Minnesota Hospital Performs Groundbreaking Treatment on Child with Rare Genetic Disease
Four-year-old Celia Grace Hammett is celebrating the holidays after becoming the first person in the United States to receive gene therapy for metachromatic leukodystrophy (MLD), a rare and often fatal genetic disease. The procedure was performed at M Health Fairview Masonic Children's Hospital, part of Fairview Health Services, a Healthcare Leadership Council member.
MLD affects just one in 40,000 children in the U.S. each year. Children with the disease often lose the ability to walk, talk and interact with the world around them. It is fatal, if untreated. It is caused by a missing enzyme in the blood. The team at M Health Fairview worked with the Food and Drug Administration to get approval for a gene therapy procedure that involved collecting blood cells from Celia Grace, sending them to Italy to be re-engineered to produce the missing enzyme, and then sent back to Minnesota. Following the infusion procedure at M Health Fairview, the cells went to work producing the missing enzyme in Celia Grace's body.
Dr. Paul Orchard, M Health Fairview Blood and Marrow Transplant Physician, said, "It's exciting to be able to offer this opportunity for Celia Grace and hopefully for many more patients like her in the future. It's a pioneering step forward for us, and hopefully for the fields of gene therapy and rare disease research."