USC, Harvard hospitals and Mayo Clinic to build national Alzheimers research system
New infrastructure could enable researchers to accelerate clinical trials and find new treatments for the disease, thanks to a grant from the National Institutes of Health
Three premier researchers from the Keck School of Medicine of USC, two Harvard-affiliated hospitals and Mayo Clinic have been awarded up to $70 million to build essential nationwide infrastructure that would remove a bottleneck in the development of techniques to treat Alzheimers disease.
The new infrastructure will implement more efficient methods to recruit participants for clinical trials. It will provide centralized services, enabling Alzheimers researchers to run innovative clinical trials, manage and analyze huge amounts of data and recruit participants from diverse backgrounds. The group will also share data, software, instruments and biologic samples such as blood, tissue and cerebrospinal fluid.
Combining brainpower to solve this intractable problem is necessary because everyone will be affected or will know someone affected by this disease in their lifetime, said Paul Aisen, one of the principal investigators of the National Institutes of Health grant and the director of the USC Alzheimers Therapeutic Research Institute (ATRI) in San Diego.
Alzheimers disease is the sixth leading cause of death in the United States.
A new therapy for Alzheimers disease has not been approved in the past 14 years, and none of the approved therapies actually change the course of the disease, Aisen said. Scientists have made great strides in understanding Alzheimers disease, and technological advances have placed us on the verge of a breakthrough. This collaboration will remove some of the barriers that have hamstrung researchers from timely completion of clinical trials in Alzheimers disease and other dementias.
Aisen from USC ATRI, Ronald Petersen from Mayo Clinic in Minnesota and Reisa Sperling from Brigham and Womens Hospital and Massachusetts General Hospital both Harvard-affiliated hospitals in Boston comprise the leadership team that will collaborate with others to create the Alzheimers Clinical Trial Consortium (ACTC).
The consortium is expected to receive nearly $70 million over five years, pending the availability of funds, to build an initial network of 35 Alzheimers disease trial sites at top universities across the nation. More sites may be added later, the NIH said.
When we announced the funding opportunity for a new publicly supported clinical trials network, we envisioned a next-generation consortium, where shared expertise could enhance the ideas and approaches of individual investigators proposing and conducting trials, said Laurie Ryan, chief of the Dementias of Aging Branch in the National Institute on Agings Division of Neuroscience, which leads NIH research on Alzheimers. I think we will have that now. I am particularly interested in how we can better engage diverse communities into research, so that trials can more effectively include and benefit everyone who is affected by Alzheimers.
Specific clinical trials would be funded separately. The consortium expects to have the capacity to manage five to seven trials over the five years of the award.
We must overhaul our current recruitment strategies for clinical trials, particularly to improve the diversity of our study participants and to reach people who do not yet have symptoms of Alzheimers disease for inclusion in future prevention trials, said Sperling, who leads the Center for Alzheimer Research and Treatment at Brigham and Womens Hospital and Harvard Medical School. The new ACTC presents a terrific opportunity to innovate in recruitment, cognitive assessments and neuroimaging for the next generation of Alzheimers trials.
Software, data and networks galore
Alzheimers disease is the sixth leading cause of death in the United States. About 5.5 million Americans are living with Alzheimers in the U.S. this year, according to the Alzheimers Association. Without successful treatments, that number is projected to rise to about 13.5 million by 2050.
As a research institution devoted to promoting health across the life span, USC researchers across a range of disciplines are examining the health, societal and political effects and implications of the disease.
The NIH allocated $1.3 billion to Alzheimers disease research in fiscal year 2017 $884 million more than in 2013. In fact, federal funds used to find a cure or treatment for this disease, which kills 1 in 3 seniors, have steadily increased over the years.
The goal of the consortium is to accelerate Alzheimers clinical trials toward finding effective interventions that will treat or prevent this devastating disease.
The consortium will help scientists intervene early, before memory loss occurs, when treatments are expected to be most effective. Early intervention requires screening thousands of volunteers to identify eligible participants a time-consuming task that can result in delayed recruitment or under-enrollment. To speed up the process, the consortium will develop new and more efficient methods for recruiting and retaining participants.
Some clinical trials fail simply because time runs out before researchers are able to finish signing collaboration agreements or recruit the requisite number of participants, Petersen said. The ACTC will do the hard work of recruiting potential participants. These trial-ready cohorts allow researchers to spend their time investigating hypotheses and gaining a better understanding of Alzheimers disease rather than dealing with tedious logistics.
Vanguards in their field
Scientists who lead Alzheimers clinical trials must assess participants cognitive state, analyze brain scans and manage data areas in which the three principal investigators excel.
Aisen has 25 years of experience designing Alzheimers disease clinical trials. He and USC ATRI are renowned for developing the gold standard for measuring study outcomes and for creating techniques to catalog massive amounts of data using biostatistics and advanced information systems. In a way, Aisen is like a traffic cop who helps direct vehicles so that drivers can avoid congestion and reach their destination without encountering roadblocks.
Sperling is a world leader in rethinking the Alzheimers problem. As the project director of global, multi-site prevention trials in Alzheimers disease, she has helped redefine the stages of Alzheimers disease, including those before symptoms arise. Her research team has developed cutting-edge cognitive and imaging tools for therapeutic trials at the preclinical stage of Alzheimers disease, before symptoms arise.
Petersen has made a name for himself in mild cognitive impairment research. He developed the first clinical trial for people suffering from this early dementia condition and has contributed a wealth of information about the stage of the disease when symptoms theoretically could be halted or reversed.
This nationwide collaboration will move us closer toward techniques to prevent Alzheimers disease, Aisen said. Perhaps one day well be able to do a blood draw to identify individuals in need of preventive measures to stave dementia and Alzheimers disease. Were not there yet, but weve had many promising studies that have provided bread crumbs on how to get there.