Georgetown researchers published a study questioning the prevalent theory of Alzheimer’s development in the journal Molecular Neurodegeneration on Oct. 31.
The research, led by investigators at the Georgetown Medical Center, explored neuron death in degenerative disorders such as Alzheimer’s disease, and found that plaque was not the cause of such neuron death. It went on to explain why people with plaque build-up in their brains sometimes do not exhibit dementia. Instead, it pointed to malfunctioning tau, a protein, as the cause of the degeneration.
Charbel Moussa, the head of laboratory for dementia and Parkinsonism in GUMC’s Department of Neuroscience, was the head researcher involved in the project, and he co-authored the study with researchers from Capital Medical University in Beijing and Merck Research Laboratories, which provided funding for the study.
“As with most research, complicated studies such as this one can take years. The process began with an idea that I was able to support with a small set of data. From there, I wrote many grants to get the funding needed to conduct the research. The project itself and the follow-up analysis took several years.” Moussa wrote in an email.
Moussa hoped that the new developments released in the study would change the current thinking about Alzheimer’s and the search for a cure.
“The key finding is that we show malfunctioning tau, not amyloid-beta (Abeta) plaque, is the seminal event that spurs neuron death in disorders such as Alzheimer’s disease. This is a debunking of the current theory of Alzheimer’s development,” he said.
According to the Centers for Disease Control and Prevention, as many as five million Americans were living with Alzheimer’s disease in 2013, but predictions estimate a threefold increase in diagnoses by 2050. The occurrence of the disease increases with age, with primary contributing factors believed to include family history. The disease remains one of the top-10 leading causes of death in the United States and the fifth-leading cause of death for adults above 65 years of age.
Crediting the Georgetown community for its contribution for the clinic, Moussa said that the study offered a culmination of his previous work in understanding Alzheimer’s. The study showed that nilotinib, a cancer treatment drug, has the potential to prevent the progression of dementias in cases where the tau is malfunctioning.
“This research builds on years of earlier work. While we inch closer to understanding the complete story of how Alzheimer’s develops, it is critical to be able to translate laboratory findings in meaningful improvements in the clinic,” he said. “At Georgetown, we are studying nilotinib in other neurologic diseases such as Parkinsonism. This clinical trial is based on our laboratory work and is supported in part, by generous Georgetown donors.”
Moussa stressed the importance of federal funding for finding a cure and the current dearth of support from Congress.
“Federal funding for biomedical research is abysmal, especially for a disease that profoundly impacts millions of people worldwide. Significant progress can be made if Congress would act now to increase funding for biomedical research,” he said.
Despite the lack of funding, Moussa saw promise in his study and similar efforts in expanding the existing understanding of the disease.
“This study suggests a completely new way to look at Alzheimer’s that might alter the way we study the disease, screen for it and ultimately treat it,” he said.