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Driving Genes to Therapies™

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Driving Genes to Therapies™ QUARTERLY REPORT: 4TH QUARTER 2014 Q4 2014 INSIDE THIS REPORT ‘Still Alice,’ Still in Search of a Cure 2 Mini Film Festival Raises Driving Genes Funds to Fight Alzheimer’s to Therapies™ 3 Financial and Research Building on its enormously successful “Whole Genome Sequencing” Project, which Update identified nearly 1,000 new genetic mutations in more than 50 different genes, Cure 4 Alzheimer’s Fund has announced a new, even more ambitious multiyear, $50 million plus program titled “Genes to Therapies” (G2T). Simply put, the new project’s goal Cure Alzheimer’s Fund is to use the most promising recent genetic discoveries to develop drugs that would Heroes stop the disease at three separate stages: 5 ••For healthy people who don’t yet have Alzheimer’s: to stop Alzheimer’s before Global Family Reunion it starts by inhibiting the production of the Abeta protein and/or clearing it from the brain after it forms. 6 ••For those exhibiting symptoms of Alzheimer’s: to stop the process early on by Sherry Sharp Joins inhibiting the formation of tau tangles and protecting neurons from undue stress. Cure Alzheimer’s Fund Board ••For those whose disease has progressed significantly: to reduce the Abeta and 6 tau-provoked inflammation, slowing down or even stopping the disease process. “This is indeed a major challenge,” said Research Consortium Chair Dr. Rudy Tanzi, “but I believe we now have the data, the technology and the funding to take it on.” The first step, he explained, will be to prioritize approximately 15 genes that fit several key criteria: ••they must have a clear impact on Alzheimer’s pathology; Don’t Miss Our ••they must be “druggable”—connected to known biological systems, producing proteins similar to those already targeted by other drugs; and Upcoming Webinar ••they must impact one of the clear intervention points—Abeta/plaque Feb. 6 at 1 p.m. EST production, tangle formation or neuroinflammation. G2T will be aided significantly by the recent “Alzheimer’s-in-a-dish” From Genes breakthrough by Tanzi and Dr. Doo Kim at Massachusetts General Hospital. Using stem cells, Dr. Kim was—for the first time—able to to Therapies create both the amyloid plaque and neurofibrillary tangle pathology Tune in live to the site of the Cure of AD in a Petri dish. This technology will enable much cheaper and Alzheimer’s Fund Research Consortium’s faster drug testing. annual meeting to hear the latest progress from our Genes to Therapies Steering “Having learned much about the pathway that leads to disease, Committee. The Alzstream™ webinar will together with the genes responsible,” said Tanzi, “we now can proceed be moderated by David Shenk, author to look for chemical compounds that correct the defects. In other words, of “The Forgetting,” and will feature each we can try to fix what is broken.” of the members of the G2T Steering continued on page 2 » Committee. No sign-up necessary. Log on at http://curealz.org/webinar. n curealz.org continued from page 1 » Genes to Therapies (G2T) Steering Committee To ensure the success of the G2T program, Cure Alzheimer’s Fund is funding a unique research facilitation process—the “infrastructure” for the project. This involves recruiting researchers with Genes to Therapies expertise in specific genes and providing them with the specialty mice and reagents they will need to do their Alzheimer’s investigations relative to their particular genes. Unlike most projects, which require six to nine months’ preparation time before any real research begins, our unique approach will save money and, most importantly, time, allowing our researchers to get started right away. Working as one, our G2T Steering Committee’s ultimate goal is to develop effective drug therapies at three different points in the pathological cascade of the disease to prevent, inhibit and cure Alzheimer’s. Here’s a quick look at each of the players. Rudy Tanzi, Ph.D., Sangram S. Sisodia, David Michael Holtzman, Chairman, Cure Ph.D., Cure Alzheimer’s M.D., Cure Alzheimer’s Alzheimer’s Fund Fund Research Fund Research Consortium Research Consortium Consortium The Andrew B. and Gretchen P. Jones Joseph P. and Rose F. Kennedy Thomas A. Reynolds Sr. Family Professor of Neurology and Head Professor of Neurology at Harvard Professor of Neurosciences, of the Department of Neurology, University and Director of the Genetics and Aging University of Chicago Director, Washington University, St. Louis Research Unit at Massachusetts General Center for Molecular Neurobiology Like Sisodia, Dr. Holtzman has a background in Hospital (MGH) A longtime collaborator of Tanzi’s, Sisodia is a biology and has helped to identify the most effective Tanzi was the pioneering scientist who discovered leading expert on the molecular and cell biology of interventions from specific gene functional variants in the linkage between specific genes and Alzheimer’s Alzheimer’s disease pathology; he has been at the Alzheimer’s disease. Dr. Holtzman is involved in clinical disease. Today Tanzi leads the Alzheimer’s Genome forefront of learning how the familial Alzheimer’s and research activities at the Washington University Project™, a groundbreaking initiative dedicated to disease (FAD) genes, including the amyloid Memory and Aging Project and the Alzheimer’s Disease finding all the genes associated with Alzheimer’s precursor protein and the presenilins, function Research Center. He has contributed greatly to our disease so we not only can repair the damage the normally and contribute to Alzheimer’s disease understanding of how anti-amyloid antibodies affect disease has caused, but prevent it from occurring. pathogenesis. His role on the committee is to help Alzheimer’s pathology and how Abeta is cleared from Tanzi’s role as chair of the G2T Steering Committee is identify other researchers, beyond the Research the brains of Alzheimer’s patients. As a member of the to coordinate the efforts of the researchers as well as Consortium, who are more familiar with designated steering committee, he will assist in identifying other to continue his innovative research on what causes genes. Sisodia was very instrumental in designing researchers with similar skills to investigate how the Alzheimer’s disease and how it develops. and implementing the unique “infrastructure” to designated Alzheimer’s genes affect the pathology, and facilitate the more rapid and cost-effective research bring his clinical experience to bear in setting priorities program for Genes to Therapies. for potential drug development. ‘Still Alice,’ Still in Search of a Cure The recently released highly acclaimed motion picture ‘Still Alice’ originated as a novel. The author, Lisa Genova of Cape Cod, Massachusetts, wrote the story of an Ivy League professor who is diagnosed with early-onset Alzheimer’s disease. As a neuroscientist who actually worked at Massachusetts General Hospital with Rudy Tanzi long before her book was published, Genova knows the complexity of the science behind the disease and the frustrations over the lack of cure. Now that “Still Alice” has become a bestseller, Genova has made a plea to her readers in a reprinting of her book to support Alzheimer’s research. To find out more, visit lisagenova.com/still-alice/. n “Still Alice” author Lisa Genova 2 Steven Wagner, Ph.D., Wilma Wasco, Ph.D. Cure Alzheimer’s Fund Associate Professor of Neurology, Research Consortium Genetics and Aging Research Unit, Principal Investigator, Department of Massachusetts General Institute for Neurosciences, School of Medicine, Neurodegenerative Disease, Harvard University of California, San Diego Medical School and Massachusetts General Hospital Wagner has an extensive background in biochemistry and drug discovery and has numerous Wasco was part of an international collaborative patents to his name. Along with Tanzi, he helped effort that identified the familial Alzheimer’s disease- “Thanks to our develop the first gamma-secretase inhibitor for associated presenilin 1 (PS1) and presenilin 2 Alzheimer’s disease, which later provided the (PS2) genes. Her research has evolved to focus on donors we’ve been platform for gamma-secretase modulators, which understanding the biological role certain AD-linked control amyloid production rather than stop it proteins play in the normal, aging and diseased able to embark on altogether. “If we administer this at the right time to brain. As research operations manager for the G2T ™ the right populations, it could help to slow down the project, Wasco will be responsible for facilitating the ambitious G2T disease process,” said Wagner. As a member of the communication among the various researchers, and project, which is steering committee, he will focus on genes that may managing and maintaining the core facilities and provide clues for early prevention. research materials, including specially engineered moving us closer laboratory mice. She also will develop and maintain Robert Vassar, Ph.D., a G2T publications resource and provide the to a cure.” operations management necessary for this complex Cure Alzheimer’s Fund —Tim Armour, president and CEO, and unique undertaking. Research Consortium Cure Alzheimer’s Fund Professor of Cell and Molecular Biology, Feinberg School of Tim Armour, President Medicine, Northwestern University and CEO, Cure Alzheimer’s Fund Vassar’s ongoing research focuses on the role of Abeta and the enzyme BACE1 in normal biological Armour brings an extensive processes and in disease mechanisms of relevance background in nonprofits and to Alzheimer’s disease. As a pioneer in Alzheimer’s fundraising to Cure Alzheimer’s research, Bob has made a number of discoveries Fund as well as an MBA from Harvard. Armour joined critical to progress in the field. As a member of Cure Alzheimer’s Fund 10 years ago to help lead the the Genes to Therapies Steering Committee, he charge in the quest for a cure. As the administrator will help ensure proper attention is paid in the G2T of the steering committee, Armour is responsible for research projects to the more basic aspects of how supporting the researchers, developing and securing Alzheimer’s-relevant genes are produced and how budgets, and being the liaison between the committee they affect the disease pathology.
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