HDSA Annual Report 2019
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GREETINGS! Dear Friends of HDSA, Thanks to the Griffin Foundation, we welcomed two youth-focused social workers to the HDSA Though 2019 may seem long ago, at times of team. Katie Dykman, LISW and Jessica Marsolek, uncertainty it’s often helpful to reflect on the LGSW are experienced professionals trained to strength and health of recent times. The 2019 serve young people and respond to the unique Annual Report for the Huntington’s Disease needs of children, teens and young adults impacted Society of America (HDSA) is a clear reminder of by HD. how strong we are as a community united in the fight against Huntington’s disease. And this is just the tip of the iceberg… your contributions have allowed HDSA to get more 2019 was one of HDSA’s most successful years hands on deck and improve our tremendous ever. Thanks to the generous contributions from support programs even more. HDSA’s telehealth people like you, HDSA t is growing in every aspect program, online support groups, HD Trialfinder of our mission services, providing more support to and helpline are FREE for everyone affected by families affected by HD. Importantly, the strides we HD because of you. made in 2019 positioned us to grow in 2020 despite the unforeseen challenges of COVID-19. HDSA continues to be a world leader in supporting HD research. In 2019, we funded more HD science In 2019, we expanded the HDSA Centers of than ever before, including more than $1.4 million Excellence program to 47 facilities and 6 clinical in direct grants to scientists. We also continued sites across America where more families can to cultivate strong relationships with leading receive expert care. With new Centers in California, bio-pharmaceutical partners to ensure that the Illinois, New York and North Dakota, HDSA HD family voice resonates in drug development supported comprehensive HD care locations in so that potential therapies are studied as quickly 53 clinics in 31 states, plus the District of Columbia. as possible. E.J. Garner, Chair, HDSA Board of Trustees and HDSA awarded a total of $1.4 million to the Centers Louise Vetter, HDSA President and CEO of Excellence program, an almost eleven percent As we continue to push forward, we cannot do increase in funding from 2018, and set us up for this without your continued support. Whether it is further expansion of care in 2020. a financial contribution, enrolling in a clinical trial, sharing a story on social media or just talking about A year ago in June of 2019, HDSA welcomed HD in your community, we are all in this together. Allison Bartlett, Esq. to serve as HDSA’s Manager of Disability Programs. With her unique Thank you and stay safe! HD-focused experience, Allison is assisting countless families through the complex disability process. By hosting Disability Chats online and providing online resources to help families take E.J. Garner advantage of federal benefits, she provides practical Chair, Board of Trustees insight into the US Social Security Administration process and what is needed for approval. Additionally, last year saw incredible growth in Louise Vetter our support programs for youth impacted by HD. President & CEO 1 2019 Highlights HDSA CENTERS OF EXCELLENCE GROW TO 47 SITES ACROSS THE U.S. 2019 HUMAN BIOLOGY PROJECT FELLOWSHIPS In 2019 HDSA announced that forty-seven out- ton’s disease that share an exemplary commitment In October of 2019, HDSA awarded five grants Huntington’s disease science in the human con- standing Huntington’s disease care facilities with to bringing comprehensive care. under the Society’s largest research initiative, the dition. Applicants worldwide proposed projects six satellite sites were awarded the designation of In December, HDSA awarded four grants under a HDSA Huntington’s Disease Human Biology addressing HD through small clinical studies or do- HDSA Centers of Excellence. This was an increase new research initiative, the HDSA Center of Excel- Project. These grants, totaling $575,000, represent nated human samples. This year’s winners hail from from 43 in 2018, and from just 20 in 2015. The four lence Clinical Research Pilot Program. These grants, HDSA’s patient-centric research focus which brings Scotland, Spain, and the United States. new 2019 Centers of Excellence are: OSF Health- totaling $50,000, represent HDSA’s patient-centric basic and clinical researchers together to facilitate Care Illinois Neurological Institute (Peoria IL), research focus by fostering novel clinical research Sanford Health, (Fargo, North Dakota), University projects within the HDSA Center of Excellence of Buffalo (New York) and University of California, network. Irvine. The HDSA Centers of Excellence are multi- disciplinary care teams with expertise in Hunting- Learn more at HDSA.org/COE Danielle Larson, MD Osama Al Dalahmah, MD Vilija Lomeikaite, PhD candidate Northwestern University Columbia University University of Glasgow Feinberg School of Medicine Dr. Al Dalahmah will examine Vilija will work on improving Dr. Larson will study whether how astrocytes, the brain’s the existing methods to detect remote “telemedicine” visits support cells, change over the an increase in CAG repeats in with a neurologist could be just course of HD to become harmful certain cells of the body and as helpful as in-person visits for versus helpful. brain during the course of HD. OSF HealthCare Illinois Sanford Health, an HD patient or family. Neurological Institute (Peoria, Illinois) (Fargo, North Dakota) Ricardo Mouro-Pinto, PhD Saul Martinez-Horta, MsC, HDCARE Clinic, Gottschalk Medical Plaza, UBMD Neurology, University of California, Irvine University of Buffalo (New York) Massachusetts General Hospital Sant Pau Hospital, Barcelona and Harvard Medical School Saul will examine what kinds Dr. Mouro-Pinto will focus on the of changes are happening in biology of CAG repeat expansion the brain and blood when HD in hopes of developing drugs to patients begin to have difficulty stop this process. with planning and memory. 2 3 2019 DONALD A. KING SUMMER RESEARCH FELLOWSHIPS 2019 BERMAN-TOPPER FAMILY HD CAREER DEVELOPMENT FELLOWSHIPS In April of 2019, four exceptional undergraduate scientists are steered towards the field, this program As part of HDSA’s commitment to developing the Dr. Byrne is working under the guidance of Dr. Ed students were awarded HDSA’s Donald A. King supports undergraduates who have committed their next generation of passionate and innovative Wild at University College London. Her project will Summer Research Fellowships. These awards are summers to training in an HD research lab. Applica- Huntington’s disease scientists, the Berman-Topper better prepare the field for future disease named in honor of Donald King, who served as HDSA tions are accepted between December and March each Family HD Career Development Fellowships prevention studies by researching how early in life Board Chairman from 1999 to 2003 and worked year and are reviewed by HDSA’s Scientific Advisory provide up to $80,000 of funding per year for three we can detect changes in the neurofilament light tirelessly to advocate for HD families until his death in Board. The 2019 Donald A. King Summer Research years to young scientists and clinicians who desire (NfL) protein in persons with the HD mutation. 2004. In order to further meaningful discoveries about Fellows completed focused HD research projects under to make HD part of their long-term career plan. Huntington’s disease and ensure that bright young the supervision of senior scientists. These prestigious awards are made possible due to Dr. Caron is guided by Dr. Michael Hayden at the the generosity of the Berman and Topper families University of British Columbia. He will investigate and CHDI Foundation. HDSA’s Scientific Advisory novel ways to enhance the delivery of huntingtin Board reviewed applications from researchers from lowering antisense oligonucleotides across the all around the world, and HDSA was able to award blood-brain barrier. two scientists the 2019 Berman-Topper Fellowship — Dr. Lauren Byrne and Dr. Nicholas Caron. Dr. Lauren Byrne and mentor Dr. Ed Wild at HDSA’s Convention in Boston. Zach Cook, Brown University Chloe LaRochelle, University of Central Florida Zach spent the summer working with Dr. Marc Tatar, Chloe worked under the guidance of Dr. Amber testing effectiveness of drugs in a fruit fly Southwell on a project investigating aggression in (Drosophila) model of Huntington’s disease. HD mice. Alexandra Putka, Duke University Colleen Strohlein, University of Pittsburgh Alexandra worked in the laboratory of Dr. Audrey Colleen spent the summer working in the laboratory Dickey to elucidate how altered function of a protein of Dr. Robert Friedlander, to investigate whether called PPAR contributes to HD pathology. phosphorylation of huntingtin protein affects its localization to mitochondria. Dr. Nicholas Caron 4 5 HDSA’S 34TH ANNUAL CONVENTION IN BOSTON SETS ATTENDANCE RECORD The 34th Annual HDSA Convention was made possible by the generous HDSA 2019 NATIONAL AWARDS support of all its sponsors: Woody Guthrie Inaugural Long-Term PRESENTING SPONSOR Advocacy Award Care Award • Genentech Seth Rotberg and Sitrin Health Care Center • Teva Pharmaceuticals Therese Crutcher-Marin HDSA Excellence HDSA Youth Award in Care Award PLATINUM SPONSOR Hallie Kile Dr. Samuel Frank • Lundbeck th Researchers of the Year Marjorie Guthrie Award The 34 Annual Huntington’s Disease Society of GOLD NONPROFIT SPONSOR Dr. Darren Monckton and Patricia McLane and America Convention was held in Boston, MA on • CHDI Foundation / Enroll-HD June 27th-29th at the Boston Marriott Copley Place. Dr. Vanessa Wheeler Morgan McLane With 1,225 guests, the Convention set a new HDSA Chapter of the Year HDSA Person of the Year SILVER SPONSORS attendance record breaking the previous record HDSA New Jersey Chapter Alexandra Boothby • Ionis (1,046) in Los Angeles, California held in 2018. The HDSA Affiliate of the Year • uniQure education tracks were jam-packed with workshops HDSA Connecticut Affiliate from world-class speakers, including Nobel Laureate BRONZE SPONSORS Dr.