There is hardly a drug that awaits humanity more than a cure for forgetting. Worldwide, around 50 million people are living with Alzheimer's or some other form of dementia. They know what it means when memories fade away. Only the new ones. Then the old ones, while the disease digs deeper into the personality.
For decades, companies have been failing to bring to market a drug that not only relieves symptoms for a short time, but actually slows the progression of the disease. This could change now. Biogen has announced that it will seek regulatory approval for the antibody Aducanumab in the US next year. It would be the first application for Alzheimer's therapy for 16 years. But the remedy is highly controversial.
Biogen announced in March that the antibody was not working. Since 2015, the company has tried to prove the effects of the drug in two studies. But the interim results seemed so hopeless that Biogen broke off both studies. Nevertheless, the company still evaluated patient data in the coming months, but its results had not yet been included in the interim results.
The big surprise came at the end of October: Biogen told his investors that the medicine was working, at least in high doses and in some patients at a very early stage of the disease. It's a lot of money, Wall Street analysts estimate that Aducanumab could bring in $ 4 billion annually. This week, Biogen unveiled details of the new results at a conference in San Diego to convince the many skeptics in the ranks of science. What is behind it?
Brain swelling as a side effect
The antibody Aducanumab can remove the protein fragment amyloid from the brains of Alzheimer's patients. However, the success of the remedy appears to depend substantially on the dose a patient receives over the period of treatment. Biogen explains that it still seemed in March that it would not work, because the dosage at the beginning of the study was too low for too many patients.
In fact, the new data suggest that high-dose aducanumab may slow the progression of Alzheimer's disease in the early and pre-stages. Surely it is not a miracle cure.
To measure the success of the drug, the researchers performed, among other things, a test that assesses cognitive performance. At the end of the treatment period of 78 weeks, patients treated with aducanumab scored 0.39 points better on average than those who received only placebo. Although this is a statistically significant difference, some experts doubt that this difference will have any significant impact on the lives of those affected. Accordingly, at least one to two points difference would be necessary.
It is also questionable how practicable the therapy would be in practice. In the treatment with the high-dose drug, more than every third patient developed a brain swelling as a side effect. Although this only led to complaints in 20 to 30 percent of those affected. Nevertheless, experts fear that the brain of the patients would have to be regularly monitored with MRIs. Thats expensive. Almost a quarter of the participants also broke off the therapy, for various reasons.
Difficult decision for the licensing authority
Biogen and experts who podium-based the company, however, expressed confidence in the drug approval process at the San Diego presentation. "All of this data indicates that the disease is affected, which means that the effects will continue to increase over time," said Paul Aisen, an Alzheimer's expert from the University of Southern California.
Scientifically proven this statement is not, but the two investigations had not lasted long enough. It is also possible that the effects only last for a short time. To clarify this, it would need another investigation, which, however, would take several more years. Many also call for this third study, because Biogen can base his findings only on one of the two studies, while the second showed no effects despite the recalculations. The company justifies this with too low dosages.
"Further studies are needed to assess the importance of aducanumab for Alzheimer's treatment," says Michael Heneka, Director of the Department of Neurodegenerative Diseases and Gerontology Psychiatry at the University of Bonn. "A therapy that can frighten the disease will probably have to consist of several very different drugs."
Judge must end up the US expert of the Food and Drug Administration (FDA), who decide on the approval of the drug. "They will have a very difficult job," said Eric Siemers, a former Alzheimer's researcher with Eli Lilly, who was not involved in the study. One analyst estimated the likelihood of aducanumab to be less than 50 percent.