Gene therapy is safe for Alzheimer's disease
Insertion of genetically modified cells into the brains of patients with Alzheimer’s disease is well tolerated and reduces cognitive decline, according to preliminary findings reported at the American
Academy of Neurology last week.
In a phase I study, researchers modified skin cells from eight patients
to produce nerve growth factor in vitro. These cells were implanted into
the patients’ brains while they were sedated or anaesthetised.
The researchers report that annual rate of cognitive decline was reduced
after the operation, with greater reductions evident at longer post-operative
intervals. Animal models have previously suggested that nerve growth
factor, an endogenous protein that prevents cell death and stimulates
cell growth, may reduce cholinergic cell loss in Alzheimer’s disease.
The researchers say these new results provide a rationale for larger
trials of nerve growth factor gene delivery for the disease. The study,
conducted by researchers from the University of California, San Diego,
was reported at the American Academy of Neurology in San Francisco on
April 27. |
The
Pharmaceutical Journal
Acrobat
Reader