's next for you? Where are you off to in your next phase of your life here and your next journey?

Emily Whitehead (30:08):
Yeah, so a week from today, I'm actually moving into my freshman dorm at the University of Pennsylvania and it's actually right down the block from where I was treated at the Children's Hospital of Philadelphia. So it's a real full circle moment but I'm really excited for this next chapter.

Gina Mullane (30:25):
Exciting. Congratulations on that. And we heard that you had a pretty adventurous summer. Anything you want to share about your experiences this summer?

Emily Whitehead (30:36):
Sure. So we were able to go to Mexico for 10 days to film for an IMAX movie that will be coming out in the spring of 2024 called Modern Medical Marvels. And for that movie, they wanted to show me thriving today after cancer. And I got to scuba dive in Cozumel and Isla Mujeres in Mexico. And I had some really cool experiences there. And I love photography, so I got to do underwater photography as well. And I took some cool pictures of sea turtles and coral reefs, and I'd always wanted to do that because my dad told me stories when I was little about him scuba diving before I was born. So it was a really, really cool trip and I hope to do it again someday.

Gina Mullane (31:20):
Oh, how fun. Sounds like it was... you made the most of it. Dad, anything to add that we didn't cover today?

Tom Whitehead (31:28):
I would just add at the end that if you're out there and you're the patient or you're the parent of a patient, you got to do all the research you can and then trust your instincts. We tell people that the doctors and nurses and staffs at the hospital are just amazing people, but they have to fix everybody that's in the hospital, and you only have to fix yourself or your loved one that you're there as a caretaker for. So you have to advocate and check everything that they put in them because they are human and they can make mistakes.

But we're very thankful for all the staff and doctors and nurses and teams that saved our family. And now we're very thankful for everyone in your industry that has taking these treatments and making the treatments and getting them there in time before these patients are running out. A lot of them are on hospice and they're on their last hope, but whenever they call me in the beginning and then maybe six months later, they call me again and say, because you took that phone call, it saved my life or my child's life. That's what keeps us going every day.

Gina Mullane (32:34):
Well, thank you both very much. It was an honor and a pleasure having you on our Vital Science podcast.

Tom Whitehead (32:40):
Thanks. Keep up the great work.

Emily Whitehead (32:41):
Thank you.

Todd Poley (32:43):
Tom and Emily Whitehead are co-founders of the Emily Whitehead Foundation. Looking ahead to our October episode of Vital Science, we'll sit down with Bo Bigelow about his daughter's rare disease diagnosis and the community he's building through the foundation for Hao-Fountain syndrome. Did you know that Charles River has a sister podcast, Eureka's Sounds of Science? This monthly podcast shares scientific patient and advocacy perspectives on trending issues in the drug development industry. You can subscribe to Vital Science and Sounds of Science on Apple Podcasts, Spotify, Stitcher, or wherever you get your podcasts. Until then, thanks for listening.