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A 21-year-old Long Island patient has been cured of sickle cell anemia thanks to a gene therapy treatment.
Medical history has recently been made in a New York City hospital. This month, 21-year-old Sebastien Beauzile became the first man in New York history to be cured of sickle cell anemia, a genetic blood disorder, thanks to a new form of gene therapy.
“Sickle cell was like a blockade for me, but now it’s just like a wall that I just jumped over,” Beauzile told CBS News.
Beauzile, who had been a patient at Cohen Children’s Medical Center since he was two months old, was treated for the genetic disease with Lyfgenia, a new approach to gene therapy developed by biotech company Bluebird Bio. The groundbreaking treatment’s technology used Beauzile’s own bone marrow in IV transfusions to create normal red blood cells.
Sickle cell disease, which impacts over 100,000 people in the U.S., is an inherited blood condition that affects the shape of red blood cells, which carry oxygen to all parts of the body. As a result of these abnormal red blood cells, people with sickle cell disease can experience a range of symptoms, including chronic pain anywhere in their bodies, stroke, and blood clots; 90% of sickle cell disease patients are Black. The genetic disorder was previously considered a lifelong disease, however, with treatments like Lyfgenia, that prognosis will hopefully change.
Since receiving the treatment in December 2024, Beauzile has not experienced any symptoms of sickle cell anemia according to Forbes.
“The cliche ‘the future is here’ is actually true in this case,” said Dr. Charles Schleien, from Cohen Children’s Medical Center, per NBC News
“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 added.
For Beauzile and his mother, Magda Lamour, words do not even begin to describe their gratitude for the medical team and the life-changing treatment. Now, cured of the once debilitating disease, the 21-year-old is looking forward to traveling, working out, and focusing on his education, with hopes of working in the medical field one day.
“To the Med 4 team, the hematology team, the transplant team, you guys have really changed my life,” Beauzile said. “I can’t wait to get back to my day-to-day life because now I feel unstoppable.”
In 1983, Kimberlin George-Wilson was the first noted case of a person being cured of sickle cell anemia by bone marrow transplant.
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