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Alzheimer’s Association Update for July 2012
Volume 8 / Issue 4 / July 2012
Advocacy Forum breaks records This year’s Alzheimer’s Association Advocacy Forum, held April 23–25 in Washington, D.C., was the most successful in Association history, with more than 750 advocates registered and all 50 states represented. Advocates participated in an intense training program and on the last day of the Forum made their way to Capitol Hill to conduct over 440 congressional visits. Advocates marched on Capitol Hill to ask Congress to support the development of a strong National Alzheimer’s Plan that includes the necessary resources to change the trajectory of Alzheimer’s disease (AD) and to cosponsor the Health Outcomes, Planning, and Education (HOPE) for Alzheimer’s Act (H.R. 1386/ S. 738), which seeks to improve detection and diagnosis of AD and other dementias and provide access to information and support for newly diagnosed individuals and their families. Since the Advocacy Forum, 25 new cosponsors signed on to the bill, for a total of 142 House sponsors. During the Forum, Secretary of Health and Human Services Kathleen Sebelius acknowledged that AD “has never attracted sufficient attention or resources” from the federal government and committed to continue to address this. She also discussed the National Alzheimer’s Plan and the Obama Administration’s commitment to the plan’s goal of preventing and effectively treating AD by 2025. She promised attendees that “This isn’t just another strategy to be published and sit on a shelf.” Secretary Sebelius told advocates that “You’ve changed the face of Alzheimer’s. You’ve restored the dignity people with the disease thought they lost. You’ve made it possible for individuals and families to speak out and ask for help. In the process, you’ve made it impossible for political figures to ignore the disease.” The final version of the National Alzheimer’s Plan was released by the Administration in May. The Association is working through its Washington office, its advocates, and other avenues to ensure that the potential of the plan is realized through adequate federal funding. Immediately after her remarks, members of the Advisory Council that was convened as part of the National Alzheimer’s Project Act (NAPA) spoke about their participation in the development of the National Alzheimer’s Plan and what they hoped would be accomplished through their work. Panel members included geriatrician Laurel Coleman, MD; Jennifer Manley, PhD, who directs a program to provide training and tools to physicians to assist with screening, diagnosis, treatment, and care of patients with AD; and David Hoffman, MEd, director of the Bureau of Chronic Disease Prevention and Control, Long-Term
Care Restructuring, and Partnership in the New York State Department of Health. The discussion was facilitated by Alzheimer’s Association President and CEO, Harry Johns, who is also a member of the Advisory Council. New to the Forum program this year were the Alzheimer’s National Dinner and a reception hosted by the Association’s strategic partner, the Alzheimer’s Impact Movement (AIM, alzimpact.org). The reception was attended by more than 350 AIM members as well as many members of Congress. The National Dinner drew more than 900 individuals and was a star-studded event hosted by television personality Meredith Vieira. The Ronald and Nancy Reagan Research Award was presented to Colonel Karl E. Friedl, PhD, for his work to advance AD research at the Department of Defense. Colonel Friedl is the Director of the Telemedicine and Advanced Technology Research Center at the U.S. Army Medical Research and Materiel Command. The award was presented to him by Congressman Jim Moran (DVirginia), who was instrumental in creating this important new research program with the support of the Alzheimer’s Association. University of Tennessee Head Coach Emeritus Pat Summitt and her son, Tyler Summitt, received the Alzheimer’s Association Sargent and Eunice Shriver Profiles in Dignity Award. Former First Lady of California, Maria Shriver, whose father Sargent Shriver passed away from AD, presented the award, which recognizes an individual, organization, or company whose actions have promoted greater understanding of AD and its effects on diagnosed individuals, families, and caregivers. Summitt, the “winningest” coach in National Collegiate Athletic Association (NCAA) basketball history, publicly shared her diagnosis of younger-onset Alzheimer’s disease in August 2011 at age 59. She and Tyler created The Pat Summitt Foundation Fund to provide grants to nonprofit organizations that raise awareness of the disease, support families, and advance research. The Alzheimer’s Association Humanitarian Award was presented to Senator Debbie Stabenow (D-Michigan) and Representative Michael Burgess, MD (R-Texas). Senator Stabenow was recognized for her continuing efforts to help produce a strong National Alzheimer’s Plan and her leadership in introducing and pushing for passage of the HOPE Act in the Senate. Representative Burgess received the award in recognition of his critical efforts to help ensure passage of NAPA and his leadership as an original cosponsor of both the Alzheimer’s Breakthrough Act (H.R. 1897) and the HOPE Act in the House of Representatives.
Alzheimer’s Association / Alzheimer’s & Dementia 8 (2012) 376–377
In 2013, the Association will hold the 25th Advocacy Forum on April 22–24. For more information, visit www. alz.org/forum.
Research Roundtable looks beyond amyloid for Alzheimer’s disease targets The Alzheimer’s Association’s Research Roundtable, a consortium of scientists from the pharmaceutical, biotechnology, imaging, and cognitive testing industries, met with scientists from academia, the National Institutes of Health, the U.S. Food and Drug Administration, and the Association on April 16–17, 2012, in Washington D.C., to consider non-amyloid targets that may offer promising strategies for the treatment of AD and other dementias. The amyloid hypothesis—which posits that excess production and deposition of the protein beta-amyloid in the brain is the key initiating event in AD—has dominated thinking about the origins and potential treatment of AD for many years; however, failed clinical trials of amyloid-based drugs has kindled enthusiasm for non-amyloid–based targets. Chaired by Karl Herrup, PhD, of Rutgers University, and Samantha Budd, PhD, of AstraZeneca, the meeting explored the multiple complex factors that contribute to the development of AD, with the goal of identifying therapeutic targets and strategies for developing new treatments. “The challenge is how to move these ideas, which are growing at a prodigious rate, into something useful for people,” said Dr. Herrup. “Non-amyloid targets represent much more of a challenge from both the biological and industrial point of view.” Current thinking is that amyloid accumulates years before symptoms appear and that subsequent neurodegeneration involves numerous other mechanisms, many of which were explored at the meeting. Among these mechanisms are cytoskeletal and tau abnormalities; mechanisms associated with aging, such as autophagy, vesicular trafficking, and cell cycle disruption; neuroinflammation; mitochondrial dysfunction, disruption of calcium homeostasis, and oxidative stress; insulin resistance; and alterations in chaperone proteins. Moreover, the pathways involved in these pathogenic processes interact, increasing the difficulty of
377
teasing out the relative importance of each and the best therapeutic targets. One subject rising to the top of discussion was tau, a protein that has been implicated in AD pathogenesis for more than 30 years and is one of two hallmarks of AD. Recent studies have shown that the pathogenic form of tau is a misfolded protein and that tau aggregation spreads along important neural networks in the brain. Studies show a similar pattern of protein misfolding in other neurodegenerative diseases, such as Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis (ALS). This pattern of protein misfolding in neurodegenerative diseases may be linked to an increased susceptibility to the misfolding in large scale neural networks in the brain. Lurking in the background of all discussions about AD pathogenesis is the question of how best to develop and test new therapies—in particular, what is necessary to promote collaboration among academia, industry, and regulatory agencies. In this regard, there is little difference between amyloid and non-amyloid targets, said researchers—both require more informative phase 1 and phase 2 studies, better use of biomarkers when determining which individuals to include in clinical trials, and clinical data to support the use of biomarkers. Further complicating the discussion about clinical trials were the widely (although not universally) accepted ideas that treatments may need to be tested in subjects who do not yet have symptoms, and that many different therapies may be needed to effectively treat AD, especially the longer one waits to intervene after the brain changes of AD have begun. While consensus was not reached about the best targets to pursue, there was widespread agreement that more collaboration is essential to move drug development forward. Perhaps more important, Roundtable participants from industry and academia coalesced around the idea of increased data sharing. “Clearly the time is ripe and the need is great, but we can’t go it alone,” said Pfizer’s Rachel Schindler, MD, the incoming chair of the Research Roundtable. “In industry, it has clearly been recognized and manifested in the increasing number of alliance partnership you see in developing compounds and sharing risks.” A more detailed report of this meeting will appear in a future issue of Alzheimer’s & Dementia.
Advocacy Forum breaks records This year’s Alzheimer’s Association Advocacy Forum, held April 23–25 in Washington, D.C., was the most successful in Association history, with more than 750 advocates registered and all 50 states represented. Advocates participated in an intense training program and on the last day of the Forum made their way to Capitol Hill to conduct over 440 congressional visits. Advocates marched on Capitol Hill to ask Congress to support the development of a strong National Alzheimer’s Plan that includes the necessary resources to change the trajectory of Alzheimer’s disease (AD) and to cosponsor the Health Outcomes, Planning, and Education (HOPE) for Alzheimer’s Act (H.R. 1386/ S. 738), which seeks to improve detection and diagnosis of AD and other dementias and provide access to information and support for newly diagnosed individuals and their families. Since the Advocacy Forum, 25 new cosponsors signed on to the bill, for a total of 142 House sponsors. During the Forum, Secretary of Health and Human Services Kathleen Sebelius acknowledged that AD “has never attracted sufficient attention or resources” from the federal government and committed to continue to address this. She also discussed the National Alzheimer’s Plan and the Obama Administration’s commitment to the plan’s goal of preventing and effectively treating AD by 2025. She promised attendees that “This isn’t just another strategy to be published and sit on a shelf.” Secretary Sebelius told advocates that “You’ve changed the face of Alzheimer’s. You’ve restored the dignity people with the disease thought they lost. You’ve made it possible for individuals and families to speak out and ask for help. In the process, you’ve made it impossible for political figures to ignore the disease.” The final version of the National Alzheimer’s Plan was released by the Administration in May. The Association is working through its Washington office, its advocates, and other avenues to ensure that the potential of the plan is realized through adequate federal funding. Immediately after her remarks, members of the Advisory Council that was convened as part of the National Alzheimer’s Project Act (NAPA) spoke about their participation in the development of the National Alzheimer’s Plan and what they hoped would be accomplished through their work. Panel members included geriatrician Laurel Coleman, MD; Jennifer Manley, PhD, who directs a program to provide training and tools to physicians to assist with screening, diagnosis, treatment, and care of patients with AD; and David Hoffman, MEd, director of the Bureau of Chronic Disease Prevention and Control, Long-Term
Care Restructuring, and Partnership in the New York State Department of Health. The discussion was facilitated by Alzheimer’s Association President and CEO, Harry Johns, who is also a member of the Advisory Council. New to the Forum program this year were the Alzheimer’s National Dinner and a reception hosted by the Association’s strategic partner, the Alzheimer’s Impact Movement (AIM, alzimpact.org). The reception was attended by more than 350 AIM members as well as many members of Congress. The National Dinner drew more than 900 individuals and was a star-studded event hosted by television personality Meredith Vieira. The Ronald and Nancy Reagan Research Award was presented to Colonel Karl E. Friedl, PhD, for his work to advance AD research at the Department of Defense. Colonel Friedl is the Director of the Telemedicine and Advanced Technology Research Center at the U.S. Army Medical Research and Materiel Command. The award was presented to him by Congressman Jim Moran (DVirginia), who was instrumental in creating this important new research program with the support of the Alzheimer’s Association. University of Tennessee Head Coach Emeritus Pat Summitt and her son, Tyler Summitt, received the Alzheimer’s Association Sargent and Eunice Shriver Profiles in Dignity Award. Former First Lady of California, Maria Shriver, whose father Sargent Shriver passed away from AD, presented the award, which recognizes an individual, organization, or company whose actions have promoted greater understanding of AD and its effects on diagnosed individuals, families, and caregivers. Summitt, the “winningest” coach in National Collegiate Athletic Association (NCAA) basketball history, publicly shared her diagnosis of younger-onset Alzheimer’s disease in August 2011 at age 59. She and Tyler created The Pat Summitt Foundation Fund to provide grants to nonprofit organizations that raise awareness of the disease, support families, and advance research. The Alzheimer’s Association Humanitarian Award was presented to Senator Debbie Stabenow (D-Michigan) and Representative Michael Burgess, MD (R-Texas). Senator Stabenow was recognized for her continuing efforts to help produce a strong National Alzheimer’s Plan and her leadership in introducing and pushing for passage of the HOPE Act in the Senate. Representative Burgess received the award in recognition of his critical efforts to help ensure passage of NAPA and his leadership as an original cosponsor of both the Alzheimer’s Breakthrough Act (H.R. 1897) and the HOPE Act in the House of Representatives.
Alzheimer’s Association / Alzheimer’s & Dementia 8 (2012) 376–377
In 2013, the Association will hold the 25th Advocacy Forum on April 22–24. For more information, visit www. alz.org/forum.
Research Roundtable looks beyond amyloid for Alzheimer’s disease targets The Alzheimer’s Association’s Research Roundtable, a consortium of scientists from the pharmaceutical, biotechnology, imaging, and cognitive testing industries, met with scientists from academia, the National Institutes of Health, the U.S. Food and Drug Administration, and the Association on April 16–17, 2012, in Washington D.C., to consider non-amyloid targets that may offer promising strategies for the treatment of AD and other dementias. The amyloid hypothesis—which posits that excess production and deposition of the protein beta-amyloid in the brain is the key initiating event in AD—has dominated thinking about the origins and potential treatment of AD for many years; however, failed clinical trials of amyloid-based drugs has kindled enthusiasm for non-amyloid–based targets. Chaired by Karl Herrup, PhD, of Rutgers University, and Samantha Budd, PhD, of AstraZeneca, the meeting explored the multiple complex factors that contribute to the development of AD, with the goal of identifying therapeutic targets and strategies for developing new treatments. “The challenge is how to move these ideas, which are growing at a prodigious rate, into something useful for people,” said Dr. Herrup. “Non-amyloid targets represent much more of a challenge from both the biological and industrial point of view.” Current thinking is that amyloid accumulates years before symptoms appear and that subsequent neurodegeneration involves numerous other mechanisms, many of which were explored at the meeting. Among these mechanisms are cytoskeletal and tau abnormalities; mechanisms associated with aging, such as autophagy, vesicular trafficking, and cell cycle disruption; neuroinflammation; mitochondrial dysfunction, disruption of calcium homeostasis, and oxidative stress; insulin resistance; and alterations in chaperone proteins. Moreover, the pathways involved in these pathogenic processes interact, increasing the difficulty of
377
teasing out the relative importance of each and the best therapeutic targets. One subject rising to the top of discussion was tau, a protein that has been implicated in AD pathogenesis for more than 30 years and is one of two hallmarks of AD. Recent studies have shown that the pathogenic form of tau is a misfolded protein and that tau aggregation spreads along important neural networks in the brain. Studies show a similar pattern of protein misfolding in other neurodegenerative diseases, such as Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis (ALS). This pattern of protein misfolding in neurodegenerative diseases may be linked to an increased susceptibility to the misfolding in large scale neural networks in the brain. Lurking in the background of all discussions about AD pathogenesis is the question of how best to develop and test new therapies—in particular, what is necessary to promote collaboration among academia, industry, and regulatory agencies. In this regard, there is little difference between amyloid and non-amyloid targets, said researchers—both require more informative phase 1 and phase 2 studies, better use of biomarkers when determining which individuals to include in clinical trials, and clinical data to support the use of biomarkers. Further complicating the discussion about clinical trials were the widely (although not universally) accepted ideas that treatments may need to be tested in subjects who do not yet have symptoms, and that many different therapies may be needed to effectively treat AD, especially the longer one waits to intervene after the brain changes of AD have begun. While consensus was not reached about the best targets to pursue, there was widespread agreement that more collaboration is essential to move drug development forward. Perhaps more important, Roundtable participants from industry and academia coalesced around the idea of increased data sharing. “Clearly the time is ripe and the need is great, but we can’t go it alone,” said Pfizer’s Rachel Schindler, MD, the incoming chair of the Research Roundtable. “In industry, it has clearly been recognized and manifested in the increasing number of alliance partnership you see in developing compounds and sharing risks.” A more detailed report of this meeting will appear in a future issue of Alzheimer’s & Dementia.