First Drug To Slow Down Alzheimer’s Disease

Biogen’s chief executive Michael Vounatsos says that he hopes their drug offers patients “the first therapy to reduce the clinical decline of Alzheimer’s disease”. Biogen has plans to file for approval with the FDA early in 2020, this discovery has the potential to be the number of drug discovery of all time. 

The drug failed in trials at first, the newer and larger analysis involved over 3,000 patients and showed that high doses reduced symptoms if caught early; in one measure of a range of mental abilities those taking aducanumab experienced 25% less decline after 18 months. Those who took the drug had reduced levels of toxic amyloid beta proteins in their brains, better cognition, memory, and language as well they were able to live independently for longer including being able to perform household chores and travelling on their own, according to Biogen.

Lead investigator, and director of the University of Rochester’s Alzheimer’s programme in New York state Anton Porsteinsson said, “There is tremendous unmet need and the Alzheimer’s disease community has been waiting for this moment.” 

Hilary Evans, chief executive at Alzheimer’s Research UK, said: “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.Taking another look at aducanumab is a positive step for all those who took part in the clinical trials and the worldwide dementia research community. As more data emerges, we hope it will spark global discussions about the next steps for delivering much-needed treatments into people’s hands.”

James Pickett, head of research at the Alzheimer’s Society, said that while they were looking forward to seeing more data, the drug had “the potential to be a transformative discovery”.

This news comes decades after many companies and those affected by this brain wasting disease have watched many other promising treatments fail in final stage trails, leading to some companies pulling out of working in the field completely. Lack of success was not just frustrating but it also implied the entire approach involving targeting amyloid beta could be wrong. 

This drug trial could re-energize work in the field, Bart de Strooper, director of the UK Dementia Research Institute at UCL said, “It is fantastic to hear of these new positive results emerging from the aducanumab trials. We have no effective treatments to slow or halt the progression of Alzheimer’s disease and I hope this signifies a turning point.”

Efforts are moving to treating people before they become symptomatic in hope that previous failures may be due to not catching the amyloid beta before it becomes too late. Biogen is now showing results among patients with first symptoms and suggests that there is merit to this approach, the ultimate goal will be to delay the disease to an extent that patients never really suffer the full effects. 

“With such a devastating disease that affects tens of millions worldwide, today’s announcement is truly heartening in the fight against Alzheimer’s,” Michel Vounatsos, chief executive officer of Biogen, said in a release. “We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer’s disease and the potential implication of these results for similar approaches targeting amyloid beta.”

After the announcement the company’s value rose close to $18 billion as investors are betting that the US FDA will give the green light to the new treatment. Hope increase even though some such as Brian Skorney, an analyst at Baird said that Biogen would “have an uphill climb” to convince regulators that positive outcomes in the new studies “were anything more than random chance”.

John Hardy, from the UCL Queen Square Institute of Neurology, was one of the originators of the amyloid beta hypothesis some 25 years ago said that he had been in the field long enough to be wary, “Can one be cautiously excited? If this is the first crack in the wall, it is wonderful.” He added that even if the benefit to patients proved to be tiny, the significance could be huge. “It will show everyone we are on the right track.”

“The FDA will now assess the new data and its strengths and limitations before deciding about whether aducanumab should be made widely available in the US. At Alzheimer’s Research UK, we are already bringing together regulators and drug developers to help speed up the delivery of promising new treatments towards patients. This important work now has renewed urgency. It’s only through a sustained commitment to dementia research that will we make breakthroughs possible that keep people connected to their families, their worlds and themselves for longer,” says Evans.