What could make a family in Lebanon travel all the way to UCLA? The answer is a unique stem cell gene therapy designed to combat SCID. The El Kerdi family welcomed a son in September 2015. His name is Hussein El Kerdi. He appeared healthy, but a missing enzyme in his immune cells put his life at risk. Keep reading to learn more about Hussein’s condition and his family’s journey to save him or follow the original story here.
What is SCID?
SCID, is short for severe combined immunodeficiency. The condition is also sometimes colloquially referred to as “bubble boy diesease” or “bubble baby disease.”
SCID is a primary immunodeficiency that is characterized by a patient having little or no immune response. This leaves a person with SCID highly vulnerable to infection from bacteria, fungi, and viruses.
There are several types of SCID, the most common form accounting for about 45% of all cases. In these cases, SCID occurs as a result of a mutation on the X chromosome. This gene is responsible for T-cell growth factor receptor and other growth factor receptors involve in the production and function of the immune system.
Common symptoms of SCID include frequent and severe infections (especially respiratory), poor growth, eczema-like rashes, and chronic diarrhea.
First Case in the Middle East
Hussein was three months old when a doctor diagnosed him with SCID. His parents, in Lebanon, had no idea whether or not a treatment for their son’s dangerous condition even existed. They were told their son’s case of SCID was not only unusual, but was, in fact, the first case of the disease in the entire Middle East. They were informed that the only place where treatment existed was Great Britain and the United States. Even then, the treatments were described as experimental.
Friends and family of the El Kerdis began to assist them. Together, they created a plan. They would journey to the United States to seek treatment for their son. This journey would ultimately lead them to Dr. Donald Kohn at UCLA.
The El Kerdis arrived at UCLA in April of 2016. Their son was six months old and his condition was getting worse.
Dr. Donald Kohn’s research pursued a treatment for SCID for over thirty years. The result of this research has been the development of a stem cell gene therapy. Stem cells are important to the treatment because they can replicate themselves and they can produce effective immune cells which SCID patients lack.
This treatment involves removing the necessary stem cells from the SCID patient. Once the missing piece of genetic code is put into the patient’s stem cells, these cells can be returned to the patient’s body. Inside the body again, the cells begin to produce healthy immune cells.
Of the 30 babies treated with Kohn’s stem cell based therapy, all 30 have been cured of SCID.
Dr. Kohn said it had been almost 20 years since the last time he’d seen a patient like young Hussein El Kerdi. There was even a period of time where the doctor didn’t think he would make it.
On July 12th of 2016, the treatment process began. Two days later, the reprogrammed cells were returned to Hussein’s body. By the time September began, Hussein was healthy enough to return home where he and his family celebrated his birthday.
Two years later, Hussein is healthy. He is not only living, but thriving.