Skylar Jones, 15, is the first patient to undergo CAR T-cell therapy at Cook Children’s. She was just 9 years old in 2014 when she was diagnosed with B-ALL. Despite undergoing intense chemotherapy, Skylar’s cancer relapsed in 2017 and again in March 2021.

CAR T-cell therapy was approved by the Food and Drug Administration (FDA) for use in refractory and relapsed B-ALL in 2017. Cook Children’s began offering the treatment for those who qualify in November 2020. For Skylar, the timing was perfect and meant she would not have to travel outside of Fort Worth and away from her long-time physicians at Cook Children’s to receive this potentially life-saving treatment.

“Had she relapsed six months earlier, we would have had to go out of town for this,” said Deby Jones, Skylar’s mother. “So for Cook Children’s to have this now and for families to not have to move to get this treatment for their kid, I mean, there’s tons of families that will benefit from this.”

Skylar’s necessary but difficult chemotherapy treatments wreaked havoc on her body. Mouth sores, headaches and rashes were common side effects. An infection required part of her lung to be removed, and the impact of chemotherapy on her bones and joints made it impossible for her to play basketball, her favorite sport.

When Skylar relapsed a second time in March, it was clear that chemotherapy was no longer the most effective course of treatment in her case. Her cancer required a different approach. This made her a good candidate for CAR T-cell therapy.

“CAR T-cell therapy is a treatment option for children with refractory B-ALL, meaning they haven’t responded to initial treatment, or kids whose cancer has relapsed,” said Holly Pacenta, M.D., a hematologist/oncologist with Cook Children’s Cellular Immunotherapy Program.

In this cell-based immunotherapy, the patient’s own T-cells, which help the body fight infection, are harvested from their blood and sent to a lab where they are reprogrammed into CAR T-cells to target their leukemia. The CAR T-cells are then reintroduced into the patient’s blood stream. From start to reinfusion, the process takes about four weeks.

The therapy begins with a procedure called leukapheresis where the healthy T-cells are removed from the patient’s blood. This takes about four to six hours and the patient is able to go home from the hospital the same day. Once the lab receives the cells, it takes a few weeks to reprogram them into CAR T-cells. Just before the CAR T-cells are reintroduced, the patient undergoes a short round of chemotherapy to lower the number of other T-cells in the body. This gives the CAR T-cells a better chance to activate inside the body to kill cancer cells. After chemotherapy, the patient returns to the hospital to be infused with their new CAR T-cells and is able to go home the same day.

Unlike chemotherapy and radiation, which affects the fast growing cells in the body, this treatment is a targeted approach that kills only the B-cells. The duration of treatment is shorter, too.

“B-ALL cells have specific markers that aren’t located on many other cells in the body, which is the part about it that’s unique,” Dr. Pacenta said. “The reason this has been so successful is because we are able to target one marker on leukemia cells to fight the disease.”

The remission rate after CAR T-cell therapy is 80%.

“Every patient is different, but we know that, in some children, this can be a long-term cure, meaning they don’t need to receive any additional treatment,” Dr. Pacenta said. “Some research studies have found that the CAR T-cells will persist in a patient’s body for as long as three and a half years, but it’s likely that the cells will be around for much longer.”

For Skylar, CAR T-cell therapy was as different from chemotherapy as night and day. She was fortunate to not experience any of the side effects associated with the therapy.

The side effects of CAR T-cell therapy include allergic reaction during and immediately following the infusion. In the weeks after, patients are closely monitored for neurotoxicity, as well as cytokine release syndrome, where the immune system becomes overactive due to the multiplying CAR T-cells releasing large amounts of chemicals called cytokines into the blood. These side effects can be very severe, with some patients requiring admission to the intensive care unit.

Now three months post infusion and Skylar's cancer is in remission. She will undergo more testing at six months post infusion, which will give her a better idea of her potential for long-term remission.

As for the possible impact of this type of cell-based immunotherapy on cancer, doctors are hopeful it could one day replace traditional treatments.

“I think there may be a day in the future where cellular therapy does replace chemotherapy, surgery and radiation for people with cancer,” Dr. Pacenta said. “But I think we are still a ways off. This therapy has been very successful initially but we still have a lot of work to do.”

“Our Joey likes to move at his own pace,” said his mother Denise Belles.

Joey arrived at Cook Children’s in June of 2019. What his family thought were headaches from recent growth spurts and allergies turned out to be a rare form of cancer called pineoblastoma. A tumor was growing in his head and spinal fluid was building up in his brain.

“At the time, he was going to football camp and loved it. Then we did the CT scan and within days, we got the diagnosis that changed our lives forever,” said Denise.

On July 3, Cook Children’s neurosurgeon Daniel Hansen, M.D. removed Joey’s tumor. Luckily, Dr. Hansen was able to get the entire mass at once. From there, Joey began a strict regimen of proton radiation therapy. In November, he was admitted to Cook Children’s Bone Marrow Transplant Unit to begin stem cell chemotherapy.

“It was really hard on his body,” Denise said. “You deplete them (patients) of everything and start from scratch. But the doctor said to Joey ‘You don’t have a shot if you don’t have the right attitude.’ So we came up with a plan and no matter what, we were going to be positive about it.”

Part of the plan came together after a visit with a physical therapist. She asked Joey to start walking and told him that if he kept track of his progress, he could win a gift card once he hit 10 miles. The incentive is part of a new program at Cook Children’s called ‘Miles in Motion’ which encourages hematology/oncology patients to get moving.

“When I first got here, I was sick and I was moving pretty slow,” said Joey. “We started walking the first day, but I could only do two laps.”

Joey would log his walks on a piece of paper in his room. Soon, the papers began piling up as he started making multiple laps around the unit.

Physical therapist Lydia Robey was part of the team that came up with the idea for ‘Miles in Motion’ as part of a quality and safety initiative to get patients out of their beds and exercising.

“We were brainstorming how to increase activity for this particular group of patients and one of our dieticians pointed out that these kids were losing muscle mass at a far greater rate than they should,” said Robey. “Some of that was due to inactivity, as well as steroids and medications that cause muscle atrophy. We started reviewing the literature and the evidence just became overwhelming of how important exercise is.”

Robey said due to varying blood counts, patients are not always able to do resistance exercise. However, she says walking is always good.

“Everyone agrees that walking is safe, so we wanted to find a way to motivate and empower families and patients to start walking as part of their daily routine,” explained Robey.

Joey and his parents did just that. They committed to walking each day during his four separate hospital stays, which spanned between two and four weeks each.

“For about a week, I did two to four laps a day and then I started getting to 10 and I thought ‘Wow, this is good!’ and then I started doing 20,” Joey exclaimed.

Before he knew it, Joey was walking more than 30 laps at a time, a feat that seemed impossible when he first started.

“There really is a noticeable difference between the first time I saw him and how he is now,” said Haleigh Schreck, one of Joey’s physical therapists. “He walks every single day on his own, no matter what he’s going through treatment-wise. His drive and motivation are very impressive.”

Last week, Joey hit a milestone that not even his physical therapists thought was possible. Joey marked off his 26^th mile in the ‘Miles in Motion’ program, which is equivalent to walking an entire marathon. Walking a marathon was never even a goal for Joey or for the ‘Miles in Motion’ program, but as he got closer to mile 26, he knew he had to go for it.

“We were just like, we’re this close to getting a marathon and I decided ‘I’m gonna do this,’” said Joey.

On Thursday, Feb. 20, Joey made his final laps around the unit were he’d been walking for the past four months. This time, nurses, child life specialists and physical therapists lined the walls. They held up handmade signs and cheered him on. With just five laps to go, Joey took one step at a time, thanking his mom and dad for their support as they finished the journey together.

At the final lap, excitement built as the medical staff cheered his name. “Joey, Joey, Joey!” they chanted until breaking into a loud roar as Joey ran through the finish line, a paper streamer strung across the hallway.

He did it. Joey walked a marathon.

“It was awesome,” he said basking in the wonder of his achievement. “I could have never done it without my family, the whole team we have. Everyone has been so supportive of me. It’s just truly amazing what we have done, me and my parents.”

This wasn’t the first marathon completed by a member of the Belles family. Denise ran the New York marathon in 2010, but she said this one was much better.

“This one is more rewarding,” Denise said. “His training was harder. I definitely feel more fulfilled with his than mine. It means so much more.”

If that wasn’t enough, finishing a marathon wasn’t Joey’s only major accomplishment of the day. It was also his final day of chemo.

“In the beginning, I really thought this was going to be devastating,” said Joey. “But everyone has been helping me and pushing me. With them, I knew I could get through this and do it with power.”

While he did have a supportive family and medical team, Joey also did what his doctor advised and had the right mindset throughout his cancer journey.

“Joey has been an amazing patient to work with,” said Haleigh. “I can’t remember a time when he wasn’t just smiling and having the most positive attitude. He blew all of our expectations out of the water.”

“I’m so proud of him. It shows how if you have the right mindset then you can overcome anything,” his mom said.

With his treatment coming to an end, Joey and his family are making plans for the future. They’re downsizing to a smaller home so they can take more trips together. This summer, they hope to go to Italy.

“We’re a close-knit family. We just love being together, but this has brought us so much closer,” said Denise. “We don’t want to wait to do stuff, like travel, anymore. We enjoy each other and we don’t take anything for granted.”

This is the daunting statistic looming over the heads of the team at the National Marrow Donor Program at Cook Children’s. Each day, this dedicated group of six sets out to see how many people they can enroll in the National Marrow Donor Program, also known as the Be the Match registry. Equally as important, they’re also tasked with contacting, scheduling and counseling donors who have been identified as a likely match.

“We have the potential to save anyone’s life,” said Jamie Kayser, the program’s director. “It’s not just blood cancers, the donors we identify also save the lives of patients with diseases like sickle cell anemia. We find donors for both adults and children.”

For Kayser, her work is more than a job. She’s one of the millions of people who have been deeply affected by cancer.

“When I was 27 years old, my first husband died of leukemia,” she explained. “He underwent two bone marrow transplants over the course of his treatment. I was not a nurse at the time, but going through it with him, I realized how important nursing was and I decided I was going to become a nurse.”

At the time, Kayser vowed to never work in oncology or in a bone marrow transplant unit. She ended up doing both. Her first role was as an oncology bone marrow transplant nurse at Cook Children’s, which she accepted in 1994. Seven years later she took over her current position as the director of the National Marrow Donor Program at the hospital.

“I say I’m doing the other side of transplant,” Kayser said. “There’s the side where the patients receive the bone marrow transplant and then there’s this side where we register donors, find matches for patients and get them ready to give.”

The National Marrow Donor Program is a nationwide registry. For those enrolled, the chance of being identified as the best possible donor for a patient is one in 300.

“There are two ways to donate depending on what the patient’s doctor requests,” explained Kayser. “The most common way is very similar to giving blood.”

Seventy-five percent of donors are asked to provide blood stem cells through a peripheral blood stem cell donation (PBSC) donation. This was the case for 21-year-old Andrew Kozman of North Richland Hills, Texas. He knew the chances of being identified as a match were rare when he signed up for the donor registry in late 2018. During his PBSC donation, he said he was shocked to receive a call just three months after he submitted his cheek swab in the mail.

“You always hear about the people who have been on the list for years and have never received a call,” said Kozman. “I was fortunate to get a call just months later. Everything has moved pretty quickly since then.”

To prepare for his donation, a nurse visited Kozman’s home for five days ahead of the appointment and injected him with a drug called filgrastim. The medication increases the number of blood-forming stem cells in the bloodstream. On the fifth day, blood is drawn and circulated through a machine that filters out the stem cells. The remaining blood is returned to the donor.

“It was just like getting a normal flu shot. I felt a little sore afterwards, but that’s it,” said Kozman. “I never really gave it a second thought. I signed up and found out that someone needed my help. I thought ‘I could sit in a chair for a few hours if it’s going to save someone’s life.’”

The second way to give is through a surgical procedure called a marrow donation or harvest. Only 25% of donors are asked to provide blood stem cells in this manner. This procedure is done at a hospital while donors are under anesthesia.

“Unfortunately, there is a lot of misconception about bone marrow harvests,” said Gretchen Eames, M.D., medical director of Hematology/Oncology and Stem Cell Transplant Program at Cook Children’s. “It is considered a minor surgery. There is no incision; no stitches or sutures are needed.”

During a marrow donation, blood stem cells are withdrawn through two small punctures in the back of the donor’s pelvic bone. Discomfort during recovery varies from person to person, but Dr. Eames says donors are usually able to manage any pain with Tylenol or hydrocodone. Most people will make a full recovery within a week.

Rebecca Proctor joined the National Marrow Donor Program registry in 2014 when she was a college student. Now a Neonatal Intensive Care Unit nurse at a Fort Worth hospital, she was also surprised to receive a call informing her that she was a match for a patient in need of a bone marrow transplant.

“I was definitely a little nervous when I first got the call,” said Proctor. “After agreeing to a marrow donation, I would have days here and there where I would panic. But I knew if this was going to save someone’s life then I could do it.”

Dr. Eames performed the procedure on Proctor at Cook Children’s, which is one of two bone marrow collection centers in Texas and the second busiest center in the U.S.

“We do approximately four to six bone marrow harvests a month,” said Dr. Eames. “Our donors come from all over the place. Many of them live here in the Dallas/Fort Worth area, but we have people who travel here from California, the east coast and the Midwest because they do not have a national marrow donor collection center where they live.”

For Proctor, the process was much easier than she anticipated.

“Besides a minimal amount of soreness, this really hasn’t been a bad recovery,” she said two days after her donation. “There’s some hoarseness in my throat from the breathing tube, but honestly, this is nothing compared to what my recipient has been going through.”

While donors don’t know who they are giving to, they are likely of the same ethnic background since donors and patients need to be a close genetic match. Given the current makeup of the National Marrow Donor Program registry, the likelihood of finding a match for a white patient is 77%. African American or black patients only have a 23% chance of finding a match.

To improve these odds, ethnic diversity among people on the registry needs to increase. To put it simply, more people are needed to join the registry.

“Everyone, when they turn 18 years old, should sign up for the National Marrow Donor Program registry,” urged Kayser. “We believe every patient should have an equal opportunity to find a match, regardless of their ethnicity.”

To increase those odds, Kayser’s team hosts booths at live events in North Texas as well as West Texas to enroll potential donors. They answer questions, collect information and cheek swabs.

Proctor, like most donors, encourages others to join the registry. Having gone through the process herself, she says she is glad she was able to help someone in need.

“This is something I am so proud to say I have done. Looking back, I would do it again in a heartbeat.”

Eleven years ago on March 17, 2003, we were blessed with a beautiful baby boy names Ja'Kiyran McArthur, also known as J.T.

Two weeks after he arrived we received a letter from the hospital where Ja’Kiyran was born, stating he had a chronic blood disorder called sickle cell disease. We were panicked, scared and very young. At our first scheduled visit with Cook Children's Hematology department we were informed that in order for a child to have this disease both parents have to carry either the sickle cell trait or an abnormal form of hemoglobin.

Over the years we have endured countless medical center stays due to sickle cell crisis*, pain episodes, blood transfusions and antibiotic therapy. Often times, we felt hopeless and  therefore received counseling when he was 6 months old.

We have learned to take it one day at a time, remain encouraged and enjoy each good day he has. Our faith teaches us that love, positivity and believing in the miraculous conquers all.

Even though we are exhausted and financially strained when he becomes ill, we wouldn't trade our struggles or this amazingly smart young man for anything in the world. We are extremely grateful to Cook Children's for providing excellent care and showing us and our son love and constant attention. The nurses are amazing and make each stay feel like being at home. The physicians are consistent and persistent when it comes to getting him well.

Everyone works together to create a loving family and superb care. We thank God for our hospital family and their tireless efforts. We want to remind everyone to get informed about this disease and help us to raise awareness and find a cure for all who have to live with it every day.

Mrs. Ashley McArthur

For more information:

• Cook Children's Sickle Cell Center
• What is sickle cell disease?
• Who gets sickle cell?
• Sickle cell disease
• My friend has sickle cell disease. How can I help?