Originally Published: 2009-02-22

Forced to live within the confines of the home, unable to have contact with the outside world or to live a normal life like other children – those afflicted by Severe Combined Immunodeficiency Disease (ADA-SCID) can finally smile.
The treatment administered by the team guided by professors Alessandro Aiuti and Maria Grazia Roncarolo at the Istituto San Raffaele, has been proven to be effective: the final results of the experimentation that began in 2000 were published in the New England Journal of Medicine.
It is a therapy that has changed the lives of many “bubble babies” – so called because they are forced to live in a sterile environment – who are now finally free to go out, whether it’s to school, or to play with friends. They are now able to lead a much more normal life.
Professor Aiuti is a medical researcher who boasts a brilliant scientific career. After acquiring his degree in medicine and surgery at the Università di Roma La Sapienza, and completing his doctorate in Human Molecular and Cellular Biology research at the same university, he went on to specialize in Haematology at the Università degli Studi di Milano. From 1994 to 1996, he did research at the Genetics Department at Harvard Medical School in Boston, and is currently associate professor of Pediatrics at the Univesità degli studi di Roma Tor Vergata. He has also published 63 scientific works in international magazines.

The stem cell gene therapy being used by your team has proven to be successful. How many children have you treated?

“Since 2000, we’ve successfully treated 12 children who came from all over the world. Among them was Parker, a Canadian child.”

We’ve been following Parker DesLauriers’ story. What can you tell us about this case?

“A year and a half has passed since Parker underwent treatment. The results are good. Parker is recovering and his immune system is returning to normal function, so we are satisfied with his response to the therapy.”

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