Historic gene therapy trial to treat Alzheimer’s disease underway at Georgetown
The Phase II study examining the safety and potential benefits of CERE-110. CERE-110 contains a gene and injected during surgery in a part of the brain affected by Alzheimer’s disease. The gene that will instruct the 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 in the brain is a medical technique called gene therapy.
“Our goal is to halt the progression of Alzheimer’s disease,” explains R. Scott Turner, MD, PhD, director of the Program Memory Disorders at Georgetown. “This is our first study of gene therapy is injected into the brain, and therefore the process requires close collaboration with our colleagues in neurosurgery at GUMC, in particular, Dr. Chris Kalhorn.
Kalhorn says Turner, an associate professor in the department of neurosurgery at Georgetown University Hospital, usually performed neurosurgical procedures similar to that used in this study.
About 50 people with Alzheimer’s disease participate in this study in less than 10 hospitals nationwide. Only persons with a mild form of Alzheimer’s disease, which are assessed and deemed competent to consent for themselves, be permitted to participate in the study. The study required each patient to select a study partner for the duration of the study. All patients in the study will undergo surgery to drill two small holes in the skull. Only patients randomized to receive CERE-110 gene therapy will be injected into the brain. Subjects randomized to the placebo group will not have the gene therapy injection.
This study is a phase II double-blind, placebo-controlled.
Phase II: The investigator has been studied in a small number of patients and this study is being conducted to determine its safety and potential benefits.
Double blind means that patients, clinical coordinators and doctors do not know whether the patient received the investigational agent until the end of the study. Only the neurosurgeon operating and equipment delivery of gene therapy will tell if the patient received the active agent.
Placebo-controlled means that patients will be randomly selected to receive either the active agent or not, but all patients undergoing surgery. This study was approved by the FDA and the Institutional Review Board GUMC.
More information: For more information about this or other studies, contact Disorders Program Memory Georgetown 202-784-6671 or visit the website at memory.georgetown.edu.
Tags: agent, alzheimer, brain, disease, disorders program, gene, gene therapy, gene therapy injected, georgetown, injected, memory, memory disorders, memory disorders program, patients, phase ii, safety and possible, therapy, therapy injected