Researchers have recently initiated a groundbreaking clinical trial aimed at treating Alzheimer’s disease in individuals with Down syndrome. This trial focuses on reducing the levels of amyloid precursor protein (APP), a key factor in the development of Alzheimer’s, in adults with Down syndrome who are genetically predisposed to the disease.
Individuals with Down syndrome are born with an extra chromosome that contains a gene responsible for triggering Alzheimer’s. This gene produces a protein that leads to the accumulation of plaques in the brain. By the age of 40, most people with Down syndrome already have these plaques, and it may take another decade or more for symptoms of Alzheimer’s to manifest. Eventually, up to 90% of individuals with Down syndrome are expected to develop Alzheimer’s disease.
In an effort to change these statistics, researchers have administered the first dose of an investigational drug as part of the HERO study. This drug is designed to prevent the formation of Alzheimer’s-causing plaques in the brain. Led by Ionis Pharmaceuticals and overseen by ATRI Medical Director Michael Rafii, MD, Ph.D., the study is set to run for two years with an initial group of 30 participants at various locations in the U.S. and Europe.
The HERO study is a significant milestone as it targets the underlying genetic cause of Alzheimer’s disease in individuals with Down syndrome. This trial represents a shift towards preventive measures rather than treatment at later stages of the disease. Additionally, it signifies a growing trend in conducting clinical trials specifically tailored for individuals with Down syndrome.
In addition to the HERO study, other trials such as the ABATE study, which focuses on a vaccine to slow down Alzheimer’s in individuals with Down syndrome, and the upcoming ALADDIN trial, which will examine the use of donanemab in this population, demonstrate the progress being made in Down syndrome research. These trials aim to provide individuals with Down syndrome with access to multiple research opportunities, a significant advancement in the field.
The HERO study is closely aligned with the Alzheimer’s Clinical Trials Consortium—Down Syndrome (ACTC-DS) and the Alzheimer’s Biomarker Consortium—Down Syndrome (ABC-DS), both of which play vital roles in advancing Alzheimer’s therapies for individuals with Down syndrome. By collaborating with advocacy groups and engaging with individuals with Down syndrome and their families, these consortia ensure that the research prioritizes the needs and voices of the community.
The investigational drug being evaluated in the HERO study, ION269, is designed to disrupt the production of amyloid plaques in the brain. Administered via injection into the lower back, the drug aims to reach the brain and inhibit plaque formation. The study will assess the safety, tolerability, and efficacy of this treatment on APP and amyloid plaques in the brain.
As the HERO study progresses, researchers are actively recruiting participants to join the trial. With sites such as Washington University School of Medicine and the University of Kansas Medical Center already enrolling participants, more research locations are expected to join in the coming months.
The HERO study, along with other ongoing trials and research initiatives, highlights the significant strides being made in understanding and treating Alzheimer’s disease in individuals with Down syndrome. By targeting the genetic roots of the disease and involving the community in research efforts, these studies offer hope for improved outcomes and a brighter future for individuals with Down syndrome at risk of Alzheimer’s disease.
