Alzheimer's gene therapy treatment enters phase II trial

Dr R. Scott Turner and his team of the Memory Disorders Program at Georgetown University, US, have begun recruiting patients to take part in a gene therapy trial, which hopes to test whether gene therapy using the nerve growth factor (NGF) gene could be used to slow the progression of Alzheimer's disease.

Alzheimer's disease is the most common cause of dementia, affecting around 417,000 people in the UK alone. Proteins in the brains of affected people begin to stick together and form what are termed 'plaques', which in turn interfere with cell function and eventually lead to cell death. At the beginning stages of the disease, patients can often feel they are having lapses of memory or simply having trouble finding the right words, but as the disease progresses the symptoms worsen and it is eventually fatal.

The new gene therapy treatment, called CERE-110, consists of the genetic information for a protein called 'nerve growth factor' (NGF), which is a survival factor for the neuronal cells in the brain. Once injected into the affected areas of the patient's brain, it is hoped that it will help protect the cells so they can continue to function properly, thus slowing the progression of the disease.

Fifty patients classified as having a mild form of Alzheimer's disease - and so competent to consent for themselves - will be eligible for the trial. All participants will undergo the neurosurgery but only half of the patients will actually receive the CERE-110 treatment, with the other half getting a non-effective placebo. The study is sponsored by the Alzheimer's Disease Cooperative Study (ADCS) through a grant from the National Institute on Aging (a part of the NIH, National Institutes of Health) in association with Ceregene, Inc.