US regulators have approved the first new Alzheimer’s treatment in nearly 20 years, paving the way for its use in Britain.
Aducanumab targets the underlying cause of Alzheimer’s disease, the most common form of dementia, rather than its symptoms.
Alzheimer’s charities welcomed the news that a new treatment for the condition had been approved.
But scientists are divided about its possible effect due to uncertainty about the results of the experiment.
If the drug is approved in Britain, it could benefit at least 100,000 people with a mild form of the disease.
The FDA said there is “strong evidence that aducanumab reduces beta-amyloid plaques in the brain” and that this is “reasonably likely to be indicative of important patient benefits.”
In March 2019, the delayed international trials of aducanumab, involving about 3,000 patients, were halted when tests showed that the drug, given as a monthly solution, was no better at slowing the decline of memory and thinking problems than a placebo.
But later that year, the US manufacturer, Biogen, analyzed more data and concluded that the drug worked, as long as it was given in higher doses. The company also said it was significantly successful in slowing cognitive decline.
Aducanumab targets amyloid, a protein that forms abnormal clumps in the brains of people with Alzheimer’s disease that can damage cells and trigger dementia, including problems with memory, thinking, communicating with others, and confusion.
‘Going in the right direction’
Aldo Sirissa, who was involved in the experiment, first noticed problems differentiating between left and right 10 years ago.
After being diagnosed, the 68-year-old, originally from Glasgow and now living in Oxfordshire, near his family, had to give up his job as a surgeon.
Sirissa was given aducanumab for two years before the trial was stopped, then had to wait a relatively long time to start another trial, at the National Hospital for Neurology and Neurosurgery in London.
“I am very happy to volunteer,” Sirissa said.
“I’m really enjoying this trip that I’m taking, and the benefits that I get from it, for which I am very grateful,” he added.
Sirissa is convinced that the drug helped him.
“I feel like I’m not confused,” he said. “Although the feeling of confusion is there, it’s not that bad.”
“I am more confident now,” he added.
Sirissa said his family has noticed improvements, too.
“Before, if I was trying to get something, I couldn’t remember where to find things in the kitchen,” he said.
“It is no longer such a big problem,” he added.
“I haven’t caught up to the level I was before, but I’m going in the right direction,” he said.
It is believed that more than 30 million people worldwide have Alzheimer’s disease, most of them over the age of 65.
For the approximately 500,000 infected people in Britain, most people eligible for aducanumab will be in their 60s or 70s and in an early stage of the disease.
Professor Bart de Strooper, director of the UK’s Dementia Research Institute, said the decision to approve aducanumab represented a “major milestone” in the search for treatments for Alzheimer’s disease.
In the past decade, more than 100 potential treatments for Alzheimer’s disease have failed.
But while de Struer hopes the drug’s approval will be a watershed for the millions of people with the condition, he said there are “still many barriers to overcome”.
“We must be clear that, at best, this is a marginally useful drug that will only help very carefully selected patients,” said Professor John Hardy, professor of neuroscience at University College London.
“A huge mistake”
Professor Robert Howard, professor of geriatric psychiatry at University College London, went so far as to call approval of the drug a “fatal mistake” and could derail the ongoing search for effective treatments for dementia “for a decade”.
The Alzheimer’s Association said the drug was “promising” but added that it was “just the beginning of the path to new treatments for Alzheimer’s disease”.
Another charity, Alzheimer’s Research UK, said it had written to Health Secretary Matt Hancock asking the government to prioritize the drug’s rapid approval process in the UK.
“People with dementia and their families are waiting too long to get new, life-changing treatments,” Chief Executive Officer Hilary Evans said.
“It is now essential that regulatory authorities evaluate the evidence to determine whether they believe the drug is safe and effective for use in the UK.”
Although many doctors are skeptical about aducanumab’s benefits, US approval could be a major boost for dementia research, which has traditionally been underfunded compared to cancer or heart disease.