Video: Behind the scenes of a life-saving gene therapy stem cell treatment

“We were so desperate. When we heard about this treatment were willing to do anything to come here.”

In the above quote from Zahraa El Kerdi, “here” refers to UCLA, a world away from her hometown in Lebanon. In September 2015, Zahree gave birth to a son, Hussein, who appeared perfectly healthy. But by six months, he was barely clinging to life due to an inherited blood disorder, ADA-SCID, also called Bubble Baby disease. The disorder left Hussein without a functioning immune system so even a common cold could prove deadly. In fact, SCID babies rarely survive past one year of age. Up until now, no treatment options existed for the disease.

But Zahraa and her husband Ali heard about a CIRM-funded clinical trial, led by Donald Kohn, M.D. at UCLA, that could modify Hussein’s blood stem cells to fix the gene problem that’s causing his disease. The El Kerdi’s 7500-mile journey to save Hussein’s life is captured in a wonderful, five-minute video produced by UCLA’s Broad Stem Cell Research Center.

With before and after scenes of Hussein’s treatment as well as animation describing how the therapy works, the short documentary is equal parts heart wrenching, uplifting and educational. Basically, what I’m trying to say is, it’s a must-see and available to view above.

2 thoughts on “Video: Behind the scenes of a life-saving gene therapy stem cell treatment

    • Dear Jose, the only way to get gene therapy is to apply to the research team behind the therapy, they are the ones who make decisions about who is eligible to be part of the clinical trial. In the case of the SCID treatment we highlighted in our blog post you have to get in touch with the researchers at UCLA and/or Orchard Therapeutics. Here’s a link to the page on the http://www.clinicaltrials.gov that has details about eligibility and how to apply: https://clinicaltrials.gov/ct2/show/NCT02999984 Right now it looks as if they have all the patients they need but they may open it up to others in the future.

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