London - The first treatment to slow Alzheimer’s disease could soon be available to millions, it was dramatically announced this week.
Drugs giant Biogen shook the medical world by unexpectedly releasing results suggesting it has developed the first effective medicine for the disease.
After years of high-profile dementia trial failures, experts welcomed the "transformative discovery" as a "hugely exciting" breakthrough that could be life changing for dementia patients.
Crucially, the company said trial data for drug aducanumab is strong enough to apply for medicine licences in the US, Europe and Japan early next year. That in itself is a huge milestone.
Despite billions of pounds spent on research, no company has got to the point of submitting an application to drugs regulators before.
Biogen said results from more than 3 000 people with Alzheimer’s showed aducanumab led to a significant slowing of the disease’s progression.
CEO Michel Vounatsos said the company had already received encouragement from US regulator the Food and Drugs Administration.
"We got clear support from the FDA," he said. "With such a devastating disease that affects tens of millions worldwide, today’s announcement is truly heartening in the fight against Alzheimer’s."
If the applications are successful the medicine could be available within two years.
It would be the first real treatment for Alzheimer’s disease, which affects tens of millions more around the world. Although drugs are available that control certain symptoms for short periods, no new treatment has been approved for 15 years, and there are no drugs at all that target the underlying cause of Alzheimer’s.
Aducanumab changes that by targeting "amyloid beta" – a toxic protein which causes plaques to build up in the brain. These plaques, or clusters of amyloid protein, interfere with the function of brain cells.
The breakthrough is all the more dramatic because it came out of the blue – and aducanumab had been all but abandoned. Biogen had announced in March that it was ending two trials for aducanumab after initial results suggested it did not work. It was the latest in a string of similar disappointing results for amyloid beta drugs.
Experts had called for researchers to go back to the drawing board in the way they think about Alzheimer’s – the most common form of dementia – and some said they doubted whether amyloid beta even causes the disease.
But Biogen said it had reconsidered their decision after pooling the results of several smaller studies to give them a cohort of 3 285 patients, 2 000 of whom had taken the drug for more than 18 months. It said that taking the highest dose of the drug was most effective, along with taking the treatment as early as possible in the course of disease.
Many experts urged caution – pointing out that the company had not yet released its full results.