Ground breaking gene therapy study results for X-linked SCID announced

This week, researchers at St. Jude Children's Research Hospital revealed some exciting study results related to gene therapy treatment options for X-linked Severe Combined Immunodeficiency (SCID), one of the most severe forms of primary immunodeficiency diseases (PI).

The gene therapy methodology examined in the study fully restored the immune system of all ten toddlers with X-linked SCID in the study group. In addition, there’s no indication that the children may develop leukemia, which has been a concern in the past for those who have undergone gene therapy. 

While the study size was fairly small and the patients who took part in the study need to be followed long term to make certain they remain healthy, this is some very promising news for the PI community.

The study results generated national and international coverage related to SCID that will help raise awareness of this and other types of PI. 

See below for links to some of the major news stories that ran in the past few days.

US News & World Report: “Gene Therapy Might Prove a Cure for Bubble Boy Disease”
(IDF President & CEO John G. Boyle quoted)

CNN: “Doctors Develop ‘Cure’ for Babies with Bubble Boy’ Disease”
(IDF Mentioned)

NBC News: “Babies with ‘Bubble Boy’ Disease Cured Using Gene Therapy”

Washington Post: “Gene Therapy Cures Infants with Bubble Boy Immune Disease”

BBC Newshour Radio: (IDF President & CEO John G. Boyle interviewed at length around 39 minute mark)

Health Day News: “Gene Therapy Might Prove Cure for Bubble Boy Disease” 
(IDF President & CEO John G. Boyle quoted)