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Published on: April 7, 2012
MUSC research could lead to Alzheimer’s treatment
by Paul Bowers for Charleston City Paper
If researchers find a treatment for Alzheimer’s disease in the next decade, it might be because of a recent study conducted at the Medical University of South Carolina.
Dr. Mark S. Kindy, a professor of neuroscience at MUSC’s College of Medicine and a research career scientist at the Ralph H. Johnson VA Medical Center, was the lead investigator in a study that focused on cathepsin B (also known as CatB), an enzyme that has been shown to generate brain plaque. The plaque, made of fibrous proteins called amyloids, is thought to be a cause of Alzheimer’s.
Alzheimer’s disease is a degenerative and fatal form of dementia that causes irritability, confusion, and memory loss. According to the Alzheimer’s Association, as many as 5.4 million Americans and 80,000 South Carolinians are living with the disease today. It is the sixth-leading cause of death in the country, and as many as 16 million people could have the disease by 2050. There is currently no cure, although drugs like Aricept have been developed to treat the symptoms.
All of that could change if Kindy’s study, which was published in the online edition of the Journal of Alzheimer’s Disease, leads to the development of a medication that treats the causes of Alzheimer’s, not just the symptoms.
“If you find a drug that doesn’t have a lot of side effects, it could be a preventive medicine like Lipitor,” Kindy says.
Much of the research on brain plaque has focused on BACE1, a gene that has long been thought to be a cause of the disease. Deleting BACE1 from lab animals’ gene sequences has been shown to reduce brain plaque, but it has also been shown to actually worsen memory deficits.
To test the effects of CatB, Kindy’s team — which also included researchers from the University of California, San Diego — injected DNA into mouse egg cells to create transgenic mice that developed brain plaque like humans. They found that blocking BACE1 was only useful in reducing the occurrence of a mutated form of amyloid precursor protein (APP), whereas blocking CatB reduced the occurrence of the more common “wild-type” APP.
The researchers have been working with a San Diego drug company called American Life Science Pharmaceuticals, and Kindy says a new drug could be on the market in five to 10 years.
“With the baby boomers coming of age now, 60, 65 years of age, even though they’ve been relatively healthy, you know, as you age, things start to happen. The body starts to fall apart,” Kindy says. “And they’re very conscious about taking care of themselves, so they’re going to want these drugs, and they’re going to want these therapeutics in order to hopefully help them out.”
PICTURE: The brain on the left is smaller due to the shrinkage and death of cells caused by Alzheimer’s Disease. A new study published by MUSC researchers could lead to an effective treatment in five to 10 years.
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