Researchers recently reported in the journal Science Advances that worms genetically predisposed to contract Alzheimer’s did not show symptoms if treated with the anti-cancer drug Bexarotene. The drug would be the first of what are being called “neurostatins,” or substances designed to reduce ailments in the brain much as statins do with the cardiovascular system. The team, led by researchers from the University of Cambridge, Lund University, and the University of Gronigen, have high hopes that Bexarotene could someday be used in human subjects to stave off the debilitating disease.
According to Elaine Healy, MD, medical director and vice president of Medical Affairs at United Hebrew of New Rochelle, Alzheimer’s “is one of many neurodegenerative diseases that causes a loss of cognitive functioning.” Alzheimer’s does so by causing a buildup of proteins in the brain. Healy notes that the researchers’ approach is “somewhat interesting in that it claims to target the very first step in the cause of the death of the nerve cells during Alzheimer’s.”
Healy explains that the entire class of neurostatins has only recently emerged as a neurological counterpoint to classic statins, which were originally designed to lower cholesterol but have since proven to have a number of benefits, including anti-inflammatory and antioxidant effects. “Statins are beginning to be viewed as drugs that have a wide range of salutary effects, and neurostatins are perhaps hoped to have the same multi-modal effect on the nervous system as statins do on the cardiovascular system.” However, Healy stresses that these two classes of drugs are not chemically related.
She also is quick to warn that such advances often take years to reach human trials and that many do not pan out. “Drugs like this have rarely been used,” she states. “In 2013, this same drug was used in research and was shown to be very promising in mice. They seemed to find that it diminished the buildup of senile plaques in the brains of the mice. There was quite a bit of publicity around this and then the results were not able to be replicated.”
Healy cautions that it may be many years, if ever, before neurostatin drugs like Bexarotene find widespread use. “We have seen news like this before and it is exciting, stimulating, and encouraging, but taking this treatment from worms that are genetically predisposed to have Alzheimer’s disease to humans could take decades—if it really is, in fact, a breakthrough.”
Nevertheless, the recent study is cause for hope. “The researchers are saying they know exactly how and when abnormal proteins form in the brain and that Bexarotene suppresses the very first step in the process of the formation of these abnormal proteins,” says Healy. “So, it is attractive in this respect, in that it allegedly could be used to prevent these abnormal processes.”