Finding a drug that actually stops the progression of Alzheimer’s disease, rather than just treat symptoms, is considered a holy grail among many drug makers. If such a drug could be developed, it would be worth billions of dollars in annual revenues. Despite many drug candidates tested over the last decade, that grail has remained elusive.
More than 5 million people currently have Alzheimer’s, and that figure could more than triple to 16 million by 2025, according to the Alzheimer’s Association, a non-profit that seeks to further research into the disease and well as improve care for patients. It’s no wonder finding a Alzheimer’s drug that could stop the disease – or at least slow its progression down — is so lucrative.
With drug development failures continuing to mount and a huge unmet medical need, the FDA is taking some action. Last week, the agency issued draft guidance essentially saying that it would try a new approach to Alzheimer’s drug development.
Pharmaceutical companies could gain accelerated approval of an Alzheimer’s medication for people who do not show any signs of the disease if the drug impacted a biomarker, or biological signal related to the disease. Although not specified by the FDA, this could include reducing beta amyloid plaque, a substance often found in the brains of those with Alzheimer’s.
While this appears at first glance to be a boon for drug companies and Alzheimer’s patients, this may not be the case. The FDA is essentially lowering the bar, at least for initial approval of a drug. Under accelerated approval, a drug comes to market, but the drugmaker still has to conduct additional tests to confirm the med has real world benefit.
However, as Ed Silverman points out in STAT, the FDA also noted that the benefit would need to include changes in cognition. This is a departure from prior guidance in that the agency previously said it was looking for changes in both cognition and function in approving Alzheimer’s treatments.
The Alzheimer’s Association is lauding the guidance. Maria Carrillo, the organization’s chief science officer, told Bloomberg News that the guidance “is a clear statement that the FDA understands that the science of Alzheimer’s has evolved.”
While I understand that the changes in the guidance could spur development of new Alzheimer’s drug candidates by researchers, I remain concerned that any potential drugs approved will do so under weakened standards of effectiveness. I want a drug developed that can truly fight Alzheimer’s – but fear drugs may be approved that have little, if any, benefi