Gene therapy may cure Alzheimer’s disease

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To stop the progression of Alzheimer’s disease, scientists are conducting gene therapy trial on patients with a mild form of Alzheimer’s Disease in US.

Researchers in the Memory Disorders Program at Georgetown University Medical Center are now recruiting volunteers for a national gene therapy trial ? the first study of its kind for the treatment of patients with dementia due to Alzheimer’s disease.

The phase II study examines the safety and possible benefits of CERE-110. CERE-110 contains a gene and is injected during surgery into a part of the brain affected by Alzheimer’s disease. The gene will instruct brain cells to produce more of a protein, called Nerve Growth Factor or NGF, which helps nerve cells survive and function properly. The transfer of this gene into the brain is a medical technique called gene therapy.

“Our goal is to stop the progression of Alzheimer’s disease,” explains R. Scott Turner, MD, PhD, director of Georgetown’s Memory Disorders Program. “This is our first study of a gene therapy injected into brain, and thus the trial requires close collaboration with our neurosurgery colleagues at GUMC, in particular Dr. Chris Kalhorn.”

About 50 people with Alzheimer’s disease will participate in this study at fewer than 10 hospitals nationwide. Only persons with a mild form of Alzheimer’s Disease, who are evaluated and deemed competent to consent for themselves, will be permitted to participate in the study. The study requires each patient select a study partner for the length of the study. All patients in the study will undergo surgery to drill two small holes in the 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.

This study is a phase II, double-blind, placebo-controlled study.

Source: Georgetown University Medical Center, USA


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