An effective treatment for sickle-cell anaemia has been found:
quote:
A reduced-intensity bone marrow transplant regimen developed by Johns Hopkins physicians provides durable engraftment with low rejection rates and also may preserve fertility, according to results of a new study conducted by Johns Hopkins Kimmel Cancer Center investigators.
“The survival rate in the long run in our study was about 95%, so we are curing the overwhelming majority of people who are transplanted, with mismatched donors,” says senior study author Javier Bolaños-Meade, M.D., a professor of oncology at the Johns Hopkins University School of Medicine. “Essentially, nearly everybody who has either sickle cell disease or thalassemia, an inherited blood disorder, can be cured, because nearly everybody has a donor.”
The study reports on long-term success among 43 patients with sickle cell disease, ages 5–41, who underwent bone marrow transplantation at Johns Hopkins from November 2014–January 2025. Seven had a full-matched genetic donor, and 36 had a half-matched donor such as a parent, child or sibling. Patients included 27 adults, ages 19–41, and 16 children, ages 5–17. All had more than two common complications associated with sickle cell disease such as a stroke or bone tissue death due to a lack of blood supply.
The probability of five-year overall survival was 95.5% at an average follow-up of 2.43 years, and the probability of disease-free survival at two years was 94.5%.
This study “is a game-changer because it’s the only curative approach that preserves fertility in many patients, and because the engraftment rate in children was over 90%,” adds study co-author Robert Brodsky, M.D., the Johns Hopkins Family Professor of Oncology Research and director of the Division of Hematology at the school of medicine. “Previous regimens with the chemotherapy agent thiotepa had lower engraftment in children compared with adults.”
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source:
https://www.hopkinsmedicine.org/news/newsroom/news-releases/2026/05/johns-hopkins-physicians-achieve-95-cure-rate-for-sickle-cell-disease-with-bone-marrow-transplant
This is why science matters, and why it matters that federal funding not be reduced: the study was in part funded by the National Institutes of Health.
About 20 million people are affected by this disease, worldwide.