The possibilities of science usually outweigh the results from science so anytime there’s a hint of a breakthrough, you have to keep from getting too excited. Still, the news that an experimental drug has appeared to slow the cognitive decline from Alzheimer’s provides something in short supply in the field: hope.
“I’ll remain cautiously optimistic,” Dr. Ronald Petersen, director of Mayo Clinic Alzheimer’s Disease Research Center, tells Stat News. “I think the data are intriguing. The effect sizes sound reasonable, the drug seems safe, and on the biological side of it, the drug seems to be working.”
The news surprised those attending an Alzheimer’s conference in Chicago this week.
After 18 months, patients who got the drug, called BAN2401, performed 30 percent better on a cognitive test than those getting placebo, Stat said.
“I wouldn’t say this is a quantum leap,” Dr. Samuel Gandy, associate director of the Mount Sinai Alzheimer’s Disease Research Center, tells the New York Times. “It is a convincing moving of the needle. But it’s not clear that the needle has moved far enough to make a difference in people’s lives.”
Other Alzheimer’s therapies have resulted in bad side effects to those taking them, but in so-called Phase 2 tests so far, fewer than 10 percent of those taking the drug had side effects from it.
What now? That depends, the NY Times says. The FDA could lower the barrier to approving the drug, but it still could take years.
The F.D.A. typically requires Phase 3 clinical trial data to demonstrate safety and effectiveness. However, the agency does have processes for expediting the review of drugs, said a spokeswoman, who declined to comment on this drug or on conditions that would be taken into consideration for an Alzheimer’s drug.
Eisai is the maker of Aricept, which is one of the few drugs that can help slow early memory decline, but which is effective for only about six to nine months. Biogen is the maker of another Alzheimer’s treatment, aducanumab, that has shown early promise in a small Phase 1 trial in both reducing amyloid and slowing cognitive decline. Many in the Alzheimer’s field are intently anticipating the outcome of two large clinical trials of aducanumab, expected to be able to report results in 2020.
Dr. Gandy said the BAN2401 results were encouraging for the prospects of aducanumab because it suggests that there are at least two compounds that may be able to attack both amyloid buildup and cognitive decline.
Still, the news provides what’s in short supply for families experiencing Alzheimer’s.