FDA pushes for drugs that block Alzheimer’s

In an effort to prevent millions of U.S. adults from developing Alzheimer’s disease during the coming decades, the Food and Drug Administration recently proposed guidelines to help drug manufacturers develop medications for treating early stages of the condition, before the onset of noticeable dementia.

The Feb. 7 draft guidance comes after a study in Neurology published online Feb. 6 projecting that the number of people in the U.S. with Alzheimer’s could triple in the next 40 years, soaring to 16 million people, due largely to the aging of the baby boom generation. The burden of caring for Alzheimer’s patients is expected to fall to primary care physicians, because too few specialists are available to meet the nation’s need, researchers said (link).

The FDA’s “ultimate goal [is] preventing or arresting Alzheimer’s disease before the brain suffers too much damage,” said Russell Katz, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, in a Feb. 7 post on the agency’s blog. Physicians and the public can comment on the proposal online for 60 days (link).

The draft guidance reflects changes in scientists’ understanding about when Alzheimer’s disease begins. It now is believed that the condition could arise as early as 15 to 20 years before clinical onset of the disease, said Dean M. Hartley, PhD, director of Science Initiatives at the Alzheimer’s Assn.

“The fact that the FDA is recognizing the early stages of the disease is very helpful for pharmaceutical companies [that are trying] to build into their trials ways of looking at these earlier components” of the condition, Hartley said.

Five drugs are licensed by the FDA to treat Alzheimer’s. In some patients, the medication temporarily improves symptoms and delays the advancement of the disease. But none of the drugs modifies the disease process. To change that, the FDA said it’s critical to identify and study patients with very early forms of Alzheimer’s disease or those at risk for developing the condition.

Understanding the draft guidance

The agency’s draft proposal explains ways researchers could identify and select eligible patients for participation in clinical trials. The guidance also discusses the types of evidence drugmakers can use to demonstrate that a new medication effectively treats Alzheimer’s symptoms or slows disease progression (link).

For instance, in studies with participants who do not yet have problems with daily function, showing evidence that a drug delays impaired thinking might provide sufficient evidence to support accelerated approval, the FDA said. After such a drug is approved, researchers would have to show that the positive cognitive effect persists over time.

“This draft guidance is intended to serve as a focus for continued discussions between the FDA and pharmaceutical sponsors, the academic community, advocacy groups and the public,” Dr. Katz said.

The proposal is part of the Dept. of Health and Human Service’s efforts under its National Plan to Address Alzheimer’s Disease. The initiative, which was launched March 15, 2012, by the Obama administration, calls for the government and the private sector to intensify efforts to treat or prevent Alzheimer’s and related dementia.

“The FDA is committed to vigorously addressing Alzheimer’s disease and will work with industry to help develop new treatments in this early population as expeditiously as possible,” Dr. Katz said.