Unprecedented: Israeli doctors deliver gene therapy directly to infant’s brain

In effort to save the life of a baby born with a devastating neurological disease, Israeli team uses experimental gene therapy to directly deliver missing gene to the child’s brain.

By World Israel News Staff

Doctors at Schneider Children’s Medical Center in Israel have treated an eight-month-old baby with an experimental gene therapy designed to replace a missing gene linked to a fatal neurological disorder, in what the hospital described as the first procedure of its kind in the world.

The baby, from an ultra-Orthodox family in Israel, was hospitalized after suffering seizures and severe developmental decline. Genetic testing identified a rare disorder caused by the absence of a functioning gene needed for normal brain function.

The treatment was carried out at Clalit Schneider Children’s Medical Center, part of Clalit Health Services.

According to the hospital, the case required coordination among doctors, researchers, regulators and a US biotech company in order to obtain special emergency approval for a therapy that had not previously been given to a human patient.

Dr. Naama Ornstein, head of the genetics unit at Schneider, recognized the disorder after the child was admitted.

She had previously treated another child with the same condition before the experimental therapy was available. That child later died.

Dr. Dror Kraus, a senior physician in the hospital’s neurology unit and an epilepsy specialist, has been monitoring and treating the infant since his arrival at the hospital.

After the diagnosis, Ornstein contacted Professor Rami I. Aqeilan of the Hebrew University of Jerusalem, whose research helped form the basis for the therapy. The treatment was later developed by a US biotech company headed by an Israeli-born physician and CEO.

The procedure was performed by Dr. Ido Ben Zvi of Schneider’s neurosurgery unit. During the operation, doctors injected a functioning copy of the missing gene directly into the infant’s brain. The gene was delivered using an engineered viral vector designed to reach cells in the central nervous system.

Doctors said the procedure required unusual medical and regulatory coordination, including special approvals from Israeli health authorities and international bodies. The dose also had to be calculated specifically for the infant’s brain size.

“This was much more than a medical procedure,” Ornstein said. “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. Doctors said they will continue to monitor his neurological development closely.

Schneider officials said the case could mark an important step for personalized genetic medicine, particularly for rare disorders in which a single missing or defective gene causes severe disease.