Whenever his lower back started to throb, Triston Tate knew a full-blown pain crisis was about to hit as a result of Sickle Cell Disease (SCD).

The pain would spread to his knee or arm, pulsing like a heartbeat. Prescription pain medication didn’t help. Episodes were severe enough to send Triston to the hospital on a regular basis.

Not anymore. Thanks to a research study underway at Cook Children’s and other sites nationwide, Triston received a particular type of genetic therapy for people with SCD. It isn’t a cure – but for Triston, the therapy has been life-changing. His cycle of pain and frequent hospitalization has stopped.

Back in September 2023, Triston’s health care team at Cook Children’s mobilized stem cells in his bone marrow to the bloodstream. Those stem cells were collected and sent to a lab on the East Coast, where the therapy manufacturer, Editas Medicine, Inc., made some tweaks in a process called gene editing.

Then in January 2024, Triston’s modified stem cells were ready. During an infusion at Cook Children’s, those cells entered his body to help make healthier blood.  

 “They took a part of me, made it better, then gave it back to me,” he explained.

The 22-year-old Burleson resident became the first patient at Cook Children’s to undergo the treatment as part of the RUBY Trial. The trial’s second patient at Cook Children’s received the therapy in July 2024, while three others have been approved to participate.

“The prior clinical trials and current experience have been very encouraging regarding the benefit of this treatment for our patients with severe sickle cell disease,” said Cook Children’s hematologist Clarissa Johnson, M.D.  “The focus on developing new treatments and improving current treatments gives us hope for the future in sickle cell disease care.”

The RUBY Trial measures the safety and effectiveness of an experimental technology for editing the genes of people with SCD. Enrollment for new patients ages 12 and up is closed. But the company anticipates recruiting patients 11 and younger, Dr. Johnson said.

Triston met the criteria for the RUBY Trial because he had one pain crisis after another. He estimates he was hospitalized at least 100 times. He couldn’t work or go to college. 

“It just got so bad,” he said. “I was hurting, taking pain medicine almost every day and being in the hospital every month for like two years straight. It was getting old. I needed to make something happen.” 

Still, he hesitated at first. He was reluctant to spend a month or so of recovery in the hospital, which the treatment requires. But Triston eventually signed on because he wanted fewer SCD complications. 

After his cells were collected and modified, they were shipped back to Fort Worth on dry ice.

Gretchen Eames, M.D., Medical Director of the Cook Children’s Stem Cell Transplant Program, oversaw the next step: chemotherapy followed by an infusion process that put Triston’s edited cells into his body, traveling to his bone marrow. 

“Patients require chemotherapy before the cell product infusion in order to ‘make space’ in the bone marrow for the manufactured cells to move in and take hold,” Dr. Eames said. “After four days of chemotherapy, the cells arrive frozen at the bedside and are then thawed out in a warm water thermal bath and then infused.”

Chemotherapy caused Triston’s throat to be sore, the only side effect of chemo he noticed. In the weeks after the transplant, he slept a lot, played card games with his mom, and passed the time on the PlayStation at Cook Children’s . He was discharged from the hospital once he could consistently create white blood cells and no longer needed frequent transfusions of red blood cells and platelets.

Like other participants in the RUBY Trial, he’ll be monitored by doctors for two years.   

Seven months later, Triston reports he’s not had another pain crisis. Now he has more energy, a job and can keep up with his friends. He described the treatment as simple, smooth and successful.   

“It works,” he said. “I don’t even take pain medicine anymore,” he said. “It’s a blessing I don’t have to go through that pain anymore.”

Dr. Eames said gene editing trials such as the RUBY Trial have been transformational in improving the quality of life and overall outcomes for patients with SCD. 

“We are so excited to be able to offer this exciting new cellular therapy here in Fort Worth, close to home for many of our patients -- and to be soon able to offer it to many more patients throughout Texas and our neighboring states,” she said.

FACTS ABOUT SICKLE CELL: 
September is National Sickle Cell Awareness Month. Here’s what you need to know:

• An estimated 100,000 people in the United States have SCD, an inherited disorder.
• A genetic mutation causes the red blood cells to curve like crescent moons rather than round discs.
• The cells get clumped up in the blood vessels instead of flowing smoothly, which causes pain and can lead to anemia, organ damage, stroke or other complications. 

RELATED STORIES:
 Clinical Trial Aims to Treat Sickle Cell Disease with Genetic Therapy

Jakera wasn’t in pain but her mom, Tiffany Ferguson, noticed her little girl had lost range of motion on her right side.

“She couldn’t open her hands, she couldn’t lift her arms. I immediately called her doctor and told her that Jakera couldn’t move anything on right side. She told me it sounded like a stroke and to call 911,” Tiffany said.

An ambulance rushed Jakera to Cook Children’s where she was treated for a stroke and diagnosed with moyamoya, a more serious disorder that developed from her sickle cell disease. Moyamoya can occur when sickled red blood cells cause repeated damage to the blood vessels in the brain. The blood vessels begin to tangle and create a “puff of smoke” appearance on an MRI.

Moyamoya can lead to more stroke activity and typically requires surgical intervention to improve blood flow around damaged blood vessels. Jakera’s medical team decided to intervene before another stroke could occur.

Jakera had revascularization surgery in February 2019, a procedure where surgeons flipped the protective covering on top of the brain (the dura) to encourage the main artery there, the middle meningeal artery, to create new collateral arteries into the brain.

Following the surgery, Jakera now receives blood transfusions every four to six weeks.

Along with interventions such as blood transfusions, sickle cell disease is also managed by medications and potentially a stem cell transplant for long term success against the disease. But finding a donor can be seemingly never ending. Physicians will look to find a match from siblings and family first before trying to find an unrelated donor, according to Cook Children’s physician Clarissa Johnson, M.D. Jakera has not yet had a stem cell transplant, but her family remains hopeful she’ll find her match.

“A stem cell transplant is a cure for sickle cell disease because you’re basically putting new stem cells in the body,” Dr. Johnson said. “Red blood cells are made from stem cells so if you put new stem cells in the body that don’t contain Hemoglobin S, you are creating a situation where you no longer have stem cells that make sickle cells.”

With sickle cell, stroke and moyamoya, Jakera has endured her share of hardships, but it wasn’t until she was diagnosed with Bow hunter’s Syndrome that she was affected by just the turn of the head.

“She started blacking out three to four times a day. It happens [when she turns] like a bow and arrow hunter when they have to turn their head to the side to aim,” Tiffany said. “She was born right handed but when she turns her body to the right, it cuts off the circulation to her brain so she actually switched hand dominance to the left.”

Jakera had a spinal fuse from the back of her neck down to her shoulders to limit the amount she can physically turn. She can no longer turn more than 90 degrees to ensure there is enough blood reaching her brain. While this is not a cure, her physicians believe it will relieve the loss of consciousness.

Jakera is not the only one in her family impacted by sickle cell disease. Her father also faces medical difficulties.

“There were days where I was running from Cook Children’s to Harris and back to Cook Children’s so that I could be with both of them during their crises,” Tiffany said. “Everyone in the family is learning to deal with it because we know it isn’t going to go anywhere right now, but they understand the most important thing is making sure they [Jakera and her father] are OK.”

Four major diagnoses and two surgeries in five years could seem daunting to most, but Jakera has flown through it all with ease, and has even made the local cheerleading squad.

“She’s like a little woman,” Tiffany said. “She takes it and keeps on moving. The stroke limited her a lot but the squad has been so good to her. She always pushes through.”

First, there’s a seven-year age difference. Then, Garrett is married and an operations manager for a company in Las Colinas, while TJ is a single, free spirt and an aspiring actor working summer stock in Kentucky with dreams of Broadway.

But get past the surface and you will find Garrett and TJ share a bond like never before – one has been saved by a bone marrow transplant and the other has provided that life giving donation to someone else.

Their story begins on an early Saturday morning in February, 2013. TJ was a junior in high school. He felt more fatigued and dizzy while performing, but felt it was probably just due to his hectic rehearsal schedule. TJ later told his mother, Sherri that he didn’t feel well and when his symptoms persisted they looked into it further.

TJ went to visit his pediatrician, Tom Rogers, M.D., on a Thursday and had blood work done on a Friday before he was scheduled to go to church camp. The next day, TJ received a tap on his shoulder. It was his parents, Tom and Sherri, telling him that Dr. Rogers called and wanted them to go to the Cook Children’s Emergency Department immediately.

“At that point I was freaking out a little bit,” TJ said. “I was definitely freaking out because I didn’t know what was going on. One of the weirdest moments was when we went to the emergency room and they gave us a private room, which generally doesn’t happen in the ER. By private room, I mean door shut kind of room. The doctor came in and asked if I knew what was going on. The doctor said, “I can tell you right now you either have aplastic anemia or leukemia. At that point, I didn’t even know what aplastic anemia was.”

Shortly after being admitted, TJ was diagnosed with aplastic anemia, which is a blood disorder where the body’s bone marrow doesn’t make enough blood cells. The disease affects approximately three in a million people.

And just like that, TJ’s life was turned completely upside down.

He was admitted to Cook Children’s and immediately pulled out of school for his junior year at Keller high school. His siblings weren’t a match for bone marrow transplant, so he initially underwent a six-month immune suppression therapy in hopes this would provide the cure for his disease.

“I was really hopeful that TJ would have a matched sibling, even though we know the chances of a sibling matching are only 25 percent,” said Richard Howrey,M.D., medical director of the Aphresis Program at Cook Children’s and the associate medical director of the Stem Cell Transplant Program. “When we got the disappointing news that TJ didn’t have a match in the family, we felt our best chance for cure was to give standard immunosuppressive therapy, in part because the high risk of serious complications associated with an unrelated bone marrow transplant.”

At the end of that timeframe, shortly after he went back to school for his senior year, his doctors told TJ the immune suppression therapy was not the long term answer they had hope for and he needed an unrelated donor bone marrow transplant.

For TJ, all of these life-changing (and life-saving) events couldn’t have come at a worse time for a young man with big plans for his future.

He had already started planning for college and a degree in musical theatre. He had 19 college theatre auditions scheduled for November, had been cast in a lead role in his high school’s musical, was rehearsing for the high school fall show, which was to be performed at the end of October and was directing his senior play.

TJ went to his doctors and asked for enough time before receiving his transplant to finish at least two of his high school obligations. Cook Children’s and Be The Match found a 10 for 10 match for TJ. His transplant took place on Nov. 8, 2013, a day he now celebrates as another birthday. TJ finished his responsibilities with proceeds from his senior directed play going to Cook Children’s.

Prior to the surgery, TJ’s parents hosted a “Shaving TJ’s Head Party” at their home. In addition to family and friends, a representative from Be The Match was invited to come and swab people who were interested in signing up with the registry. The age range for donors at the time was between 18 and 40 years of age, which eliminated many of the guests who were high school age or parents of high school students who were older. But Garrett decided to sign up and was swabbed that evening.

“The event was a party. It was fun. It was celebratory,” Garrett said. “I weighed the cost of swabbing, but at the time I didn’t fully know the weight of that decision. I really wanted to get on the registry though. The thought going through my head was when TJ was diagnosed, they tested me and our younger brother, Austin, but neither one of us was a match. I found out that it’s very common for siblings to not to be a match. That’s sad being the older brother and I can’t give TJ what he needs to get healthy. I thought, ‘I would love to be able to do this for somebody else’s brother.’”

TJ has a lot to celebrate now. He’s come a long way from the days of chemotherapy, radiation treatment and 108 transfusions.

“During that time it was very much about what do I have to do now to pursue theatre in the future,” TJ said. “I had to set aside acting for a while to get healthy, but I think it was the right choice. It worked out perfectly.”

TJ is currently entering into his junior year at Coastal Carolina University in Conway, S.C. He is pursuing a degree in musical theatre and is in Italy this fall studying Physical Theatre. As it turns out, TJ donor was from Germany. If both parties agree, donors and recipients have the option to meet. TJ and his donor have contacted each other through Facebook and texts, and plans are underway for TJ and his donor to finally meet face to face during his trip abroad.

“We had pretty much known from the beginning that we would want to keep in contact with this person,” TJ said. “I had to wait two years and then it was sign this form and sign that form. He had to sign a consent as well. He actually reached out to me first. I will meet him in the fall and it’s going to be awesome. It will be really interesting. I’m excited for sure. He seems very down to earth and very understanding.”

So as TJ’s transplant story was coming to the kind of happy ending any actor would want to play, Garrett’s story was just beginning.

Earlier this year, Garrett received a call to say he was a match for someone. Garrett admits to becoming nervous as he read about the procedure. As an analytical person by nature he couldn’t help but think about all the details of the procedure. Plus, he’s not a fan of needle sticks or blood. But after talking and praying with his wife Sheila, he knew this was something he sincerely wanted to do – to pay it forward for the help TJ received.

“It was such an incredible coincidence that Garrett wasn’t able to help his brother, but then had the opportunity to save the life of a complete stranger,” Dr. Howrey said.

In July 2017, Garrett drove to Cook Children’s for the first time since TJ had been discharged. Garrett said it brought back a flood of memories of when his younger brother was a patient there.

He arrived at 6 a.m. for the 8 a.m. procedure. The last thing Garrett remembers was him laughing and saying to the anesthesiologist, “I like this guy.”

The medical team drew more than a liter of bone marrow and everything appears to be a success. A year will go by before Garrett will have the opportunity to meet the person who received his bone marrow.

“I would love to meet that person,” Garrett said. “TJ had to wait two years to get in contact with his donor because he was outside the United State. In the U.S., it’s only a year. So my wife and I are definitely looking forward to making contact when that time frame’s up.

Garrett said his soreness continued for a month or so, but he has since returned to a 100 percent and he’s so glad that he took the time out to swab his cheek at TJ’s party.

“TJ and I have a quite an age gap between us,” Garrett said. “I remember growing up, playing video games and my younger brothers wanting to hang out or whatever with me. I thought they were such pests and I would get upset with them. But seeing them grow up and be in high school, I thought I want to be more a part of their lives. Then seeing TJ go through all of this, I was just like, ‘Man, I really want good quality time with him. It made me want to be closer to him.”

As they sit across from each other talking about their experiences, there’s a brief pause. Neither looks at each other but the feeling is there. They are closer than ever before … a perfect match.

The transplant program started at Cook Children’s under the direction of Paul Bowman, M.D., with a goal of keeping patients in need of transplant at the medical center instead of sending them across the country to other transplant sites far away from their home.

Over the last 30 years, thanks to burgeoning technology, participation in clinical trials and being a National Marrow Donor Program center for transplant, collection and donation, Cook Children’s has become the third largest transplant program in Texas.

It has grown from only a handful of matched sibling transplants into approximately 40 transplants annually.

“Where a transplant 20 years ago would have been a treatment of last resort, this is absolutely not the case today. It is now a very intense treatment we are offering patients early on in their therapy with the hope that we can save more children and they can live long and productive lives,” said Gretchen Eames, M.D., M.P.H, medical director of Cook Children’s Hematology and Oncology Center.

The marrow inside our bones is responsible for making the blood cells our bodies need. When this process is interrupted or fails, it can cause a wide variety of health problems, some of which can be life threatening.

The hematology and oncology team ask families to think of bone marrow as a big factory that makes billions of stem cells for your body to use:

• Bone marrow is the spongy stuff inside all of our bones.
• The stem cells are stored in the bone marrow until they are needed.
• When they grow up, the stem cells leave the bone marrow and move into the blood where they can do their jobs.

If a child’s bone marrow factory is not working right because of cancer, a blood disorder or other kind of damage, a stem cell transplant will replace the “old” factory and give him or her a brand new factory that works the right way.

Doctors decide who will be the source of stem cells for the child’s transplant. The decision is based on the child’s diagnosis and special blood testing called tissue typing. In general there are two types of stem cell transplant:

Autologous:

• The child will receive his or own stem cells and the transplant is needed to support the patient during very intensive chemotherapy.
• This type of transplant is often used for children with neuroblastoma; this type of transplant might also be used for Hodgkin lymphoma or certain types of brain tumors.

Allogenic:

• The child will receive stem cells from someone else who has a similar tissue type. This donor might be a brother or sister or maybe someone the child has never met through a donor program.
• This type of transplant is often used for children with leukemia, lymphoma, severe aplastic anemia or other blood disorders, certain metabolic disorders or certain types of inherited problems of the immune system.

Through the program’s affiliation with the National Marrow Donor Program (NMDP), Cook Children’s has access to donor registries from all over the world. These registries can find donors for bone marrow, peripheral blood stem cell and umbilical cord blood transplants.

The Cook Children’s Bone Marrow and Stem Cell Transplant program is actively involved in clinical research as well. The program is a member of the Center for International Blood and Marrow Transplant Registry (CIBMTR), the Pediatric Bone Marrow Transplant Consortium (PBMTC), Clinical Trials Network (CTN) and the Children’s Oncology Group (COG), as well as early investigational consortiums, including New Approaches for Neuroblastoma Therapy (NANT) and Therapeutic Advances for Child Leukemia (TACL).

“Seventy percent of patients in need of a bone marrow transplant don’t have a match in their family,” Dr. Eames said. “If they don’t have a match within their family, the only recourse is to look through national and international donor registries to find a match. In 1993, when we did our first unrelated donor transplant, that was a big step for our program and allowed us to treat more kids right here at home. Nowadays, nearly 50 percent of our transplants are from unrelated donors.”

At Cook Children’s, the primary patient diagnosis is Acute Lymphoblastic Leukemia (ALL) with 60 percent of the patients having a malignancy of some type and 40 percent being nonmalignant disorders, such as bone marrow failure syndrome, immune deficiencies, red blood disorders and metabolic disorders.

For more information:

Certain diseases and treatments can deplete a child's healthy stem cells. Sometimes the body needs help to replenish those cells. When this happens, your child may require a very complex process called a stem cell or bone marrow transplant. Click to learn more about the program.

Since 1986, Cook Children's Bone Marrow and Stem Cell Transplant program has performed nearly 1,000 transplants in children with cancer, blood disorders or inherited conditions. That's what makes this program one of the more diverse and experienced pediatric transplant programs in the Southwest.

Cook Children's is a member of:

• The Center for International Blood and Marrow Transplant Research (CIBMTR)
• Pediatric Blood and Marrow Transplant Consortium (PBMTC)
• The Children's Oncology Group (COG) stem cell transplant section.
• We are accredited through the Foundation for Accreditation of Cellular Therapy (FACT).

Over the last three years, 30 to 40 transplants were performed every year for a variety of diseases, with leukemia being the most common primary diagnosis.

#Erasekidcancer

September is Childhood Cancer Awareness Month. This month, we're pulling back the curtain to share what Cook Children's is doing to fight pediatric cancer. Stories will be shared to reflect the hearts of our caring professionals and volunteers, the brains of our clinicians and researchers and the courage of our patients and families.

Here are three articles about the kids who fight cancer and the work Cook Children's does to hopefully someday #erasekidcancer for future generations:

When it comes to battling cancer: she rocks!

Tori Pence has loved music since she was 7 years old when her grandfather gifted her with her first guitar. But it wasn’t until she was admitted to Cook Children’s that her passion for music flourished. Click to read her story.

Luke's story

On Tuesday, Sept. 1, Cook Children's began this year's #erasekidcancer campaign for Children's Awareness Month with a ceremonial 1k walk. Fifth grader Luke Lange, who’s in remission for Hodgkin’s Lymphoma, stepped up to the podium and lowered the microphone to fit his 10-year-old height to share what Cook Children’s means to him. We found it so inspiring, we thought everyone should hear his story.Click to read Luke's words. 

Our efforts to #erasekidcancer

The Cook Children's Hematology and Oncology Center works every day on medical treatments and research to help make the blood disorders and cancers that affect children and teens, disappear. Here are some of our efforts to #erasekidcancer: Click to watch great videos about our MIBG program, Bone Marrow and Stem Cell Transplant Program and Complex Blood Disorders and Diseases program.

More about Hematology and Oncology

As a specialty, hematology and oncology was formed because there are so many instances where blood diseases and cancer cross paths. Just like adults, children get cancer, but the cancers children have are very different in many ways than adult cancers. Because of the unique nature of children with cancer, they must be evaluated and treated by pediatric specialists. Our top priority is providing the best care for your child.