More than a half-century after John F. Kennedy committed the U.S. to landing a man on the moon in less than a decade, Washington has set another, far more ambitious deadline for itself: prevent and effectively treat Alzheimer disease (AD) by 2025.
That goal is one of five “building blocks” of the new National Plan to Address Alzheimer’s Disease. The others are: optimize care quality and efficiency, expand support for people with AD and their families, enhance public awareness and engagement, and track progress and drive improvement. The 69-page report by the U.S. Department of Health and Human Services (HHS) calls itself “the initial blueprint for achieving the vision of a Nation free of AD.”
“HHS will prioritize and accelerate the pace of scientific research and ensure that as evidence-based solutions are identified, they are quickly translated, put into practice, and brought to scale so that individuals with Alzheimer’s disease can benefit from increases in scientific knowledge,” the National Plan declared. “HHS will identify interim milestones and set ambitious deadlines for achieving these milestones in order to meet this goal.”
To date HHS has taken two steps toward fulfilling the plan: creating an implementation-monitoring tool and publishing a Request for Information inviting comment from public agencies and private organizations on their interest in contributing to the development of an International Alzheimer’s Disease Research Portfolio. HHS has said it will compile portfolio information and make it accessible through a searchable online database.
Drug Development Hurdles
Some 5.4 million Americans among about 36 million people worldwide have AD. These numbers are expected to balloon to 15 million Americans among 115.4 million people worldwide by 2050.
It’s fair to say the disease and its related illnesses constitute one of the last untapped markets for new blockbuster drugs. The size of the market has been estimated at $5.4 billion in 2010, set to nearly triple to $14.3 billion by 2020, according to Decision Resources.
Yet only five medicines are approved by FDA for the disease: Cognex, Aricept, Exelon, Razadyne, and Namenda. None are a cure; all treat only symptoms, and none for more than a few months. Several potential new medicines have been grounded in recent years by failed clinical trials. In January, Pfizer and Medivation ended a co-development and marketing collaboration on Dimebon (latrepirdine) after a Phase III trial did not meet either of its co-primary endpoints of improving cognition or self-care and daily function. Pfizer had paid $225 million up front and agreed to pay $500 million tied to milestones.
In 2010, Eli Lilly ended development of the peptide semagacestat after a Phase III trial showed that the gamma secretase inhibitor failed to improve disease progression. Additionally, the treatment was associated with worsening of clinical measures of cognition and the ability to perform activities of daily living. Similar Phase III failures ended development of Myriad Pharmaceuticals’ nonsteroidal anti-inflammatory drug tarenflurbil earlier that year and of Neurochem’s amyloid B antagonist tramiprosate in 2007.
“Perhaps the disease is being treated too late, when damage is irreparable?” The Lancet posited in an editorial. “The best time to treat Alzheimer’s disease is likely to be before memory loss and tissue destruction occurs, but this is hard to model in animals. That means identifying people at risk of developing the disease, perhaps because of a genetic predisposition or by measuring biomarkers.”
A key factor in the dearth of treatments for AD has been lagging federal basic research funding compared with diseases whose stakeholders have shown more activism and Washington savvy. During FY 2012, the agency expects to spend $5.451 billion on cancer research and another $3.075 billion on HIV/AIDS. That’s more than $8.5 billion combined.
By contrast NIH spent $448 million in FY 2011 on Alzheimer disease research and expects to spend about the same this year and $449 million in FY ’13. President Barack Obama has asked NIH to squeeze out another $50 million from its current budget and $80 million next year. Of the total for FY 2012, $25 million will come from ongoing research on whole genome and whole exome sequencing at the National Human Genome Research Institute. The $80 million for next year would come from the Administration’s FY 2013 budget for the Prevention and Public Health Fund, which was created by the Affordable Care Act to focus on pressing public health needs. Next year’s budget proposal also includes another $26 million for other agencies for caregiver support, education, and public awareness.
Lack of funding isn’t the only hurdle for AD research. So too is the difficulty of getting patients and their families to participate in clinical trials. The National Plan calls for increasing enrollment in trials “through community, national, and international outreach.”
A Plan Needed for the Plan
“For a long time we just naturally thought there would be a silver bullet somewhere that would take care of this disease,” Eric J. Hall, president and CEO of the Alzheimer’s Foundation of America and member of the advisory council advising on the National Plan, told GEN. “For the last decade that has not been the case.”
“The Plan by itself really sends out a message: This is no longer an orphan disease. This is center stage in the government’s purview, where resources are being brought to bear,” Hall added. “The Plan itself, I think, sends hope to families that they don’t suffer in silence or isolation. There’s a lot of hope in that families know that someone actually cares about what it is they’re struggling through.”
Other than creating patient registries and helping states build skills in serving people with AD, the Plan leaves many needed action steps to future meetings among “representatives from across the federal government, state and local governments, academic medical research institutions, and the private sector.” Meetings for the trials enrollment action plan are envisioned for June, with the to-do list set to emerge in August and be implemented by December.
Stakeholders deserve more specifics rather than the promise of another plan within a Plan. Some details exist, however, on who will conduct the additional clinical research. Public-private partnerships will carry out “voluntary disclosure of new and ongoing clinical trials” and coordinate “annual review of the status and progress of the trials and emerging opportunities” by January 2013.
In addressing multiple constituencies, the National Plan was bound to disappoint in spots, since many of its action steps will require months of coordination before anything happens. Yet the Plan remains valuable both for its vision of stopping AD and related dementias and especially for its spotlight on the need for more research, patient participation, and especially funding if the disease is to be conquered. Money will be both the biggest need and the hardest to secure since both political parties have committed to cutting spending.