UCLA Doctor Credited With Curing Child With Bubble Baby Disease

LOS ANGELES (CNS) - An infant whose family traveled 7,500 miles to save his life from a life-threatening immune disorder is thanking the UCLA doctor who treated him. 

In 2015, the child, Hussein El Kerdi, was diagnosed at three months old in his native Lebanon as having the immune disorder adenosine deaminase-deficient, also know as bubble baby disease, according to a statement from UCLA. 

The boy's immune cells lacked an important enzyme that could have made even a common cold deadly. Babies with the disease must remain isolated in germ-free environments to avoid exposure to viruses and bacteria and many with the condition typically do not survive past their second birthday. A relative of Hussein's parents, who is a doctor in Michigan, put them in touch with Dr. Donald Kohn, a physician-scientist at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, who has been perfecting a stem cell gene therapy for bubble baby disease for more than three decades. 

In April 2016, the family traveled 7,500 miles with then six-month-old Hussein to meet with Kohn in Los Angeles and begin treatment. 

The treatment consists of extracting blood-forming stem cells from the patient's bone marrow and correcting the genetic mutation that causes bubble boy disease by inserting the gene responsible for making adenosine deaminase. The corrected stem cells are then infused back into the patient and they begin producing a continual supply of healthy immune cells capable of fighting infection.``I hadn't seen a patient like Hussein in 15 or 20 years,'' Kohn said. ``About three to four weeks in, I thought he wasn't going to make it through. But he did.''In July 2016, Hussein began treatment and his recovery progressed so rapidly, he was healthy enough to return to Lebanon in September, just before his first birthday. 

His parents celebrated Hussein's first birthday with Dr. Kohn and the nurses who cared for him and expressed their gratitude.``I hope that when Hussein grows up, he comes to the States and gets educated to be a doctor at UCLA,'' his father, Ali El Kerdi, said. ``On behalf of myself and my wife and child, I want to say thank you to Dr. Kohn and to UCLA and to all the people who helped bring this miracle to life.''Nearly two years since the procedure, Hussein is healthy and living with his family in Lebanon. 

Kohn, whose work focuses on genetic blood disorders, received approval from the U.S. Food and Drug Administration in 1993 to test the treatment in clinical trials. 

Since then, 30 out of 30 babies with the condition have been cured in six trials run by Kohn. Data from a seventh trial is currently being analyzed.

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