NEW ORLEANS – On Friday, Oct. 23, 15-year-old Phillip Mullin rang the bell at Manning Family Children’s to celebrate the functional cure of his sickle cell disease through gene therapy. Phillip is the second person and first child to receive the groundbreaking treatment in Louisiana, and the first in the state using LYFGENIA – a one-time gene therapy to treat sickle cell disease in patients 12 years of age or older with a history of vaso-occlusive events – also known as pain crisis.
For most of his life, Phillip Mullin never knew when a sickle cell crisis would occur. By age 15, he had been hospitalized more than 100 times as his family searched for answers and treatments that could finally change the course of his disease.
That search led them from their home in Alexandira, Louisiana to Manning Family Children’s, where Phillip underwent groundbreaking gene therapy. LYFGENIA is made specifically for each patient, using the patient’s own blood stem cells (from which red blood cells are produced). It adds functional copies of the beta-globin gene to cells leading to production of anti-sickling hemoglobin that may decrease or stop painful medical events.
Phillip’s journey included months of preparation, stem cell collection, blood exchange procedures, chemotherapy, infusion of his genetically modified cells, and a challenging recovery. But today, the long road that has led Phillip to this moment is now full of hope, as he celebrates his re-birthday and future free of pain and limitations.
“The true measure of what we accomplish is not simply what we cure,” said Dr. Ben Watkins, director of the Stem Cell Transplant and Cellular Therapies Program at Manning Family Children’s and Section Chief of Pediatrics Hematology/Oncology at Tulane University School of Medicine. “It is the lives that people get to live because of it. Today, we get to celebrate all that has been made possible for Phillip and his family, and there is no greater joy than this.”
One hundred days after receiving his genetically modified cells, and surrounded by family and friends, Phillip rang the bell with the highest hemoglobin in his life. During the bell ringing ceremony, the hospital unveiled a custom Lego display created by Bricks and Minifigs in honor of Phillip’s love of Legos and heard remarks from NFL legend and hospital namesake Archie Manning, who introduced NFL alumni ambassador Tevin Coleman. Coleman’s own daughter was diagnosed with sickle cell disease as an infant, and the family hopes to raise awareness, while creating a better future for those living with sickle cell disease.
“Today, we are celebrating giving a 15-year-old kid his childhood back, and a future no longer limited by the weight of sickle cell disease,” said Lucio A. Fragoso, president and CEO for Manning Family Children’s. “This was made possible by our extraordinary doctors, care teams, medical partners, and those who have supported Phillip along the way – and now, we get to enjoy watching him be a kid, while dreaming big about his future.”
Phillip hopes to become a pediatric hematology-oncology physician so that he can care for children facing the same challenges he has overcome with such remarkable courage and resilience.
The Center for Cancer and Blood Disorders at Manning Family Children’s has the largest team of pediatric hematologists oncologists in the Gulf South, along with the most advanced therapies. It’s nationally ranked hematology oncology program provides advanced treatments for children with leukemia, lymphoma, Sickle Cell Disease, hemophilia, and other childhood cancers and blood disorders. As the only accredited pediatric stem cell transplant program in Louisiana, the team at Manning Family Children’s performs advanced treatments including bone marrow transplants, CAR-T cell therapy, and newly-approved gene therapy for treatment of Sickle Cell Disease.
For more than 70 years, Manning Family Children’s has proudly served as Louisiana’s number one ranked, and only, freestanding comprehensive children’s hospital with more than 600 pediatric-trained providers and 3,000 employees solely dedicated to the care of kids. In addition to comprehensive services offered at its main campus in New Orleans, Children’s operates a robust network of more than 30 satellite locations, pediatric affiliations across Louisiana and the Mississippi Gulf Coast, and robust and long- community programs designed to meet the health needs of the diverse communities it serves. At the heart of its mission, Manning Family Children’s is committed to delivering expert care for every child, every time – regardless of the family’s ability to pay.