Israeli Researcher Patents Cure for 'Bubble Boy' Disease
by Karin Kloosterman
(IsraelNN.com) The film The Boy in the Plastic Bubble opened America's eyes to the rare genetic disorder -- severe combined immunodeficiency (SCID) -- afflicting up to 100 babies every year in the US. David Vetter, whose story the movie is based on, was a Texas boy who succumbed to the fatal disease known as Bubble Boy disease at the age of 12, after living years behind plastic barriers that protected his immune system from germs.
Thanks to research developed by an Israeli doctor, Prof. Shimon Slavin, babies and children everywhere may be spared from this fatal disease. Recent research based on Slavin's ground-breaking gene replacement approach developed in Israel, was tried and tested by a team of scientists from around the globe. The results were a success.
Using Slavin's patented approach, the international team, including researchers from Italy, found and then reported in the New England Journal of Medicine that Slavin's gene therapy protocol -- reduced-intensity conditioning (RIC) -- does not only treat, but appears to have cured eight out of the 10 children they treated for the fatal Bubble Boy disease.
"The first time the treatment was successful was our patient in Israel, based on my protocol, in my laboratory," says Slavin. "Based on that first patient, this is the basis of the [new] medicine," being reported in the journal, he says.
Slavin's story starts back in 2000, when he met an Arab family from East Jerusalem. Having a child who had already died from the disease, the family turned to Slavin to see if he could treat their new baby girl, also diagnosed with SCID.
He wanted to go for a cure
Currently the only treatments for SCID are either bone marrow transplants, which do not always work, or enzyme injections, which can cost up to half a million dollars a year. Any solution only prolongs life: "Even with those who have money, they will eventually die," Slavin tells ISRAEL21c.
Due to an internal disagreement between medical authorities over payments for the Jerusalem baby's treatments, the girl did not get enzyme therapy -- a mistake that would eventually save her life.
"Our child did not have any injection of enzyme," says Slavin, "I came up with the idea that we should go for a cure, and not repeat any failures. Based on our work with the biology of stem cells, we had a lot of experience.