Oral drug could repair brain damage caused by Alzheimer’s disease

In the United States, nearly 6 million adults have Alzheimer’s disease. The disorder usually strikes before the age of 60 and there is no cure, although there are medications that can alleviate symptoms of memory and other cognitive changes.

However, scientists may be on the cusp of a treatment that can stop Alzheimer’s disease in its tracks. In a study published on Wednesday in the review Science Translational Medicine, a group of researchers led by neuroscientists at Yale University have discovered an experimental drug taken by mouth that appears to reverse brain damage in mice with Alzheimer’s disease and may protect the brain from future damage for at least less than a month. If its remarkable success and safety can be replicated in future human clinical trials, the new drug, called BMS-984923, could save countless Alzheimer’s disease patients. It might even prevent disease for those at risk.

To treat Alzheimer’s disease, it is first important to understand what causes it. A decade or two before a person with the disease shows overt symptoms, abnormal levels of a naturally occurring protein form structures called amyloid plaques. These sticky deposits build up between neurons and disrupt normal brain function, Dr. Stephen Strittmatter, a neurologist at Yale University and co-author of the new study, told The Daily Beast.

Around the same time, there is a buildup of another tau protein, which builds up inside neurons and damages them. This is followed by inflammation, which prevents the brain from being able to eliminate toxic amyloid plaques and tau proteins on its own.

“So you have three bad things going on. But what really matters for people’s symptoms is the neural network — the synapses that connect one neuron to another that are really needed by the brain to do its job,” Strittmatter said.

Many efforts to design effective treatments for Alzheimer’s disease have focused on targeting amyloid plaques. But Strittmatter thinks it may be more crucial to consider therapy that protects the brain’s precious synapses, which transmit information between neurons. Enter BMS-984923, a drug that appears to shut down the mechanism that allows tau proteins to build up inside neurons and sound the alarm for inflammation.

BMS-984923 isn’t a new drug – it’s known in the Alzheimer’s disease research community – but it hasn’t really been tested before to treat the disease. In mice genetically engineered to develop Alzheimer’s disease, Strittmatter and his colleagues found that BMS-984923 targeted tau protein accretion without affecting other receptors and also prevented inflammation. This gave the damaged synapses time to heal and the benefits lasted for up to a month after stopping treatment.

These early results are promising, but it may take a few years before the drug is prescribed to patients with Alzheimer’s disease. While BMS-984923 appeared to be safe in large rodents and monkeys, a human safety trial is underway and nearing completion, Strittmatter said. The drug might also not be useful for patients in the later stages of Alzheimer’s disease – the lab mice used by the researchers looked more like people with early to mild symptoms, so it’s hard to say without other research. For healthy people who may be at risk of developing Alzheimer’s disease, Strittmatter said BMS-984923 could one day be taken as a preventative, but the jury is still out.

“I think the synergy and the combination [therapies] may be the end result,” Strittmatter said. “We need to have a few small successes before we start combining them. I think in the longer term, if we can make progress on two fronts and put them together, we could make much more substantial progress for Alzheimer’s disease.

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