ATRI Researchers have dosed the first participant in a clinical trial of an investigational medicine designed to lower the amount of amyloid precursor protein (APP) for the potential treatment of Alzheimer’s disease (AD), which is initially being studied in adults with Down syndrome (DS) who have a genetic risk of developing AD. Those with Down syndrome are born with an extra chromosome that carries a gene pivotal to causing Alzheimer’s. The gene produces a protein that causes a buildup of plaques in the brain. By the time most people with Down syndrome hit age 40, they have already developed these plaques. It may take another decade or more to develop symptoms, but eventually up to nine in 10 people with Down syndrome are expected to develop Alzheimer’s disease. Seeking to change these odds, last month, researchers gave the first dose of an investigative drug to a participant in the new clinical trial, called the HERO study. The drug is designed to stop Alzheimer’s-causing plaques from forming in the brain. Led by Ionis Pharmaceuticals, with investigative oversight by ATRI Medical Director Michael Rafii, MD, PhD, the study is expected to run for two years with an initial 30 participants at sites across the U.S. and Europe.

Researchers from Boston University Chobanian & Avedisian School of Medicine have found a new role for the transcription factor (proteins that regulate the transcription, or copying, of genes). In the fruit fly, this transcription factor, named Traffic Jam, activates a non-coding piRNA gene named Flamenco to promote female fruit fly (drosophila) fertility. The discovery solves the 30-year-old mystery of how Flamenco gets activated to protect fruit fly ovaries from a series of genetic parasites called retroviral transposons, and may one day help with infertility issues in humans.

A McGill University-led study found that people with cannabis use disorder (CUD) had elevated dopamine levels in a brain region associated with psychosis.

“This could help explain why cannabis use increases the risk of hallucinations and delusions, key symptoms of schizophrenia and other psychotic disorders,” said first author Jessica Ahrens, a PhD student in McGill’s Integrated Program in Neuroscience.