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Hemophilia Gene Treatment

Hemophilia Gene Treatment

More than 400,000 people suffer from hemophilia around the world, and currently, there is a global effort to eradicate this life-threatening bleeding disorder. Hemophilia gene treatment in Israel, based on genetic engineering, is breaking new ground as a way to treat the disease for life with just a single dose. Sheba is the only medical center in the world to offer innovative hemophilia gene treatment to international medical tourists.

Hemophilia Gene page
How Conditions Side Effects Candidate Why Sheba Team

How does hemophilia gene treatment work?

Healthy genes are engineered in a laboratory, and then a large number of them are inserted into the genetic material of a virus. Typically, the common human adenovirus is used, because it is important that the virus can be removed from the body without leaving damage after it has completed its mission.

The virus is infused intravenously into the patient. Once it travels to the liver, the virus will be cleared from the body – but the healthy genes will remain. The goal is that within a few months, enough of the liver cells that have been “infected” with the healthy genes will divide and multiply, replacing the defective genes that cause the coagulation disorder – and thereby resolving the hemophilia.

The treatment’s success is evident when the virus is no longer present in body fluids, the liver remains healthy, and the liver cells with the healthy gene begin to produce the missing clotting factors. Ultimately, the desired effect is that patients bleed less.

Infusing the gene into a patient with hemophilia is performed as a one-time treatment. Weekly follow-up evaluations are carried out during the first year, with long-term monitoring over five years.

What conditions can be treated with hemophilia gene treatment?

There are two types of hemophilia:

  • Hemophilia A (Classic Hemophilia) – caused by a lack of clotting factor VIII
  • Hemophilia B (Christmas Disease) – caused by a lack of clotting factor IX

Presently, hemophilia gene treatment is available in Sheba only for Hemophilia type A. However, Sheba Medical Center is already recruiting participants for clinical trials of this new therapy for Hemophilia type B; the trials are expected to begin next year.

What are the possible side effects from hemophilia gene treatment?

No serious side effects have been reported, although long-term data is still unavailable. A small group of patients who underwent this therapy experienced a transient increase of liver enzymes, which resolved efficiently by a simple drug treatment.

Who is and is not a candidate for hemophilia gene treatment?

This treatment is generally suitable for patients with hemophilia and a healthy liver over the age of 18.

Patients with liver illnesses or those who have developed antibodies for blood clotting factors cannot receive this treatment. To enable the gene treatment to work properly, patients should also have no antibodies to the virus that is used (therefore specific screening tests are performed prior to enrollment).

Why choose Sheba for hemophilia gene treatment in Israel?

Sheba Medical Center is one of the few hospitals in the world to offer this rare and revolutionary treatment to patients with hemophilia. Moreover, Sheba is the first healthcare facility to make hemophilia gene treatment available to international medical tourists.

Hemophilia gene therapy is performed at Sheba’s National Hemophilia Center. We are a world-recognized center staffed by experienced physicians and study coordinators who actively lead many state-of-the-art clinical studies. Our team has been rated as excellent by FDA evaluators. When you come to Sheba for hemophilia gene treatment, a premier team of medical specialists will design your individualized therapy.

An underlying foundation of all treatments at Sheba is the hospital’s deep dedication to holistic healthcare. In addition to administering gene treatment for your hemophilia, multidisciplinary doctors will consider your overall well-being – optimizing your physical comfort and tending to your psychological needs. Additionally, to ensure that your entire experience at Sheba goes smoothly, a personal coordinator from the International Medical Tourism Division will provide assistance every step of the way – including travel arrangements, language interpretation, and follow-up exams.

Gene therapy for hemophilia is affordable at Sheba. We are a government institution, with transparent fees and no surprises when it comes to cost. When compared to the price of treatment at other leading medical centers worldwide, you will benefit from maximum quality medical care at a minimal cost at our hospital in Israel.

Meet the Team

Gili Kenet
Prof. Gili Kenet
Director of The National Hemophilia Center and Institute of Thrombosis & Hemostasis Professor and Chair of the Hematology Department at the Sackler Medical School, Tel Aviv University
Prof. Kenet is a pediatric hematologist, director of the Israeli National Hemophilia Center and the Amalia Biron Thrombosis Research Institute of Tel Aviv University. Prof Kenet is currently leading Sheba’s clinical research studies on treating hemophilia with gene therapy. She is also involved in advanced research projects focused on neonatal hemostasis, pediatricthrombosis and novel non replacement therapies for patients with severe bleeding disorders. She has chaired the scientific subcommittee (SSC) of ISTH for pediatric and perinatal hemostasis and is now co-chair of the SSC for FVIII/ FIX and rare bleeding disorders. She has been awarded many international grants for her varied research and authored more than 200 manuscripts published in high impact medical journals, as well as several chapters in books. Professor Kenet received her medical degree from the Hebrew University in Jerusalem.
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Tami Barazani-Brutman, PhD
The National Hemophilia Center and Thrombosis Institute
Dr. Barazani-Brutman leads the study coordinators team at our National Hemophilia Center. She joined the center in 2009, and has since been responsible for all the clinical trials in the field of Hemophilia and Thrombosis. Dr. Barazani-Brutman has also been involved in dozens of international multicenter hemophilia studies, including pharmacokinetic studies. She is the author of many publications, and has served as an invited speaker for several research conferences.
Dalia Bashari, RN
Head nurse of the Israeli National Hemophilia Center
Dalia Bashari attended Nursing School at Sheba Medical Center, Tel HaShomer, and later trained in the specialized field of Advanced Oncological Nursing, also at Sheba. Over the past few decades, she has studied in many high level international nursing workshops for hemophilia, and attended symposiums in hemophilia care held in the United States, England, Ireland, Austria, Switzerland, and Spain. Ms. Bashari has also delivered numerous lectures on topics related to contemporary hemophilia therapy.

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Sheba Medical Center provides innovative, personalized medical care to patients from around the world. We are the largest, most comprehensive hospital in the Middle East and dedicated to providing advanced and compassionate medicine for everyone.

We welcome all cases, including the rarest and the most challenging. Our medical teams collaborate to provide the best possible health outcomes. From your initial inquiry through the long-term follow-up care, we are here for you.

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