Stanford University researchers have reported mixed results ofPhase I clinical testing of their myoblast transfer technique totreat Duchenne's muscular dystrophy.
The therapy is aimed at compensating for a missing ordefective muscle protein, dystrophin, by injecting patients withhealthy muscle cells. In the disease, a genetic defect results ininsufficient quantities of the protein, leading to severe musclewasting and death in early adulthood.
The researchers reported last week in Nature that transplantedmyoblasts from healthy relatives persisted and produceddystrophin in the muscle fibers of three of eight boys with thedisease.
One patient showed evidence that the donor myoblastscontinued to express the gene for dystrophin at six monthsafter transplant.
However, the patients have yet to show improved musclestrength. "The efficiency of myoblast transfer remains low," theStanford team said.
-- Roberta Friedman, Ph.D. Special to BioWorld
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