A team of researchers at the Perelman School of Medicine have reported a reversal of certain Alzheimer's Disease symptoms through the implementation of a new treatment. One of the key researchers, Nirinjini Naidoo, Ph.D. at Penn Medicine, elaborated on these encouraging findings.
Naidoo stated, "By generally improving neuronal and cellular health, we can mitigate or delay disease progression." She further explained, "In addition, reducing proteotoxicity—irreparable damage to the cell that is caused by an accumulation of impaired and misfolded proteins—can help improve some previously-lost brain functions."
Alzheimer’s Disease is a neurodegenerative disorder characterized by an accumulation of protein fragments in the brain. Currently, it affects over six million people in the United States. By 2060, it is estimated that this debilitating disease will afflict over thirteen million Americans. A group of Penn Medicine researchers including Naidoo observed the effects of a new Alzheimer’s Disease treatment on mice, according to a news release from Penn Medicine.
The mice used in this study are referred to as APPNL-G-F mice and exhibit common characteristics of Alzheimer’s Disease such as severe memory loss and build up of protein fragments in the brain. The researchers implemented a treatment involving 4-phenylbutyrate (PBA), a fatty-acid molecule known for blocking protein accumulation. PBA is already approved by the Food and Drug Administration as a safe treatment option for an unrelated illness. Previous tests have shown that PBA improves sleep and cognitive abilities in mice without Alzheimer’s Disease symptoms.
Jennifer Hafycz, a graduate student at Perelman School of Medicine, treated both young and middle-aged mice with PBA. Hafycz found that PBA improved proteostasis in crucial brain regions related to memory. The treatment also enhanced performance on the Spatial Object Recognition test which assesses the ability to distinguish between moving and non-moving objects. Mice with Alzheimer’s symptoms had previously lost their ability to perform well on this test, but PBA treatment restored this ability. Even when the mice were treated later in life, researchers observed positive impacts on memory retention. In fact, PBA inhibited the formation of amyloid beta plaques, a common protein accumulation in Alzheimer’s patients.
