Groundbreaking Gene Therapy Cures 21-year-old of His Sickle Cell Anemia: ‘I’m not in pain anymore’
A New York hospital has declared a patient cured of sickle-cell anemia, a debilitating genetic disorder that mostly affects individuals of African heritage.
Considered to be cured, other genetic treatments have proven successful in reducing or ending the bouts of pain and occasional surgeries that plague the lives of those who suffer from the disease.
Cohen Children’s Medical Center said their patient Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New York Post.
Developed by Bluebird Bio., Beauzile received the treatment in December of 2024. Since then, the young man has been seemingly cured of the disease which caused him skin ulcers, back pain, hip pain, joint pain, and severe chest pain.
“Growing up with sickle cell, it’s kind of been over all my life,” Sebastien said, according to Gene Online. “So now that I’m cured, I’d say it’s my new birthday, because now nothing’s going to stop me.”
Part of a genetic mutation that humans developed to protect themselves against the malaria parasite, sickle cell disease occurs when blood cells, through forming sickle shapes, have trouble reaching the heart.
Dr. Jeffrey Lipton, the center’s director for pediatric hematology, predicts Lyfgenia will replace bone marrow transplants as the primary method for addressing sickle cell anemia, and called the treatment “a fix.”
Lygenia takes a sample of the patient’s bone marrow and introduces healthy adult hemoglobin from a donor. Hemoglobin is a protein that helps transport oxygen through the body via red blood cells, and is at the heart of sickle cell disease. Tinkering the two, they are then infused back into the patient’s body, where the donor hemoglobin is gradually coded for rather than the affected hemoglobin of the patient.
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“Sebastien’s recovery has been amazing, and we hope he is just the first of many patients we treat with Lyfgenia,” said Charles Schleien, MD, senior vice president of Cohen Children’s Medical Center.
In January, GNN reported that base editing, another form of gene therapy that, like Lygenia, doesn’t involve the more famous CRISPR technology, was able to seemingly cure 20-year-old Brandon Baptiste, who is now “going to the gym every day, doing cardio and weight lifting.”
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Baptiste became eligible for an experimental trial of base editing called BEACON. By October 2023, after a year of tests to ensure he was physically capable in his diminished state to handle the procedure, it began with a sample of his blood stem cells.
These were then transferred to a separate facility where the base editing would take place. Using chemotherapy, his team then killed off all the diseased blood stem cells in his bone marrow, after which he was ready to receive his own stem cells back in November.
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