In first, Israeli doctors deliver gene therapy into brain of 8-month-old baby

In a medical world first, Israeli doctors have delivered gene therapy into the brain of an eight-month-old baby suffering from a rare, life-threatening form of genetic epilepsy, the Hebrew University of Jerusalem says.

The procedure, which replaced the missing critical gene, was performed at the Schneider Children’s Medical Center in Petah Tikva.

Prof. Rami Aqeilan of the Lautenberg Center for Immunology and Cancer Research at the university’s Faculty of Medicine laid the groundwork through his research into the WWOX gene.

Children born with this severe genetic defect suffer from intractable epilepsy, profound developmental delays, and a high risk of premature death. While mutations of this gene are found worldwide, the specific catastrophic mutation treated in this case is particularly prevalent among people of Yemeni Jewish ancestry.

Dr. Naama Orenstein and Dr. Dror Kraus of Schneider Children’s Medical Center performed the surgery last month. The doctors used a single injection to deliver healthy copies of the WWOX gene directly into the child’s brain neurons.

The child has since been discharged from the hospital and is in stable condition.

“What began as a scientific effort to understand the biological function of a gene has now become a potential treatment for children suffering from one of the most severe forms of epilepsy,” says Aqeilan.