New gene therapy protocol: First successful treatment for 'bubble babies'

Jerusalem (June 21, 2002) -- A lifesaving protocol that has wide ranging consequences for the successful treatment of genetic diseases has been developed by a team of Israeli and Italian researchers for the treatment of children born without immune systems. A description of the first successful application of the new treatment appears in the June 28 issue of the journal Science.

The procedure, which improves an existing protocol to give a biological advantage to genetically altered stem cells, resulted in a complete reversal of severe combined immunodeficiency (SCID), a condition caused by the lack of an essential enzyme, adenosine draminase (ADA). Children born with an acute form of SCID are known as "bubble babies" and have to be kept in isolation units to prevent them from coming in contact with infectious elements.

The research was led by Prof. Shimon Slavin, Dr. Shoshana Morecki and Dr. Memet Aker of Hadassah-Hebrew University Medical Center in Jerusalem, in collaboration with Dr. Allesandro Aiuti and his colleagues from the San Raffaele Institute for Gene Therapy in Milan, Italy.

According to Slavin, Head of Department of Bone Marrow Transplantation at Hadassah Medical Center, the significance of this new development extends beyond those immediately affected by SCID and has the potential to be equally effective in curing similar genetic diseases, such as Gaucher's and other diseases caused by enzyme deficiency.

"The concept can be applied to all genetic diseases where there is a need to engineer stem cells to produce normal products, especially when patients have no matched donor available for safe bone marrow transplantation," Slavin said.

Conventionally, such patients are usually treated with ADA replacement therapy in an attempt to rescue the immune system. This replacement therapy, however, is not a long-term solution; it is only effective for one or two days at a time and is extremely expens

Contact: Roberta Elliott
Hadassah, the Women's Zionist Organization of America

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