FDA to Alzheimer's researchers: Time to focus on very early stage patients

One of the hottest conversations in drug research circles right now concerns the future of Alzheimer's drug research. Following the high-profile Phase III failures of bapineuzumab and solanezumab last year, there has been growing buzz among investigators that they need to shift their attention to early-stage patients, whose brains are not yet severely damaged by the disease. And in case anyone hasn't been listening, the FDA added its voice to the move, offering a new draft guidance today aimed at shooing investigators toward a more realistic goal.

"The scientific community and the FDA believe that it is critical to identify and study patients with very early Alzheimer's disease before there is too much irreversible injury to the brain," said Russell Katz, M.D., director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, in a release. "It is in this population that most researchers believe that new drugs have the best chance of providing meaningful benefit to patients."

That's exactly in line with a number of closely watched trials now taking shape, including Genentech's study for crenezumab, in-licensed from AC Immune. Eli Lilly ($LLY) had tried to gain the FDA's attention with its secondary analysis of early stage patients in its big solanezumab study. But the agency clearly decided to show Lilly the door, sending its scientists out to do a new Phase III that will exclude the moderate-stage patients who hadn't responded in the trial.

Curiously, even after the trend to refocus investigators on early stages of the disease emerged in recent months, Merck ($MRK) announced an unusual Phase II/III study of its Alzheimer's candidate that will go after the same mild-to-moderate population that has frustrated all other earlier attempts. So the agency may have some additional lobbying to do before everyone wakes up to the switch.

It won't be easy to shift, though. Investigators have been working on new biomarkers and clinical measures that can be used to identify early-stage patients for clinical trials. But with millions of people suffering from the disease and millions more threatened, wasting more time and effort on misguided studies is in no one's interest.

- here's the FDA's release