Studies show that Alzheimer’s disease can be stopped if treatment begins before symptoms are revealed. The participants were the best candidates that scientists could find: still healthy, but with rare genetic mutations, they confirmed they would develop dementia.
For five years, volunteers received monthly blood tests, brain scans, spinal cord and cognitive tests, as well as monthly infusions or injections of one of two experimental drugs.
Now, the verdict is on: drugs have done nothing to slow or stop cognitive decline in these subjects, dashing the hope of scientists.
Neurologist and principal investigator of the study at Washington University in St. Louis. Randall Batman says he was “shocked” when he first saw the information: “It was really crushing.”
The results are a profound disappointment, scientists said – but not a knockout punch. The drugs don’t work, but the problems can be fixed: perhaps the dose was too low, or their patients should have been given too young.
Very few experts want to abandon the hypothesis that amyloid plaques in the brain are closely involved in Alzheimer’s disease.
The data from this international study, known as DIAN-TU, are still being analyzed and will be presented at scientific conferences in Vienna on April 2nd and Amsterdam in July.
The test was sponsored by the University of Washington, St. Louis, a subsidiary of Eli Lilly and Roche – a subsidiary of the National Institutes of Health and Charities, including the Altihar Association.
The study was short, with only 194 participants: 52 took a drug called Gantenerumab made by Rant, and an equal number of Solenzumab were made by Eli Lilly. Most of the subjects had no symptoms; Some were experiencing very mild symptoms at the very beginning. About 40 family members served as a comparison group and did not receive any drugs.
Volunteers cause excessive production of amyloid as a result of gene alterations that result in strong brain damage. Always enough for Alzheimer’s disease. The experimental drugs attacked amyloid, and scientists like Dr. Batman hoped they would prevent cognitive decline.
Participants in DINAU-TU were generally younger than Alzheimer’s patients, and other brain abnormalities, such as mini-strokes, had not yet been detected. The success of this experiment, it was hoped, showed that Alzheimer’s could be overcome.
Eli Lilly’s chief scientific officer. “This was a courageous test run by patients with a deep unmet medical need,” said Daniel Skovronsky.
Now companies and academic researchers must face a worrying question: Is it time to emphasize the development of anti-amyloid drugs for Alzheimer’s disease?
Studies of anti-amyloid drugs are still underway in older people. Scientists are testing drugs in the same group as DIAN-TU: A large family of Colombians also carry gene mutations that lead to Alzheimer’s at first.
Until now, studies of anti-amyloid drugs have repeatedly failed. Companies have spent billions of dollars on drugs. Someone like Pfizer is completely out of the race.
Many researchers have stated that they are not yet ready to leave.
The disease, they note, always progresses in the same way: amyloid accumulates in the brain and then a tangled, spaghetti-like protein, Tau appears, and neurons die.
“Amyloid and tau define the disease. Bingo, “said Dr. Ronald Petersen, director of the Mayo Clinic Alzheimer’s Disease Research Center. “It doesn’t make any sense not to attack the amyloid.”
Director of the National Institute of Aging. How well the two experimental drugs cleared the brain’s amyloid in subjects was not yet known, Richard Hodges said. This will require further analysis.
However, the institute has already donated another study to people with rare genetic mutations. This time, treatment with anti-amyloid drugs will begin even earlier – they are likely to have symptoms decades ago.
Nevertheless, Dr. Hodges says investigators must target other drugs.
Alzheimer’s drugs are currently in phase 3 trials – which are meant to imply that a drug actually works – essentially an anti-amyloid drug. But early-stage studies have focused on potential new approaches to the prevention and treatment of Alzheimer’s, said Dr. Hodges.
“Out of 46 pharmacological trials, 30 have targets other than amyloid,” he added. “We are moving in the direction of these other potential targets.”
Dr. Batman at the University of Washington at St. Louis knows the disturbing history of Alzheimer’s studies, however, is pushing for a failure to study.
“Over 300 unsuccessful trials – okay, what’s the reaction” that probably succeeded him, Dr. Batman wondered. “In terms of numbers, you have to say that they are not good. However I really thought we stacked the deck. “
His heart goes out to those participants who are destined to receive this critical condition. All four drugs were designed to reduce the risk of Alzheimer’s disease, but no one could change the course. “We have nothing now to treat these people,” he said.
Investigators also don’t have a quick way to notify participants of the results. Sending out party notifications requires the approval of an institutional review board, and this can take some time.
It would not be possible to call every participant, University spokesman Judy Martin Finch said. Instead, the university posted a news on Sunday at midnight.
Marty Riswig (1), a study participant living in Denver, learned the results this morning while searching online and through a private Facebook group with rare genetic mutations leading to Alzheimer’s.
For five years, a nurse has been coming to give him solenzumab at home, and once a year he travels to Washington University in St. Louis for extensive clinical and cognitive testing.
He and his wife were shocked to see the news. The average age of the onset of Alzheimer’s disease in his family is 47.
“We were very optimistic,” Mr Reswig said. “The devastating part of it for me is that I was really hoping it would change the world.”