Doctors of the Sheba Tel-HaShomer Medical Center made history Wednesday by performing for the first time in Israel a surgery that delivers gene therapy directly to the brain.
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The procedure, which involved injecting the gene directly into the brain of the patient – 4-year-old Adiroop Kumar from India – lasted seven hours, and with the cost of 10 million shekels ($2.7 million) per vial was the single most expensive single surgery ever performed in Israel.
The groundbreaking treatment was conducted as part of a global study on the Upstaza gene therapy medicine, with 30 more children participating in Taiwan, Japan, China, Germany, England, France, and the United States.
In Israel, the surgery was conducted by Dr. Zion Zibly, director of the Department of Neurosurgery at the Sheba Medical Center and Dr. Lior Ungar, a senior neurosurgeon in the department. Dr. Bruria Gidoni-Ben-Zeev, head of the Pediatric Neurology Department at the Edmond and Lily Safra Children's Hospital, oversaw all the treatments, and the follow-up and medical care.
Adiroop had arrived with his mother from India specially for the procedure, which is conducted as part of the study, free of charge. A few years earlier, he was diagnosed with AADC deficiency, an incredibly rare genetic disease that is caused by changes in the gene that produces the AADC enzyme needed to produce certain substances vital for the normal functioning of the brain and nerves, such as dopamine and serotonin. The condition makes it nearly impossible for a child to lift his or her head, let alone walk and talk.
Until now, there has been no cure for AADC deficiency, which most often leads to death by the age of 10. In Israel, 10 children have been diagnosed with the disease in recent years, with four fatal cases. Around two new cases are diagnosed in Israel yearly.
Upstaza is a first-of-its-kind treatment that involves introducing a healthy gene into the patient's brain, into the area that misses the necessary gene. It has already been authorized in Europe, with Israel and the United States to follow.
"We work with hundreds of patients, and each and every one of them has a unique story. The same is true in this case, which fills us with the hope that we will be able to save many more lives in Israel and around the world. We believe that this scientific breakthrough will also serve us in other areas, and will allow us to bring relief to many patients,' Zibly said.
Adiroop's mother told Israel Hayom, "Adiroop is our second child, and in a genetic test we did at the age of six months, he was diagnosed with the disease. The doctors warned us that his life expectancy might be short. Therefore, every morning and all the time I check his breathing to make sure he is alive. I really hope that there is a chance that he will survive this terrible disease.
"We hope that in Israel we will start a new life and path for our son. We also feel in Israel and in the hospital the humanity and the kindness, we no longer feel like strangers. We love the people, the culture, the tradition, the language and the food. Indeed it is the Holy Land."
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