ALZHEIMER'S BREAKTHROUGH
Alzheimer's disease robs people of their memory and their ability to learn and to make judgments. It can also cause hallucinations, anxiety and agitation. Medications can treat the symptoms, but they don't target the cause. Now, a one-of-a-kind therapy that goes after the cause in hopes of a cure.
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Each year, more than four million Americans will be told they have Alzheimer's disease.
Neurologist Zoe Arvanitakis says we've made an impact on the disease, but more is needed.
Zoe Arvanitakis, M.D.
Neurologist
Rush University Medical Center
Chicago, IL
"There are several FDA approved medications to treat Alzheimer's disease, so they help with the symptoms of the disease, but they don't seem to change the underlying course of the disease."
Researchers from Rush University hope to do just that.
Zoe Arvanitakis, M.D.
"We're going after something completely new and using a very novel method as well."
It's called gene transfer. It uses the drug Cere 110 to send growth factors deep into the brain.
Zoe Arvanitakis, M.D.
"The issue up until now has been how to safely and effectively deliver nerve growth factors to the brain."
Now, using very thin needles, surgeons inject the drug into the area of the brain that deteriorates very early on in Alzheimer's.
Ron Shellady and his wife Sue knew they needed to act fast when ron got his diagnosis.
Ron Shellady
"I didn't want to get any worse. And I, you know, talked to Suzy about it, and I basically says, 'I don't care. I'll do anything.'"
And he did. Last July, he was the first patient in the world to receive this new drug.
Sue and Ron Shellady
"He's always been kind-of number one." "I like to win. There's no fun in losing."
Since the treatment, Ron says he's no longer losing his memory, but only time will tell how effective the treatment really is.
Ron Shellady
Had experimental treatment
"It's easier now not to forget; yes, I would say that's true. I pay more attention. I'm more attentive to things that I have, that I know I have to pay attention to."
With six grandkids, he'll have a lot to keep his eyes and his mind on.
Rush University in Chicago is the only center in the world participating in this research. To participate in the trial, patients must be between 50 and 80 years old in the earlier stages of Alzheimer's disease.
BACKGROUND: Alzheimer's disease affects more than 4.5 million Americans. It is caused by brain degeneration and is marked by the death of cells, particularly the cells in the basal forebrain. These cells are the primary source of the brain chemical messenger called acetylcholine. Alzheimer's disease impairs the production of acetylcholine, one of the main chemical messengers in the brain that is important for memory and cognitive function. The number of Americans with Alzheimer's has more than doubled since 1980 and will continue to grow. By 2050, researchers say the number of people with the disease could range from 11.3 million to 16 million.
STANDARD TREATMENT: Currently, there are five FDA-approved medications that treat cognitive symptoms in patients with Alzheimer's disease. Four of these try to boost the brain levels of acetylcholine by blocking the enzyme that breaks down acetylcholine. The fifth drug acts on another brain chemical. However, none of the available drugs are known to change the underlying neurobiology of the disease.
NEW SCIENCE: Investigators at Rush University Medical Center in Chicago have successfully initiated a new technique that uses gene therapy to deliver nerve growth factor into regions of the brain where neurons are degenerating. Their hope is to prevent cell death and reverse cell atrophy, two hallmarks of Alzheimer's disease. The new technique uses the drug CERE-110. CERE-110 carries the nerve growth factor (NGF) gene encased in a harmless viral coating, which protects the gene. A key objective of the study is to deliver the CERE-110 directly to the part of the brain that is almost universally affected by Alzheimer's disease, the basal forebrain, and not to other parts of the brain where it may cause side effects.
NEW HOPE: Rush researchers are studying CERE-110 as part of a phase I study to evaluate its safety and tolerability using two different doses. Memory and cognitive function will be assessed regularly during the two-year study. Between six and 12 subjects with mild to moderate Alzheimer's disease will be enrolled in the study. Researchers use a sophisticated means of delivering NGF to the brain that does not require a skin biopsy or the use of skin cells. Zoe Arvanitakis, M.D., from Rush, says, "If you can positively affect the basal forebrain, it may have a widespread effect on the entire brain because projections from that area reach out to all other parts of the brain, delivering the important neurotransmitter acetylcholine." Until recently, there was no practical method to deliver NGF to the specific areas of the brain where neurons were degenerating in Alzheimer's disease. Indiscriminate delivery of NGF to the brain would cause side effects, such as weight loss and pain.
Researchers say extensive studies in animals, including primates, showed that NGF gene delivery to the basal forebrain prevented the death of the same group of cells (cholinergic neurons) that undergo severe degeneration and death in Alzheimer's disease patients. NGF restored shrunken brain cells to near-normal size and quantity and also restored axons connecting the brain cells, essential for communication between cells.
Veronica Cech
Research Coordinator
Rush Alzheimer's Disease Center
600 South Paulina St. Suite 1029
Chicago, IL 60612
(866) 761-7806
veronica_ngf@rush.edu
Copyright © 2004 Ivanhoe Broadcast News, Inc.
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