Gene therapy cures life-threatening lung infection in teenage boy
Posted March 2, 2012on:
A 16-year-old boy who was born with a life-threatening immune disorder has become the first patient to benefit from a new form of gene therapy.
Doctors at Great Ormond Street Hospital in London said the treatment had only a temporary effect but was sufficient to help the boy overcome a lung infection. Remy Halbawi, who is at school in Wimbledon, south London, had been ill for two years with the fungal infection and would not have survived without radical treatment, doctors said.
Remy was born with a genetic condition called x-CGD (Chronic Granulomatous Disorder) that affects boys. Patients with the disorder have a faulty gene that disrupts a subset of immune cells called neutrophils, which act as the body’s first line of defence against infection.
Children born with x-CGD struggle to fight infections caused by fungi or bacteria, and are often hospitalised with severe infections in the first few years of life. A bone marrow transplant from a suitable donor is the only cure for the condition.