INDIANAPOLIS – A possible Alzheimer’s disease treatment from drugmaker Eli Lilly missed the main goals of two late-stage studies, but the treatment showed some potential for slowing memory decline in patients with mild cases of the mind-robbing condition.
Shares of Lilly climbed Friday after the Indianapolis company announced what could be a step toward a long-awaited breakthrough in the fight against a disease that has no cure and ultimately proves fatal. But doctors and Lilly officials cautioned against overreacting to the study’s initial results.
Lilly said Friday that its treatment, solanezumab, failed to slow cognitive decline, which involves a person’s ability to remember things, in two late-stage studies of about 1,000 patients each. But when data from the trials were combined, scientists saw a statistically significant slowing of the rate of cognitive decline in that bigger population.
They also saw a statistically significant result when they examined a subgroup of patients with mild cases of Alzheimer’s disease. The studies focused on patients with mild to moderate Alzheimer’s cases.
The studies fell short of their main goals, and that means the drug “isn’t going to the (Food and Drug Administration) tomorrow to be approved for sale,” said William H. Thies, chief medical and scientific officer for the Alzheimer’s Association, which was not involved in Lilly’s research. Still, he said the statistical significance of the combined results is important.
“If that can be replicated, that is a major finding,” he said. “It’s the first time we’ve been able to change the course of Alzheimer’s disease or any part of Alzheimer’s disease in people.”
Lilly said it will discuss the results with regulators and plan the next steps. Full results from the studies will be presented at two scientific conferences in October, the company said.
It’s unclear how the Food and Drug Administration will view the results, given that the drug missed its main goals.
If you look through “rose-colored glasses” at the results, there may be a sign of potential benefit on cognitive tests, but it is not clear whether that is enough to make a real difference clinically in how patients do, said Dr. Ronald Petersen, director of the Mayo Clinic’s Alzheimer’s Disease Research Center.
The key will be details the company will present later on brain imaging and other tests, he said. If those show that the drug is helping clear plaque, “it might mean that the drug is doing what it’s supposed to be doing, but we’re just attacking the disease too late,” said Petersen, who heads a safety monitoring panel for two companies working on a different Alzheimer’s treatment.
It’s up to the FDA to decide how much faith to put in secondary analyses or results in subgroups, he added.
“The danger would be an over-interpretation of a small finding or a subtle effect,” Petersen said.
About 35 million people worldwide have dementia, a term for brain disorders that affect memory, judgment and other mental functions. Alzheimer’s is the most common type. In the United States, more than 5 million people have Alzheimer’s, which is the country’s sixth-leading cause of death.