ARTICLE IN BRIEF
In June, the Department of Human Health Services provided an update on federal initiatives to promote the prevention and treatment of Alzheimer's disease — a mandate of the National Alzheimer's Project Act.
Two and a half years after the passage of the National Alzheimer's Project Act (NAPA), the federal government remains committed — at least on paper — to promoting a national agenda around the prevention and treatment of the disease.
In June, the Department of Health and Human Services (HHS) published an update on the National Plan to Address Alzheimer's Disease, an initiative to: identify prevention and treatment strategies for Alzheimer's disease (AD) by 2025; improve AD patient care; enhance support networks for individuals with AD and their families; increase public awareness; and carefully track data for these efforts.
In a telephone interview with Neurology Today, Richard J. Hodes, MD, director of the National Institute on Aging, the NIH agency spearheading the research goals of the initiative, highlighted areas where progress had been made over the past year, and outlined future priorities. Challenges remain, he acknowledged.
“In the case of Alzheimer's disease, we have a clinical public health imperative that all of us understand, and the restrictions in resources are something that all of us feel very strongly,” he said.
A YEAR IN REVIEW
Since 2012, training grants by the Health Resources and Services Administration have also been issued to more than 10,000 health care providers on topics from dementia diagnosis to effective behavior management for individuals with dementia and their caregivers, to ensure an expanded and better informed AD workforce. Additionally, the HHS launched its own Alzheimer's website (http://www.alzheimers.gov), offering resources to individuals with AD and their caregivers and increasing public awareness of the disease.
In fiscal 2012, $50 million in additional funding was allotted for some specific Alzheimer's initiatives, Dr. Hodes reported.
- Whole-exome and whole-genome sequencing to explore additional risk factors and protective factors for Alzheimer's disease: “The samples are now being sequenced, so soon we will have a dramatically increased set of data to allow genetic risk factor and protective factor identification.” (More information on this project can be found here: http://1.usa.gov/12dBLNBl.)
- Intranasal insulin for treating people with mild cognitive impairment or mild AD: “This was based on a pilot study that was successful in showing positive outcomes in a small group in the short-term using this intervention, which delivers insulin directly into the brain without reducing blood sugar systemically.” He added that there has been previous research suggesting that individuals with AD may have abnormal “glucose metabolism and insulin resistance in the brain, which could contribute to the poor viability and survival of certain cells in the brain.” A full-scale clinical trial is now in progress (clinicaltrials.gov/ct2/show/NCT01767909).
- Clinical trial for AD Prevention: conducted in Colombia, South America in a group of extended families with early-onset familial AD, to look at whether drug therapy has an effect in preventing the advance of disease as indicated by abnormalities on brain imaging and clinical assessments. This clinical trial has begun: http://1.usa.gov/12gG3aD.
- Utilizing pluripotent stem cells that can be differentiated into neuronal cells to help to recognize differences in pathological mechanisms between normal- and Alzheimer's-affected individuals and possibly provide a way to screen for new interventions. This research is under way: http://1.usa.gov/1bj0FSS.
One of the major accomplishments in research was bringing together international AD experts for the Alzheimer's Disease Research Summit 2012: Path to Treatment and Prevention (http://1.usa.gov/1506EX>), Dr. Hodes said, which developed the recommendations on how best to advance AD research that have helped shape the 2013 priorities.
Based on the Alzheimer's Disease Research Summit and a series of meetings with institute directors at the NIH, a new set of specific initiatives for AD was established, he added. The RFAs were posted, and applications are currently being reviewed; funding will be announced later in the summer. The new initiatives are as follows:
- An interdisciplinary and systems biology approach to better identify and validate new targets for Alzheimer's disease with an emphasis on novel targets that have not been extensively studied. “This will combine techniques like genomics, identification of gene expression, proteomics, and systems biology, to see if we can unravel the genetically and epigenetically determined abnormal functions in the cells and brains of individuals with Alzheimer's. We've received a good number of very exciting applications.”
- Full-scale, definitive Alzheimer's prevention trials. “There's currently a great deal of support for the notion that the best prospects for successfully intervening in Alzheimer's disease are to intervene early before there's been irreversible damage.”
- Early safety trials of new interventions that have a preclinical rationale or promise, but have not yet been tested in humans.
- Promote development of potential interventions through preclinical stages, “so researchers with candidate therapeutic compounds for novel targets who need to show efficacy in appropriate animal models and who need to establish the optimization of compounds for efficacy and delivery to the brain can be supported through this initiative at various stages of development.”
OPPORTUNITIES, CHALLENGES AHEAD
“As we're looking to translate better understanding of basic neuroscience into clinical interventions, neurologists will be very critical to these new efforts,” Dr. Hodes told Neurology Today. Additionally, neurologists have a role to play in educating the public, he said. “I think that neurologists who have direct interactions with patients and who have a clinical voice — one that is highly valued by policymakers — can play a pivotal role here.”
The number one research challenge, he added, in the national fight against AD is “paying attention to the best science and deciding which avenues hold the most promise — not just the obvious or favored theories, but also investigating a wide variety of alternatives. Until we have success, we don't know the right direction, so we have to keep our intellectual perspective very open.”
Obtaining adequate resources is a significant hindrance, he acknowledged. “We have scientific opportunities that we've never had before and we remain committed to doing all that we can with the resources available. But resources are limiting, and the change in NIH, NIA, and, therefore, Alzheimer's budgets in the past year have added to that challenge.
“We planned the series of announcements of RFAs this year based on the proposal that there would be actually an 80 million dollar increase in funding available for AD in fiscal 2013 — those funds did not develop, but NIH director Francis Collins did commit 40 million dollars from his discretionary funds to Alzheimer's research, so part of the funding is now accounted for,” Dr. Hodes explained. The announcement of additional research supported by these funds will be made at the end of the summer. The President's fiscal 2014 budget proposal calls for an additional $100 million for AD, with $80 million for research and the remainder for care and services.
Dr. Hodes remains optimistic: “We will continue to do our best to encourage and fund new research under these circumstances.”
Even with the challenges, experts said, the creation of the NAPA is a move in the right direction. Before the creation of NAPA and the National Alzheimer's Plan, said David J. Gill, MD, Alzheimer's really was not a priority in the US. “Now, people are starting to realize the huge crisis we're going to be in in the next 20 to 40 years as the number of people with AD at least doubles — if not increases by 4 to 5 times — by 2030; that's really going to dominate US medical care,” added Dr. Gill, a cognitive and behavioral neurologist at Unity Rehabilitation & Neurology in Rochester, NY, and a member of the Neurology Today editorial advisory board.
Dr. Gill also praised the expanded goals of the Update, but questioned the ability to fund these promising new priorities. Especially difficult to finance, he said, will be the research into lifestyle modifications for AD prevention because pharmaceutical companies would not have a stake in such studies.
NATIONAL ALZHEIMER'S UPDATE: 2013 ACTION ITEMS
In the 2013 update, the Department of Health and Human Services (HHS) outlined new action items that would involve the entire national Alzheimer's community:
- Develop an Alzheimer's disease training curriculum for primary care providers to deliver high-quality dementia care.
- Investigate preventable hospitalization and emergency department use among individuals with Alzheimer's disease and the best interventions for reducing them.
- Improve detection of elder abuse and neglect through aging networks and program providers.
- Award demonstration grants to help promote legal services groups that assist families and communities impacted by Alzheimer's.
- Develop an expanded Dementia Capability Toolkit for state and local health networks to improve dementia services in their communities.