Last month, on June 7, the US Food and Drug Administration (FDA) approved a new medication for the treatment of Alzheimer’s Disease—for the first time in 18 years. The drug, developed and produced by Biogen, and known as aducanumab, will be sold under the brand name Aduhelm at a cost of $56,000 a year, with additional costs in the tens of thousands of dollars relating to ongoing monitoring (through brain scans) and ongoing diagnostic testing that will necessarily accompany the use of the drug. It will be given through a monthly intravenous infusion (IV drip). It accompanied with five other drugs that are currently approved in the U.S. for the treatment of Alzheimer’s. Since most patients who will be given the drug are seniors, coverage would fall under Medicare Part B. However, Medicare has not yet announced how the overall cost of the treatment will be covered.
There is considerable controversy over the FDA’s hasty approval of the drug through its “accelerated approval” (or “fast-track”) program, with many specialists and doctors who treat Alzheimer’s patients expressing reservations and concerns about the effectiveness of the drug in slowing down or halting the cognitive decline that is a characteristic of Alzheimer’s. What is different about the new drug is, earlier drugs merely sought to alleviate dementia symptoms, Aduhelm addresses what has long been thought to be the cause of Alzheimer’s, that is, the amyloid beta plaques that accumulate in the brain (discussed in previous newsletters). The new drug is a monoclonal antibody that attacks and breaks down the amyloid plaques. The clinical trials of the drug showed that it did in fact reduce the quantity of the plaques significantly. However, past experience with other amyloid-reducing drugs has shown that simply reducing the amount of amyloid in the brain does not necessarily slow down cognitive decline. The concern with the new drug is that in two Phase 3 trials, one trial indicated a slight slowing of cognitive decline, but the other showed no effect at all. The trials were stopped when it was noticed that the drug was showing no signs of being beneficial, and more than 33 percent of the more than 3,000 participants were thus unable to complete the trail. So, there is very little evidence that the drug is effective as a treatment for Alzheimer’s, even if it is effective in reducing amyloid plaques.
Another issue that concerns specialists and doctors is that the new drug may cause swelling or bleeding in the brain, accompanied by symptoms such as blurred vision, confusion, headaches, dizziness, or nausea, much like earlier amyloid-reducing drugs that have been tried in the past. In the Phase 3 trial of the new drug, these side-effects were experienced by 40 percent of the participants receiving the high dose, with the result that 6 percent of the high-dose participants dropped out of the trial. While these side-effects are manageable with careful monitoring, doctors are concerned that such monitoring will be costly and difficult to do in a regular practice and in care homes and assisted living facilities, where an estimated 42 percent of residents are afflicted with dementia.
Finally, the people who participated in the trial were all in the early stages of Alzheimer’s and had mild cognitive impairment. However, the FDA’s label for the drug says: “for the treatment of Alzheimer’s disease,” thus creating the impression that it is an effective treatment across the board, even for patients in advanced stages of the disease. Several doctors who are treating Alzheimer’s patients have expressed concern that they are now ethically obliged to prescribe the drug for all their Alzheimer’s patients if these patients request it, even if the patient is in the later stages of Alzheimer’s and will almost certainly not benefit from it, according to the available evidence from the clinical trials.
The best approach is to proceed with caution and to examine all the facts before making the decision about taking Aduhelm.