An experimental treatment has shown early signs of delaying Alzheimer’s symptoms in individuals genetically predisposed to develop the disease in their 40s or 50s. This promising development comes as part of ongoing research led by Washington University in St. Louis, though funding delays tied to political challenges threaten to disrupt further progress.
The study focuses on families with rare genetic mutations that virtually guarantee the onset of Alzheimer’s at a specific age. These families provide a unique opportunity for researchers to track the disease’s progression and assess the impact of early intervention.
Jake Heinrichs, a participant in the study from New York, has been part of the research for more than a decade. Despite inheriting an Alzheimer’s-causing gene that claimed the lives of his father and brother at a similar age, Heinrichs remains symptom-free. He credits the treatment for giving him “an extension to my life that I never banked on having.”
However, Heinrichs, now 50, and his wife, Rachel Chavkin, express concern over the potential loss of access to the treatment due to funding cuts. “How much time do we have?” Chavkin said. “This trial is life.”
Currently, two FDA-approved drugs in the U.S. can slow the progression of early-stage Alzheimer’s by targeting amyloid plaques in the brain, a hallmark of the disease. However, this new study investigates the possibility of preventing or delaying Alzheimer’s by removing amyloid much earlier in the process—before the first symptoms appear.
The research, which involves a subset of 22 participants, showed that long-term treatment with amyloid-targeting drugs halved the risk of symptom onset. These results, published in Lancet Neurology, mark a significant breakthrough, though researchers caution that further studies are needed to confirm the long-term effectiveness of the treatment.
Dr. Randall Bateman, who leads the Dominantly Inherited Alzheimer’s Network at Washington University, is particularly focused on understanding how prolonged amyloid removal impacts the development of symptoms. His team is now shifting participants from an experimental drug to Leqembi, an FDA-approved intravenous treatment, to further explore its protective effects.
“What we want to determine over the next five years is how strong the protection is,” Bateman said. “Will these individuals ever develop Alzheimer’s if we continue treatment?”
However, political and funding challenges are casting a shadow over these efforts. Bateman’s attempt to secure National Institutes of Health (NIH) funding for the next phase of the study has been delayed due to canceled reviews and ongoing issues at the agency.
This delay comes as NIH director nominee Dr. Jay Bhattacharya raised concerns about the agency’s focus on amyloid-based research, questioning whether a wider range of hypotheses should be considered. His remarks have fueled worries that NIH may reduce support for amyloid-focused studies, despite ongoing evidence of its role in Alzheimer’s.
While researchers remain divided on whether amyloid is the sole cause of Alzheimer’s, there is consensus that it plays a significant role in the disease’s development. Amyloid plaques trigger the accumulation of tau proteins, which ultimately lead to neuron death and cognitive decline. Current research is also investigating other factors such as inflammation, immune cells, and viral connections.
Despite the uncertainties, families like Heinrichs’ and June Ward’s, another study participant, are hopeful. Ward, who is 64 and healthy, is particularly excited by the possibility that Alzheimer’s may not be her fate, as her mother began showing symptoms at 62.
“We need the NIH to be not politicized,” Chavkin added. “It’s about keeping people alive and helping them live better.”
As the study continues, the looming question remains: Will future funding allow these critical discoveries to progress, or will political challenges stall the potential to delay the devastating effects of Alzheimer’s for future generations?
Related Topics
