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Cancer drug prevents build-up of toxic brain protein
Date:5/9/2013

s tried anything like this before," he says.

Moussa, and his two co-authors graduate student Michaeline Hebron and Irina Lonskaya, PhD, a postdoctoral researcher in Moussa's lab searched for cancer drugs that can cross the blood-brain barrier. They discovered two candidates nilotinib and bosutinib, which is also approved to treat CML. This study discusses experiments with nilotinib, but Moussa says that use of bosutinib is also beneficial.

The mice used in this study over-express alpha-Synuclein, the protein that builds up in Lewy bodies in Parkinson's disease and dementia patients and which is found in many other neurodegenerative diseases. The animals were given one milligram of nilotinib every two days. (By contrast, the FDA approved use of up to 1,000 milligrams of nilotinib once a day for CML patients.)

"We successfully tested this for several diseases models that have an accumulation of intracellular protein," Moussa says. "It gets rid of alpha synuclein and tau in a number of movement disorders, such as Parkinson's disease as well as Lewy body dementia."

The team also showed that movement and functionality in the treated mice was greatly improved, compared with untreated mice.

In order for such a therapy to be as successful as possible in patients, the agent would need to be used early in neurodegenerative diseases, Moussa hypothesizes. Later use might retard further extracellular plaque formation and accumulation of intracellular proteins in inclusions such as Lewy bodies.

Moussa is planning a phase II clinical trial in participants who have been diagnosed with disorders that feature build-up of alpha Synuclein, including Lewy body dementia, Parkinson's disease, progressive supranuclear palsy (PSP) and multiple system atrophy (MSA).


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Contact: Karen Mallet
km463@georgetown.edu
Georgetown University Medical Center
Source:Eurekalert

Page: 1 2

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