Dementia research has seen more than 100 failed attempts to develop a disease modifying treatment for Alzheimers since 1998, with pharmaceutical giants including Eli Lilly, Pfizer, and Roche all reporting setbacks with experimental therapies. Against that backdrop, a 100 million dollar Genentech clinical trial based in Colombia is taking a fundamentally different approach: treating patients before symptoms appear, targeting the amyloid plaque believed to be the primary driver of Alzheimers disease in people who carry a rare gene mutation that makes early onset dementia virtually certain.
Why This Dementia Research Trial Targets a Colombian Family
The study focuses on members of a Colombian family who carry an unusually rare genetic mutation linked to early onset Alzheimers, causing dementia symptoms to appear as early as age 45. This genetic specificity gives researchers an exceptional opportunity in dementia research: a population where the disease is highly predictable, the timeline is known, and intervention before symptom onset is feasible.
The trial enrolled approximately 300 participants. Of those, around 200 are suspected carriers of the rare gene mutation, with half receiving the anti amyloid injection being tested and half receiving a placebo. The remaining 100 non carriers serve as a control group and receive a placebo. Final results are not expected until approximately 2020, when the last participant completes five years of testing, though an interim analysis is expected to be published before that date.
The Amyloid Hypothesis in Dementia Research
Genentech’s trial is built on the amyloid hypothesis, the theory that accumulation of amyloid plaque in the brain is the primary cause of Alzheimers disease. This has been the dominant framework in dementia research for decades, though it has generated significant controversy given how many amyloid targeting drugs have failed in clinical trials.
The most compelling recent evidence in support of the hypothesis came from Biogen’s early trial results for aducanumab, an antibody that targets amyloid plaque. The drug produced measurable reductions in brain amyloid, with more pronounced effects at higher doses and over longer treatment periods. Biogen announced plans to advance aducanumab to a Phase III trial. Following those results, Roche researchers expressed renewed confidence in their own amyloid targeting compounds, announcing plans to reexamine two experimental drugs that had experienced setbacks in 2014.
Why Early Intervention Is Central to Dementia Research Progress
The core insight driving the Colombian trial is timing. Researchers told investigators they believe better results can be achieved by starting treatment before the onset of symptoms and before severe brain damage has occurred, in people who would otherwise be certain to develop the disease. This rationale aligns with the broader realization in dementia research that previous trial failures may have been attributable not to flawed drug targets but to treating patients whose disease was already too advanced for amyloid removal to produce cognitive benefit.
An Australian study tracking amyloid accumulation over time found that amyloid begins building up in the brain approximately 15 years before symptoms appear. Treating patients with existing clinical disease means targeting a process that has already been underway for a decade and a half, well past the window where intervention may be most effective. The Colombian trial attempts to close that gap by identifying genetically at risk individuals and intervening at the earliest biologically measurable stage.
The Scale of the Dementia Research Challenge
Alzheimers disease currently affects approximately 15 million people worldwide, with the total dementia burden reaching around 50 million. That number is projected to grow to 75 million by 2030, driven in large part by aging global populations and the absence of any approved treatment that addresses the underlying cause of the disease rather than managing its symptoms. Every currently available Alzheimers drug manages symptoms only.
The urgency of finding a disease modifying treatment in dementia research is therefore not merely scientific but demographic and economic. Clinical trial participation, particularly in studies targeting the earliest stages of disease, represents the most direct path available for accelerating that discovery.
To read more about Alzheimers and neurodegenerative disease research, visit the FOMAT blog. FOMAT conducts dementia and CNS clinical trials at sites across the United States. To learn more about active studies, visit FOMAT’s patient studies page.
For the full source, see the original article at BioSpace.