September 23, 2009

Alzheimer’s gene therapy trial starting

Georgetown University researchers say they've started recruiting volunteers in the first gene therapy trial for dementia due to Alzheimer's disease.

The phase II study will examine the safety and possible benefits of CERE-110, which contains a gene and is injected during surgery into a part of the brain affected by Alzheimer's disease.

Researchers said the gene will instruct brain cells to produce more of a protein called Nerve Growth Factor, which helps nerve cells survive and function properly.

Our goal is to stop the progression of Alzheimer's disease, said Dr. R. Scott Turner, director of Georgetown's Memory Disorders Program, who is leading the research.

About 50 people with Alzheimer's disease will participate in the study at fewer than 10 hospitals nationwide. Only people with a mild form of Alzheimer's disease, who are evaluated and deemed competent to consent for themselves, will be permitted to participate, officials said.

All patients in the study will undergo surgery to drill two small holes in their skull. Only those patients randomly assigned to receive CERE-110 will have the gene therapy injected into the brain. Those subjects randomized to the placebo group will not have the gene therapy injected.

The study is supported by a grant from the National Institute on Aging in association with Ceregene, Inc, which will provide the active agent used in the research.