A complete cure for Alzheimer’s may be on the horizon, as researchers have revealed a drug that can turn off the death of brain neurons in mice. Calling the finding a “historic turning point,” scientists hail the new study as extremely “significant” in Alzheimer’s research. Numerous brain diseases that cause degeneration have the same underlying cause: proteins that are folded the wrong way. When too many of these rogue proteins accumulate, brain cells can begin to die.
Previously, scientists have attempted to stop the proteins from accumulating in the first place, but in this study, the researchers went after the death of the neurons directly. By giving mice a drug they developed, they were able to “switch off” the death of the brain cells completely. The medicine was delivered to the mice through their stomachs.
This new potential Alzheimer’s cure is particularly exciting because it was a replication of an earlier study. Science is built through replication of study results, and the reproduction of such a major outcome has set the medical community abuzz with excitement. King’s College professor Roger Morris said he was thrilled about the study outcome. “This finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer’s disease,” he said, “I’m very excited, it’s the first proof in any living animal that you can delay neurodegeneration. The world won’t change tomorrow, but this is a landmark study.”
The study was performed at the University of Leicester and was manned by staff from the Medical Research Council’s (MRC) Toxicology Unit. It has been published in the journal Science Translational Medicine. The new drug has the potential to treat a host of diseases that cause brain degeneration, including Huntington’s, Parkinson’s, Alzheimer’s and other similarly-acting illnesses. In many brain diseases such as these, the misfolded proteins cause the brain to cease making healthy proteins. That’s because the bad proteins don’t disappear and end up acting like a virus. Viruses can cause the brain to simply close down its protein factory in an attempt to stop the replication of the virus. In cases of Alzheimer’s and other degenerative brain diseases, the brain treats the misfolded proteins like viral cells and acts the same way in defense against them. However, when the mice were given the new drug, the degeneration stopped entirely. And what’s more; the mice remained healthy at a five-week follow up.
Giovanna Mallucci, the head researcher on the study, said, “They were absolutely fine, it was extraordinary. What’s really exciting is a compound has completely prevented neurodegeneration and that’s a first. This isn’t the compound you would use in people, but it means we can do it and it’s a start. (It’s a) new pathway that may well give protective drugs.” She seems to indicate that the importance of this breakthrough can’t be overstated:
It’s the first time a substance has been given to mice that prevents brain disease. The fact that this is a compound that can be given orally, that gets into the brain and prevents brain disease, is a first in itself… We can go forward and develop better molecules and I can’t see why preventing this process should only be restricted to mice. I think this probably will translate into other mammalian brains.
Scientists say that the drug is in an early form and it will need a lot more research and funding before it can be developed into a drug to treat humans. They say that stage could be about ten years away, or more. One of their primary concerns is the risk of side effects. The drug did cause the mice to get a form of diabetes, which in turn, caused weight loss and elevated levels of blood sugar. Researchers say any drug approved for human use would need to be targeted only to the misfolded proteins and not carry the risk of diabetes.
A complete cure for Alzheimer’s may be on the horizon, and scientists are excited by the possibilities. This new study shows mice can be cured of the disease, and researchers hope that a human cure could soon follow. It may take a decade or more of research, but there’s no doubt scientists will be hard at work to expedite the process.
By: Rebecca Savastio