New Sickle Cell Therapies Highlight Equity Gaps and Treatment Progress

Racial and ethnic disparities in health care persist in every facet related to equitable access, quality, and progressive research of diseases that disproportionately impact minority patients, especially sickle cell disease (SCD).

SCD is a rare inherited hemoglobin (Hb) disorder caused by a point mutation in the β-hemoglobin gene, resulting in the sickling of hemoglobin that distorts and injures red blood cells.¹ SCD affects approximately 100,000 people in the US as of 2024, yet more than 90% of patients are non-Hispanic Black or African American. Patients with SCD often experience vasoocclusive episodes (VOEs), hemolytic anemia, organ damage, and failure, thus reducing their expected life span by 20 years when compared with the national average. ^1,2 Despite this, patients’ extensive health care needs remain disproportionate. Treatments and therapies for SCD are often expensive, and patients report that their symptoms and pain are frequently dismissed or minimized. However, newer therapies, despite decades of stagnating research, can potentially improve access and increase patients’ life expectancy.

“[It’s an] obvious disease that affects minorities, and they have received less attention than diseases that affect nonminority populations,” Martin H. Steinberg, MD, a hematologist and internal medicine specialist at Boston University Chobanian & Avedisian School of Medicine, said in an interview with The American Journal of Managed Care^® (AJMC^®). “But I think over the last 50 years, that's changed dramatically so that there's a high degree of interest in research and sickle cell disease because it's a disease that's very amenable to treatment.”

FDA-Approved Treatments for Sickle Cell Disease Are Expanding Beyond Hydroxyurea

Recent therapies like hydroxyurea (Droxia, Bristol-Myers Squibb), the first FDA-approved drug for SCD in 1994, increase fetal hemoglobin (HbF), which, through unknown pathways, improves SCD symptoms by reducing pathogenic sickle hemoglobin.³ However, it requires lifelong medication and maintenance due to HbF reactivity.

“The goals of the approved gene therapies are to produce enough HbF, or fetal-like hemoglobin, in the red blood cells so that about a third or more of their hemoglobin is this HbF, and then the pain episodes...and the anemia go away,” Steinberg said. “What we need is an oral agent, a pill that you could take that does this. Hydroxyurea does it, but for complicated reasons, it doesn't do it enough, especially when it started later in life.”

Other FDA-approved therapies for SCD since then include L-glutamine (Endari; Emmaus Medical, Inc), voxelotor (Oxbryta; Global Blood Therapeutics), and crizanlizumab (Adakveo; Novartis), which alleviate VOEs.³ Conversely, a more recent therapy for treating SCD, isoquercetin, shows promise in targeting thromboinflammation in adults with SCD.

Gene Therapy and Thromboinflammation Research Signals a New Era for SCD Care

Targeting thromboinflammation in patients with SCD addresses the adverse events associated with hydroxyurea. Venous thromboembolism (VTE), when it occurs, requires lifelong anticoagulation, which increases patients’ risk of life-threatening bleeding. This new therapy broadens the scope of research for SCD by shaping it as a thromboinflammatory disease, as many of the downstream symptoms of SCD are exacerbated by thromboinflammation.⁴

“Targeting those systems, whether it's inflammation or thrombosis, may lead to a beneficial effect for patients with sickle cell disease,” Ted Wun, MD, chief of hematology and oncology at UC Davis Health, said in an interview with AJMC. “There have been studies of using platelet inhibitors, which are much more commonly used in patients with heart disease, to prevent recurrent strokes, heart attacks, or even primary strokes and heart attacks, that have been tried in SCD patients, and also anticoagulation to try to prevent thrombosis.”

However, despite its introduction as a new potential therapy for SCD, there is limited research on SCD therapies targeting inflammation or thrombosis that have positive outcomes related to pain, Wun said.

Steinberg further expanded on the use of thromboinflammation-targeted therapies and said that they’re more likely to be useful in conjunction with hydroxyurea.

“There might be a place for these agents and people who are taking hydroxyurea; I don't see these as single-use agents,” he said. “I think most patients, at least adult patients, who are on hydroxyurea need other drugs, because, as I said, hydroxyurea is not good enough, but they're not primary therapies.”

Cost and Provider Education Remain Key Barriers to Equitable Sickle Cell Treatment Access

As research within SCD continues to advance, Wun and Steinberg both agree that there are challenges to implementing new therapies successfully, especially regarding access and cost.

“There are many steps and challenges all the way from systems to practitioners who are familiar with hydroxyurea and how to use it to resistance by or concerns with patients, some of it very legitimate, but a lack of understanding,” Wun explained. “There was very low uptake of those in the community, partially because of access to a practitioner who isn’t even aware of them or doesn't know really how to use the drugs.”

Attempts to improve access, Wun said, have been curated by the American Society of Hematology. The organization has invited practitioners and physicians to participate in SCD workshops that are now sponsored by national organizations dedicated to SCD, like the Sickle Cell Disease Foundation.

“[They] train teams to take care of patients with sickle cell disease. And they're not just talking physicians, but nurse practitioners, social workers, nurses, and pharmacists,” Wun said. “Over the 4 or 5 years, those operating 58 new teams throughout the country were trained in optimal care for patients with SCD.”

Improving awareness and promoting continuing education are only 2 ways health institutions aim to improve access, but cost remains a significant barrier. Gene therapies were FDA-approved to treat SCD in 2023, but CRISPR-based gene-editing therapy can cost anywhere between $1 million and $3 million.⁵

“It turns out that the patients with sickle cell anemia in this country are both medically and economically disadvantaged,” Steinberg said.

At Boston University Chobanian & Avedisian School of Medicine, where Steinberg works, he and his team were able to provide gene therapy for their patients, albeit not many. He also said that although the upfront costs for health care funding agencies or payers may be higher, it may ultimately equate to less over the life of a patient with SCD.

“Equitable health care is a laudable goal, which we're really far from in this country, not just for the patients with sickle cell, at least for everybody,” Steinberg said.

References

1. Solovieff N, Hartley SW, Baldwin CT, et al. Ancestry of African Americans with sickle cell disease. Blood Cells Mol Dis. 2011;47(1):41-45. doi:10.1016/j.bcmd.2011.04.002

2. Data and statistics on sickle cell disease. Centers for Disease Control and Prevention. May 15, 2024. Accessed February 16, 2026. https://www.cdc.gov/sickle-cell/data/index.html

3. Ball J, Bradleyu A, Le A, Tisdale JF, Uchida N. Current and future treatments for sickle cell disease: from hematopoietic stem cell transplantation to in vivo gene therapy. Mol Ther. 2025;33(5):2172-2191. doi:10.1016/j.ymthe.2025.03.016

4. Lizzarralde-Iragorri MA, Goplan BP, Merriweather B, et al. Isoquercetin for thromboinflammation in sickle cell disease: a randomized double-blind placebo-controlled trial. Blood Adv. 2024;8(1):172-182. doi:10.1182/bloodadvances.2023011542

5. Pagliarulo N. Pricey new gene therapies for Sickle Cell Pose Access Test. BioPharma Dive. December 8, 2023. Accessed February 16, 2026. https://www.biopharmadive.com/news/crispr-sickle-cell-price-millions-gene-therapy-vertex-bluebird/702066/