Israeli breakthrough: gene replaced in baby’s brain

In a historic medical breakthrough, doctors in Israel have become the first in the world to treat a baby born with a fatal genetic brain disorder by injecting a healthy gene directly into his brain.

The eight-month-old baby has become the first human ever to receive the experimental gene therapy, designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival.

The pioneering effort at Clalit Health Services – Schneider Children’s Medical Center in Petah Tikvah, united an Israeli physician, an Arab scientist and the Israeli-born CEO of an American biotech company in a race to save the life of an ultra-Orthodox infant.

The breakthrough began with Dr Naama Ornstein, head of genetics unit at Clalit-Schneider, who recognised the rare disorder after the baby was hospitalised with seizures and severe developmental decline.

Years earlier, she had treated another child with the same condition, but at the time, the experimental therapy had not yet been available, and the child died. Alongside the genetic team, Dr Dror Kraus, senior physician in the Neurology unit at Clalit-Schneider Children’s Medical Center and an epilepsy specialist, has been closely monitoring and treating the child since the day he arrived at the hospital.