By Meredith Wadman of Nature magazine
In December 2010, the US Congress passed the National Alzheimer's Project Act. The law instructs the US government to develop its first-ever strategic plan for battling Alzheimer's disease, the dementia-inducing brain disorder that is expected to afflict at least 11 million US citizens by 2050.
Earlier this month, an advisory committee to Kathleen Sebelius, US Secretary of Health and Human Services (HHS), issued a draft framework for the strategic plan. Its research goals are ambitious: slow the progression, delay the onset and ultimately prevent the now-incurable disease.
After the committee's quarterly meeting yesterday, Ronald Petersen, chairman of the Advisory Council on Alzheimer's Research, Care and Services, tells Nature how his panel is helping to shape the fight against Alzheimer's.
What is the advisory council and what is its task?
The advisory committee was mandated by the National Alzheimer's Project Act to advise the [HHS] secretary on the development of a national plan for Alzheimer's disease.
The committee has around 25 members. Roughly half are from federal agencies that are involved with Alzheimer's disease. And half are non-federal members. They include caregivers, care providers, state-agency employees, voluntary health organizations, patient advocates, and a couple of researchers: Jennifer Manly from Columbia University Medical Center in New York, and myself.
When is your final plan expected to be published?
The law requires that we generate a plan and revise it annually. The first iteration of the plan, for this year, will probably be out in early May. We will revisit it again at each meeting; we are required to meet four times each year. All along the way it's going to be informed by experts in the area as well as the general public.
What will the plan mean for researchers?
It will set an ambitious and a bold plan for attacking this disease on the research front over the next eight to ten years. We are going to advocate for -- at the very least -- increased funding for research. Of course it couldn't be at a worse time, with the federal government talking about reduced budgets and austerity. Regardless of that, we feel that the public-health crisis is here now.
Will the National Alzheimer's Project Act actually deliver any more money for research?
No. There is no appropriation with the act itself or with this committee. Now, this committee may very well advise the secretary of HHS -- this is a very preliminary number -- that there should be an annual federal research budget for Alzheimer's of about US $2 billion. Right now it's less than half a billion dollars a year.
If that is in fact a recommendation, then the secretary will have to respond. It doesn't mean that she's going to give you $2 billion tomorrow. But what it does provide is a platform for advocacy groups then to go to Congress and say: `Look, the National Alzheimer's Project Act, which you passed, required a national advisory board to be constituted. This is their recommendation. Now, what are you going to do about it?'
What research goal will the plan address?
The research goal, as we are putting it out there today, is to develop an effective treatment strategy, delay the onset and slow the progression of the disease. Part of the discussion this morning was: what's the time frame for that? We're leaning towards a 2020 goal as opposed to, say, 2025. The shorter time frame adds a sense of urgency.
On the other hand, we have to balance that with scientific credibility. Are we going to cure the disease by 2020? Of course not. But can we make some impact? Can we define better molecular targets? Can we compress the process for drug development, evaluation and approval? Things like that would be realistic targets.
We are also looking broadly at what it would take to really meet our goal. For instance, can we do something to increase the incentives for public-private partnerships like the Alzheimer's Disease Neuroimaging Initiative?
What research areas do you feel are most in need of attention?
The validation of biomarkers. If we are going to develop protective therapies for Alzheimer's, we have to aim to do it earlier in the disease process. By the time a person develops dementia, the damage done to the central nervous system might be irreversible.
We are learning that the biological basis for the dementia is laid down fifteen years before the appearance of clinical symptoms. How do you know who's at risk? Is it family history? Genetic predisposition? Are there biomarkers in the blood? In the spinal fluid? We need studies that will tell us which people are at risk for developing the clinical symptoms later on.
What research areas present the greatest opportunities?
We need to broaden our potential therapeutic targets. A lot of the field is focused on amyloid. Amyloid is definitely a player in the disease. Yet if you look at people who develop the clinical syndrome of dementia, especially later in life, yes, they have amyloid in the brain but they also have other pathologic entities -- vascular disease; synucleinopathies [insoluble fibrils of the normally soluble protein, alpha-synuclein]; a tauopathy [which is marked by disease-inducing, insoluble tangles of another protein, tau]. The most common pathology in someone who develops clinical dementia in their late 70s or 80s is the presence of multiple pathologies.
Many diseases afflict large numbers of Americans. Why does Alzheimer's need a special panel and strategic plan?
The first reason is demographics. The estimate is that there are 5.4 million people in this country with Alzheimer's disease. And that's going to triple by the middle of the century. The costs associated with that will be enormous. And there are no treatments. There are no survivors. And the leading risk factor is age. We've got that baby-boomer population that is reaching that age. So it's a public-health crisis. It couldn't be at a worse economic time. But diseases don't choose the right economic times to manifest themselves. We've got to deal with that.