Researchers from the Zilkha Neurogenetic Institute at the Keck School of Medicine published a groundbreaking paper that highlights the underlying causes of Alzheimer’s disease and reveals a potential treatment for it and other similar brain conditions.
The research, a five-year-long study with more than 150 patients in clinical trials, has successfully identified the blood brain barrier as the root of the disease, which affects an estimated 5.7 million Americans.
Institute Director Berislav Zlokovic, the primary investigator and author of the study, partnered with the Mark and Mary Stevens Neuroimaging and Informatics Institute to conduct the research.
“Our recent paper is about the blood brain barrier breakdown,” Zlokovic said. “If there are leaky blood vessels in the brain … then we should work to fix it … and acting on the vascular system may have important effects on delaying or even stopping cognitive decay.”
According to Zlokovic and his researchers, as the brain and its vessels deteriorate, toxic proteins and other harmful molecules make their way into active brain tissue, damaging areas of the brain that are essential for memory.
Researchers from the institute conducted the study in collaboration with neurologists from the Washington University School of Medicine in St. Louis and the Huntington Medical Research Institutes in Pasadena. Each research organization was responsible for bringing in the trial’s clinical patients and collecting the resources and data the USC team used to inform its findings.
“We collect blood, spinal fluid and urine to try and look for … what people like to call biomarkers of Alzheimer’s disease,” said Michael Harrington, a director of neuroscience and research at HMRI. “Our focus is to look at these groups [of patients], whether they’re cognitively healthy, impaired or demented and … look for biomarkers that will detect the disorder and also reveal the mechanism behind it.”
The findings, which were published in the Jan. 14 issue of Nature Medicine, can potentially help with early Alzheimer’s diagnoses and new drug development. Since the study’s publication, the neurologists are planning to expand their research to include more patients.
“Under our program right now, we have close to 700 patients,” Zlokovic said. “The objective of the study is to know the predictive value of vascular changes in the blood brain barrier in predicting cognitive decline in people who have Alzheimer’s disease. After that … we will expand this to other genetic diseases like small vessel disease.”
Alzheimer’s is only the beginning, as researchers plan to apply their work to other brain diseases.
According to Axel Montagne, an assistant professor of research physiology and neuroscience at Keck, successful diagnoses of Alzheimer’s will lead to advances in medical technology to treat Parkinson’s disease, Huntington’s disease and, most notably, amyotrophic lateral sclerosis.
“We know for sure that the blood brain barrier is contributing to the evolution of each of those diseases in different ways.” Montagne said. “If we find a cure, if we find a way to seal the blood barrier, we may be able to help cure other diseases.”