A trial of a potential Alzheimer's drug failed to prevent or slow cognitive decline, another disappointment in the effort to find solutions for the disease.
The first time a drug was given to people who were genetically destined to develop the disease was in the decade-long trial. About 1,200 of the 6,000 people in the family have a genetic variation that makes them more likely to develop Alzheimer's in their 40s to 50s.
The disease has taken away the ability of many members of the family to work, communicate and carry out basic functions. People die in their 60's.
169 people were given either a placebo or a drug in the trial. The placebo was given to 83 people who were not carriers of the disease in order to protect their identities.
The trial investigators had hoped that intervening with a drug years before memory and thinking problems were expected to emerge would hold the disease at bay and provide important insights for addressing the more common type of Alzheimer's that is not driven by a single genetic flaw.
The leader of the research team said at the news conference that they were disappointed with the results. Everyone who would benefit from an effective Alzheimer's prevention therapy is in our hearts. We take solace in the fact that this study continues to help shape a new era in Alzheimer's prevention research.
The results are a blow to the drugs that target the amyloid in the brain of patients with Alzheimer's. There have been years of studies with various drugs that attack amyloid. The studies of crenezumab in people in the earliest stages of Alzheimer's disease were stopped in 2019.
Aduhelm was approved by the FDA last year in a highly controversial decision. It was unclear if Aduhelm could help patients, but the F.D.A. approved it under a program that allows authorization of drugs with uncertain benefit if they are for serious diseases with few treatments and if the drugs affect a biological mechanism that is reasonably likely to help patients. Many Alzheimer's experts criticized the decision because of the poor track record of anti-amyloid therapies. The trial results add to the disappointing evidence.
Sam Gandy, the director of Mount Sinai's Center for Cognitive Health, wished there was more positive to say.
According to Dr. Gandy, the patients who were most likely to respond to anti-amyloid were the members of the Colombian family.
Dr. Pierre Tariot, the director of the Banner Alzheimer's Institute and a leader of the Colombian research, said some of the data did suggest that patients receiving crenezumab fared better than those receiving the placebo.
Many anti-amyloid therapies, including Aduhelm, have been found to cause brain bleeding or swelling in some patients.
There will be additional data presented at the conference. Thursday's results didn't include more detailed information about the drug's effects on the brain and other parts of the biology of Alzheimer's They did not include increases in the dose of crenezumab, which was given to patients as they learned more about the drug. Some patients in the trial received up to two years of the highest dose.
Dr. Francisco Lopera began working with family members decades ago and helped determine that they were afflicted with a form of Alzheimer's. The trial convinced him that prevention is the best way to find a cure for Alzheimer's disease even if we don't have a good result.
The contribution to the investigation of Alzheimer's disease was made by us. We are ready to begin looking at the solution for this disease.
Maria Areiza said her husband was one of the first patients to sign up for the trial. About eight years ago, Hernando started to develop symptoms of cognitive decline. He can still hold a conversation even though he has been diagnosed with Alzheimer's dementia. His family was hoping that he would benefit from the trial.
His wife said that he had put all his hopes in the study.
She reported from Medelln.
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