Before Jake Gyllenhaal starred in the 2001 comedy Bubble Boy, there was a 1976 version starring John Travolta called The Boy In The Plastic Bubble. Both movies were inspired by the lives of David Vetter, who had severe combined immunodeficiency (SCID), and Ted DeVita, who suffered from severe aplastic amenia. Because of their compromised immune systems that needed to be protected from infections, both were required to live in completely sterile environments. David Vetter spent his short life in a plastic bubble (an isolator containment centre designed by NASA engineers). (David’s parents had another son who was born with SCID and died in infancy from the disease.)
In 1983, at the age of 12, David underwent a bone marrow “transfusion” from his sister, although she wasn’t a perfect match. It was a new way to treat immune disorders, but it wasn’t a success for David. He died four months later from lymphoma. Ted DeVita lived in a special, sterile room at the National Institute of Health Clinical Center. Like David, he could only leave his sterile space when wearing a spacesuit and helmet. He died, before his 18th birthday, from iron overload from too many blood transfusions and no chelation.
Earlier this week, Britain’s National Institute for Health and Care Excellence (NICE) approved GlaxoSmithkline’s gene therapy Strimvelis for USD $700,000, its gene therapy for adenosine deaminase deficiency-severe combined immunodeficiency, or ADA-SCID – also known as bubble boy syndrome. The single infusion is expected to be a lifetime cure. In 2016, Strimvelis was granted marketing authorization by the European Medicines Agency, but it is awaiting commercialization pending reimbursement decisions.
Meanwhile, UCSF Benioff Children’s Hospital San Francisco is testing a new gene therapy treatment for infants with SCID. The clinical trial has USD $11.9 million from the California Institute for Regenerative Medicine (CIRM) and uses technology developed by St. Jude Children’s Research Hospital that, according to UCSF, “delivers a functional gene into the patient’s blood-producing stem cells.” This new therapy would provide an alternative to stem cell transplants using donor cells, which can result in serious infection, graft-versus-host disease or rejection of the donor cells. CIRM provides more details here.
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