"Strimvelis: La terapia genética ignorada por la industria farmacéutica que ha transformado la vida de Aitana"

En marzo de 2021, mientras el mundo comenzaba a recuperarse de la pandemia gracias a las vacunas contra la Covid-19, Mari Carmen Gómez se encerró con su recién nacida en una sala de aislamiento del Hospital Reina Sofía en la ciudad española de Córdoba. “Pasamos seis meses dentro de esas cuatro paredes. Aitana era una bebé con tubos, así que no podía hacer mucho con ella, pero sentía una profunda angustia. Netflix y mi teléfono me salvaron,” bromea Gómez, una cosechadora de aceitunas de 30 años originaria de Santaella, a 48 kilómetros de Córdoba.

Aitana, born two months earlier, had been diagnosed with severe combined immunodeficiency of adenosine deaminase (ADA-SCID), a rare genetic disease — occurring in just one newborn per year in Spain — that destroys the immune system’s cells. It is one of the most severe forms of what is colloquially known as “bubble baby syndrome,” where patients are forced to live in complete isolation because even a minor infection could be fatal.

Today, Aitana is a lively, happy, and curious child who is about to complete the first term of her first year at nursery school. Her immune system is fully functional, thanks to gene therapy — an innovative treatment in which a virus, in this case a retrovirus, is modified to deliver a healthy gene into the patient’s body, acting like a Trojan horse to replace the defective gene.