New drug prevents hallmark of Alzheimer’s disease in animals
Scientists have identified a fresh drug that is effective at inhibiting the development of plaques, an integral sign of Alzheimer’s disease, in the brains of animals.
The study, which appears in the Journal of Experimental Medicine, lays the groundwork for further studies. If these corroborate the findings, they could pave just how for clinical trials in humans.
Alzheimer’s facts
Alzheimer’s is a developmental neurodegenerative disease that typically occurs in persons aged 60 or older.
In 2014, about 5 million people in the U.S. had Alzheimer’s disease. By 2060, this figure increase to around 14 million, the Centers for Disease Control and Prevention (CDC) estimate. Alzheimer’s happens to be one of the top 10 leading factors behind death in the U.S.
As the National Institute on Aging note, the condition typically starts with milder symptoms, such as for example memory loss and confusion. A person with extreme Alzheimer’s may not recognize close family or friends and be unable to communicate.
Scientists are not yet sure why some individuals, rather than others, develop Alzheimer’s disease. Experts assume that it results from a characteristic buildup of protein plaques in the mind. These plaques then affect the functioning of brain cells, which in turn causes slow cognitive decline.
The plaques can appear a decade before the person experience symptoms.
Currently, there is absolutely no cure for Alzheimer’s disease. Treatments address symptoms without tackling the primary cause.
Modulation or inhibition?
The team behind the recent study discovered and tested a new drug that are effective and safe at reducing the buildup of plaques in animal models of the disease.
Prof. Steven Wagner, of the Department of Neurosciences at the University of California NORTH PARK School of Medicine, notes, “Alzheimer’s disease can be an extraordinarily complex and multifaceted condition that has, up to now, defied effective treatment, aside from prevention.”
“Our findings suggest a potential therapy that might prevent among the key components of Alzheimer’s disease,” Prof. Wagner, also the study’s senior author, explains.
The therapy functions by modulating an enzyme called gamma-secretase, which is paramount to the production of plaques in the mind of a person with Alzheimer’s.
In previous research, scientists inhibited gamma-secretase. However, this yielded significant adverse effects, like the “worsening of cognitive abilities relative to placebo,” according to Prof. Wagner and his co-authors.
In the recent study, the team tried a slightly different approach. They used a gamma-secretase modulator (GSM), a drug that adjusts rather than inhibits the action of the enzyme.
This allowed gamma-secretase to keep fulfilling its other important roles but reduced its contribution to the buildup of plaques.
Effective and safe in animals
After testing the GSM in mice, rats, and macaques, the researchers discovered that it significantly reduced the buildup of peptides that induce plaques. Also, the drug produced no toxic unwanted effects in any animals.
Administering the GSM before or after the animals began to build up plaques reduced overall plaque formation. In addition, it reduced inflammation connected with plaque buildup - which is key because scientists assume that this inflammation plays a role in the disease’s progression.
The researchers hope that, eventually, the GSM might become a therapy that may inhibit the development of Alzheimer’s disease in humans.
According to review co-author Prof. Rudolph Tanzi, director of the Genetics and Aging Research Unit at Massachusetts General Hospital, in Boston, “In this study, we've pharmacologically characterized a potent GSM that, based on its preclinical attributes, seems to equal or exceed the potency of any previously tested GSMs.” He continues:
“Future clinical trials will determine whether this promising GSM is safe in humans and may be utilized to effectively treat or prevent Alzheimer’s disease.”
Source: www.medicalnewstoday.com
