From Morocco to San Diego: One-Person Clinical Trials, Gene Therapies and CIRM
The good news for gene therapy and a cure for congenital deafness could be heard, one could say, all the way from Morocco.
The news involved a clinical trial for one, 11-year-old boy, who is now hearing as a result of the treatment. The success of the trial has implications for a proposed $1 million, one-person clinical trial being considered by the governing board of the California Institute for Regenerative Medicine (CIRM) on Thursday.
Two San Diego researchers sought the CIRM funding to test a gene therapy for an extremely rare genetic defect that has caused intractable epilepsy and severe neurodevelopmental delay in a child. Their application was sidetracked last November when CIRM board members raised policy questions about a clinical trial for only one person. Such trials are called “n of one,” statistical jargon meaning that the total number of patients (n) equals one.
The New York Times carried the story this morning about the deafness trial and 11-year-old Aissam Dam. The excellent article by Gina Kolata said,
“On Oct. 4, Aissam was treated at the Children’s Hospital of Philadelphia, becoming the first person to get gene therapy in the United States for congenital deafness. The goal was to provide him with hearing, but the researchers had no idea if the treatment would work or, if it did, how much he would hear.
“The treatment was a success, introducing a child who had known nothing of sound to a new world.
“‘There’s no sound I don’t like,’ Aissam said, with the help of interpreters during an interview last week. ‘They’re all good.’ (He communicates with a sign language he invented.)
Joseph Gleeson and Olivia Kim-McManus, both of UC San Diego, are co-principal investigators on the CIRM application. A few hours ago, the California Stem Cell Report asked Gleeson via email for his thoughts on the implications of the deafness trial in connection with their application (CLIN2-15085) He replied,
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