A Louisiana Teen Had Over 100 Hospital Stays With Sickle Cell Disease And Gene Therapy Just Functionally Cured Him
Phillip Mullin is 15 years old, from Alexandria, Louisiana, loves Legos and Spider-Man, and has had more than 100 hospital stays in his life. Sickle cell disease, which causes abnormally shaped red blood cells that can block blood flow and lead to severe pain, organ damage, stroke, and infection, has defined the terms of his existence since birth. He is now the first child in Louisiana to be functionally cured of the disease through gene therapy, specifically through Lyfgenia, a stem cell-based gene therapy developed by bluebird bio that received FDA approval in December 2023. The treatment was administered at Manning Family Children's Hospital in New Orleans. At a ceremony celebrating Phillip's treatment on October 24, Phillip's mother Shataka Figgins described what this moment means after years of watching her son endure the disease. She said she would now be unpacking every fear, every negative emotion she had ever carried, every time she thought her baby was not going to be there with her. She said she would remember every sleepless night and every moment she wondered how much more his body could take, and she would also see the young man who made it through. Phillip's speech at the ceremony brought most of the room to tears, including New Orleans Saints legend Archie Manning, who said he had never seen anything quite like Phillip detailing his journey and called him a wonderful young man whose mind is really something.
Phillip's speech was about sacrifice and strength. He said sacrifices are necessary and that you cannot gain anything without first giving something in return, though if you can endure the pain and walk away from it, you find that you now have a heart strong enough to overcome any obstacle. He called his sister Paige his superhero, acknowledging that even when he pushed her away, she was always there and always waiting, and that she is the greatest gift God ever gave him. Paige said at the ceremony that she never thought her brother would have this opportunity, and that it just feels great. Lyfgenia works by extracting a patient's own blood stem cells, using a viral vector to insert a functional copy of the beta-globin gene that sickle cell patients are missing or have mutated, and infusing those corrected cells back into the patient after a round of chemotherapy to make room for them. When the corrected cells engraft and multiply, they begin producing normal hemoglobin, effectively ending the sickling process from within. The FDA approval in 2023 followed a clinical trial in which all 28 evaluable patients achieved complete resolution of severe vaso-occlusive events within six to eighteen months of treatment. Phillip Mullin is the first patient in Louisiana to receive it, and the first child in the state to be functionally cured. He said he plans to go back to school and become a hematologist and oncologist so he can relate to sickle cell patients and actually know what they are going through. He is going home to sleep in his own bed and build Legos with his baby sister first.
