Sheba News Page New 2019 7

Revolutionary Gene Therapy at Sheba: Coming Closer to a Cure for Hemophilia

Researchers at Sheba Medical Center are advancing rapidly towards a cure for hemophilia, a bleeding disorder that prevents normal blood clotting. At present, more than 400,000 individuals suffer from hemophilia around the globe.

Using breakthrough gene therapy to eradicate hemophilia with a single dose of treatment, Sheba is one of the first hospitals worldwide to conduct this Phase III clinical trial on humans. Currently, six hemophilia patients are participating in Sheba’s clinical trial and receiving the treatment. The results of the trial will be known within two months.

The innovative, experimental gene therapy contains the common human “adenovirus,” which was developed by a bio-pharma company in the USA. The virus is used to deliver a healthy copy of a gene that compensates for the defective gene responsible for the excessive bleeding. Ultimately, the goal is to cure hemophilia with just one dose of this treatment.

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Prof. Gili Kenet, director of the National Hemophilia Center and Institute of Thrombosis & Hemostasis

Prof. Gili Kenet, pediatric hematologist and director of the National Hemophilia Center and Institute of Thrombosis & Hemostasis at Sheba, and a professor of hematology at Sackler Medical School, Tel Aviv University, is leading this study.

“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,” said Prof. Kenet. “I am cautiously optimistic that this new type of gene therapy will finally help eradicate this disease.”

Currently, treatment for hemophilia entails receiving infusions every few days with medicine to promote blood clotting. One participant in the clinical trial, a 23-year old male patient, has been undergoing infusions for hemophilia at Sheba since birth. He shares, “Though I like coming to Sheba for treatments over the years because of Prof. Kenet’s professionalism and dedication, which includes her entire doting staff, I would love to get rid of this disease – so I can go ahead and live a normal life.”