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Dr. Kim Mulvihill ReportingMedical researchers may have discovered a new approach for treating brain disorders like Alzheimer's and Parkinsons. The discovery comes out of methods to treat a childhood disease with a peculiar name - niemann pick type c.
For families, a diagnosis is devastating. The disease usually strikes very young children and causes a progressive deterioration of the nervous system. It interferes with the child's ability to metabolize cholesterol. With niemann pick type c, brain cells accumulate fat and die.
Synthia Mellon, M.D., UCSF Researcher: “it's 100 percent fatal disease. All children and adults who have this disease will die from it. And there is no treatment for this disease.”
But a possible new strategy for treatment - involving mice -has emerged in a lab at UCSF. The mice come from a family that naturally develop the disease. A mouse with the illness cannot perform the simplest task of crawling across a piece of string. The mouse barely holds on.
But another mouse also with the disease can complete the task with no apparent difficulty. The difference between the two? The mouse who can shimmy across the string received a single dose of a treatment.
Dr. Mellon and her team discovered the mice with the disease made less and less of an important hormone: a neurosteroid called allopregnanolone. Neurosteroids appear to play a role in how our nervous system develops.
Synthia Mellon, M.D.: “If neurosteroids are important in the development of he nervous system, could that contribute to their neurodegenerative demise, and could replacement or treatment alleviate some of the demise in those animals?”
A single treatment of the hormone provided dramatic results. And researchers discovered, the earlier they administered the treatment, the longer the mouse lived. It's not a cure, but the treatment did delay the onset of neurological problems.
The researchers caution their findings must still be evaluated in humans. But their research could have implications for treating other neurodegenerative diseases -- diseases such as parkinson's.. and alzheimer's. Dr. Mellon believes their results suggest they should start looking.
The hormone in question is also produced naturally in humans, and produced no apparent side effects in the mice.
