Encouraging findings in Alzheimer’s prevention trial lead to the next phase

Families with the genetic form of early onset Alzheimer’s disease are rare, but contribute greatly to our understanding of the condition in the wider community.

About thirty families are known in Australia, where several members have had Alzheimer’s disease over multiple generations.

People with brothers, sisters and parents who have one of these rare genetic mutations have been participating in research at NeuRA for several decades.

About the DIAN Study

In 2009, NeuRA was selected as a site in the Dominantly Inherited Alzheimer Network (DIAN), an international collaborative study led by Professor John Morris and Professor Randall Bateman at Washington University in St Louis, Missouri. The Sydney site is led by Professor Peter Schofield, CEO of NeuRA.

In 2012, results from DIAN clearly showed that the onset of symptoms in Alzheimer’s disease is preceded by the accumulation of amyloid (abnormal protein) in the brain over a period of 15 to 20 years.

Prevention trial

A prevention trial was then arranged to see whether drugs could prevent or reduce brain amyloid, and, if successful, whether this would prevent or delay the onset of memory and thinking problems.

NeuRA was one of 24 international sites for the trial, in which family members at risk of developing Alzheimer’s disease were randomised to receive one of two drugs, or a placebo. As part of the trial, they received a monthly injection or infusion and had annual visits to NeuRA for several days of assessments including MRI scans, a lumbar puncture for spinal fluid analysis, and special PET scans at Royal Prince Alfred Hospital. Dr Bill Brooks is the Site Principal Investigator of the NeuRA site.

Trial results

After seven long years, the trial finished in November 2019, by which time all the participants had had at least four years of treatment. The persistence and dedication of the trial participants was exceptional and unprecedented.

The primary endpoint of the study was a slowing of cognitive decline as measured by multiple tests of thinking and memory. Although in the initial analysis neither drug met the primary outcome or demonstrated cognitive benefit, one of the two drugs did have a significant effect on the accumulation of amyloid in the brain.

Because of this encouraging finding, an Open Label Extension of the trial was announced in mid-2020.

What next

All the participants involved in the Open Label Extension have been offered active medication, with no placebo group. The Extension also allows further data to be collected on the effect of active medication over a longer period of time, and in a larger group of participants.

NeuRA participants were eager to join in this new phase, and several have begun their monthly injections.

COVID-related border closures have made visits to interstate participants difficult but it is expected participants will have begun their trial treatment by the end of March.

The Open Label Extension is expected to last at least two years.

A word from Dr Bill Brooks himself

NeuRA trial participants have just completed four years of monthly injections or infusions aimed at preventing Alzheimer’s disease.

Based on the encouraging results of the placebo-controlled trial, all our participants have put their hands up for a further two years during which everyone will receive active medication.

For families with the genetic form of Alzheimer’s disease, these dedicated family members at last offer hope for the future and we salute them!”

15 March 2021