Drug for rare form of Alzheimer’s “scientifically promising”

For the first time in a clinical trial, findings indicate that early removal of amyloid plaques in the brain years before symptoms are observed, may delay onset of Alzheimer’s dementia. Researchers investigated the effects of the experimental anti-amyloid drug gantenerumab to determine whether it could prevent participants developing dementia. The data offers new evidence supporting the amyloid hypothesis of Alzheimer’s.

The international study evaluated 73 participants with rare, inherited genetic mutations that cause the overproduction of amyloid in the brain. These individuals had originally enrolled in the world’s first Alzheimer’s prevention trial in 2012 (the Knight Family DIAN-TU-001). They were further included in an extension of the trial investigating an anti-amyloid drug, the researchers noted.

“Everyone in this study was destined to develop Alzheimer’s disease and some of them haven’t yet,” stated senior author Dr Randall Bateman, the Charles F. and Joanne Knight Distinguished Professor of Neurology at WashU Medicine.

“We don’t yet know how long they will remain symptom-free – maybe a few years or maybe decades. In order to give them the best opportunity to stay cognitively normal, we have continued treatment with another anti-amyloid antibody in hopes they will never develop symptoms at all. What we do know is that it’s possible at least to delay the onset of the symptoms of Alzheimer’s disease and give people more years of healthy life.”

Gantenerumab is discontinued and no longer in development, so most participants in the extension study have begun to receive US FDA-approved anti-amyloid drug lecanemab. Analysis from this phase of the extension trial is yet to be conducted, according to the researchers.

“While this study does not conclusively prove that Alzheimer’s disease onset can be delayed and uses a drug [gantenerumab] that will not likely be available, the results are scientifically promising,” explained Professor Tara Spires-Jones, Director of the Centre for Discovery Brain Sciences at the University of Edinburgh, Group Leader in the UK Dementia Research Institute, and President of the British Neuroscience Association.

“Whether these findings apply to current approved anti-amyloid treatments needs further work, but these results highlight the importance of early intervention and longer-term follow-up in determining the benefits and risks of dementia treatments,” Dr Susan Kohlhaas, Executive Director of Research, Alzheimer’s Research UK stated.

She continued, emphasising the importance of further research: “We’re in a period of uncertainty for global investment into research. Now, more than ever, we must collaborate to collect valuable long-term data.”

Details of the study are published in The Lancet Neurology.