An eight-month-old baby from Israel has become the first human to have an experimental gene therapy directly injected into his brain neurons to cure a rare form of epilepsy.
The treatment, which inserted a missing gene, was performed at Clalit-Schneider Children’s Medical Centre in Peta Tikvah.
The surgery is intended to fix a severe genetic defect that can cause intractable epilepsy, profound developmental delays, and a high risk of premature death. It took place after an extraordinary international effort involving physicians, researchers, regulators and biotech leaders.
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 was not available, and the child died.
Alongside the genetic team, Dr. Dror Kraus, senior physician in the neurology unit 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, Dr. Ornstein reached out to the scientist who originally developed the therapy, Professor 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 US biotech company led by an Israeli-born physician and chief executive.
Together, the team crossed borders and bureaucracies to secure special emergency approval for the treatment, although it had never been administered to a human being.
In a delicate neurosurgical procedure, Dr. Ido Ben Zvi injected a functioning copy of the missing gene directly into the baby’s brain.
The operation required unprecedented medical and regulatory coordination, including special approvals from Israeli health authorities and international oversight bodies. Doctors say even the dosage calculations were extraordinarily sensitive, as the amount had to be tailored precisely to the infant’s brain size.
Dr Ornstein said: “This was much more than a medical procedure. It was a fight for a child’s chance to smile, develop and live.
“Families often ask us: if a child is missing a working gene, why can’t we simply give them one? Usually, we have no answer. This time, for the first time in the world, we could.”
The baby has since been discharged home, while doctors continue closely monitoring his neurological condition. The case is already being described as a landmark moment in the future of personalised genetic medicine and a rare story of science, humanity and cooperation overcoming every barrier in order to save a child’s life.
Clalit Health Services is Israel’s largest health organisation, running 14 hospitals and 1,600 community clinics nationwide.
