A Boston neuroscientist who has spent more than four decades chasing the genetic roots of Alzheimer’s disease is now positioned to test whether a cheap daily pill could stop the disease before it starts. Dr. Rudolph Tanzi, who heads the Henry and Allison McCance Center for Brain Health at Massachusetts General Hospital, is at the center of two major funding announcements this month that together total tens of millions of dollars aimed at shifting Alzheimer’s treatment from reactive to preventive.
Mass General Brigham was awarded up to $41 million over four years by the Advanced Research Projects Agency for Health, a division of the US Department of Health and Human Services, to launch a national platform trial called the Platform Trial for Biomarker-Guided Alzheimer’s Disease Prevention, according to Mass General Brigham. The effort is co-led by Tanzi and Dr. Gene Bowman. As The Boston Globe reports, Tanzi and his research team are also slated to receive $25 million from philanthropists, with the combined federal and philanthropic funding supporting a four-year study testing five to 10 drug combinations the team developed.
From a Petri Dish to a National Trial
The project traces back to a tool Tanzi’s team created in 2014 known as Alzheimer’s in a Dish, a human-cell 3D model that develops the sticky plaques and tangles characteristic of the disease, per the Globe’s reporting. That system let researchers screen potential treatments faster. Tanzi’s team has used it to screen approved drugs and natural plant compounds. The planned trial will test five to 10 drug combinations.
The first five drug combinations will be tested in 300 patients whose blood biomarkers test positive for Alzheimer’s, the Globe reports, with 60 patients assigned to each combination and 60 to a placebo group. Researchers will then assess whether those biomarkers improve in blood samples collected after patients take the experimental drugs. The University of California San Diego School of Medicine is also partnering on the ARPA-H initiative, leveraging its Alzheimer’s Disease Cooperative Study network to accelerate nationwide patient recruitment using blood biomarker screenings, according to UC San Diego.
A Startup Built on Decades of Research
Separately, Boston-based startup Acta Pharmaceuticals announced $24 million in total seed financing this month, backed by the Alzheimer’s Disease Research Foundation, Dolby Family Ventures, and Gamma Bio Investors, to commercialize Tanzi’s early-intervention drug pipeline, per BioSpace. Tanzi cofounded Acta. The company has begun an early-stage clinical trial of a gamma-secretase modulator and is recruiting 72 trial participants, according to the Globe.
Why a Pill, and Why Now
Tanzi has said he hopes a daily pill can block harmful amyloid plaque buildup before cognitive decline sets in, comparing the strategy to taking statins early to prevent heart disease, per the Globe. If the clinical trial succeeds, he has said, people at risk of Alzheimer’s could take the medicine to prevent the disease altogether, rather than treating it after symptoms emerge…