In decades of research, scientists have focused on eliminating the signature plaques of Alzheimer’s to fight the devastating disease. Now, a drug in phase-II trials is taking a new approach, focusing on strengthening cells’ protection against neurological attacks, which may be a game changer, Time reported.
Dr. Frank Longo of Stanford University School of Medicine and his team are studying the treatment LM11A-31, or C31, as his team calls it, in combination with anti-amyloid and anti-tau therapies, which Time reported could be a potent counterpunch to neurological problems from memory loss to confusion to loss of language.
“The field is taking a step back and re-examining where we are with regard to what we know, what we don’t know and what might be some of the best avenues going forward to look for treatments,” Dr. Ronald Petersen, director of the Mayo Clinic Alzheimer’s Disease Research Center and the Mayo Clinic Study of Aging, who is not involved in the LM11A-31 research, told Time.
In the past, scientists focused on treating the degradation of the brain caused by Alzheimer’s—normally orderly and uniform cells are cut off from their nutrient supply by amyloid plaques that accumulate in an afflicted-brain. Treatments tested tried to find ways to absorb excess amyloid or break it down and while these treatments worked in animals, they were less successful in memory and cognitive function improvement in people, TIME reported.
Researchers then considered the timing of drug administration, but that required knowing when the amyloid first develops— about 30 percent of people over 70 have amyloid in their brains but no signs of dementia, TIME reported. Therefore, not everyone who has amyloid has Alzheimer’s.
According to TIME, similar treatments against tau, another Alzheimer’s protein, which tends to appear in later stages when memory, organized thinking and language begin to fail.
“We think that tau may incite the whole process of neurodegeneration,” Dr. William Jagust, a professor of public health and neuroscience at the University of California, Berkeley, told TIME. “That’s important if you think of Alzheimer’s as moving through standardized group stages. The first stage is [depositing] of amyloid. In the second stage, something probably happens with tau. Somewhere in there we begin to see neurodegeneration.”
As Time reported, C31 presents itself as a drug that can intervene at any or all of the stages. Longo has found that C31 can disrupt at least 10 of the 14 amyloid-triggered brain signals that can ultimately lead to neuron deterioration.
According to Petersen, theoretically a patient at high risk of developing Alzheimer’s could be injected with nerve growth factors that prevent or lessen nerve cell damage.
C31, which passed phase I clinical trials for safety and minimal side effects, is currently in phase II, human testing.
C31 is being tested on 72 healthy people who don’t have any signs of Alzheimer’s and is promising, but now researchers are watching to see if it affects memory and thinking, according to TIME.
If successful, the treatment would be groundbreaking, but some have reservations, TIME reported.
“If approved, these could be the first drugs that will change the course of the disease” rather than just treat its symptoms, James Hendrix, director of global science initiatives at the Alzheimer’s Association, told TIME. “To bring back neurons that have been destroyed by plaques and tangles–to me that still seems almost like science fiction,” he continued. “I have a hard time getting to that point.”
According to TIME, more than 200 Alzheimer’s drugs have been tested since 2000 and none has proven to be the miracle cure. Longo’s work with C31 is the first to focus on increasing levels of nerve-growth factors to potentially give people protection against damage and minimize effects.
The open question is figuring out which people can benefit from which types of treatments, TIME reported. Should older people be screened for signs? When should monitoring begin? Perhaps a risk score, as with heart disease, is a solution.
What is clear, is that Alzheimer’s begins decades before symptoms start and the best treatments occur early, TIME reported, suggesting that C31 may become the first drug in an Alzheimer’s “cocktail.”
“In an ideal world, you want to take a 78-year-old and say, I think in your brain amyloid is contributing to 20 percent of your cognitive problems, so I’ll give you an anti-amyloid therapy. You also have tau proteins contributing to about 35 percent of your problems, and so on,” Petersen told TIME. “You’d want to design a therapeutic regimen based on the different components and their contributions to that patient’s disease.”