The Biogen laboratory announced at the end of October that it was going to ask the American health authorities for authorization to market the “first treatment which will reduce the decline in cognitive functions and autonomy” in Alzheimer’s patients. Announcement effect or real hope? Why Doctor questioned Doctor Stéphane Epelbaum, specialist in pathology, on the subject.
“We hope to offer Alzheimer’s patients the first treatment that will reduce the decline in their cognitive functions and autonomy”. At the end of October, the Biogen laboratory said it wanted to file an authorization request for Aducanumab, a new drug against Alzheimer’s, with the American drug agency, the FDA, in early 2020. For twenty years, research has multiplied in this field without any drug capable of effectively curbing this degenerative disease which affects more than 35.6 million people. every year in the world does not seem to see the light of day. Also, this press release has of course had the effect of a bomb. So much so that the Vaincre Alzheimer association released a video titled Alzheimer’s: hope for a treatment is reborn. Announcement effect or real glimmer of hope? Why Doctor questioned the Dr Stephane Epelbaumneurologist at Pitié Salpetrière, at the Institute of Memory and Alzheimer’s Disease and researcher at the Brain and Spinal Cord Institute (ICM), rather optimistic on the subject.
“In the past, there have been a lot of publicized announcements of supposedly finally effective treatments for Alzheimer’s disease. These announcements were made on often very weak evidence and there were not enough arguments to support the effectiveness of the treatments previously advanced. Today, from my point of view, the result presented by Biogen is true”, begins the specialist.
At the end of March, Biogen had however announced the cessation of clinical trials testing its treatment based on a monoclonal antibody, Aducanumab, on 2,000 patients for lack of convincing results. “A few side effects had been reported which clinically resulted in headaches in 20% of patients treated with Aducanumab, but that was not enough to stop a study. They decided to stop their study on the basis of trivial analysis, an interim analysis, because they had no strong signals at that time indicating that the drug was going to be effective. They began to have weak signals but judged that it was insufficient to continue the study which cost them very dearly. You should know that it is very expensive to conduct studies against Alzheimer’s disease”, explains Stéphane Epelbaum.
Slow down the loss of autonomy by up to 40%
“This so-called futility study was carried out on data acquired until the end of 2018. The analysis took four months to be carried out and during this time other patients continued the study. On March 21, 2019, Biogen announced that the results were not conclusive, but by then combining the data of the additional four months with those of the futility study, the company finally decided that the drug was effective according to the criteria predefined at the time. start of the study”, specifies the neurologist.
Today, the laboratory promises a treatment that would reduce the aggregates of beta-amyloid, a protein that accumulates and creates plaques in the brain tissue of patients with Alzheimer’s, causing their loss of autonomy. In detail, this drug would reduce cognitive decline by 23% compared to a placebo and slow down the loss of autonomy by up to 40%. “It would stop the disease and allow people to continue shopping or leaving their homes alone, which are very important things,” enthuses Stéphane Epelbaum.
“Now Biogen is relaunching the program that was dead and the researchers want to see what happened to the people who took part in the studies, in order to examine whether those who were on placebo are doing worse than those who were on treatment until March. . This will give them a longer-term view of the potential effect of the product,” he continues.
Bringing public opinion into the FDA’s decision
As for FDA approval, “it’s a gamble,” Epelbaum admires. “By making its announcement, Biogen wants to raise the interest of the public and associations on Alzheimer’s disease. The laboratory wants public opinion to weigh in the balance and make the FDA flinch”. Even if the product would be approved in the United States, it is not ready to arrive in France. Indeed, in our country, “the marketing authorization for a drug often takes longer than in the USA. It may take another year or two.”
There is also the question of reimbursement, because the drug offered by Biogen will undoubtedly be very expensive. “Biogen has put considerable cost into the development of this product. It will cost several thousand euros a year, maybe 20,000. This is a monthly injection that is done in the hospital. At first, this will probably only be done in specialized centers”, specifies the specialist who, if this drug arrived in France, would probably prescribe it to his patients.
“At the moment, the results seem convincing to me but they would have to be confirmed by follow-up studies which should start very soon. I need a little more evidence to establish my certainty. There, we are 90% sure but I remain cautious because we have been talking about Alzheimer’s disease for a very long time and looking for an effective treatment”, explains the researcher.
“The most advanced treatment in research”
A position shared by the France Alzheimer association, according to whom “the disease is gaining ground day by day” (by 2020, France will probably have 1,275,000 people with Alzheimer’s, according to his figures). If the news from Biogen is “synonymous with hope for the many families confronted with the disease and who find themselves to this day without a therapeutic solution (…). However, it is advisable at this stage to be cautious”, writes the organization on its site. If the additional analyzes carried out by the health authorities “on the basis of the information communicated by Biogen” prove to be positive, it will remain “thereafter to determine which patients will be able to benefit from this treatment and when.
While waiting to learn more, Aducanumab is today “the most advanced treatment in research against Alzheimer’s”, concludes Stéphane Epelbaum.
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