Gene Therapy’s Potential for Treating Neuroblastoma

A new, experimental gene therapy demonstrated promising results for the treatment of cerebral adrenoleukodystrophy (CALD). The treatment, called LENTI-D, is being developed by bluebird bio (BLUE). Results from the study, called STARBEAM, were presented at the American Academy of Neurology meeting.

The primary endpoint of the study is freedom from "major functional disabilities, " such as the losing ability to communicate or wheelchair dependence (MFD-free), and will be assessed two years following treatment. In this interim report, all 17 treated patients remained free of major functional disabilities, with three patients completing two years follow-up and the remaining 14 patients completing at least six months of follow-up. While encouraging, the results are preliminary, and more follow up is needed prior to FDA approval.

This is the second therapy based on blood stem cell modification to make news in recent weeks. The first, called Strimvelis, received a positive opinion from the European CHMP, which recommended approval to the European Commission for the treatment of severe combined immunodeficiency.