The first treatment to slow Alzheimer’s disease could soon be available to millions, it was dramatically announced last night.
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 last night 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.
On Tuesday, Biogen Inc (pictured) says it plans to file approval for a drug to treat early Alzheimer’s disease, called aducanumab, with the FDA in early 2020
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.’
Biogen is said to have had a breakthrough after years of high-profile dementia trial failures (stock image of patient above)
If the applications are successful the medicine could be available within two years – a prospect that saw Biogen shares soar 35 per cent yesterday, adding $14.9billion (£11.6billion) to the company’s market value.
It would be the first real treatment for Alzheimer’s disease, which affects 500,000 in Britain and 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 last night urged caution – pointing out that the company had not yet released its full results.
But Dr James Pickett, of the Alzheimer’s Society, said: ‘After the trial being stopped earlier this year because it appeared not to work, further analysis suggests that it does benefit people with dementia in the earliest stages.
‘We’re waiting for further data but this could be the first new treatment for Alzheimer’s disease in over 15 years, and as such, has the potential to be a transformative discovery.’ Hilary Evans, at Alzheimer’s Research UK, added: ‘People affected by Alzheimer’s have waited a long time for a life-changing new treatment and this exciting announcement offers new hope that one could be in sight.’
Professor Bart De Strooper of the UK Dementia Research Institute, added: ‘I hope this signifies a turning point.’
But others were more cautious. Professor Rob Howard of University College London said: ‘We have been down this road before. We are only being allowed to see a cherry-picked selection of data and I suspect once we see the full results we will see that the clinical effect is very small indeed.’
Samuel Gandy of the Mount Sinai Alzheimer’s Disease Research Centre in New York, added: ‘I want to believe, but I’m not ready to suspend disbelief.’