Researchers for a clinical trial searching for a sickle cell disease cure, which included two patients treated at Cleveland Clinic Children’s, have presented their latest findings at the European Hematology Association 2024 Hybrid Congress in Spain.

What You Need To Know

  • Researchers for clinical trial searching for a sickle cell disease cure, which included two patients treated at Cleveland Clinic Children’s, have presented their latest findings at the European Hematology Association 2024 Hybrid Congress in Spain
  • The researchers spoke about the data they collected on 18 patients using an experimental gene-editing cell therapy called renizgamglogene autogedtemcel

  • The patients had their stem cells taken for gene editing before undergoing chemotherapy “to clear remaining bone marrow” before having their cells replaced

  • According to the researchers, there have been no serious side effects and treatment has been “well-tolerated"

According to a Cleveland Clinic press release, the researchers spoke about the data they collected on 18 patients using an experimental gene-editing cell therapy called renizgamglogene autogedtemcel.

“This innovative treatment modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease,” the release reads.

The patients had their stem cells taken for gene editing before undergoing chemotherapy “to clear remaining bone marrow” before having their cells replaced.

According to the release, there have been no serious side effects and it has been “well-tolerated.” The researchers state that the patients have regained white blood cell and platelet counts and have not had any pain events since undergoing the treatment.

They also note that the patients they’ve watched for five months or more “have seen their anemia resolve.”

“It’s encouraging that this gene-editing treatment continues to show promising efficacy for sickle cell patients,” said Dr. Rabi Hanna, chairman of the Division of Pediatric Hematology Oncology and Blood and Marrow Transplantation at Cleveland Clinic Children’s and the RUBY trial’s presenting investigator, in the release. “These latest results offer hope that this new experimental treatment will continue to show progress and get us closer to a functional cure for this devastating disease.”

The Cleveland Clinic states that around 100,000 people in the U.S. have the disease and that anywhere from 1 to 3 million people carry the trait. African Americans have a higher risk, with 1 out of every 365 babies being born with the disease.

This disease is genetic, and Cleveland Clinic explains that it causes an individual’s red blood cells to be sickle-shaped, hence the name. This blocks the flow of blood and causes them to break easily, leading to pain and issues with the heart and liver alongside a shortened life expectancy.

“Medications can help manage the disease, but a cure is possible only through a blood or marrow transplant, which has risks and often requires a sibling donor,” the release reads.

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