How St. Jude Has Helped Transform Sickle Cell Disease Care

Growing up, Justin Flowers was admitted to the hospital countless times for blood clots, infections, and episodes of crippling pain.

Flowers has sickle cell disease, a blood disorder that is passed down in families. It causes red blood cells to become stiff and misshapen. These sickled cells can block blood flow, damage organs, cause severe pain, and shorten a person’s life.

As a child, Flowers received care at different locations around Memphis, Tennessee. By 2008, services for children with sickle cell disease were housed at one place: St. Jude Children’s Research Hospital.

“The care at St. Jude was a million times different,” Flowers says. “If I woke up hurting at 3 a.m., my mom could call St. Jude, and they would say, ‘We’ll be expecting him.’ By the time we got there, my room was ready, and the team already knew what I needed. It felt like everyone knew me and understood what worked best for me.”

Today, Flowers’ experience reflects decades of work by St. Jude physicians, scientists, and advocates. Their efforts helped transform sickle cell disease care and pave the way for advances such as gene therapy. 

Barriers of race and inequity

St. Jude is widely known for its work with childhood cancer. But the hospital’s connection to sickle cell disease dates to its founding.

One person who helped shape that work was Lemuel Diggs, MD. A faculty member at the University of Tennessee College of Medicine in Memphis, Diggs advised hospital founder Danny Thomas as plans for St. Jude took shape. Diggs also received the hospital’s first research grant  to support his sickle cell disease work.

Diggs moved to Memphis in 1929. He was one of only a few scientists studying sickle cell disease, a condition that primarily affects people of African, Central and South American, Middle Eastern, and Indian descent.

Diggs was a White physician in the segregated South studying a disease that primarily affected Black patients. At a time of widespread racial discrimination, he conducted research, published scientific papers, and helped define many of the disease’s clinical features.

“The medicine of sickle cell disease is the medicine of civil rights,” says Mitch Weiss, MD, PhD, chair of the Department of Hematology at St. Jude. “When Dr. Diggs was here, the Tuskegee Institute syphilis experiments (that withheld lifesaving treatment from Black men with syphilis) were at the height of activity. In the South, hospitals were segregated. St. Jude was involved in changing that.”

Because sickle cell disease primarily affected people of color, it historically received less attention and funding than many other diseases. Many patients and families had reasons to distrust the health care system because of past discrimination and harmful experiences.

“The injustice of the inequity led to a lack of trust between providers and patients,” says Deepa Manwani, MD, medical director of the Comprehensive Sickle Cell Disease Program at St. Jude. “There was a wariness to participate in clinical trials. But if an institution has a good team and good providers, you can overcome that.”

As the first fully integrated pediatric hospital in the South, St. Jude helped build trust with families and expand access to care.

Building a clinical program

St. Jude began treating children with sickle cell disease in the 1960s. 

One early leader in patient care was Rudolph Jackson, MD, one of the hospital’s first Black physicians. He moved to Memphis in 1968 after leading a sickle cell disease program in Philadelphia. 

At the time, care providers focused on treating and managing the infections, pain, fatigue, stroke, and severe lung problems caused by the disease. Jackson went into Memphis neighborhoods to work with children and families affected by sickle cell disease.  

In the 1970s, the National Institutes of Health (NIH) chose Jackson to head the federal government’s sickle cell disease program.

Hydroxyurea offers hope

Sickle cell disease was hard to treat because there was no way to prevent the “sickling” of cells. 

That changed with a medicine called hydroxyurea, a drug developed to treat certain cancers. Researchers discovered that it increased fetal hemoglobin in the blood.

Fetal hemoglobin does not sickle in the same way that sickle-cell hemoglobin S does. At first, some people worried that the medicine might not be safe for young children.

An early champion of using hydroxyurea in children was Winfred Wang, MD, who joined St. Jude in 1979. He led many studies at St. Jude and played a key role in national trials involving other institutions.

“He ushered in what I would call the more modern era of sickle cell disease therapy at St. Jude,” Weiss says. “Probably the most important thing he did was the Baby HUG trial.”

The Baby HUG study showed that hydroxyurea was safe for children as young as 9 months old. The results supported national recommendations to offer the medicine early in life.

Wang’s work also led to many clinical advances, including newborn screening, stroke prevention, and routine penicillin treatment to prevent infections.

“That has led to more comprehensive care,” says Yvonne Carroll, JD, RN, director of educational outreach for the St. Jude Department of Hematology. “It has also led to people living longer. But what’s more important is their quality of life.”

From treatment to cure

Even with these advances, a cure remained elusive.

That changed in 1983 when a St. Jude patient had a bone marrow transplant to treat her leukemia. She also had sickle cell disease. The transplant cured both diseases.

St. Jude built a transplant program for sickle cell disease and continues to perform them today.

Transplants have transformed lives such as the Dorle family. Three of the family’s six siblings received transplants for their sickle cell disease. Their siblings who did not have sickle cell disease were their marrow donors.

“It made a huge difference,” says Barinedum Dorle, who had a transplant 4 years ago. “Before the transplant, it felt like every year I was being admitted to the hospital for pain, pneumonia, or something else. Since the transplant, I haven’t had to be hospitalized.”

While effective for some patients, transplants require compatible donors and intensive treatment, limiting their availability.

Gene therapy opens new doors

A new era began in 1993 when gene therapy pioneer Arthur Nienhuis, MD, came to St. Jude as director.

Sickle cell disease was a good candidate for gene therapy. In 1949, researchers discovered that the hemoglobin in people with sickle cell disease was different from normal hemoglobin. This finding helped scientists better understand how the disease works. It also made sickle cell disease the first disease understood at a molecular level. This discovery laid the foundation of modern molecular medicine.

Nienhuis believed gene therapy could treat diseases caused by faulty genes. He recruited scientists, created programs, and built the infrastructure needed to advance the field. 

His  work with Brian Sorrentino, MD, of St. Jude Experimental Hematology, helped lead to a cure for SCID-X1, a life-threatening disorder of the immune system also known as “bubble boy disease.” This milestone paved the way for advances in gene therapy for sickle cell disease.

“It didn’t happen in his lifetime, but much of what we’re doing today stems from Dr. Nienhuis’ work,” says Akshay Sharma, MBBS, of Bone Marrow Transplantation and Cellular Therapy. “St. Jude is among the few institutions that took part in the key early clinical trials of gene therapy for sickle cell disease. Today, we have the technology to do more than just treat the symptoms of this disease. We can eliminate it in some patients. That’s an opportunity to make a meaningful impact that few institutions have ever had.”

Gene therapy has had a meaningful impact on the lives of patients such as Donte Johnson, who had gene therapy at St. Jude several years ago.

“They were telling me I wasn’t going to make it to see 25,” Johnson says. “I just turned 25 in January, and I’ve accomplished a lot of things since then.”

Comprehensive care and research

Today, St. Jude treats more than 750 children with sickle cell disease each year. The program offers expert care for patients from infancy through the teen years. A team of specialists supports patients and families. St. Jude also coordinates newborn screening referrals across western Tennessee, Mississippi, and Arkansas.

“Any child in West Tennessee who is born in the hospital with sickle cell disease is referred to us,” Carroll says. “We ensure that 100% of those families know that their child has sickle cell disease, and we get them to follow-up care. Families need access to care and education about available services.”

Researchers continue to focus on improving quality of life. One key area is pain, a hallmark of sickle cell disease. Scientists are using brain imaging and genetic studies to better understand why some patients have more severe pain than others.

Researchers are also studying why organ damage develops and how to prevent health problems earlier in life.

Transitioning to adult care

Another major focus is ensuring patients remain connected to care as they become adults.

Years ago, clinicians noticed that many patients stopped getting regular care after leaving children’s hospitals. Jane Hankins, MD, director of St. Jude Global Hematology, helped create a program that helps young adults move from pediatric care to adult care. The program was developed with Methodist University Hospital and Regional One Health.

“After they left St. Jude, we did not know what was happening to them,” Hankins says. “We referred them to adult doctors, but we did not have a mechanism to ensure that they went.”

Hankins led efforts to structure services and align national and international guidelines. A federal grant helped fund the hiring of staff members focused on the transition to adult care.

“With sickle cell disease, you have to keep up with medical care to prevent complications,” Hankins says. “If patients aren’t followed any more, they may stop taking medicines. They risk medical complications such as organ damage. I have seen patients deteriorate greatly because they did not have adequate follow-up care. Some of them even died.”

Educating the community

Many health care workers outside sickle cell disease programs get little training about the disease.

To address this, St. Jude started a program called Sickle Cell Nursing Excellence Train-the-Trainer Boot Camp in partnership with the University of Tennessee Health Science Center College of Nursing and the International Association of Sickle Cell Nurses and Professional Associates. The program teaches nurses and other health care providers about sickle cell disease.

Boot Camp started in Memphis and expanded to Brazil, Nigeria, and India. It has trained more than 300 nurses, who have trained more than 3,000 other health care providers.

“We hope this snowball effect continues,” says Carroll, one of the program’s founders.  

The power of information

The transition program brought hundreds of patients into continuous care, creating a large group for long-term research.

That effort helped establish the Sickle Cell Clinical Research and Intervention Program (SCCRIP) in 2014. It continues today.

SCCRIP is a multi-center study that follows people with sickle cell disease throughout their lives. Researchers want to better understand how the disease affects health, daily life, and long-term outcomes. More than 1,600 patients across the Southeast and Midwest have joined the study.

“SCCRIP is a standardized way to understand what is happening to people with sickle cell disease at every stage of life,” Carroll says. “We’re learning why organs become damaged over time and how we can prevent those problems and help patients.”

SCCRIP also helps guide future research. By studying its extensive data, scientists can identify new genetic targets for treatment.

“We can’t keep using the same tools we’ve always used,” Manwani says. “Can we use modern tools and move beyond a one-size-fits-all approach to treating sickle cell disease?”

Looking ahead

The future of sickle cell disease treatment is unfolding on multiple fronts.

Drug companies are investing in new therapies. Researchers are working to make gene therapy faster, safer, and easier to deliver. Current treatments that can provide a cure are too expensive for patients or health care systems. They can take months and require highly specialized facilities. One day, scientists hope to deliver these treatments more simply, such as through an injection or a pill.

At the same time, St. Jude is sharing knowledge with health care providers worldwide. Many children worldwide still lack access to medicines and screening. For Hankins, expanding access is as important as developing new technologies.

“How do we get this to the rest of the world?” she asks. “How do we get penicillin, hydroxyurea, and basic care to people in very remote areas? How do we reach the poorest of the poor? How do we train community health workers to identify patients and provide treatment?”

For decades, children like Justin Flowers faced a future defined by pain, hospitalizations, and shortened life expectancy. Today, Flowers chairs the Community Advisory Board for the American Society of Hematology, helping strengthen patient-centered and community outreach programs for sickle cell disease and other blood disorders.

St. Jude physicians and scientists say their work is far from finished.

Their next challenge is ensuring that every child with sickle cell disease, regardless of where they are born, has access to the same advances that transformed care in Memphis.

“We are in the field not just to treat children with catastrophic diseases, but to cure them,” Sharma says.

From Lemuel Diggs’ pioneering research during segregation to today’s work in gene therapy and global health, the St. Jude sickle cell program has been shaped by one simple belief: Every child deserves the chance to live a healthy life.