"The revolutionary treatment is administered in just one single dose, with no need for further doses, but it is so sophisticated that it can cost around one million euros. Eliana received it a decade ago and now leads a completely normal life. She gets great grades in school, plays basketball, and has even joined the school choir. It's incredible, her father recounts with emotion."
"For me, a cure means the absence of the disease for life. We can't say that yet. However, the observation period has already lasted between seven and 12 years, and the clinical benefits have remained completely stable, so I hope it will continue to be that way for life, Kohn, of the University of California, Los Angeles, explains to EL PAIS."
An experimental one-dose gene therapy has saved 62 children with ADA-SCID, a genetic childhood disease that severely weakens the immune system and permits fatal infections such as pneumonia, meningitis and chickenpox. The therapy uses autologous blood or bone marrow stem cells into which a healthy copy of the defective gene is inserted and then returned to the patient. The single-dose treatment can cost around one million euros. Recipients like Eliana have returned to normal lives with schooling and sports. Follow-up in some cases spans seven to twelve years with stable clinical benefits and reported 100% survival in five long-term cases, while researchers remain cautious about declaring a lifelong cure.
Read at english.elpais.com
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