Glimmers of hope radiate from the Medical Research Council in Leicester, following scientists’ recent success in fully stopping brain cell death in lab mice. Scientists used a chemical compound that could one day lead to patient-friendly drugs capable of curing Alzheimer’s disease.
The chemical has a long road ahead before it can penetrate the market as a standalone drug. But scientists have set high expectations for it already, as the initial tests have shown immense success in preventing brain tissue death in neurodegenerative diseases thus far, with many experts already seeing the findings as a watershed moment in the field of neuroscience.
"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,” Prof. Roger Morris, from King's College London, told the BBC.
While Morris cautioned that the breakthrough won’t result in a cure anytime soon, he agreed that the study marked a new frontier for scientists to explore on their way to curing a range of devastating diseases that have afflicted millions and affected even more.
"The world won't change tomorrow,” he said, “but this is a landmark study."
Neurodegenerative diseases are marked by dying cells in the brain and spinal cord, and due to the mass extermination of neurons, bodily functions such as muscle use and movement control, and cognitive functions such as speech and memory, often become impaired, if not immobilized. The most common diseases are Alzheimer’s, Parkinson’s disease, multiple sclerosis, and Huntington’s disease.
The breakthrough research came from the Medical Research Council Toxicology Unit, based at the University of Leicester. A team of scientists analyzed the way viruses attack a group of brain cells and force the production of viral defense proteins, which sometimes get produced as faulty “misfolded” proteins. These misfolded proteins do more damage than the regular defense mechanisms, lingering in the brain cells longer and sapping neurons of their ability to function. The cell essentially starves, and, when replicated across many, many neurons, brain functions deteriorate in the process.
In the end, mice that didn’t receive the compound saw continued cellular breakdown and often died within 12 weeks. Meanwhile, those that had received it saw neurodegeneration come to a halt and survived with healthy brain tissue for the duration of the study.
"They were absolutely fine, it was extraordinary,” said lead researcher, Prof. Giovanna Mallucci, to the BBC. "This isn't the compound you would use in people, but it means we can do it and it's a start."
The trial was not without wrinkles. Many of the mice developed a mild form of diabetes as a result of their pancreas failing. Admittedly, drugs that target issues in the brain must specifically work in that region.
Alzheimer’s is the most common form of dementia in adults, robbing 5.4 million Americans — 5.2 million of whom are over the age of 65 — of precious mental faculties, including speech, memory, reasoning skills, and overall cognition.
“The hope for something like this is that we are able to arrest the process of cell death, of brain cell death,” Mallucci said, “and that’s what is so exciting.”
The next step for Mallucci and her colleagues is bridging the gap between clinical trial and full drug development — to do further testing and tinkering in order to minimize side effects and maximize localized efficacy.
"Targeting a mechanism relevant to a number of neurodegenerative diseases could yield a single drug with wide-reaching benefits, but this compound is still at an early stage,” said Dr. Eric Karran, the director of research at the charity Alzheimer's Research UK. “It will be important for these findings to be repeated and tested in models of other neurodegenerative diseases, including Alzheimer's disease."
Source: Moreno J, Halliday M, Molloy C. Oral Treatment Targeting the Unfolded Protein Response Prevents Neurodegeneration and Clinical Disease in Prion-Infected Mice. Science Translational Medicine. 2013.