In a development that's being hailed as a major breakthrough in the search for an Alzheimer's disease cure, British researchers say they have identified a drug-like compound that combats the debilitating condition and may also work against other neurodegenerative illnesses like Parkinson's.
In a new study, published today in the journal "Science Translational Medicine," scientists from the Medical Research Council Toxicology Unit at the University of Leicester have found the compound can effectively "switch off" the defective neurological processes in the brains of dementia patients that lead to the death of brain cells.
The discovery could pave the way for new treatments for the condition that strikes millions of Americans and tens of millions more around the world.
"We’re still a long way from a usable drug for humans — this compound had serious side effects," noted lead researcher Giovanna Mallucci, in a university press statement. "But the fact that we have established that this pathway can be manipulated to protect against brain cell loss first with genetic tools and now with a compound, means that developing drug treatments targeting this pathway for … neurodegenerative diseases is now a real possibility."
Prior research by the MRC team had found the buildup of certain misshapen proteins in the brains of mice can trigger the kind of brain cell death seen in Alzheimer's patients. By blocking the production of those proteins, they found they could effectively protect the brain cells of the mice and allow them to live longer.
For the new study, the researchers gave an oral drug-like compound to mice with dementia-like symptoms, hoping to block the defective brain-signalling processes in the same way. The compound, which had originally been developed by GlaxoSmithKline for a different purpose, was able to enter the brain from the bloodstream and halt the disease, throughout the whole brain. It also produced weight loss in the mice and mild diabetes, due to damage to the pancreas, the researchers said.
Although the compound was tested in mice, researchers said the same principles apply to the brains in people with debilitating brain diseases such as Alzheimer's or Parkinson's.
"Our previous study predicted that this pathway could be a target for treatment to protect brain cells in neurodegenerative disease," said Mallucci. "So we administered a compound that blocks it to mice with [dementia]. We were extremely excited when we saw the treatment stop the disease in its tracks and protect brain cells, restoring some normal behaviors and preventing memory loss in the mice."
Hugh Perry, chair of the MRC’s Neuroscience and Mental Health Board, said he is confident future research will determine ways to blunt the negative side effects of the compound and that the findings have broad implications that go beyond treating Alzheimer's patients.
"Misshapen proteins in prion diseases and other human neurodegenerative disorders, such as Alzheimer's and Parkinson's, also over-activate this fundamental pathway controlling protein synthesis in the brains of patients," he said. "Despite the toxicity of the compound used, this study indicates that, in mice at least, we now have proof-of-principle of a therapeutic pathway that can be targeted.
"This might eventually aid the development of drugs to treat people suffering from dementias and other devastating neurodegenerative diseases."
According to the American Institute on Aging, as many as 5.1 million people in the U.S. have Alzheimer's disease. There is no current cure and studies of drugs to treat the condition have been disappointing in recent years. Existing treatments may slow the advance of the disease or modify the symptoms.
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