After a decades-long process, Cambridge-based biotech company Biogen has guarded what experts say could be the “first real treatment” for Alzheimer’s disease at the door of the Food and Drug Administration.
It’s called aducanumab. In Biogen’s clinical trials, which had a bumpy road and some mixed results, participants who received the treatment were able to retain their memory and perform daily tasks for longer than those who received the placebo.
On Friday, Biogen announced that the FDA accepted its application, complete with phase one to three clinical trials, for approval of the treatment. The federal agency will decide if the new treatment is approved for use in the clinic by March 7, 2021.
“I think there’s a good chance that aducanumab, if approved and we do not yet know it, will be the first disease-causing drug for Alzheimer’s, and that it will be a huge milestone and patients of mine and others can access it. medicine, “says Dr. Dennis Selkoe, professor of neurological diseases at Brigham and Women’s Hospital and Harvard Medical School. He did not work with Biogen, but some of his patients were involved in clinical trials with aducanumab.
Before saying aducanumab, Selkoe says doctors could only prescribe drugs to Alzheimer’s patients that would slow down some of the symptoms of the disease but then lose its effectiveness. They could do nothing to slow the progression of the disease itself. But by changing the course of Alzheimer’s itself, aducanumab can offer patients something that no other drug candidate has achieved: more time.
‘If you had 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 her decline, that will keep her in a better place for a longer period of time, ”he says. “This is what patients and their families were desperately looking for.”
I talked to Selkoe about the drug and how it works. This interview has been edited for length and clarity.
What does aducanumab do?
“Aducanumab is an antibody. It binds to the amyloid beta protein, especially aggregate forms of the amyloid beta protein that we consider the principle bad guys in Alzheimer’s disease. Alzheimer’s starts in many cases, if not most cases, with the buildup of this small protein called amyloid beta and the formation of amyloid beta plaques.There is ample evidence that people with too much buildup of this small protein develop Alzheimer’s.Now aducanumab binds to amyloid beta protein. It mobilizes the body’s immune system against the amyloid beta plaques, and seems to clear the plaques itself.
“The next step in Alzheimer’s is the buildup of toxic tangles of a protein called tau, as well as the accumulation of tau in an abnormal form called phospho-tau. In participants in Biogen’s clinical trials, amyloid plaque load went down and tau load went down.
Importantly, in phase three clinical trials, aducanumab helped the rate of decline in cognition in patients – issues such as memory and other cognitive functions that are gradually lost. There was evidence that aducanumab helped maintain household chores. “, cleaning up, preparing some food and doing other daily tasks that we expect people to do relative to placebo. Aducanumab stunned that decline. It did not stop the decline, but it slowed it down, that is what we hope for.”
Is Fucadabab approved by the FDA?
“It can not yet be concluded that it will be approved. It was a very important step for the FDA to announce that they will review Biogen’s application and expedite this review. An FDA advisory committee will also examine the data and give an opinion. give – it could be a split opinion.It all depends on what the experts believe is a sufficient degree of delay of the decay to make it suitable for approval and [administer] however, to many hundreds of thousands and millions of people presenting with Alzheimer’s disease.
“Some experts may think that although it shows a clinical benefit and no question removes the abnormalities of Alzheimer’s from the brain, the degree of benefit is not that great. It is a small benefit that one sees. Then some will say, even if it is a small advantage, it is one that my loved ones and patients should be offered.
“In my opinion, it’s very impressive that it can actually do that, and it has to be approved based on the data. This is a very complex disease, and I do not think anyone believes that aducanumab will solve patients’ problems. “It’s a very important step in the right direction. You have to run before you run, and without that crucial step we will not make as much progress towards the second and third antibodies.”
What would the approval of this medicine mean for people with Alzheimer’s?
“This is a remarkable development. Again and again I will tell patients that I see evidence that you have symptoms and signs of Alzheimer’s, but I have no treatment that reduces that. We only have symptomatic treatments that can help you for a few weeks or months, then lose effect.
“This is a game changer, although I and others acknowledge that it is not ideal. There is still more work to be done. What we need to do is gain experience with how early we should use aducanumab. It will be remarkable be to turn to families and say, ‘We have a drug.’ “
