A patient on Long Island is the first in New York to be cured of sickle cell anemia, doctors say.
Medical history made at Cohen Children's Medical Center
For 21 years, Sebastien Beauzile suffered chronic pain from debilitating sickle cell anemia.
"Sickle cell was like a blockade for me, but now it's just like a wall that I just jumped over," he said.
He is now making medical history in New York state.
"The cliche 'the future is here' is actually true in this case," said Dr. Charles Schleien, with Cohen Children's Medical Center.
"We feel blessed to be able to offer, be the first ones to be able to offer this," Dr. Jonathan Fish added.
Groundbreaking Lyfgenia treatment used Beauzile's own bone marrow in IV transfusions to create normal red blood cells.
"When we could use Sebastien's own stem cells to do this therapy, we were delighted," Dr. Banu Aygun said.
"Sickle cell disease was described in modern medicine in 1910, and here we are over 100 years later, and this is the first cure you are seeing," Dr. Jeffrey Lipton said.
In the United States, sickle cell amenia most commonly affects people of African, Mediterranean and Middle Eastern descent.
Cohen Children's Medical Center hopes to target those communities with this life-altering therapy.
"To the Med 4 team, the hematology team, the transplant team, you guys have really changed my life," Beauzile said.
"Sebastien been coming to this hospital since he was 2 months old. There is not enough words to show you how grateful I am," said Magda Lamour, Beauzile's mother.
Beauzile will soon be able to travel, work out and focus on his education. He hopes to work in the medical world.
"Amazing, and I can't wait to get back to my day-to-day life because now I feel unstoppable," Beauzile said.
Civil rights attorney Benjamin Crump on Thursday announced a $100,000 donation to Northwell Health to further the health system's efforts to treat sickle cell disease.
ALLoftheFancyPants on March 15th, 2025 at 21:36 UTC »
Sickle cell is fucking awful and this is amazing. I hope this treatment becomes widely available. And by “available” I mean actually available both physically and financially.
mces97 on March 15th, 2025 at 20:37 UTC »
Ok, so being a biology and medicine geek, this is super interesting, not to mention an incredible moment for humanity. But I was confused reading the article why they called this a cure. Before I read it I assumed CRISPR or a similar gene therapy would had been used. I was correct, but the article really didn't mention that.
Here's the technology they used.
"Lyfgenia is a personalized gene therapy that uses the patient's own blood-forming stem cells. These cells are modified in the laboratory to contain a healthy copy of the beta-globin gene, which is responsible for producing sickle hemoglobin. The modified cells are then infused back into the patient, where they establish themselves in the bone marrow and produce healthy hemoglobin. This helps to reduce or eliminate the production of sickle hemoglobin, which is the underlying cause of SCD."
kokopelleee on March 15th, 2025 at 20:31 UTC »
This is why medical research should be funded
but we know how that’s going