A draft plan from an expert panel convened by the U.S. Department of Health and Human Services sets an audacious goal of having a means to effectively treat or prevent Alzheimer’s disease by 2025.
The development of such a plan is mandated by the National Alzheimer’s Project Act, which was signed into law at the start of 2011. Though the act called for the creation of a coordinated national plan to address the rising toll of Alzheimer’s disease and an evaluation of national research efforts, the law did not provide any new funding for such research.
What makes the goal so audacious is not only the long development times needed to bring drugs to market, but the lack of understanding about the underlying causes of the neurodegenerative disease, which afflicts as many as 5.4 million Americans. Efforts to bring a drug to market have been stymied by spectacular late-stage failures including the failure of two high profile late-stage drug candidates during 2010.
Eli Lilly quit developing semagacestat when it found the drug not only failed to slow the disease’s progression, but also worsened the cognitive function of trial patients and heightened their risk of skin cancer. Pfizer and Medivation watched Dimebon prove no better than a placebo, much to their surprise after the drug showed promise in early testing. The partners said on January 18 they would cease development of Dimebon after it failed to meet two primary endpoints in a late-stage trial.
The issue is urgent. The Alzheimer’s Association estimates the economic toll from the disease will rise to more than $1 trillion by 2050 from $183 billion today. It is already the sixth leading cause of death and the only one for which there is no way to cure, prevent, or slow its progression, the group said.
“This process is about changing the course of Alzheimer's disease,” say Harry Johns, president and CEO of the Alzheimer's Association and member of the Advisory Council that produced the draft report. “It is about setting the path for that change right away with an aggressive timeline. Developing an urgent, achievable and accountable strategy for Alzheimer’s is about hope for millions of people today and tomorrow. What we need now is a meaningful plan with appropriate resources that, when fully implemented, will bring us from possibility to reality.”
The draft plan calls for the setting of research priorities, enhanced research, coordination of efforts with international public and private entities to leverage resources, and facilitating the translation of discoveries into treatments.
The draft plan also calls for enhancement of early diagnostics, expanded support for caregivers, better care and education for patients; expanded support for caregivers, and improvements to early diagnosis.
Some have criticized the draft plan for creating a deadline and setting up a perception of failure should the goals of the plan go unmet. But disease advocates, long critical of the relatively small investment being made in Alzheimer’s research, see this as an important step forward. The National Institutes of Health in fiscal 2011 was expected to spend just $480 million in Alzheimer’s research. That compares to $2.1 billion for heart disease research and $6 billion for cancer research.
“There’s no doubt that we have no time to waste, but it will be vital to engage Congress and sustain their commitment in order to achieve a national plan to defeat Alzheimer’s disease and help countless families now and in the future,” says Eric Hall, president and CEO of the Alzheimer’s Foundation of America and a member of the Advisory Council. “Much work lies ahead, but I am confident that we are on the right path toward the resolution of this crisis.”
January 20, 2012
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