Revolutionary Gene Therapy Could Eliminate Hemophilia


Israel’s Sheba Medical Center, Tel HaShomer Could Eliminate Hemophilia Disease with Revolutionary Gene Therapy

World Hemophilia Day is today, April 17 and Israel’s Sheba Medical Center, Tel HaShomer is making significant progress in the global battle to cure this bleeding disorder. More than 400,000 individuals suffer from hemophilia worldwide.

Sheba is one of the first hospitals in the world to conduct a Phase III clinical trial on humans using innovative gene therapy that contains human “adeno virus” (a common virus). It was a developed by a bio-pharma company in the United States. Six hemophilia patients are receiving the revolutionary treatment as part of this clinical trial.

This experimental treatment uses a virus to deliver a healthy copy of a gene to make up for one that causes the excessive bleeding. The goal is to cure the disease for life with just a single dosage.

Results of this trial will be known within two months.

The project is being led by Professor Gili Kenet, a pediatric hematologist and director of the National Hemophilia Center and Institute of Thrombosis & Hemostasis at Sheba Medical Center and a professor of hematology at Sackler Medical School, Tel Aviv University.

“We have worked with this particular bio-pharma company on a variety of treatments for hemophilia because they value our first-class research facilities and our dedication to finding ways to help our patients. I am cautiously optimistic that this new type of gene therapy will finally help eradicate this disease,” says Kenet.

A 23-year-old male patient, who is part of this clinical trial, has been treated at Sheba Medical Center since birth. “Though I like coming to Sheba for treatments over the years because of Professor Kenet’s professionalism and dedication, which includes her entire doting staff, I want to get rid of this thing out of my body so I can go ahead and live a normal life,” he said.

The current treatment for hemophilia requires patients get infusions every few days with medicine that helps their blood clot.

About Sheba Medical Center, Tel HaShomer

Born together with Israel in 1948, Sheba Medical Center, Tel HaShomer is the largest and most comprehensive medical center in the Middle East. Sheba is the only medical center in Israel that combines an acute care hospital and a rehabilitation hospital on one campus, and it is at the forefront of medical treatments, patient care, research and education. As a university teaching hospital affiliated with the Sackler School of Medicine at Tel-Aviv University, it welcomes people from all over the world indiscriminately. In 2019, Newsweek magazine named Sheba one of the top ten hospitals in the world. To learn more, visit: eng.sheba.co.il.

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