Doctors are getting closer than ever before to finding the cure for sickle cell disease. The disease causes the red blood cells in an infected person to become misshapen and begin to break down. The disease causes pain in the joints, in the chest, dizziness, fatigue, and low oxygen in the body.
The disease affects less than 200,000 people in the U.S per year. Seven sickle cell patients were reportedly cured with stem cells from donors, and now doctors are taking a new look at the disease after the discovery.
In order for a patient to undergo a stem cell transplant, the cells had to have come from someone with the same human leukocyte antigens (HLA), which means that their tissues are compatible. Since HLA markers are hereditary, it is best that close relatives with compatible tissues are the donors. Unfortunately, it is difficult to find a relative of the patient that has compatible tissues.
Between January 2014 and March 2017, a total of 50 adults with sickle cell disease were screened as potential candidates for a half-matched stem cell transplant. After two unsuccessful attempts, scientists adopted the new protocol, which modifies one originally developed at Johns Hopkins University. The new protocol included an increased dose of radiation before the transplant and by infusing growth factor-mobilized peripheral blood stem cells instead of bone marrow cells.
Eight patients underwent the new transplant. One patient developed chronic graft-versus-host disease, where the donor cells see the patient’s cells as foreign and begin to attack them. The patient died of what researchers called “unknown causes” about a year after the transplant. The seven other patients were alive at a follow-up after 16 months and showed a stable 95 percent or greater acceptance of donor cells. These patients also showed improved blood work at least one year following the transplant.
“These patients are cured of sickle cell disease,” Damiano Rondelli, the study’s senior author, said. “This transplant protocol may cure many more adults patients with advanced sickle cell disease.”
In the future, sickle cell anemia could be fully eradicated by this new cure.