First patient to receive breakthrough sickle cell gene therapy leaves hospital
First patient to receive breakthrough sickle cell gene therapy leaves hospital
The first patient to undergo recently approved gene therapy for sickle cell disease, Kendric Cromer, has left the hospital. The 12-year-old has been receiving treatment at the Children’s National Hospital in Washington, D.C., United States of America, for the past 44 days, but left the facility on Monday, reports the New York Times.
Sickle cell disease, caused by a genetic mutation in haemoglobin, is said to affect approximately 100,000 people in the U.S.
Until December 2023, there seemed to be no way out for those affected. However, the U.S. Food and Drug Administration approved two revolutionary gene therapies: a $3.1 million treatment developed by Bluebird Bio and a $2.2 million therapy from Vertex Pharmaceuticals.
These therapies now offer patients a chance to permanently alter the genetic mutation that causes the disease, potentially giving them a life free from its burdens.
Reflecting on how the disease had stolen his childhood, Cromer said, “I thought I would have sickle cell for the rest of my life.”
His parents explained that the decision to pursue therapy was not an easy one. They said that despite numerous consultations with doctors and careful study of medical texts, they were not fully prepared for the emotional and physical toll the treatment would take. “We knew it would be hard, but it was harder than we could have imagined,” said his mother.
