Gene therapy trial wrenches families as one child’s death saves another
July 20, 2016
MIT Technology Review reports on a gene therapy trial in
Italy that has reported strong early results for an inherited neurodegenerative
disease that leads to paralysis, dementia and, ultimately, death. The trial led
by Alessandra Biffi, MD – now director of gene therapy at Dana-Farber/Boston
Children’s – helped a number of babies identified through newborn screening
after an older sibling died of metachromatic leukodystrophy (MLD).
The experimental gene therapy works best if treatment is given before symptoms
appear. It appears to be the first gene therapy designed to be given
preventively, Tech Review reports, and, according to recently published
results, freezes or prevents the rapid onset of the disorder.