Experts have begun a clinical trial that could take decades to complete but has the potential to change the lives of millions.
اضافة اعلانResearchers are testing a new drug designed to remove amyloid plaques from the brains of young individuals who are genetically predisposed to early-onset Alzheimer's disease.
The study is led by researchers from the Washington University School of Medicine in collaboration with pharmaceutical company Eli Lilly, which developed the experimental drug remternetug.
This drug is intended to be a successor to donanemab (commercially known as Kisunla), which received approval for the treatment of early Alzheimer’s in July 2024.
Both drugs target misfolded beta-amyloid protein, which accumulates in the brains of Alzheimer’s patients, forming solid plaques that contribute to cognitive decline.
In addition to amyloid, misfolded tau protein also plays a crucial role in disease progression.
While anti-amyloid drugs are the first approved treatments believed to slow Alzheimer’s progression, their current impact remains limited.
However, evidence suggests that amyloid plaques begin accumulating in the brain 20 years or more before Alzheimer’s symptoms appear. Researchers believe these drugs could be more effective if administered early to those at risk—potentially even preventing the disease entirely. This hypothesis is what the new trial aims to test.
The Primary Prevention Trial
This study, known as the Primary Prevention Trial, will enroll 240 participants from families carrying mutations in one of three genes known to cause early-onset Alzheimer’s.
Participants will be divided into two groups:
- One group will receive the experimental drug
- The other will receive a placebo as a control group
The drug will be administered every three months for two years, with the option of continuing treatment for an additional four years.
Eligible participants are aged 18 to 25 and are at least 11 to 25 years younger than their expected Alzheimer’s onset based on family history (which typically occurs in their 30s, 40s, or 50s).
Due to the participants’ young age, researchers do not expect to observe cognitive changes during the trial. Instead, they will monitor whether the drug slows or prevents the buildup of amyloid plaques in the brain.
Long-Term Study and Potential Impact
The placebo-controlled phase of the trial is expected to last four to five years, while the entire study is set to conclude by 2034. However, researchers plan to track participants for many years beyond that.
If successful, this trial could pave the way for new preventive strategies against Alzheimer’s—not just for those at risk of early-onset disease but also for individuals vulnerable to late-onset Alzheimer’s. The findings will provide valuable insights into the disease’s mechanisms and contribute to the development of more effective treatments in the future.
