Is Charlotte Becoming a Center for Sickle Cell Treatment? – Charlotte Magazine

By the time Dominick Bibbs entered high school, the episodes of excruciating pain—“crises,” as sickle cell disease patients call them—had increased in frequency from twice a month to twice a week. The pain would target his knees and elbows, sometimes his lower back. At times, it was sharp, stabbing; others, it was a dull, diffused ache.

When the crises came, Bibbs knew he’d be down for days, unable to function. No school, no play, no socializing—just pain medicine and waiting for the agony to ease. He’d already had his enlarged spleen, another consequence of his condition, removed at age 4. It was in his late teens, when the agony seldom relented, that Bibbs started to understand the broader implications of the disease. Tens of thousands of others suffer through similar pain, and most, like him, are Black. Bibbs got clear on two important things: He had an opportunity to help others; and he couldn’t—and almost certainly wouldn’t survive to—take much more of this.

“That’s when I wanted to get into the trial to help people other than me,” Bibbs explains. We’re speaking via Zoom, and his mother, Nicole Searles-Bibbs, has joined us. Bibbs is 22, completing his nursing school prerequisites at Prairie View A&M University in Texas. He mentions that he has one more spring-semester finalexam to take after this call.

“That’s how I got introduced to Dr. (Michael) Eckrich, and Dr. Eckrich talked to me and explained that I could change or be a factor in changing, like, the—what’s the word?”

“Change the world of sickle cell,” his mother interjects. She’s at home in San Antonio, where Bibbs grew up and later underwent the treatment that allows him to live.

“Yeah. ‘Change the world of sickle cell,’” Bibbs echoes. “I didn’t want people to have to go through what I went through.”

A doctor in Chicago discovered sickle cell disease in 1910. Until a generation ago, a diagnosis typically meant a painful life and early death, usually before adulthood. The disease mainly affects Black people. A study published last year examined nearly 75,000 U.S. patient records from 2016 through 2018 and found that 93.4% of SCD patients were Black, compared to 4.8% Hispanic and 1.8% white. About 100,000 Americans have the disease, and it affects one in every 365 Black births, according to the Centers for Disease Control. (By comparison, it occurs in one of every 16,300 Hispanic births.)

Medical researchers have concluded that the sickle cell trait is a genetic modification that evolved to combat the malaria parasite. That’s why people of African ancestry are so susceptible to the disease. Both Searles-Bibbs and Bibbs’ father, Derell, knew they carried the sickle cell trait. But, she says, not having kids “wasn’t an option for us.” They learned their son had the disease three months after his birth.

Eckrich, who oversaw Bibbs’ life-altering treatment in 2021, still keeps in touch. That’s partly out of necessity. Eckrich is in Charlotte now, and Bibbs has to fly in every few months for checkups. But the two have developed a personal bond, too. “He was a doctor,” Bibbs says, “who always told me the truth.”

Eckrich, 47, is a pediatric hematologist and oncologist who began his faculty career in 2012 at Levine Children’s Hospital, now owned by Atrium Health. He left for Methodist Children’s Hospital in San Antonio in 2016 and, over the next five years, led a team that conducted clinical trials to develop a new treatment for sickle cell disease. Levine Children’s recruited him back in 2021, along with members of his team, and Eckrich now leads the Lifespan CuRED (pronounced Q-red) Hemoglobinopathy program in Charlotte. Bibbs took part in the clinical trials in San Antonio, which joined others around the country and led to the Food and Drug Administration’s (FDA) approval of the new treatment in December.

“I just saw him a couple of weeks ago. It’s been a privilege to care for him,” Eckrich says. “He was one of the first few patients brave enough to go through this when there were a lot of unknowns. We explained that to him at the time, and he was committed to make this journey not only for himself but for other folks with sickle cell disease.”

The treatment, which goes by the brand name Casgevy, uses a technology called CRISPR/Cas9 to edit patients’ stem cell DNA in a laboratory before reinjecting it so it can produce healthy blood cells. If the treatment succeeds over the long haul—and early results show promise—it could offer SCD patients a chance at long, productive, agony-free lives.

“Sickle cell disease was the first genetically described disease to be linked to a specific gene, and that gene has a single nucleotide change,” Eckrich says, referring to the basic structural component of DNA. “So, in the whole genome, one nucleotide changes, and you have the difference between a patient who has a debilitating disease for their whole life, or they don’t have anything. We’ve known about that for a long time. So this new technology has always been a little bit of a holy grail.”

The disease has been a terror for parents and children for more than a century. SCD encompasses a range of inherited blood disorders that cause a defect in hemoglobin, a protein that carries oxygen in red blood cells. The defect produces hard, sticky, sickle-shaped red blood cells instead of soft, round, healthy ones. The defective cells die early, which leads to a shortage of red blood cells; and their shape and rigidity make them clog blood vessels, which inhibits blood flow and can cause intense pain, anemia, organ damage, and stroke.

Patients’ odds of surviving beyond childhood began to improve after the Civil Rights Movement highlighted racial disparities in health care. In 1972, Congress approved federal programs for sickle cell education, research, treatment, and detection. Screening for newborns, which allows treatment to begin a short time after birth, was in place in all 50 states by the mid-2000s. In 1998, the FDA approved hydroxyurea, a drug developed for cancertreatment that can halve the rate of severe pain episodes in SCD patients.

But as effective as it is, hydroxyurea only manages symptoms. It doesn’t cure SCD. All the while, the pain is apparent only to the sufferer. “If somebody’s having sickle cell pain, there’s no swelling, no redness,” says Dr. Lewis Hsu, a pediatric hematologist, director of the Pediatric Sickle Cell Program at the University of Illinois Chicago, and chief medical officer for the Maryland-based Sickle Cell Disease Association of America. “In many cases, there’s no change in lab tests. All you have is a patient report. So, there’s a lot of misunderstanding and stigma where people just don’t believe the person is having this bad pain.”

Since the early 1980s, the only cure for SCD has been a bone marrow transplant from a donor, often a patient’s relative. But less than 20% of eligible patients can find matching donors, Hsu says, and even those run the risk of immune rejection or graft-versus-host disease, in which donor immune cells attack the recipient’s tissues.

That’s what makes the CRISPR/Cas9 treatments so important—potentially revolutionary, Eckrich says. The use of the patients’ own cells dramatically reduces the possibility of immune rejection and eliminates the risk of graft-versus-host disease. The FDA’s approval of Casgevy means Levine Children’s can begin to treat patients as an authorized treatment center this summer, he says.

“For the last 20 years, our approach has always had to be, for patients with severe disease who would consider having a transplant, to discuss all the risks of having a transplant from somebody else,” he says. “Now, we’re able to consult and see patients and discuss the risks of what we call an autologous transplant—basically getting your own stem cells, after they’ve been edited, back to you.

“Many of the key parts of the operation are still as difficult and high-risk, but we have expertise in doing them. We’ve been doing autologous transplants for patients for a long, long time, and we really understand how to do those safely. Some of the complications that patients face afterwards are, frankly, much easier to deal with, so we can do these procedures with much less risk to the patient.”

One of those risks comes from chemotherapy. SCD is not a cancer, but treatment for it often resembles treatment of leukemia; both are diseases in which patients produce insufficient or defective blood cells. When SCD patients prepare to receive stem cell transplants, doctors administer chemo drugs to kill defective stem cells and make room for healthy, modified ones.

But the effects of chemo can be as debilitating as SCD. (For example, Bibbs received doses of the common chemo drug busulfan and spent much of the next year recovering more from the drug than from SCD.) Eckrich and other researchers and clinicians want to refine Casgevy treatment so it requires less chemo.

“If we can treat a patient with sickle cell disease, improve their quality of life, and have the net impact that we’ve had for many of these patients—and hopefully will continue to have—the future for many of these (blood) diseases is going to be bright,” Eckrich says. “When you get rid of those barriers—and CRISPR will allow us to genetically get rid of some of those barriers—transplantation becomes much, much easier.”

When Eckrich left San Antonio to return to Charlotte, Dr. Lyndsay Molinari, who had recently completed a pediatric blood and marrow transplantation fellowship at Duke, took over his role as principal investigator for sickle cell gene-editing research at Methodist Children’s Hospital. After about a year, Levine Children’s reached out to her to see if she wanted to join Eckrich, whom she considers a mentor, in Charlotte.

Molinari is originally from Syracuse, New York, and her husband is from New England. “Texas was just farther from my family than I wanted to be,” she says, “even though I loved the program and the work that I was doing there.” She joined the team at Levine Children’s in April 2023. Reuniting with Eckrich motivated her, too, and so did her sense that Atrium Health, and the health care industry in Charlotte, was on the rise.

“I think Atrium’s at a really interesting crossroads right now with the merger with Wake Forest,” says Molinari, 39. “The academic and research opportunities that are becoming more readily available were attractive to me.”

That growth includes the Wake Forest University School of Medicine in Charlotte, expected to open in Midtown in 2025, and builds on some of the advances that Atrium and Novant Health— Charlotte’s other major health care provider—have made in recent years. Even before Levine Children’s invited Eckrich back to establish the gene-editing program, the adjoining Atrium Health Levine Cancer Institute had earned a reputation for treating SCD in adult patients, Hsu says: “It’s one of the bigger and better sickle cell centers.” Eckrich’s team, which works with children, exchanges information with its counterparts at the Cancer Institute—which means Charlotte is on a short list of American cities where doctors research and treat SCD in both children and adults.

Dr. Lyndsay Molinari had worked with Dr. Eckrich before in San Antonio, and joined the team at Levine Children’s Hospital in 2023. Courtesy

The Atrium team also gets patient referrals from an unlikely source—the St. Jude Affiliate Clinic at competing Novant Health Hemby Children’s Hospital, which has treated blood disorders in children since 2001. For the past decade, the clinic has taken part in Memphis-based St. Jude Research Hospital’s study of SCD patients over years of treatment, from birth to death—research possible only since early detection and new treatments have lengthened patients’ lives.

Charlotte’s is one of five St. Jude clinics that have tracked about 1,300 patients in the Sickle Cell Clinical Research and Intervention Program (SCCRIP), which gathers data on the long-term effects of current SCD treatments.

“This just gives you an independent way of tracking all sorts of things,” says Dr. Felipe Bautista, the Ecuador-born pediatric hematologist and oncologist who leads the clinic’s sickle cell team. “If you’re following a patient just during childhood, then you’re missing all the badness that happens during the adult years. This is lifelong. I’m not aware that there’s anything comparable.”

Among the patients enrolled in Charlotte are Anthony Shipp, 12, and his sister Jennie, 10. When she got pregnant with Anthony, their mother, Shannon Shipp, knew that both of her adult daughters had the SCD genetic trait, present in one of every 13 Black births. “But I didn’t know what it meant,” she says.

Anthony was born in 2011 and was prone to illness during early childhood. But his mother didn’t realize how dire his condition might be until her granddaughter, Naiome, died of SCD in July 2012, a month short of her second birthday.

Jennie And Anthony Shipp, Courtesy

“When she passed, it just opened my eyes. I was like, ‘You know what, I’d better pay attention. I need to start reading these pamphlets that they’re giving me,’” Shannon says. “You know, they explained it to me, but I didn’t understand, and I wasn’t asking questions.”

Naiome had been treated at the St. Jude clinic, and Shannon took her son there. Anthony was enrolled in the SCCRIP when he was “2 or 3,” Shannon says, and began taking hydroxyurea. By then, she had given birth to Jennie, an unplanned second child with her husband. “I felt disappointed in myself for a moment. But then I snapped out of it,” she says. “I’m like, ‘Well, she’s here now, and it’s time to raise her.’” She took Jennie for treatment at St. Jude, too, and enrolled her in the study: “I said, prayerfully, ‘They will come up with a cure.’”

Gene-editing therapy may be in the future for both Anthony and Jennie. But the treatments they’ve received at the St. Jude clinic allow them to live near-normal lives: Jennie attends Bruns  Elementary School and takes dance classes, and Anthony, who’ll enter seventh grade at Ranson  Middle School next month, hopes he can make the soccer team.

Shannon and I are talking on the phone as she picks Anthony up from school. I ask her if I can talk to him. He tells me life’s much better since the days when the pain made him double over—“like when you crumple up a piece of paper,” his mother says—and miss school.

“I barely got any education, and most kids would make fun of me when I was at school,” Anthony says. “But now, I’m in school, like, the whole, entire year, and I’m really getting good in class.” His classmates, he adds, don’t make fun of him anymore.

Dominick Bibbs met Dr. Eckrich as he approached his high school graduation. Even with his crises, during which he couldn’t make it to class and teachers had to visit his home in San Antonio, he was still on track to graduate on time in 2020. Another doctor treating Bibbs learned about Eckrich’s clinical trials at Methodist Children’s Hospital and recommended it. Bibbs had to wait until his 18th birthday to enroll. He applied and was accepted. Then COVID delayed many medical procedures, and Bibbs was left with his pain.

At first, Bibbs was hesitant to enter the trials. “I had to think about it for a little minute,” Bibbs says. “But then, once it started getting worse, it was at the point where I didn’t even think I was going to make it past 18 or 19. … I said, ‘There’s nothing worse that can happen, so I might as well try it.’”

Bibbs decided to take the 2020-21 school year off for treatment rather than enroll in college. When his treatment finally began in early 2021, he spent 39 days in the hospital and made visits to an apheresis clinic that withdrew his stem cells. Doctors installed a catheter in his chest for removing and reinjecting blood and cells and for his chemo.

Between visits, he endured more rounds of chemo. In his already weakened state from years of SCD, Bibbs endured the chemo with difficulty. He had no energy, and he developed mouth sores, a common side effect, that made eating difficult. “Not very fun,” Bibbs says. Meanwhile, doctors in a lab made precise modifications to the DNA in his stem cells so they would produce fetal hemoglobin to prevent “sickling” and improve blood flow—at least theoretically, curing the disease.

When we speak in early May, Bibbs has just passed his third “birthday,” April 19, 2021, the day doctors returned the edited cells to him. “We actually have a video of it,” Searles-Bibbs says. “They’re in this Hydro Flask-type thing, and when you open it, smoke comes out, and then it’s just three little vials that his stem cells are in, and they just inject him through his IV to go back through his port into his body.”

The crises stopped that day. Bibbs says he hasn’t had one since. But it took him nearly a year to get over the effects of the chemo. He enrolled at Prairie View in the fall, not recovered yet. He got winded just walking to class. “I was still bald,” he says. “I got tired very quick.”

Dominick Bibbs, Courtesy

By spring 2022, though, he felt his energy coming back. He started to hang out with friends. He joined the historically Black fraternity Kappa Alpha Psi and threw himself into its community projects. In his rare free time, he takes occasional trips to Houston, about an hour away, to eat out. “I’m a big steak connoisseur,” he says with an impish grin. Before his treatment, he took 10 medications every day. Today, he takes an occasional Motrin for mild chronic pain.

Now comes a mystery that only time will solve: Whether the treatment has cured his SCD or is only a temporary fix. Researchers have no specific reason to suspect it won’t last. But it’s a brand-new treatment, and, until recently, there was no such thing as a long-term SCD survivor. This is where the St. Jude lifetime study can add value—by tracking whether gene editing and other treatments have long-term or secondary effects, like increased risk of cancer, that researchers don’t know about yet.

“There is an enormous amount of ongoing clinical research still needed in this field for us to understand that these treatments are durable, and potentially curative, without the trade-off. Right now, it appears that’s the case,” Eckrich says. “To understand those questions, we really do need the longitudinal approach, where we have a lot of data over a lot of time to help inform us about which way to go and how to strategize. So, I’m grateful for centers that make that commitment.”

Bibbs is still a young man, and given what he’s endured and how little he socialized growing up, he’s a bit shy. He’s quick to downplay the significance of his treatment, not just for him but others. “Dominick is a very humble person, and I don’t think he realizes the impact he has on the sickle cell world. We’re always telling him, ‘You need to tell your story.’ But he’s so nonchalant about it,” Searles-Bibbs says as her son listens. “He’s just, ‘OK, I just did what I needed to do,’ and it’s like he doesn’t realize how serious and how much of an impact this has in the sickle cell community and for African Americans.”

Bibbs beams and shakes his head. “I see the importance of it,” he says with a grin, “but I guess I don’t let it go to my head or anything crazy.”

Before he enrolled at Prairie View, before he could be sure he even had a future, Bibbs thought he might study engineering. A hospital stay that coincided with his 18th birthday changed his thinking. This was an especially bad crisis that landed him in intensive care, and two male nurses there did something life-altering: They spoke to him as a fellow human being and not, Bibbs says, “as a statistic.” He decided on nursing school, which he’ll enter in the fall. He wants to be what those two men were for him, and to treat patients like the younger, afflicted version of himself. I ask him what he thinks he can bring to those interactions, and he hesitates, unsure of what to say.

One more time, his mom puts her finger on it. “Compassion,” she says, “and relatability.”

“Yeah,” her son says. “That’s the word I’m looking for.”

GREG LACOUR is the editor.

Want to Know More?

SCD patients interested in Casgevy treatment can contact Atrium Health at PedsBMTNewPatientReferral@atriumhealth.org or 980-442-2900.