An Alzheimer’s drug that could halt the progression of the disease has been hailed a “game changer”.
The memory-robbing disorder has no cure, with existing treatments working to ease its symptoms.
In severe cases, patients may become aggressive, struggle to move or even hallucinate.
Hopes have been raised after the US Food and Drug Administration (FDA) granted the medication aducanumab fast-track approval.
Trials have demonstrated aducanumab helps to untangle Alzheimer’s tell-tale protein clumps in the brain, potentially reversing the disease.
The FDA has not considered a new treatment for the condition in 17 years, with aducanumab’s fate being decided by 7 March 2021.
If licensed, it would be the first therapy approved to delay Alzheimer’s progression.
‘A new era for Alzheimer’s treatments’
“The FDA’s acceptance of the aducanumab with priority review is an important step in the path to potentially having a treatment that meaningfully changes the course of Alzheimer’s disease,” said Michel Vounatsos, CEO of the manufacturer Biogen, as reported by Global News Wire.
“We believe aducanumab marks the beginning of a new era of potential treatments for Alzheimer’s disease that will inspire even more discovery and innovation to bring hope to those affected by this devastating disease.”
More than 5 million people in the US have Alzheimer’s, which is expected to increase to over 13 million by 2050.
In the UK, around 850,000 people have dementia – the umbrella term for conditions that cause a gradual decline in brain function.
Alzheimer’s is the most common type of dementia, affecting between 50% and 75% of patients in the UK.
The disease is thought to be caused by the abnormal build-up of proteins in and around brain cells.
Two of these are amyloid – which forms plaques, and tau – which develops into tangles.
Over time, the chemical messengers that send signals between brain cells decline.
Different areas of the vital organ then shrink, usually starting with those responsible for memories.
Trials have shown aducanumab works to untangle these clumps, slowing memory loss.
The drug was also linked to improvement in a patient’s ability to carry out day-to-day tasks, like household chores and leaving the home alone.
“I am heartened by what this progress may mean for people living with Alzheimer’s disease and their families,” said Dr Christopher van Dyck, from the Yale Alzheimer’s Disease Research Center.
“If aducanumab is determined to be effective in reducing the decline in cognition and activities of daily living resulting from progression of this disease by addressing the underlying disease pathology, it will deliver meaningful benefits to those who most need them.”
Keeps patients ‘in a better place for a longer time’
Aducanumab has had a bumpy road. Studies were scrapped in March 2019 after two late-stage trials suggested it was of no benefit.
Several months later, Biogen looked at a larger amount of data that became available after the trials stopped.
It found the drugs were effective providing they were given early enough and at a sufficient dose.
Experts hope aducanumab will benefit those with early signs of dementia, helping to halt or slow the disease before patients became incapacitated.
The FDA has been set an “action date” of 7 March 2021, but “plans to act early on this application under an expedited review”.
Michael Yee, an analyst at the investment bank Jefferies, expects a verdict in the first quarter of 2021, adding the data demonstrating the drug’s effectiveness is “mixed and still inconclusive”, Fierce Biotech reported.
Nevertheless, the fast-track approval suggests the FDA is “comfortable with the totality of the data, recognises high unmet need and really wants to get an Alzheimer’s drug approved”, he added.
Analyst Brian Abrahams, from RBC Capital Markets, has given aducanumab a 30% chance of approval.
Dr Dennis Selkoe from Harvard told wbur: “I think there’s a reasonable chance that aducanumab, if approved and we don’t know that yet, will be the first disease-modifying agent for Alzheimer’s, and that will be a huge milestone, and patients of mine and others can access the drug.
“Having cared for hundreds of patients with this disease, this would be a game changer.
“At least for some of my patients, if aducanumab is approved, [it would be] something that will slow down their deterioration, that will keep them in a better place for a longer time.”
The FDA only accepts drugs for priority review if they offer major advances in treatments or provide a therapy where none existed.
If approved in the US, experts are confident other health watchdogs around the world will consider doing the same.
In the meantime, Biogen is planning further studies, which will offer high doses to all eligible participants from the earlier trials.
Speaking of the fast-track review, Fiona Carragher from the Alzheimer’s Society said: “This marks another crucial step forward in the development of the first ever treatment to slow down the progression of Alzheimer’s disease.
“While news that the application to the US regulatory authorities has been fast-tracked should certainly give people affected by dementia a boost, this is a world first for dementia research, the journey is not yet complete.
“Currently treatment options are limited so people may ask ‘what is there to be lost?’, but it’s critical we fully understand the impact and safety of this drug which will now be down to the US Food and Drug Administration to determine.”