Alzheimer's Disease: Drugs Targeting Dementia-Causing Toxic Particles Could Be Trialed in Two Years

Scientists have developed an "innovative" method to attack the toxic particles that lead to brain disorders such as Alzheimer's disease.

The team hope drugs developed as a result of their research could be in clinical trials in just two or three years from now, one researcher told Newsweek. The team reported their findings in the journal Proceedings of the National Academy of Sciences of the United States of America.

Alzheimer's disease is a form of dementia that sees nerve cell death and tissue loss across the brain. As it shrinks, patients experience symptoms including personality change, memory failure and difficulties performing everyday tasks.

The disease affects some 5.7 million Americans, the Centers for Disease Control and Prevention (CDC) reports. Associated with aging, it's the fifth-leading cause of death in people aged 65 and above. In 2016 there were 49 million people aged 65 or older in the U.S., and the CDC expects that number to double by 2060.

At the molecular level, Alzheimer's is linked to the buildup of dangerous protein clumps in the brain, study author and University of Cambridge researcher Michele Vendruscolo told Newsweek. Small and soluble "toxic particles" form during this process and kill neurons, he explained.

Vendruscolo and his team used an "innovative drug discovery approach" to develop small molecules that can slow down the formation of these toxic particles, he said. His team's method was "the first" to directly target those particles and "dramatically" reduce their numbers.

So far, researchers had tested the molecules in laboratory assays and in roundworms. "We are now in the process of testing them in a mouse model," Vendruscolo said, adding that his team may see the first results of clinical trials just two or three years from now.

Conceptual image of neurons in the brain. Andrii Vodolazhskyi

Although hundreds of clinical trials have focused on Alzheimer's disease, none have yet taken aim at the toxic particles thought to cause the condition. "Although this is currently the most widely accepted hypothesis, it has not been proven yet," Vendruscolo added. "Our small molecules are providing a powerful route to prove or disprove this hypothesis."

The "novel" research could provide a "streamlined pathway" for the discovery of drugs that prevent the early-stage protein accumulation seen in Alzheimer's, Cleveland Clinic neurologist Jagan Pillai, who was not involved in the research, told Newsweek. "It remains to be seen if promising compounds could also be evaluated for other [related] disorders including Parkinson's and Huntington's disease."

Nikhil Palekar, medical director of the Center of Excellence for Alzheimer's Disease and associate professor of psychiatry at Stony Brook Medicine, called the findings "significant." Current treatments "are minimally effective," he told Newsweek, and "recent attempts at drug development have failed to show improvement in symptoms or slow the progression of the disease."

Read more: 'Zombie' brain cells could hold key to combating Alzheimer's, mouse study suggests

Although Alzheimer's therapies have seen a recent uptick in excitement and interest, Pillai added, targeted study is urgently needed to develop a sustainable cure.

The new research is promising in theory, Palekar said, but it "needs to translate into successful drug development." The complex nature of diseases like Alzheimer's could make this tricky, he added. "Alzheimer's disease is a hugely important public health issue, with millions afflicted worldwide. The need for more research towards novel therapeutic targets cannot be overstated."

"We hope that our research will help shift the public opinion from considering Alzheimer's disease as a death sentence to dealing with it as a curable or preventable condition," Vendruscolo said.