Investigational therapies for familial Alzheimer’s disease
Last Reviewed: Feb 2, 2026Navigating Alzheimer's is a challenge for any family, and it can be particularly challenging when adults in the prime of their life are taken town by Alzheimer’s symptoms. When they learn they’re a part of this unique community, the question most families have is, “What can we do to treat it?”
Members of the familial Alzheimer’s disease (FAD) community have played an important role in understanding the disease and testing investigational therapies.
“I now honestly believe that we are within 10 years of finding a cure.””
In spring of 2025, a member of the Youngtimers community who has been involved with research for 27 years said: “I now honestly believe that we are within 10 years of finding a cure. There are a multitude of different avenues of research occurring simultaneously, ranging from modifying DNA to eliminating the proteins we believe cause cognitive decline.”
Although the approval of Leqembi in 2023 and Kisunla in 2024 were exciting, the work is not done. As our understanding of the disease develops, Alzheimer’s researchers continue to identify new therapeutic targets for Alzheimer’s disease, giving tremendous hope to FAD families.
Targeting Tau
Neurofibrillary tangles of tau protein are an important component potentially responsible for the symptoms that present in Alzheimer's disease. The abnormal accumulation of tau protein shows a strong relationship with symptom severity, and researchers suggest that once cognitive decline begins, focusing on tau could be more effective than focusing on amyloid.
Researchers are exploring different strategies to control the abnormal accumulation of tau, including the use of treatments with antisense oligonucleotides (ASOs) ⧉ or anti-tau antibodies (similar to amyloid-targeting antibodies). One example in this category of monoclonal antibodies for tau regulation is E2814 ⧉ .
Gene editing technology
The ability to target the underlying cause of a disease (i.e. a mutation) and make a lasting correction makes gene editing technology an attractive approach for rare genetic disorders like FAD.
The recent discovery that antisense oligonucleotides (ASOs) treatment can effectively target and treat spinal muscular atrophy¹, signals the potential to use ASOs as treatments for other neurological disorders. Innovations like CRISPR ⧉ and siRNA ⧉ could offer groundbreaking new ways to modify or even prevent the disease at the genetic level.
Early intervention
Many Alzheimer’s drug trials have shown no observable benefit, and some believe this is due to the fact that they were tested on people with advanced stages of Alzheimer's disease. When too much damage has accumulated in the brain, it may be too late for treatments to make a difference.
Researchers believe that the earlier people can be treated, the better the outcomes and clinical benefits will be.
DIAN-TU’s primary prevention trial aims to learn whether treatment can slow the rate of progression of cognitive/clinical impairment or improve disease-related biomarkers in people at-risk of FAD.
Learn more about the DIAN primary prevention trial. ⧉
The forefront of Alzheimer’s research
Many of today’s treatment options are available because FAD families have chosen to participate in Alzheimer’s research.
Families with FAD genetics face unique struggles, but they also hold a unique opportunity: by participating in Alzheimer’s research, they can help scientists understand the disease better and uncover therapies to slow and stop it.
Sources
O'Brien RJ, Thomas K, Martin B, et al. The AHEAD study: a randomized controlled trial of an anti-amyloid drug in preclinical Alzheimer's disease. Alzheimer's Dement (N Y). 2021;7(1):e12122. doi:10.1002/trc2.12122. Link ⧉