A Breakthrough Treatment for Sickle Cell Disease Offers Curative Potential

Sickle cell disease (SCD) is an inherited, lifelong and painful blood disorder in which red blood cells are shaped like crescents or sickle-shaped instead of discs.

These damaged sickle-shaped blood cells generally clump together, impeding the flow of blood and reducing the ability for blood to carry oxygen to tissues and organs. This results in episodes of acute pain as well as long-term damage, causing a range of complications throughout life and increasing the risk of premature death.[1][2]

A common complication of SCD is abnormally high cerebral arterial velocities which is an indicator of stenosis presence or reduced oxygen delivery to the brain and is associated with a greater risk of strokes and cognitive problems.[1]

According to the U.S. Centers for Disease Control and Prevention (CDC), SCD affects approximately 100,000 Americans and millions worldwide, and disproportionately 90% affects those of non-Hispanic Black, African, Caribbean, Middle Eastern, and South Asian descent, with an estimated 3%–9% are Hispanic or Latino.[3]

The estimated life expectancy of those with SCD in the United States is more than 20 years shorter than the average expected, which is, according to data from the CDC caused by the patients with SCD not receiving the recommended healthcare screenings and treatments.

A Patient’s Story
KeAndra Hickman, from Virginia’s Eastern Shore, was diagnosed with SCD at birth.

“I had too many hospital visits to count,” Hickman noted. “I would wake up in pain almost days. Every day, before I put my feet on the floor, I’d reach into my nightstand for medicine.”

Extreme pain, known as a pain crisis, is one of the hallmark symptoms of SCD. With frequent hospitalizations, chronic pain and damage to vital organs, people living with SCD often experience a greatly diminished quality of life.

“Physically, I couldn’t always do what my friends did, or sometimes just things required to get through the day,” says Hickman.

Although long-standing research has shown that bone marrow transplants can cure SCD, most patients lack a perfectly matched donor, making treatment options inaccessible.

That changed for Hickman in September 2022, during Sickle Cell Awareness Month, when Robert Brodsky, M.D., director of the Division of Hematology at the Johns Hopkins University School of Medicine and the Johns Hopkins Family Professor of Medicine and Oncology, visited her community college and spoke about a new treatment option.

For the first time, Hickman felt hope.

In a national, multicenter clinical trial (BMT CTN 1507; NCT03263559) of which results were presented during the 2023 annual meeting of the American Society of Hematology (ASH), Brodsky and his team demonstrated that a reduced-intensity haploidentical bone (low dose) marrow transplant which uses a partially matched stem cell donor, such as a parent, sibling or cousin, offers a potential treatment for the patients diagnosed with severe sickle cell disease.[3]

In this type of bone marrow transplant, the donated bone marrow comes from a half-matched donor, such as a patient’s parent, sibling or child.

Other relatives, including a nephew, niece, aunt or uncle, are also often suitable donors. Patients were treated with pre-conditioning hydroxyurea to prepare them for transplant, and were given post-transplant cyclophosphamide, a drug that earlier research has proven to stave off graft-versus-host disease, a complication in which the immune cells in the donor marrow attack their new host, the patient.

“Our findings show that matched donors — available to only a minority of patients with sickle cell disease — are no longer required for cure of the disease with allogeneic transplantation,” Brodsky expained.

“Reduced-intensity haploidentical bone marrow transplant is a safe, curative therapy suitable for adults with sickle cell disease who also have complications related to their disease, such as organ damage, stroke or pulmonary hypertension, making it a treatment option for many more patients.”

This approach, curative in 90% of adult patients treated for serious sickle cell-related health complications, and it is now reshaping what is possible for people with SCD

The study included 42 individuals with sickle cell disease. The majority of the participating patients were African American (92.6%), and the median age was 22. Of the 42 participants, 95% were still alive two years after transplant, and 88% have not seen their sickle cell disease return and are considered cured.

“These data confirm results from Johns Hopkins and Vanderbilt, where longer follow-up demonstrates that virtually all patients beyond two years from transplant without disease are cured,” Brodsky noted

“It’s very rare for a patient to have a perfectly matched donor, but virtually everyone has a half-matched donor,” Brodsky explained.

50 years of transnational research
This breakthrough “half-matched” bone marrow transplant therapy is the result of more than 50 years of translational research at Johns Hopkins. It offers a more accessible alternative to traditional high-dose conditioning, which has long limited transplants to younger patients with minimal organ damage. Unlike fully matched transplants or emerging gene therapies, which are often not available to adults with complications, this method is tailored to meet the needs of the broader sickle cell population.

Hickman underwent the half-matched bone marrow transplant procedure in 2022.

“I pursued going through bone marrow transplant because I felt like nothing could be worse than what I faced every day with sickle cell,” Hickman said.

Today, she wakes up without needing pain medication. The little things, like taking out the trash or showering without pain, are invaluable, Hickman says. Looking ahead, she hopes to attend college and become an oncology nurse.

“I want to get my story out to help as many people as I can,” Hickman noted. “I’m now living a life that previous doctors said I never would. I feel like I went through this to help others,” she added.

Hickman and Brodsky both share a goal to honor the strength of patients and accelerate awareness of real, life-changing solutions.

“We need to change the paradigm for managing sickle cell disease,” Brodsky said.

“We’ve got to educate patients, primary care doctors and parents about this so they don’t lose hope, and so we move toward advancing care together,” he concluded.

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Clinical trial
Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507) – ClinicalTrials.gov ID NCT03263559

Reference
[1] Garcia D. Stem Cell Transplant for Sickle Cell Disease in Childhood Improves Health-Related Quality of Life a Decade Later. Onco’Zine. December 10, 2024 [Link]
[2] What Is Sickle Cell Disease? National Heart, Lung and Blood Institute. Online. Last accessed on December 4, 2024.
[3] Sickle-cell disease (SCD). U.S. Centers for Disease Control and Prevention Online. Last accessed on July 9, 2025
[4] Kassim AA, Walters MC, Eapen M, Ritzau N, Smith M, Solh MM, McKinney C, Nieder M, Ross M, Kent M, Abusin G, Mallhi KK, Silva JG, Shaughnessy P, Kanter J, Haines H, Farah RJ, Khaled Y, Abraham A, Bollard CM, Cooke KR, De La Fuente J, Hanna R, Horowitz MM, Jordan LC, Krishnamurti L, Leifere E, Mahadeo KM, Shenoy S, Ritzau NM, DeBaun MR, and Brodsky RA. LBA-4 – Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507. Late-Breaking Abstracts Session, American Society of Hematology (ASH) December 12, 2023, 9:00 AM-10:30 AM. [Presentation]

Featured image: From left to right, Robert Brodsky M.D., KeAndra Hickman, and KeAndra’s mother Stephanie Scarborough. Photo courtesy © 2025 KeAndra Hickman/Johns Hopkins University School of Medicine.

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