State targets ‘bubble boy’ syndrome

California’s stem cell agency is ready to award $20 million on Thursday to a UCLA researcher to assist in his 30-year search for a widely available cure for what has come to be known as the “bubble boy” syndrome– severe combined immunodeficiency (SCID).

The California scientist is Donald Kohn, of the Broad Stem Cell Research Center at UCLA, who said Tuesday that 30 out of 30 babies have already been cured using his type of therapy. Kohn said that the research involving the extremely rare disease could lead to progress in treating other afflictions ranging from sickle cell disease to cancer.

The progress in treating the “bubble boy” disease is much changed from decades ago when the case of David Vetter captured the nation’s attention as the boy who was “born into a world he could not touch.”  He ultimately died at the age of 12.

Kohn’s proposal for an early stage clinical trial would be co-funded with $8.9 million and would test a stem cell and gene product known as OTL-101. It could replace what the stem cell agency said were the “suboptimal,” current treatments for  Adenosine Deaminase Severe Combined Immunodeficiency.

The affliction is extremely rare and occurs in less than one in 100,000 births worldwide, according to Wikipedia. Without treatment, children can die before the age of two.

The agency’s application review summary said that reviewers were “highly enthusiastic” about Kohn’s proposal during their closed-door session last month. The summary said that reviewers, who are from out-of-state and do not publicly disclose their economic interests, “applauded the move to a cryopreserved product that will allow improved patient access to the therapy.” They also noted that the costs of the trial are “exceedingly high.”