ALS THERAPY DEVELOPMENT INSTITUTE 10 YEAR ANNIVERSARY 1999 - 2009 ANNUAL REPORT 2009 Letter from the CEO The ALS Therapy Development Institute is unlike any other research center in the world. As you will see from the following pages, the Institute that you have helped to build has overcome many challenges throughout the years. It wasn’t a gift from the government or major corporations that formed ALS TDI; it was the accumulated generosity and sense of urgency shared by hundreds of families from all over the world that laid the foundation, fueling research year after year. This dedication has provided nearly $60 million to ALS TDI and created a center of excellence solely dedicated to responding to the ALS crisis. Just as we were driven and inspirited by ALS patients ten years ago, so are we today. The face of the Institute has changed dramatically over the years as well. New families have joined those whom have been here since the beginning. New technology and researchers have become part of the team, allowing us to do more with every dollar we receive. Each and every ALS patient, caregiver, family member, donor, friend, employee, collaborator, consultant, and partner plays an important role in the advancement of our mission. Every new step taken toward that goal is only possible because of those that came before, and we continue to honor past contributions by never wavering in our resolve. In order to begin to tell the story of ALS TDI we must recognize the families that have funded our research, and as such this annual report starts by first listing the Family Funds that have made our work possible. The generosity of the ALS community and our partners at the MDA and the Department of Defense, along with the dozens of academic and industry collaborations that we work with have significantly shortened the length of time and cost of drug development for ALS. This annual report contains a brief history of ALS TDI over the past 10 years. The core values that drive us today have not changed from what they were ten years ago: courage and love, challenge and respect, hope, an adventurous spirit and patients today. We will always make decisions with those battling ALS today at the forefront of our minds. It is a privilege to report to you, that over the past year, we have made great strides, however more work is needed if we are to turn incremental advancements into treatments for patients today. I am daily reminded of the responsibility I shoulder as I look back at ALS TDI from the beginning. What this place means to those that have been part of its creation and growth, the loss that has been incurred, and the hope we must work daily to make real. As a scientist it is my duty to look at ALS in an unbiased way and ask the tough questions about ALS. As the head of ALS TDI it is my profound responsibility to ensure our progress, for all those that supported us and for those counting on us today. This is your lab and I thank you for your support. Sincerely, Steve Perrin, Ph.D. CEO & CHI E F SCIENTIFIC OFFI ce R Augie Nieto, CHAIRMAN O F TH E BO ARD BO ARD O F DIR ec T O R S Leslie Michelson, Vice Chair, Theodore Reich, Treasurer, James Allen Heywood, John Heywood, Spiros Jamas, Robert Rodin, Alexander Cappello, John Sutherland, Keith Melanson, Stan Appel ALS TherapY DEVELOPMENT INSTITUTE Pull out 10 YEAR ANNIVERSARY Will Hubben, pALS, First annual established the ALS/MND Sean Forrester Scott Belly Dance Research Mail List (later, after joins ALS TDF as FORUM An volunteer following James Allen Heywood starts Extravaganza ALS Therapy Development Institute his death, it is renamed in the ALS Therapy Development takes place his honor as the Hubben anonymous his mother’s Foundation three months at Karoun in The virtual, web-based bulletin Digest). Within its first year, donor makes diagnosis. ALS runs board, “The ALS Forum,” is in his family and after his brother Stephen is Newton, MA. the mailing list grows to over ALS TDF opens ALS TDF’s Founder and CEO, James Allen $1 million diagnosed with amyotrophic launched. grant to Sean himself has The Foundation applies for 1,000 email addresses. first lab inside New Heywood, begins to hold “ALS 101” sessions for lateral sclerosis (ALS, Lou ALS TDF. a 50/50 chance of Phase I Stem Cell Transplant “Neurobase” developed England Medical ALS patients. The goal is to provide information 1999 Gehrig’s disease). A nonprofit developing it. trial. If approved, it would to track leads and Center in Boston. and encourage discussion among stakeholders biotech, ALS TDF is dedicated to First therapy concept www.als-tdf.org launched. 2000 Alex & Brit be the first of its kind for gather information to regarding drug development. drug development for patients developed: replace d’Arbeloff ALS in the world. support research and make $300,000 2001 today. Stephen is the first donor, EAAT2 protein using development. writing a check for $10,000 and gene therapy. donation to telling his brother to “get going.” ALS TDF. “ALS In-Vivo Screening Program” The Uptake Therapy launched in the Fall. Alex & Brit ALS TDF raises ALS TDF’s “Friends & Family Project kicked off Scaled up to become d’Arbeloff $434,761 in its first Community” goes live. Web ALS Hope/Hobler Foundation in partnership with largest in the world. Heywood brothers ALS TDF receives approval www.als-tdf.org gets facelift; make $100,000 year of existence. based fundraising community 27 drugs tested in SOD1 makes $500,000 gift to ALS TDF. The Dr. Jeffrey Rothstein and ALS TDF featured to conduct the world’s designed to include results from donation to launched as one of the first in mouse; 2 hits. Foundation renames its main drug- at John Hopkins in New Yorker ALS TDF raised first stem cell trial for ALS. research trials and better connect ALS TDF. the non-profit world. screen program the “ALS Hope Drug University. 1st annual “Night at Fenway” event held. magazine exposé $1,562,126 in 2000. Stephen Heywood and 2 patients and researchers together. 2001 Discovery Center”. “Curing the Incurable.” others participate. ALS TDF screens Dapsone, Established 4 external research collaborations WHI-P131, Celebrex, with academics and more than 25 partnerships Chlorpromazine, Copasone, 58 drugs with biotechs and pharmaceutical companies. Gusperimus, Quinacrine, evaluated Riluzole, Thalidomide, CVF, throughout DDC IIG, Indomethacin, 2003, including The Friends & Family Leflunomide, Memantine 8 advanced Community, the Foundation’s and others. efficacy screens. 2002 main fundraising arm, doubles Ten ALS 101 sessions held 2003 2001 throughout the year. New funding allows the lab its income to $1.8 million. ALS TDF executes a combination ALS TDF moves across the to move to a larger space at drug trial of Ritonavir and Charles River into a larger 28 drugs evaluated and the Massachusetts College of Hydroxyurea at the University of Ronya Kozmetsky ALS TDF raised research facility located in the 3 preliminary hits in the Pharmacy by the end of the year. San Francisco in Partnership with makes $1 million SOD1 mouse model. $4,018,069 in 2001. Harvard Partners building. Alex & Brit d’Arbeloff the Larry L. Hillblom Foundation. pledge to ALS TDF An anonymous donor provides a $300,000 make $1,000,000 ALS TDF raised after the passing of her grant funding the development of a new donation to ALS TDF. $4,335,417 in 2002. husband, George, to 2003 bioinformatics system at ALS TDF. ALS a year earlier. Passion For Life 1st Annual Leadership Fund, inspired Summit held in Scientists at the Institute in 2000 by Cambridge at the innovate a novel device A Biosafety Level 2 Lab is pALS Mary Lou Massachusetts known as an intrathecal constructed at ALS TDF. Krauseneck, Biotechnology Council The Fran Delaney catheter, which delivers The secure lab allows reaches its followed by a lobster 2003 PGA Tour Golfers Jeff Julian and Fund hits their $1 potential therapeutics expansion of gene Mass spectromety core $1 million dinner and Awards at Tom Watson, together with Tom’s million cumulative directly to the spinal therapy and cell-based created. The goal is fundraising the Heywood’s home Watson’s caddy Bruce Edwards, start fundraising ALS TDF staff attend 2004 to monitor drug levels milestone. in Newton, where the cord of the mouse. ALS TDF hires industry treatment pipelines. “Driving 4 Life,” a fundraising effort milestone in less Society for Neuroscience in attained in any tissue type. Foundation first began ALS TDF research team trained discovery experts, based on golf. Campaign raises $1 than 3 years. San Diego. to operate. establishing a dedicated million by end of the year. attends Society for 2004 2005 Neuroscience meeting Discovery Biology Core. ALS TDF purchases a in New Orleans. powerful Zeiess X1 microscope and builds up its molecular imaging ALS TDF raises The 1st Annual “A His Brother’s capabilities thanks to an $3,509,483 - spends “Friends Cure is Coming!” walk Keeper, a book allocation from the Jeff 79% on R&D. and Family is held. Dozens of by Jonathan Nearly 100 cyclists, including Hadley Fund. This makes the Community” patients and families Weiner, many pALS, participate in the Foundation the grows to include walk from Lexington chronicles Tri-State Trek, a 270-mile bike largest funded R&D 70 separate and Battle Green to the program exclusively Stephen’s ride from Boston to NY. Jamie articulates the active funds. ALS TDF moves to its Old North Church in focused on ALS.