Drug that stops the progression of Parkinson's disease goes to human trials



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An exciting experimental drug developed by scientists at Johns Hopkins Medicine has been found to stop the progression of Parkinson's disease in living mouse models. The new drug may be the first drug to specifically slow the progression of the devastating disease as opposed to current treatments that target only the symptoms.

Microglia are a kind of immune cell mainly found in the brain. One of the neurodegenerative processes that occurs in the brain of patients with Parkinson's disease is when microglial cells send chemical signals to another type of brain cell called astrocytes. This signal repels these astrocytes in more aggressive behaviors, gnawing the connections between the neurons.

"The activated astrocytes we focus on are going to revolt against the brain," says Ted Dawson, one of the project's researchers, "and this structural degradation contributes to dead areas of brain tissue in people with Alzheimer's disease. of Parkinson's disease, the idea being that if we could find a way to calm these astrocytes, we could slow down the progression of Parkinson's disease. "

A fascinating new drug developed by researchers NLY01 calls and it is similar to a group of drugs already used in humans to manage insulin levels for type 2 diabetes. Early experiences of the Johns Hopkins team have shown that NLY01-treated human microglia effectively deactivated its negative signals and ceased to transform healthy astrocytes into aggressive and damaging astrocytes.

The next step was to check the effectiveness of the drug in mouse models simulating Parkinson's disease. Two distinct mouse models were used to evaluate the effectiveness of the drug, and in both models, NLY01 was able to slow neurodegenerative progression or stop it altogether. An experiment with transgenic mice designed to simulate the degeneration of the disease showed that the animals lived 120 days longer than their untreated counterparts, a 30% longer life expectancy.

The next step in the process will be human clinical trials. test the safety profile of the compound. Dawson is confident that early safety tests should be fast because of the similarity of the compound with existing drugs that have already proven safe for humans. It is not yet known whether the compound acts effectively on human subjects, but it is undoubtedly an exciting step forward in the development of a drug capable of slowing the onset of this a devastating disease that affects one million people in the United States alone

. study was published in the journal Nature Medicine .

Source: Johns Hopkins Medicine

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