In a historic medical breakthrough, doctors in Israel’s Clalit Health Services -Schneider Children’s Medical Center, became 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, in a pioneering effort that 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.

Recognition of disorder

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

The treatment was an extraordinary international effort involving physicians, researchers, regulators and biotech leaders who moved quickly to make the impossible possible for one small child.

The breakthrough began with Dr. Naama Ornstein, head of genetics unit at Clalit- Schneider, who immediately recognized the rare disorder after the baby was hospitalized 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.

Determined not to lose another patient, Ornstein reached out to the scientist who originally developed the therapy, Prof. Rami I. Aqeilan, an Arab researcher from the Hebrew University of Jerusalem, whose academic discovery had since evolved into a clinical treatment acquired by a U.S. biotech company led by an Israeli-born physician and CEO.