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Neuroprotective Compounds Appear to Work - at Least in Mice

by Lynn Shapiro, Writer | November 05, 2008

"Tissue culture and mice are easy to work with but far removed from patients," he says. He adds, "It's difficult to say which compound will ultimately work, ours or others. So far, the track record hasn't been good although I'm optimistic that effective compounds will be found for patients."

Fortunately, treatments are available to ameliorate symptoms, especially in the case of Parkinson's disease, which causes a tremor due to the loss of the neurotransmitter dopamine.

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"The symptoms associated with Parkinson's disease can temporarily be stopped with a combination of L-Dope and COMT, or with MAO inhibitors, so that patients (notably including actor Michael J. Fox and Newsweek columnist Michael Kinsley) may do well on the medicine for a decade or more," Dr. D'Mello says.

"However, such drugs are not a cure. They don't slow down the loss of neurons, which cause the disease. The attractive feature of the new compounds is that they may be able to stop the root cause of the disease."

For Alzheimer's disease patients, Aricept and other drugs on the market improve learning and memory, but the gains are modest.

There is no cure for either condition or for other neurodegenerative diseases such as ALS or Huntington's disease. After a while, the progressive death of nerve cells results in a loss of brain function and eventually, the patient dies. "Our major thrust is to find out what happens in a neuron that's degenerating and then as a biologist, my interest is investigating what molecules and genes are responsible for degeneration and which are involved in preventing neuronal loss," he says.

"Once we understand the workings of neurons in greater detail, we can prevent them from dying, by designing drugs."

The Society for Experimental Biology and Medicine said in its press release heralding Dr. D'Mello's discovery of indolones that "because the population is aging and people are living longer, the identification of small-molecule inhibitors of neuronal death is of urgent and critical importance."

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