2020 Postcard from Palm Springs
Highlights from the 2020 CHDI HD Therapeutics Conference are now here! This year’s Postcard covers HTT-lowering therapeutics, including an
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Emmy Award-Winning TV Journalist / HD Advocate
“How Far We Have Come and Where We Are Heading”
Charles Sabine’s talk at the Huntington Society of Canada (HSC) National Conference will describe the lessons he learnt from his time at NBC, his family’s shocking Huntington disease (HD) story, and his discovery of the hugely underestimated prevalence of the disease due to centuries of shame and stigma. He will also relate the story of the unprecedented global collaboration between researchers and HD families that has made research into this field the most thrilling anywhere in medicine.
Emmy Award-winning TV journalist Charles Sabine OBE worked for the U.S. network NBC news for 26 years.
That career saw him cover twenty-four wars, six revolutions, and four earthquakes. He was present for most of the news events of Europe, the Middle East, Africa and Asia since the early 1980s. There, he learnt first-hand the extraordinary limits that the human spirit is capable of reaching in the face of tragedy inflicted by both nature and humankind.
In 2008, he decided to put the lessons of those experiences to a different use and he became a pioneering spokesman for freedom of scientific research, as well as sufferers of degenerative brain illnesses – in particular, Huntington disease, which has ravaged his family.
That role has led to Sabine speaking at prestigious venues across the world, including the European and British parliaments, the Royal Institution in London, the World Congress on Freedom of Scientific Research, and the Vatican.
In her last New Year Honours list, Queen Elizabeth II awarded the Most Excellent Order of the British Empire (OBE) to Sabine, “Global Campaigner, Huntington’s Disease”. This was the first time in the history of these awards that the words ‘Huntington’s disease’ were used in a citation for an OBE.
“Huntington Disease Before Disease”
Empowered by clinical trial data and data from human-derived cells at multiple stages of Huntington disease (HD), we now define the earliest steps in HD pathology that occurs decades before disease onset. We have recently focused on the question of “how much and how soon?” when we design clinical trials and define the best time to treat people gene positive for HD. Innovation in lab science, pharmaceutical development and the drug approval process will lead us to a new paradigm of how we treat HD, with focus on preventing the onset of disease.
Dr. Ray Truant started his Huntington disease research lab at McMaster University in Hamilton, Ontario in 1999. His lab receives funding grants from CIHR and the Krembil Foundation, as well as the Huntington Society of Canada, the Huntington’s Disease Society of America, and the Hereditary Disease Foundation.
Dr. Truant is currently Full Professor in the Department of Biochemistry and Biomedical Sciences, and was Chair of the Scientific Advisory Board as well as a National Board Officer with the Huntington Society of Canada 2007-2021.
In 2012, he was awarded the Queen Elizabeth II Diamond Jubilee Medal for public service and in 2014, the Michael Wright Community Leadership Award from the Huntington Society of Canada.
Dr. Truant’s lab has been focused on developing new methods of microscopy and nanoscopy using biophysics and computer machine learning to study huntingtin function at the single cell level using cells derived directly from HD patients and those at risk of disease. This system was developed in the lab in 2018. His group has been involved in the development of huntingtin lowering splice modulation drugs. His current research focus is on the pre-disease state of HD and defining biomarkers that will aid in defining this stage as the optimal therapeutic strategy to prevent disease onset.
SATURDAY, NOVEMBER 18
HD Researchers/HD Buzz Founders and Contributors: Dr. Jeff Carroll, Dr. Rachel Harding, Dr. Sarah Hernandez and Prof. Ed Wild
“HD Buzz Global Research Update”
Old friends of HSC Ed and Jeff are back! And they are joined by members of the newly expanded HDBuzz editorial team to bring you an entertaining, informative and inspiring run-down of what’s hot in clinical trials, drug development and cutting edge lab research into Huntington’s disease – and what you can do to help.
Dr. Jeff Carroll works in the Department of Neurology at the University of Washington, as well as the n-Lorem foundation, whose goal is to develop and deliver antisense oligonucleotide (ASO) drugs to patients with nano-rare diseases for free, for life. Jeff serves on the access to treatment committee to vet submitted cases for n-Lorem. Jeff himself comes from a family affected by HD. In 2003, genetic testing revealed that Jeff had inherited a mutant copy of the HD gene from his mother, which means that in the absence of new treatments, he will eventually develop symptoms. In addition to his work in the lab, in 2011, he co-founded HDBuzz.net, the first and leading source of accessible, impartial research news for the HD community worldwide.
Dr. Rachel Harding is the Principal Investigator of the Huntington’s disease team at the Structural Genomics Consortium, University of Toronto. Rachel and her team research the 3D structure and shape of the huntingtin protein to try and understand how this molecule works and how this Huntington’s disease mutation might affect this big and complex molecule.
Dr. Sarah Hernandez is the Director of Research Programs at the Hereditary Disease Foundation (HDF). She carried out her postdoctoral work with Dr. Leslie Thompson at the University of California, Irvine, where she used stem cells and fruit flies to define HD-related changes that exist outside the cell, in the “extracellular matrix”. At the HDF, Sarah oversees the Foundation’s scientific research portfolio by managing the grants program, acting as the scientific liaison, and coordinating scientific programming through webinars, workshops, and conferences. Sarah enthusiastically supports the mission of the HDF, which is to identify the most promising research and accelerate treatments for Huntington’s disease.
Prof. Ed Wild, FRCP, PhD – Ed is Professor of Neurology at UCL Queen Square Institute of Neurology, Associate Director of UCL Huntington’s Disease Centre, and a Consultant Neurologist at the National Hospital for Neurology and Neurosurgery. Ed has worked on HD since 2005 and leads a team focusing on clinical trials of new HD treatments and studying cerebrospinal fluid to understand HD. He has authored seven book chapters and over 90 peer-reviewed scientific publications, and is Chief Investigator of HDClarity, the first and largest multinational CSF collection study in HD. He received the Huntington’s Disease Society of America Researcher of the Year Award, and Huntington Society of Canada Community Leadership Award. He co-founded HDBuzz, the leading source of accessible, impartial research news for the HD community.
President of Woody Guthrie Publications/Daughter of Woody and Marjorie Guthrie
“She Came Along To Me: The Marjorie and Woody Guthrie Story”
This program chronicles the journey of Woody Guthrie’s battle with Huntington’s disease and his wife, Marjorie, and her tireless fight to find a cure! When Woody and Marjorie met in 1941, Marjorie was a dancer with the Martha Graham Dance Company and Woody was an up-and-coming folk balladeer living in New York City. As Marjorie said, “We were like any young couple in love…and …our kids were going to grow up and be world changers and hopers.” Huntington’s cruelly interrupted their dreams. However, their belief in “world changers and hopers” remained when Marjorie founded the grassroots organization The Committee to Combat Huntington’s Disease, now the Huntington’s Disease Society of America. This multimedia program incorporates archival photographs, historic and personal film footage, along with music by Woody Guthrie.
Nora Guthrie, the youngest child of Woody and Marjorie Guthrie, is the president of Woody Guthrie Publications, Inc. She served as president of the Woody Guthrie Foundation for over 30 years, founded the Woody Guthrie Archive in 1992, and co-founded the Woody Guthrie Center in Tulsa, OK in 2013. Working with her father’s archive, Nora has given unprecedented access and visibility to his creative works through new recordings, publications, films, and exhibitions. She is the executive producer of numerous GRAMMY Award-winning albums, recording hundreds of her father’s previously unknown lyrics set to music by contemporary musicians. Her most recent projects include “Woody Guthrie: People Are the Song,” a major exhibition presented at the Morgan Library & Museum in NYC, and the ASCAP Deems Taylor/Virgil Thompson Award-winning publication, “Woody Guthrie: Songs and Art • Words and Wisdom” (Chronicle Books). Since Woody’s passing in 1967, Marjorie, and now Nora, have consistently reminded us that Woody’s work is enlightening, inspiring, and edifying – spotlighting Woody Guthrie as a significant guiding voice and citizen of the world.
Join our 50th Anniversary campaign, Route 50: Driving Hope Forward and work toward a world free from Huntington disease: www.huntingtonsociety.ca/hsc50/