NIH Funding Boosts New Alzheimer’s Research on Prevention, Novel Drug Targets

Researchers will test promising drugs aimed at preventing Alzheimer’s and identify and validate biological targets for novel therapies, with approximately $45 million in new funding from the National Institutes of Health. The initiative will support innovative new studies as part of an intensified national effort to find effective interventions for this devastating degenerative brain disease.

The studies are among the first to be developed with direction from the 2012 NIH Alzheimer’s Disease Research Summit: Path to Treatment and Prevention and reflect research goals in the National Plan to Address Alzheimer’s Disease External Web Site Policy. Of the funding, $40 million is from an allocation from the Office of the NIH Director, Dr Francis Collins, with additional funding from the National Institute on Aging (NIA), the lead Institute within NIH for Alzheimer’s research.

“As many as 5 million Americans face the challenge of Alzheimer’s disease, which robs them of their memories, their independence, and ultimately, their lives,” Dr Collins said. “We are determined, even in a time of constrained fiscal resources, to capitalise on exciting scientific opportunities to advance understanding of Alzheimer’s biology and find effective therapies as quickly as possible.”

The clinical trials investigate possible ways to stop the progression of the disease. The translational research study awards are focused on identifying, characterszing and validating novel therapeutic targets.

“We know that Alzheimer’s-related brain changes take place years, even decades, before symptoms appear. That really may be the optimal window for drugs that delay progression or prevent the disease altogether,” said NIA Director Dr Richard Hodes. “The clinical trials getting under way with these funds will test treatments in symptom-free volunteers at risk for the disease, or those in the very earliest stages — where we hope we can make the biggest difference.”

"Basic and genetic studies of the disease — from the abnormal proteins involved, to genetic influences, to inflammation and other Alzheimer’s-related brain changes — have advanced our knowledge. This has given us new insights into the biological underpinnings of this extremely complex disorder," Dr Hodes said.

These awards support the following clinical trials. (Individual investigators can be contacted about when these studies will recruit participants.):

• The Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) Trial — Dr Randall Bateman, Washington University, St. Louis, and co-investigators. $1.5 million in fiscal 2013, with the potential for $6 million over 4 years.

• The Alzheimer's Prevention Initiative APOE4 Trial — Drs Eric Reiman and Pierre Tariot, Banner Alzheimer’s Institute, Phoenix, and co-investigators. Fully funded in fiscal 2013 at $33.2 million.

• Allopregnanolone Regenerative Therapeutic for MCI/Alzheimer’s: Dose Finding Phase 1 — Drs Roberta Brinton and Lon Schneider, University of Southern California, Los Angeles. Fully funded in fiscal 2013 at $2.4 million.

• Pathway Discovery, Validation and Compound Identification for Alzheimer’s Disease — Drs Philip De Jager, of the Brigham and Women's Hospital, Broad Institute, Harvard University, Boston, and David Bennett, of Rush University Medical Center, Chicago. $1.7 million in fiscal 2013, with the potential of $7.9 million over 5 years.

• Integrative Biology Approach to Complexity of Alzheimer’s Disease — Dr Eric Schadt of Icahn School of Medicine at Mount Sinai, New York City, and a team of investigators. $1.6 million in fiscal 2013, with the potential of $8.2 million over 5 years.

• A Systems Approach to Targeting Innate Immunity in Alzheimer’s — Dr Todd Golde, University of Florida, Gainesville, and colleagues. $1.6 million in fiscal 2013, with the potential of $7.7 million over 5 years.

One additional award made possible by this new funding is pending.