Game-changing treatment next to be used for sickle cell disease; clinical trials to begin in early 2015

  • UCLA stem cell researchers cured 18 children born without a working immune system due to life-threatening ADA-deficient Severe Combined Immunodeficiency (SCID)
  • Breakthrough stem cell gene therapy developed by Dr. Donald Kohn and team identifies and corrects faulty gene in children with ADA-deficient SCID using child’s own cells

UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called “Bubble Baby” disease, a life-threatening condition that if left untreated can be fatal within the first year of life.

The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patient’s own cells with no side effects.

To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health.

Good Morning America: UCLA Researchers Announce Gene Therapy Cure for 18 ‘Bubble Baby’ Patients

Today Show: New 'bubble baby' treatment means kisses for 18 kids

TIME Magazine: ‘Bubble Boy’ Disease Cured With Stem Cells

CBS: UCLA Doctors Hail Potential Cure For ‘Bubble Baby’ Syndrome

Fox: Bubble Baby: Gene Therapy Cured 2-year-old Girl Of Deadly SCID At UCLA

Media Contact: 

Mirabai Vogt-James
(310) 983-1163
mvogt@mednet.ucla.edu